EUROPEAN COMMISSION

Brussels, 26.5.2026

SWD(2026) 450 final

Addendum to
COM(2025) 1022 and COM(2025) 1031 adopted on 16.12.2025

COMMISSION STAFF WORKING DOCUMENT

European Biotech Act

Accompanying the documents

Proposal for a Regulation of the European Parliament and of the Council on establishing a framework of measures for strengthening Union's biotechnology and biomanufacturing sectors particularly in the area of health and amending Regulations (EC) No 178/2002, (EC) No 1394/2007, (EU) No 536/2014, (EU) 2019/6, (EU) 2024/795 and (EU) 2024/1938 (European Biotech Act)





and





Proposal for a Directive of the European Parliament and of the Council amending Directives 2001/18/EC and 2010/53/EU as regards the placing on the market of genetically modified micro-organisms and the processing of organs

{COM(2025) 1022 final}
{COM(2025) 1031 final}

Table of contents

1Introduction

2Background

2.1Political and legal context

2.2Related EU legislation

2.3Background on the sector

3Problem definition

3.1Problem: biotech companies struggle to innovate, raise capital, bring innovations to market, produce and grow in the EU, while maintaining high level of protection and safety

3.2What are the problem drivers?

3.3Consequences

3.4What is the baseline from which measures are assessed?

4Approach to the Biotech Act

4.1Objectives of the proposal

4.2Choice of the legal instruments and legal basis

4.3Subsidiarity: necessity for EU action and EU added value

4.4Intervention logic of the proposal

5Description of the proposed measures and analysis of their main impacts

5.1Interventions for regulatory simplification: measures and expected impacts

5.2Interventions on industrial enablers: measures and expected impacts

6Cumulative Economic, social, environmental and other impacts of the proposal

6.1Regulatory simplification and administrative burden

6.2Competitiveness and investment attractiveness

6.3Innovation and research

6.4Public health and safety

6.5Environmental impacts

6.6Digital by default principle

7Monitoring and evaluation

ANNEXES:

Annex 1: Procedural information

Annex 2: Stakeholder consultation

Annex 3: Who is affected and how? 

Annex 4: Analytical methods

Annex 5: Additional information on background on the sector and problem definition

Annex 6: Overview of the proposed measures and articles of the proposed regulation and directive

Annex 7: Additional information on measures and expected impacts

Annex 8: COMPETITIVENESS CHECK

Annex 9: SME check

Glossary

1Introduction

This staff working document summarises the analysis supporting the proposal for a Regulation 1  (the ‘proposed Regulation’) and the proposal for a Directive 2 (the ‘proposed Directive’) which were adopted by the Commission on 16 December 2025. These two proposals (collectively referred to as the ‘Biotech Act’) aim to strengthen the EU’s biotechnology and biomanufacturing sectors, particularly in the area of health. They amend key legislation relevant to these sectors.

Biotechnology and biomanufacturing, supported by artificial intelligence (AI) and digital tools, could help modernise entire parts of the EU economy. They hold huge potential to boost competitiveness and innovation, while delivering for patients, public health, food safety, security and other areas. Despite its world-class scientific base, the EU is trailing its competitors when it comes to translating biotechnology research into development, market deployment and manufacturing at scale. Fragmented ecosystems, financing constraints, complex regulatory pathways, and a lag in adapting regulatory frameworks to technological advances, hinder EU biotechnology companies’ potential to innovate, bring innovation to market, produce and grow in the EU. As a result, the EU’s biotechnology sector does not meet its full potential to tackle major societal challenges and faces a widening global competitiveness gap.

The proposed Regulation and Directive present a series of measures primarily for the health biotechnology sector, with targeted actions addressing other biotechnology areas (food and feed, industrial and agricultural biotechnologies). The two proposals aim to accelerate the way biotechnology innovations, products and services are developed and placed on the single market, while maintaining the highest safety standards. The proposed Biotech Act is one of the flagship initiatives of the Competitiveness Compass 3 , designed to unleash the EU’s innovation potential. It also contributes to regulatory simplification and administrative burden reduction goals.

The Biotech Act is in line with the United Nations’ Sustainable Development Goals (SDGs), in particular SDG 3 ‘Good health and well-being: Ensure healthy lives and promote well-being for all at all ages’; SDG 9 ‘Industry, innovation and infrastructure: Build resilient infrastructure, promote inclusive and sustainable industrialisation and foster innovation’; and SDG 12 ‘Responsible consumption and production: Ensure sustainable consumption and production patterns’. More details on the relevant SDG targets and indicators are available in Annex 3. Regarding specialisation in terms of scientific output, the EU leads inter alia in SDGs 9 and 12. 4

2Background

2.1Political and legal context

A ‘European Biotech Act’ was announced by the President of the Commission in the 2024 - 2029 Political Guidelines of the European Commission 5 , to make it easier to bring biotechnology products from the laboratory to the factory and then onto the market, while maintaining high safety standards. It was further reflected in the mission letter of Commissioner Várhelyi 6 .

In the Commission Recommendation on critical technology areas for the EU’s economic security 7 , biotechnology was recognised as having “an enabling and transformative nature in areas such as agriculture, environment, healthcare, life sciences, food chains or biomanufacturing”. The Commission Communication on Biotechnology and Biomanufacturing 8  highlighted the high growth potential and labour productivity of biotechnology and biomanufacturing. This makes the sector pivotal to the competitiveness and modernisation of the EU’s economy, as well as conducive to its strategic autonomy and resilience. While the EU’s strengths were recognised (i.e. research and innovation base and capacities), the Commission stressed the need to address the challenges faced by companies, users and consumers. It called for both a supportive regulatory environment and a coordinated approach, particularly for developing infrastructures, fostering the use of AI, and encouraging private and public investments. Additionally, the EU’s Economic Security Strategy 9  identified biotechnology as one of the 10 technology areas most likely to present sensitive and immediate risks related to technology security and technology leakage. This strategy launched an EU joint risk assessment with Member States on biotechnology, which identified relevant biosecurity risks that require mitigation measures.

The seminal reports by Enrico Letta 10 and Mario Draghi 11  also underscored the need to take action in the biotechnology and biomanufacturing sectors. They identified the need for a stronger, more dynamic European industrial policy, including measures for the biotechnology and biomanufacturing sectors to scale up and get products on the market faster. The report by Sauli Niinistö 12  also pointed to the need to strengthen biological defence capabilities and preparedness against emerging biological threats, including synthetic pathogens.

In line with this growing political impetus, reducing the EU’s strategic dependencies in sensitive sectors has become a priority of the European Council 13 , including in health and food, as identified in the Versailles Declaration of 2022 14 . The Council 15  has urged the Commission to unlock the potential of biotechnologies for the EU’s competitiveness through the European Biotech Act, while maintaining environmental and safety standards. In particular, the Council has encouraged actions to advance the use of regulatory sandboxes, promote research and development (R&D) in advanced therapy medicinal products (ATMPs) and address the falling global share of EU clinical trials (CTs). It also supports the greater use of AI and the development of appropriate skills. In parallel, three Important Projects of Common European Interest (IPCEI) in the biotechnology domain are currently under design 16 , reflecting Member States’ interest in coordinated, large-scale investment approaches.

The European Parliament’s recommendations in its resolution ‘Future of the EU biotechnology and biomanufacturing sector’ are in line with these priorities, asking to facilitate “a fast and efficient uptake of biotechnology and biomanufacturing through clear regulatory frameworks” 17 . Recommendations include facilitating the uptake of biotechnology and biomanufacturing; enabling more efficient scale-up and commercialisation of innovations; streamlining and simplifying the time-to-market for biotechnology throughout their life-cycles; streamlining the clinical trials framework by minimising administrative burden and delays; and using regulatory sandboxes. It also recognises the need for a skilled workforce, dedicated support for SMEs to access funding, and biosecurity screening standards. The European Parliament has also prepared an own-initiative report on the ‘Public health aspects of biotechnology and life sciences’ 18 , where it puts forward similar recommendations. It has called for the Biotech Act to review, simplify and optimise the regulatory framework for biotechnology in healthcare to foster innovation, as well as for structured EU support to help excellent biotechnology innovation districts in the EU grow and ensure that they have sufficient capacity, resources and the scientific edge to promote new groundbreaking biotechnology discoveries, innovation and commercialisation.

2.2Related EU legislation

2.2.1Existing EU legislation

Health biotechnology products are subject to several EU legislative frameworks, in particular Regulation (EU) No 536/2014 on clinical trials on medicinal products for human use  19 , Regulation (EC) No 1394/2007 on ATMPs 20 , Regulation (EU) 2024/1938 on standards of quality and safety for substances of human origin intended for human application (SoHO) 21 , Directive 2010/53/EU on quality and safety of human organs intended for transplantation 22 , and Regulation (EU) 2019/6 on veterinary medicinal products (VMPs) 23 . These legislative frameworks govern different stages of the product’s lifecycle, including research, clinical development, authorisation and the placing on the market of biotechnology products and therapies. Accordingly, the EU biotechnology sector can only flourish if they are efficient, coherent and innovation-friendly. 

For food and feed safety, the placing on the market of biotechnology products must comply with requirements set out in the General Food Law 24  and with specific legal frameworks, where applicable 25 . Placing products on the market that contain or consist of genetically modified organisms (GMOs), other than food and feed, falls within the scope of the legislation on the deliberate release of GMOs 26 . These legislative frameworks ensure high levels of human and animal health, and environmental protection. To foster biotechnology innovation, it is important to keep these objectives front and centre and maximise procedural efficiency and speed, because risk-assessment and authorisation procedures directly affect the time and cost for biotechnology products to reach the market.

The proposed Biotech Act reviews and seeks synergies with other legislation to ensure coherence with the overall EU regulatory system. It complements the Critical Medicines Act (CMA) 27  in strengthening EU-based biotechnology research and manufacturing. It is in line with the pharmaceutical strategy for Europe 28  and complements the recent revision of the EU pharmaceutical legislation 29 . It is also complementary to the Commission’s proposal to revise the EU medical device legislation 30 , sharing the same overall objectives and including common measures such as the single authorisation process for combined device-medicine clinical studies. It also aims to improve coordination and consultation between relevant competent authority groups established under other frameworks, such as medical devices 31 , medicines, SoHO, health technology assessment 32 , etc.

2.2.2Aligning with the broader priorities of the Commission

As a flagship initiative under the Competitiveness Compass 33 , the Biotech Act aligns with the EU’s broader innovation and competitiveness agenda, turning its priorities into actions in the strategic sector of biotechnology. In particular, the Biotech Act aims to reduce the administrative burden, improve coordination, simplify rules and streamline procedures, in line with both the Competitiveness Compass and the Commission’s Communication on implementation and simplification 34 . The Biotech Act forms part of the Commission’s Life Sciences Strategy 35 which recognises biotechnology as a strategic cross-sectoral technology and seeks to strengthen the EU’s biotechnology ecosystem, streamline regulatory pathways and boost the EU’s competitiveness more broadly in life sciences.

The Biotech Act complements other policy initiatives announced in the Competitiveness Compass. For example, it aims to improve access to risk-tolerant capital for biotechnology firms, aligning with the Savings and Investments Union 36  and the Start-up and Scale-up Strategy 37 , which establishes the Scaleup Europe Fund 38 . Its biosecurity provisions reflect the link between competitiveness and security by strengthening safeguards for biotechnology products of concern. They address the risks identified by the biotechnology joint risk assessment launched under the EU’s Economic Security Strategy 39 and complement Regulation (EU) 2022/2371 on serious cross-border threats to health 40 . The provisions aim at helping to prevent and prepare for such threats and to ensure a coordinated EU-level response, also for arising public health emergencies (including any arising from the misuse of emerging biotechnologies). They also complement the Medical Countermeasures Strategy 41  for preparing and responding to health threats, including human-made biosecurity threats.

The Biotech Act reflects the emphasis on talent and contributes to the Union of Skills 42 . It aligns with actions under the European Strategy on Research and Technology Infrastructures 43 . It aligns with EU funding and investment initiatives that support biotechnology research, innovation and industrial scale-up. It takes into account EU financial support available, including cohesion policy programmes, InvestEU, the European Investment Bank (EIB) Group’s TechEU programme 44 , 45  and the European Innovation Council. The strategic importance of biotechnology is also acknowledged in preparations for the next Multiannual Financial Framework (MFF), namely the next iteration of the Horizon Europe programme (2028-2034). It is acknowledged in the section on collaborative research activities in the Competitiveness component, as well as the proposed European Competitiveness Fund, which includes a dedicated ‘Health, Biotech, Agriculture and Bioeconomy’ policy window.

The Biotech Act is designed to be coherent with related digital policies and pursue digital transformation objectives. It aims to support secure, data-driven biotechnology ecosystems and contribute to the EU’s technological sovereignty by promoting greater data use and AI integration in the biotechnology sector. Its emphasis on AI is consistent with the AI in Science Strategy 46 , the Apply AI strategy 47 , the AI continent action plan 48 , the European AI Act 49 , the Data Union strategy 50 , as well as the EU Cybersecurity framework  51 . The Biotech Act is also consistent with the objectives of the European Health Data Space Regulation (EU) 2025/327 52 and of the Data Union Strategy  53 , in particular with regard to data and metadata semantic interoperability and quality accelerators, data labs, and AI factories. It should also be read in coherence with ongoing interoperability and standardisation work for common European data spaces under Article 33 of Regulation (EU) 2023/2854 54 , including the European Trusted Data Framework, insofar as these initiatives are relevant to metadata, discoverability, semantic interoperability and the cross-border re-use of data.

The Biotech Act is consistent with the Vision for Agriculture and Food 55 , in particular because it amends the General Food Law to make the regulatory environment more supportive of innovation and competitiveness in the agri-food sector. The Biotech Act is also consistent with the Commission proposal on plants developed by certain new genomic techniques  56 as it continues to adapt the GMO framework to ensure that innovative products are regulated proportionately. The Biotech Act is also designed to build synergies with initiatives such as the recently-adopted Bioeconomy Strategy  57 . 

Climate change has highlighted the need to prioritise the EU’s resilience, so the Biotech Act reflects this priority too. It is in line with the Commission’s climate neutrality objectives set out in the EU Climate Law 58 and the EU’s Strategy on Adaptation to Climate Change 59 . It also ensures alignment with the ‘do no significant harm’ principle. Biotechnology products have the potential to support adaptation to climate change, contribute to health and food security through sustainable biomanufacturing, and protect biodiversity. They may also replace products potentially more harmful for the environment and provide benefits for consumers and users.

The Biotech Act will not affect EU legislation on the Registration, Evaluation, Authorisation and Restriction of Chemicals (REACH) 60 , Regulation (EU) 2024/1735 on sustainable biogas and biomethane technologies and biotechnology climate and energy solutions 61 or the Directive 2010/63/EU on the protection of animals used for scientific purposes. 

2.3Background on the sector 

For the purposes of this document, and as per the proposed Regulation, biotechnology 62  means “the application of science and technology to living organisms, as well as parts, products and models thereof, to alter living or non-living materials for the production of knowledge, products and services”. On this bases, health, food and feed are the main scope of the proposals. 

Biotechnology and biomanufacturing are major contributors to the EU’s economy. In 2022, the overall biotechnology sector accounted for EUR 38.1 billion of EU GDP (ca. 1.58% of the European industrial sector), with health biotechnology being the dominant contributor. Additionally, it contributed to 913,160 jobs, with around 77% 63 of those jobs coming from the health biotechnology sector. The sector in the EU is characterised by a large base of small and medium-sized enterprises (SMEs) (47%) and micro enterprises (35%), complemented by a smaller number of large companies (18%) 64 . The sector’s dynamism is illustrated by the 5,000 R&D-active biotechnology companies in the EU 65 . Furthermore, there were 3,232 unique biotechnology startups founded since 2015 that are still operative in 2025, largely in health 66  (See Annex 5, Figure 1).

The sector’s strategic importance is underscored by its rapid expansion. Over the last decade, the EU biotechnology industry has almost doubled to the share of GDP it generates, growing by around 7% per year on average between 2015 and 2024 67 (See Annex 5, Figure 2 for details on the sector GVA comparison). 

From a global perspective, the EU has significant strengths in biotechnology, but its overall competitiveness position is under increasing pressure (see section 3 on problem definition). The EU is the largest global exporter of biotechnology products, demonstrating the sector’s strong industrial base and integration into global value chains (see Annex 5, Figures 3 and 4) 68 . It is also a leader in biotechnology science, reflected by a publication record comparable to that of the US and China, with all three regions having between 20 – 25% of the top 10% most cited publications across biology, biomedical research and clinical science 69 . The EU also represents 15% of global patent families in the biotech field (see Annex 5, Figure 5 for details on the distribution across the EU) 70 . At the same time, other major jurisdictions are accelerating policy and investment efforts to strengthen their biotechnology ecosystems, as shown by recent initiatives in the US at both congressional and federal level, which frame biotechnology as a core economic and national security priority requiring coordinated action to sustain global leadership 71 .

In terms of projections, biotechnology and gene technologies are some of the most impactful technology trends expected to influence business models over the next five years 72 (i.e. the second most impactful technology in the medical and healthcare devices industry, with similar trends expected in agriculture, forestry, and fishing). In terms of job creation, a strong positive trend has been observed 73 . In the public consultation on the European Biotech Act stakeholders agreed that biotechnology and biomanufacturing products could positively impact the EU’s economy 74 . 

Finally, biotechnology applications have a significant potential to generate positive societal and environmental outcomes, making this sector a pillar of the EU’s societal wellbeing in key areas in particular in health and food. This potential is positively perceived by stakeholders, recognising in the public consultation the contribution of biotechnology and biomanufacturing products to society (90%) and to the environment (79%) and considered that biotechnology and biomanufacturing products that reach the single market are safe and secure (77%) 75 . Moreover, around a third of respondents (38%) agreed or strongly agreed that the EU regulatory environment ensures a higher level of safety and security than in some other countries 76 .

Health biotechnology encompasses a wide range of applications, from the development of innovative medicines and new medical devices addressing unmet medical needs, to combating epidemics and transforming the treatment of rare diseases. Gene and cell therapies, for example, hold great promise for treating genetic disorders 77 and restoring biological functions. Although the timeline for developing conventional vaccines is lengthy, current biotechnology tools allow scientists to develop new vaccines or adapt existing vaccines much faster than before 78 .

Agri-food biotechnology innovations support farmers and food producers. Environmental and industrial biotechnology solutions contribute to the sustainable transformation of our economy. Genetically modified micro-organisms (GMMs) play a decisive role in the development of industrial, environmental and agricultural biotechnology, both as a tool for manufacturing and in products. Products containing GMMs often yield innovative and beneficial product characteristics that other products cannot achieve or can achieve but with a higher cost or environmental impact. Examples of products containing GMMs already on the market outside the EU or in development include novel biofertilisers, biopesticides, and bioremediation products (i.e. to remove hazardous and toxic substances from the soil or wastewater).

3Problem definition

Figure 1. Problem tree

The Commission has refined and clarified the problem tree and intervention logic (see Section 4.4) from the version presented in the Explanatory Memorandum of the Biotech Act. These adjustments do not alter the underlying problem definition or policy rationale but provide a more structured and detailed explanation of the issues and causal pathways.

3.1Problem: biotech companies struggle to innovate, raise capital, bring innovations to market, produce and grow in the EU, while maintaining high level of protection and safety

Although the EU benefits from a sizeable biotechnology sector and strong scientific capabilities (see section 2.3), it does not meet its full potential for innovation in biotechnology and biomanufacturing. The EU lags behind other regions when it comes to translating its world-class science and innovation into commercially viable products in the EU, and even more so in manufacturing such products at scale. As a result, biotech companies often end up investing, growing, employing, creating value and placing their products on markets abroad. This pattern weakens the EU’s ability to retain value creation and industrial capacity from its research base 79 . 

The EU faces a substantial gap in venture capital with limited investment by pension funds and other institutional investors. Biotech is especially affected by this market failure due to the particular risk profile of biotech start-ups. Long delays between the initial innovation and first revenue generation due to lengthy regulatory process, massive upfront investments needed for CTs, and a high risk of failure of individual projects deter venture capital investors.

In addition to the fact that the EU accounts for 22% of biotech start-ups, against 49% in the US, the EU’s scale-up capacity also remains limited. Only a small share of EU biotech firms reaches significant funding levels (12% of agricultural biotechnology start-ups, 10% of health biotechnology start-ups, and 8% of industrial biotechnology start-ups secure over EUR 10 million 80 ). Very few exceed EUR 200 million 81 . As many companies struggle to scale in the EU, they increasingly rely on mergers and acquisitions as a common exit route. Around 40% of acquisitions of EU biotech start-ups are executed by US acquirers, highlighting how EU biotech innovation is frequently integrated into global pharmaceutical and life-science value chains instead of growing into large firms in the EU.

AI and data are now foundational to competitiveness in biotechnology, driving rapid advancements in discovery, development and manufacturing. While 20% of EU enterprises with 10 or more employees adopted AI by 2025 - up from 13.5% in 2024 - adoption in EU pharma and biotech manufacturing has surged even further, with large companies integrating AI at scale and shifting from pilot projects to system-level transformation 82 . However, in-house AI development and scaling remain constrained by data and compute bottlenecks - particularly for SMEs. AI biotech platforms are inherently capital-intensive, relying on costly model training and large-scale data generation, and depend on access to high-quality data and advanced compute infrastructure. As the industry matures, the gap between large pharma industry and smaller players is widening, underscoring the need for equitable access to technology and robust regulatory frameworks.  

3.2What are the problem drivers?

The problem is caused by three main related factors. Firstly, the regulatory and administrative operating environment for biotechnology in the EU is complex which in turn impacts speed.

Secondly, the EU’s biotechnology ecosystem operates in a fragmented manner, failing to tap the full scale of the EU research or testing infrastructure, markets, access to capital or production capacities, thus limiting companies’ ability to grow and compete internationally.

Thirdly, technological advances in biotech can result in obsolete frameworks to govern this fast-moving sector.

3.2.1Driver 1: the complexity of the EU regulatory framework, leading to slow time-to-market

A first set of challenges relates to the regulatory environment, which stakeholders consistently perceive as complex, time-consuming and difficult to navigate. In the EU, 60% of companies see regulatory burden as a key obstacle to long-term investment 83 . This finding applies to the biotechnology and biomanufacturing sector, as reflected in responses to the public consultation. Complex regulatory pathways and divergent implementation of EU legislation were identified as barriers to the expansion of EU companies, particularly start-ups, spin-offs and other SMEs 84 . Respondents largely agreed that EU rules create regulatory barriers, particularly when products move closer to market entry and formal approval processes 85 .

In addition, the perception of the EU regulatory environment compared to other countries showed a limited agreement about the level of predictability of the EU regulatory environment 86 . Also, a small number of respondents agreed or strongly agreed that the EU regulatory environment accelerates access to the market, is less complex and clearer, or leads to lower compliance costs than in some other countries 87 .

Such issues linked to the regulatory environment are confirmed across a variety of biotechnology areas. Detailed descriptions are presented in Annex 5.

With regards to the authorisation of clinical trials, the EU faces growing challenges in maintaining its competitive edge due to (i) lengthy regulatory timelines (particularly for the authorisation of multinational trials), and (ii) higher administrative requirements and costs in the EU/EEA associated with the authorisation and conduct of clinical trials, compared to other regions. As a result, sponsors 88 increasingly favour jurisdictions that offer faster regulatory timelines, simpler and more streamlined approval processes, and improved access for the recruitment of patient populations. This contributes to widening the competitiveness gap. On ATMPs, delays and increased costs are caused by administrative complexity and overly stringent requirements for GMO-based gene therapies. This stems from dual regulatory compliance requirements i.e. under the Clinical Trials Regulation and with the fragmented implementation of GMO legislation by Member States, further complicated by a blanket requirement for environmental risk assessment that does not take into account the specificities of low-risk gene therapies.

In the EU framework for VMPs, two main challenges are observed: (i) duplicative, not-fit-for purpose regulatory framework applicable to VMPs that contain or consists of GMOs and (ii) disproportionate administrative burden in the handling of variations not requiring assessment.

There is an increasing focus in the EU and globally on the authorisation of biosimilar medicines, where approval is increasingly based on analytical and functional characterisation to demonstrate similarity to the reference product 89 . However, the EU’s regulatory framework can still lead to the conduct of lengthy and costly multi-country comparative efficacy studies, even when robust analytical methods may demonstrate comparability and where a more tailored, risk-proportionate approach could therefore be justified. In the food area, EU law requires pre-market authorisations/approvals for several categories of food and feed products and related food chain inputs, the so-called ‘regulated products’ 90 . In those cases, the European Food Safety Authority (EFSA) must conduct a scientific risk assessment under the applicable sectoral legislation before risk managers at EU and/or national level decide to grant a pre-market authorisation/approval. Delays have been observed in both validation and risk assessment phases as also confirmed by the findings of the ongoing evaluation of the Commission on EFSA’s Performance covering the period 2017-2024. These have been linked to: (i) dossier deficiencies and limited effectiveness of pre-submission advice, (ii) procedural consequences linked to non-compliance with study notification requirements; and (iii) governance constraints relating to EFSA’s Scientific Committee/Scientific Panels.

As a cross-cutting element of complexity, developers of AI applications in biotechnology face a rapidly evolving regulatory landscape due to sectoral legislation and AI-specific requirements, including validation, documentation, explainability and risk-management obligations. The increasing integration of AI across the biotechnology lifecycle requires clarity on how existing EU frameworks apply across different development stages. Limited capacity, especially among SMEs and start-ups, to navigate applicable regulatory frameworks, further constrain the uptake of AI-enabled solutions in biotechnology. Public consultation responses 91 confirm that stakeholders see a need for greater clarity in the practical application of AI-related requirements across the biotechnology lifecycle (58% agreement). Stakeholders report uncertainty regarding how AI-related requirements interact with existing medicinal product and clinical research frameworks, particularly in areas such as model validation, documentation and lifecycle monitoring 92 and highlight the importance of guidance. Taken together, these patterns show that the EU biotech companies’ difficulty to thrive is not a science deficit, but rather a lack of translation and a scale-up deficit shaped by long, complex and capital-intensive pathways from research to market.

3.2.2Driver 2: Fragmentation of enabling ecosystems and suboptimal incentives for private investment constraining the scale-up of EU biotechnology and biomanufacturing 

The EU’s ambition to scale and industrialise biotechnology is currently limited by challenges in key enabling factors, in particular fragmented thus insufficient access to finance, development of infrastructures (e.g. clusters), availability of specialised talent and the deployment of AI and use of data.

First, with regards to access to finance, the EIB estimates the sector investment gap at EUR 40 billion annually. 93 US biopharma start-ups received around nine times more late-stage funding than EU biopharma start-ups, with around EUR 219 billion of venture capital focused on health biotechnology invested in the US compared to EUR 25 billion in the EU, between 2015 and June 2025. 94  Even larger gaps in access to late-stage capital have pushed biotech companies to find funding abroad, notably in the US. Between 2015 and 2021, 67 EU biotech companies going for a public listing have targeted the US NASDAQ rather than the European stock markets, with 60% of them only listing on NASDAQ without any listing on an EU exchange. 95

Similarly, in earlier stages of capital, although the EU biotech venture ecosystem has expanded materially in recent years, it remains a second-tier venture capital market in global biotechnology. Annual investment increased from EUR 0.84 billion (2015) to a peak of EUR 5.47 billion (2021) during a rapid expansion phase, before partially normalising to EUR 3.89 billion (2025) (around 29% below the 2021 peak) consistent with post-pandemic slower deployment (see Annex 5, Figure 7). As a comparison, the US operates at a different order of magnitude across the cycle (peaking at around EUR 45 billion in 2021) 96 . In the public consultation 97 , stakeholders reported difficult access to private and some public investment instruments in the EU in equity, debt, and commercialisation support (for more information, see synopsis report in Annex 2). In their answers, companies also noted that public funding mechanisms, while supportive for early R&D stages are also insufficiently tailored to the needs of biotech scale-ups. Specific challenges of start-ups and SMEs were also underlined from both representatives of business associations and public authorities. Respondents from academic and research also pointed the fragmentation in funding rules and tax incentives across Member States, limited availability of large venture capital funds, and a lack of banking expertise in biotechnology. Non-Governmental Organisations (NGOs) also reported challenges related to fragmented and insufficient public funding and a lack of significant grant-making institutions. This shows a need to reinforce de-risking financial instruments and budgetary guarantees in the EU.

Biological VMPs benefit from two protection mechanisms: regulatory data protection (Articles 39-40 of Regulation 2019/6) and intellectual property (IP) protection (patents and SPCs) under Regulation (EC) No 469/2009 98 . Nevertheless, the veterinary pharmaceutical market presents specific challenges for innovation, such as fragmented species-specific markets, low price levels, and a small overall market size. In this context, there is a need for enhanced incentives to support the development of biotechnological VMPs to diagnose, treat or prevent zoonotic diseases.

Second, with regards to biotechnology clusters, their outreach in the EU remains fragmented, with significant disparities in scope and resources. Biotechnology clusters foster collaboration across entities at different stages of the biotechnology value chain and across biotech sectors. They enable the transformation and evolution of, for example, university spin-offs into companies ready for the commercialisation of their product 99 . While clusters compete for investment, talent and projects, there is substantial scope for collaboration, exchange of best practices allowing the realisation of scale effects that cannot be achieved at regional level. The few clusters that have an EU-wide relevance provide networking and coordination support, yet the landscape remains fragmented, risking duplication of efforts and limiting impacts. As a result, EU biotechnology clusters cannot fully leverage their collective potential to compete with clusters in other regions, and the EU as a whole underperforms in the field of biotechnology.

Respondents to the public consultation 100  identified five main barriers to clusters: insufficient financial support (58%), insufficient public support (54%), incapacity to reach a critical mass of stakeholders (46%), insufficient collaboration among existing clusters (46%) and insufficient start-up incubators or business support infrastructure (45%). Other barriers were identified, with representatives of companies pointing to the limited involvement of end-users (patients, healthcare providers) and public authorities underlining the insufficient support for product development. In the area of ATMPs, the lack of innovation hubs that combine emerging ATMP specialisation with infrastructure capabilities, regulatory expertise and collaboration among clinical centres, research bodies, industrial participants and commercialisation services, obstructs development in the EU.

Third, productivity gains, acceleration of innovation and efficiency improvements associated with the use of AI and big data remain below their potential in the EU’s biotechnology ecosystem 101 . Fragmented, heterogeneous, and high-dimensional data is one of the challenges, as datasets generated in healthcare or research contexts are often not readily usable for the training, testing and validation of AI systems without significant curation and standardisation efforts. For health data, ongoing EU initiatives, such as the European Health Data Space, are expected in the coming years to facilitate access and cross-border use; at the same time ensuring that such data are sufficiently curated, annotated and interoperable to support AI development remains a key challenge. Data access challenges affect non-health data relevant to biotech, such as datasets on chemical reactions used in drug discovery and biomanufacturing processes 102 . Public consultation 103 results confirm these challenges. Companies, business associations and academic or research institutions report the highest levels of difficulty. Around two-thirds of companies and research institutions rely on data sourced from outside the EU/EEA, primarily due to higher reliability and quality of available datasets, as well as clearer legal frameworks for data access and lower perceived access costs. This suggests that improving the quality, usability and interoperability of datasets available within the EU could significantly strengthen the competitiveness of the European biotechnology ecosystem.

Fourth, the EU’s ambition to scale and industrialise biotechnology also depends on the availability of specialised talent. While the EU maintains a strong scientific base, stakeholder evidence points to persistent gaps in the workforce segments critical for scale-up and deployment.

In the EU’s biotechnology sector, stakeholders have identified gaps in the workforce segments needed for scale-up, progression to commercial scale and manufacturing 104 . There is a significant shortage in the total number of higher education and vocational education and training graduates entering the biotechnology sector 105 , with shortages particularly observed in the following competences: biomanufacturing and bioprocessing, regulatory and compliance (e.g. ATMPs and pharmacovigilance), and digital skills 106 (e.g. bioinformatics, AI-enabled processes, data governance). Furthermore, for some profiles, stakeholder consultations indicated that technical skills would need to be complemented by additional competencies 107 . This challenge can be explained by several factors 108 , such as constrained hands-on training capacity due to the scarcity and high cost of specialised facilities, the fragmentation of qualification systems across Member States, limiting skills portability and talent mobility and the lack of entrepreneurial skills in the EU (less than 50% of EU students have access to entrepreneurial education in secondary and higher education 109 ). Barriers to the upskilling of workers are also relevant.

3.2.3Driver 3: Rapid technological developments & regulatory adaptation lag

EU biotechnology companies also face barriers to manufacture, place products on the market and grow due to EU regulatory frameworks not always keeping up with rapid technological advances. This perception was confirmed in stakeholder consultations, as regulatory frameworks that insufficiently account for the innovative and rapidly developing nature of the biotechnology sector were also identified as a barrier to the expansion of EU companies 110 .

One major reason brought forward by stakeholders and some Member States is the complex, not innovation friendly and in part ill-fitting EU legislation on GMOs, in particular as regards GMMs. The EU’s legal framework for GMOs (including GMMs 111 ) originates from the 1990s and was primarily tailored to genetically modified plants. The framework as it currently stands is less suitable to handle GMMs given the biological differences compared to plants and the variety of micro-organisms and their applications 112 . In a recent scientific opinion, EFSA concluded that the potential risk of a microbial product relates to the changes introduced in the micro-organism itself regardless of the method used to introduce them, supporting a more holistic approach to the assessment of micro-organisms based on the characteristics of the final product. 113  It further concluded that for certain GMMs less assessment requirements would be sufficient to ensure safety. In this respect, considering the risks of typical GMMs and derived products and the variety of risk profiles, the current GMO regulatory procedures are considered by many stakeholders and some Member States as disproportionate, overly rigid and time consuming, rendering the EU unattractive for investing and bringing such GMMs to the market as products. In addition, the particularly short development cycles of GMMs, with one product frequently being based on a previous product, mean that a quick, efficient authorisation path is very important. 

There is also a regulatory gap between the existing EU legal framework on the quality and safety of human organs intended for transplantation and current medical practice on organ preservations, leading to diverging interpretations across Member States and hence legal uncertainty. The current EU framework was designed for preservation rather than processing and does not provide a clear, harmonised approach for these activities. It does not include a particular framework for authorising processing operations, nor a mechanism for benefit-risk assessment of these interventions, and no provisions for oversight of transplantation centres that seek to apply a specific processing technique. Furthermore, the current framework does not contain a mechanism for coordination between the organ transplant competent authority and the competent authorities operating under other legislative frameworks (e.g. on medical devices, medicinal products, or on SoHO). In 2024, although over 32,000 organ transplants were performed across the EU, the gap between the supply of transplantable organs and clinical demand remained, with over 52,000 patients registered on transplant waiting lists across the EU (as of 31 December 2024) 114 .

Similarly, in the ATMP area, outdated definitions and technological lag in the regulatory framework are leading to discrepancies between the legal framework (classification uncertainty) and progressing science, hampering market access for these products (e.g. affecting in vivo tissue generation, acellular therapies, and bio-synthetic hybrids).

The rapid technological advances characterising the biotechnology sector challenge existing regulatory frameworks. While these frameworks ensure high safety standards, they offer limited regulatory flexibility in a context of rapid technological advances. As a result, innovative biotechnology applications may struggle to progress efficiently from development to market deployment across these sectors when regulatory frameworks are not sufficiently adapted to emerging technologies. In the health area, existing regulatory frameworks are not fully adapted or adaptable to complex and hybrid emerging health biotechnology products, leading to barriers such as a lack of legal certainty on products classification and evaluation. In the VMPs sector, regulatory uncertainty also represents a serious hurdle to the marketing or use of novel technologies, methods or products. In the food chain sector, EU legislation requires pre-market authorisations for several categories of food, feed and related food chain inputs but also provide for limited regulatory flexibility.

Biotechnologies are critical for the Union's defence and security. And while rapid advances in biotechnology over the last decades have brought considerable benefits for healthcare, research and the economy, such advances also make biological agents more widely accessible, increasing risk of their misuse. For example, the price of synthetic nucleic acids (such as DNA or RNA) has decreased more than 100-fold over the past 20 years 115 , making it increasingly feasible to synthesise potentially dangerous genomic sequences, such as the DNA or RNA of viruses, at limited cost.

While some commercial nucleic acid synthesis providers screen orders to identify potentially dangerous sequences and verify the legitimacy of their customers, there are currently no EU-level rules on this. National rules diverge 116 , failing to offer a level playing field, fragmenting the single market and undermining the EU’s prevention and biosecurity efforts. 117 In stakeholder consultations, several industry actors, including SMEs, have therefore called for harmonised EU biosecurity rules.

Finally, the potential of data and AI remains largely untapped in the EU. Despite new regulatory frameworks (European Open Science Cloud, AI regulatory sandboxes mandated in the AI Act, EuroHPC/AI factories/gigafactories), some challenges remain. AI biotech platforms are inherently capital-intensive, relying on costly model training and large-scale data generation, and depend on access to high-quality data and advanced computing infrastructure, which remain comparatively limited in the EU 118 . Furthermore, the scaling of AI 119 in biotechnology requires sophisticated experimentation environments capable of integrating high-performance computing, biological laboratories and advanced data infrastructures. 120  However, the availability of such integrated environments remains limited in the EU. Stakeholders underlined the importance of collaborative environments that facilitate the responsible testing, validation and deployment of AI-enabled biotechnology solutions while ensuring compliance with EU legislation 121 . 

3.3Consequences

3.3.1EU is facing a major competitiveness gap in biotech, despite being a leader in science

The problem presented in section 3.1. results in a gap between the EU and the US in terms of entrepreneurial dynamism. The EU only represents 22% of the global biotech start-up activity (i.e. start-ups founded since 2015) while the US accounts for nearly half (49%) of them (see Annex 5, Figure 6) 122 .

This competitiveness gap is observed across the health and agri-food biotechnology sectors:

The EU is becoming less attractive to sponsors for conducting clinical trials in global comparison, affecting the economy, public health, and skill capacity building. The number of clinical trials conducted worldwide has increased substantially over the past decade, rising from approximately 13,000 in 2013 to 22,000 in 2023. Over the same period, the geographical distribution of clinical trials has shifted markedly. While the EEA’s share of global clinical trials declined from 18% in 2013 to 9% in 2023, China’s share increased from less than 10% to nearly 30% 123 . A similar trend is observed in cell and gene therapy trials: between 2013 and 2023, Europe’s global share steadily declined, whereas China experienced a dramatic rise over the same period, emerging as the leading region. 124  This growth also reflects a different portfolio structure, with China placing stronger emphasis on early-stage and post-marketing development, particularly mononational Phase I and Part IV trials, compared to the EEA’s stronger focus on multinational Phase II and Phase III trials 125 . Despite the strong growth in global clinical trial activity, the level of activity in terms of number of clinical trials submissions in the EU remained broadly stable between 2013 and 2025 126 . Consequently, the EU did not capture a proportional share of this growth, leading to a widening competitiveness gap relative to other regions with potential implications for economic performance and timely access by patients to innovative treatments 127 . 

Figure 2. Clinical trial activity in the EU 128

Note: Figure 2 describes the trend in the submission of clinical trial applications in the EU between 2013 and 2025. The bars illustrate the total number of applications per year, while the remaining lines show the share of the applications for mononational, commercial, paediatric and rare-disease trials.

The gap in clinical trial activity between the EU and other markets, particularly China, is also reflected in changes in annual pharmaceutical R&D expenditure of which approximately 60% can be on average allocated to clinical trials 129 . Using overall pharmaceutical R&D expenditure as a proxy, China experienced a significantly larger increase in R&D expenditure compared to the other regions 130 .

Figure 3. Pharmaceutical R&D expenditure in selected regions in 2010 and 2022 131 .

Note: Figure 3 illustrates annual pharmaceutical R&D expenditures in 2010 and 2022 for China, Europe, Japan and the USA.

These trends show that Europe’s position in global pharmaceutical R&D, including its clinical trial activity, is facing increasing challenges in maintaining its competitive edge 132 .

The relative decline in clinical trial activity is likely to come along with adverse effects on the public healthcare systems, the economy and the development of skills, and capacity within the Union. Although estimating the economic impact of clinical trial activity is inherently complex and dependent on multiple model assumptions, recent studies based on national and EU-wide data indicate that each euro invested in clinical trials generates between EUR 1.60 and 2.50 of added value for the economy, along positive effects on employment 133 . A recent report estimates that commercial trials, which account for 53.1% of the trials authorised in the Union 134 , create annually about EUR 35.7 billion in gross value added to the economy in the EEA and support the creation of over 165,000 jobs 135 .

In addition, clinical trial activities are estimated to reduce costs for national healthcare systems. For example, two studies conducted in Italy indicate a leverage effect of avoided costs between 2.2 and 3.5; that is, for every euro invested in clinical trials or disbursed by sponsoring companies to health facilities, the Italian national health system saved more than two euros 136 . Finally, clinical trial activities also affect the health of European citizens. For example, patients participating in clinical trials can access innovative medicines up to 5 to 10 years before commercial launch in the EU. 137  

The innovation and competitiveness gap in the EU’s ATMP sector is also becoming increasingly evident. Although Europe currently holds a 22.1% share of the global gene therapy market (2025) and the sector is projected to grow at a compound annual growth rate of 14,6% (2026–2033), the region risks falling behind North America and the Asia-Pacific region. Forecasts indicate that North America will generate the highest revenue in the sector by 2033, while the Asia-Pacific market is expected to reach USD 4,193.1 million in the same year 138 . This trend, also highlighted in the Draghi Report 139 , underscores the EU‘s struggle to strengthen its R&D capabilities and address the innovation and competitiveness gap in the region’s ATMP market. Slow progress in ATMP development threatens the EU's position in the global healthcare and biotechnology sectors, as innovators may increasingly choose to operate in jurisdictions with more agile regulatory frameworks.

When it comes to biosimilars, the EU’s biomanufacturing capacity can be further utilised. The EU has significant untapped potential in biosimilar manufacturing. The EU retains the largest cumulative authorisations of biosimilars (151 products vs. 72 in the US and 66 in Canada) and leads the global market in revenue valued at approximately EUR 25.6 billion in 2024, representing around 6% of the total pharmaceutical industry's contribution to GDP. Sales grew at 15% annually between 2020 and 2024 across five core therapeutic areas, and the sector supports around 81,000 jobs in Europe: 16,000 direct (20%), with the remainder split between indirect roles such as clinical research (30%) and induced employment (50%). The sector depends on a highly skilled workforce, with 40% of direct employees (~7,000) in technical roles and 70% holding university degrees. 140  However, manufacturing activity is increasingly migrating to Asia, particularly South Korea and India 141 . While the EU remains a key innovation hub — supplying over 75% of active ingredients for innovative biologics — it must develop more dedicated and cost-efficient manufacturing infrastructure to keep pace with international competitors. 142  

Similarly, challenges to bring novel health biotechnology products to the market have been observed 143 , and are generally expected to increase given the increasing complexity and hybrid nature of novel products. These challenges further weaken the EU’s position in the global innovation race, delaying the availability of these products to patients across the EU.

Regulatory uncertainty also has practical implications for cross-border exchange of processed organs and for the scaling of machine perfusion technologies, contributing to uneven uptake and fragmentation within the EU 144 , and eventually reducing the potential for optimal matching of organs and recipients. Over the last three years, the cross-border exchange rate of allocated organs fluctuated between 20-23% 145 .

In the field of GMMs, recent evidence shows that there is significant activity in the development of innovative GMMs by both private and public/academic entities. However, the majority of the identified GMMs originates from the US or China, while only a limited number were developed within the EU 146 . Notably, there are currently no products on the single market that contain or consist of GMMs, except for medicinal products which are, however, not authorised under the GMO legal framework but under the EU framework on pharmaceuticals. This is particularly affecting the competitiveness of SMEs in the GMM field, since they are typically among the most innovative companies in the biotechnology sector but may lack the regulatory expertise and financial resources to navigate complex legal frameworks that may not adequately fit their innovative products. At the same time, this lack of products on the markets, or prospect to bring products efficiently to the market, is discouraging investments in R&D, thus negatively impacting the wider science community in the EU. EU economic sectors, foremost the agricultural, industrial and environmental sectors, are negatively impacted as they cannot easily make use of novel tools available in third countries that make operations more efficient.

As a consequence of the issues outlined under drivers 1 and 3, a competitiveness gap is also observable for biotechnology products in the food chain (food and feed products), including substances (e.g. additives, enzymes etc), and other food chain inputs (e.g. food contact materials).

Finally, as AI continues to expand across the biotechnology lifecycle in the health and food sectors, the bottlenecks innovators face when attempting to develop, validate and deploy AI-enabled biotechnology solutions in the EU slow the transition from scientific discovery to real-world deployment. As a result, the Union risks failing to fully capture the productivity gains, efficiency improvements and innovation opportunities associated with the large-scale use of AI in biotechnology. This under-utilisation of AI may therefore translate into a growing competitiveness gap, as other jurisdictions more effectively integrate AI into biotechnology research, development and production across multiple biotech sectors.

3.3.2Biotech’s potential to help address major societal challenges is underexploited 

Despite its innovation and growth potential, and expected benefits for patients and society at large, the potential of biotechnology remains largely underexploited in the EU, across the different biotechnology areas.

In the health sector, ATMPs – such as gene therapies – offer a transformative potential for treating a broad spectrum of diseases, from rare genetic disorders to common chronic conditions. Despite this promise and the innovative potential in the EU, patient access in the EU remains severely limited.

Organ transplantation also increasingly involves ex-vivo organ processing, such as machine perfusion to assess and recondition donor organs prior to transplantation. These techniques can expand the pool of transplantable organs, improve matching of organs and recipients, improve clinical outcomes and strengthen health-system resilience. However, there is limited cross-border exchange and slow EU-wide uptake of innovative processing technologies 147 .

In the VMP sector, the potential of biotechnology to improve animal health and strengthen food supply chain resilience remains underdeveloped. Favourable regulatory conditions for the use of biotechnology in animal health will not only benefit the biotech pharmaceutical industry, but also farmers, animal health, the food supply chain and human health (zoonotic diseases). When disease spreads in food-producing animals, the impact on farmers can be dire and food security can be put at risk. Vaccination is a powerful tool to prevent and control animal diseases and contributes to reducing the use of antimicrobials. As a large share of emerging infectious diseases in humans has a zoonotic origin, preventing animal diseases also has positive effects on human health. Novel biotechnology tools enable the development of safer and more effective vaccines, as well as the distinction of vaccinated animals from those infected, and allow vaccines to be developed or adapted much faster than with conventional approaches, which is critical to prevent and control animal diseases.

When it comes to innovative GMMs 148 , their non-deployment in the EU market, in addition to the loss of competitiveness opportunities outlined under the driver 3, result in an unfulfilled potential for GMMs to contribute to other social and environmental objectives. For example, GMMs can now be used as or in biofertilisers, biostimulants, biopesticides, biocides, bioremediation, wastewater treatment, biomining and bioleaching, offering benefits in the wider agri-food, industrial, marine and environmental sectors. As GMM-based products that would be more efficient or environmentally friendly than the alternatives are not being used, EU society and consumers are negatively impacted as they are being deprived of innovative solutions to important challenges linked to climate change, environmental pollution and food and feed security.

Moreover, in the food and feed sector, the potential of biotechnology remains underexploited and experimentation in real conditions (e.g. real-world food production and processing environments) is constrained. Biotechnology tools can support more efficient production processes across the food chain. These technologies can improve the safety and quality of food products, for example by reducing pathogens and toxins and enhancing nutritional properties 149 . Enzymes and biocatalysts can reduce environmental impacts by replacing chemical processes and improving process efficiency 150 . As regulatory drivers constrain the deployment of innovations in such areas, the single market as well as the contribution of biotechnology to food safety and security, resource efficiency, environmental sustainability and the EU’s strategic autonomy, are not fully realised.

Furthermore, the rapid advancement of technology, including AI, alongside increased data collection and use are cross-cutting enablers with huge potential across the different areas of the biotech sector, yet to be fully utilised. AI and advanced data use act as key enablers of biotechnology innovation across health and agri-food applications. AI-driven tools support target identification, molecular design, optimisation of biological processes, CT design and manufacturing efficiency, while in the agri-food sector they enable advanced fermentation, strain optimisation and resource-efficient production. These applications can accelerate innovation cycles, reduce development costs and strengthen resilience in areas such as medical countermeasures, disease detection and sustainable food systems. However, the full potential of AI-enabled biotechnology remains underexploited in the EU. The effective deployment of these technologies requires clear lifecycle guidance, as well as access to integrated environments combining experimental and computational capabilities for testing and validation, and high-quality, well-curated datasets that allow AI systems to be trained and deployed reliably. The absence of these enabling conditions limits the EU’s ability to fully harness biotechnology to address major societal challenges and strengthen its strategic autonomy in health and food systems.

Regarding the application of biotechnology to defence and security, the primary challenges identified by stakeholders in the public consultation were the risks to strategic autonomy in biomanufacturing and availability of countermeasures (50%), cybersecurity risks (e.g. biotechnology infrastructure, AI tools used) (45%), vulnerabilities in the resilience of biotechnology supply chains (44%), and threats related to biosecurity and biosafety including misuse of biotechnology (40%). Finally, opportunities in biosecurity were also particularly underlined in the public consultation 151 : the development of new innovative medical countermeasures (45%), detection of biological threats (45%), as well as increased food security (43%).

3.3.3Potential for misuse of biotechnology poses growing biosecurity risks

In addition, the rapid progress in and increasing accessibility of biotech innovations also increase the likelihood and impact of biosecurity incidents, as certain applications of technologies and products may have the potential for misuse, posing threats to public health and safety.

The exponential decrease in the cost of synthesising DNA and RNA has made the physical material needed to create biological agents, including dangerous pathogens, increasingly accessible. Studies by RAND Europe 152 and CLTR 153 , two independent non-profits, estimate the current annual probability of a large-scale biological attack using synthetic nucleic acid at 1%, and accidents from their use at 1.5%, with expected economic harm to the EU of almost EUR 20 trillion for a large-scale event.

The EU joint biotechnology risk assessment carried out under the EU’s Economic Security Strategy argues that the likelihood and impact of such incidents is further amplified by advancements in AI and computational tools in biological applications, which can lower the technical barriers to the design, optimisation and misuse application of biological agents.

These advances could significantly expand the pool of individuals capable of conducting technical tasks with biotechnology products that could be misused for malicious purposes, such as synthesising dangerous pathogens. Modelling published in 2025 suggests that, under a set of assumptions about near-term AI capabilities, could increase the annual probability of an epidemic from biological misuse by a non-state actor roughly sixfold, from 0.15% to 1% 154 . This reflects broader concerns that AI tools developed for beneficial applications in life sciences may also present dual-use risks if misapplied. 155

3.4What is the baseline from which measures are assessed? 

The baseline trajectory through 2038 156 is expected to show a widening of the competitiveness gap between the EU and its main competitors. This is driven by the compounding interaction of the identified drivers. Regulatory complexity deters investment; market fragmentation, in particular capital scarcity, constrained innovation; and the expanding pipeline of frontier technologies amplifies administrative burdens and regulatory barriers, within an ecosystem that is not positioned to absorb them. The thematic sections that follow detail these dynamics across five interconnected dimensions 157 . 

Regulatory landscape and administrative burdens

Under the baseline, biotechnology developers will be confronted with an EU regulatory landscape characterised by three mutually reinforcing deficiencies: the proliferation of overlapping and partially incompatible regulatory frameworks, the persistent national heterogeneity in the implementation of procedures, and the absence of structured pathways for innovations that do not fit neatly within existing legislative frameworks.

The most pervasive source of regulatory burden is procedural duplication across intersecting frameworks. Under the baseline, developers of GMO-containing ATMPs will continue to incur in up to 50 additional days in CT authorisation timelines beyond conventional medicines, with further extensions for substantial modifications. For VMPs, GMO-containing products will continue to face approval timelines two to three times longer than non-GMO equivalents despite the fact that no GMO-containing VMP has ever received a negative opinion on the basis of its GMO component, triggering an 'innovation tax', estimated at EUR 269,000-840,000 per year in FTE burden. Such a burden is set to compound, reaching EUR 403,000-1,119,000 by 2040, as the pipeline is projected to encompass approximately 100 GMO-containing VMPs in various stages of development at any point through 2040. In addition, CT applications subject to dual-track requirements will increase from 8-15 to 12-20 per year and annual GMO-containing marketing authorisation applications are expected to rise from the current 3-5 to approximately 5-8 per year by 2040. Meanwhile, the administrative burden from variations not requiring assessment (VNRA) is projected to scale proportionally with the expanding marketing authorisation base: the number of active veterinary Marketing Authorisations is expected to grow to approximately 55,000-60,000 by 2040, with annual VNRA volumes scaling accordingly. The aggregate annual VNRA fee burden is projected to grow in proportion.

In the biosimilar domain, the disproportionality of certain regulatory requirements will also persist. Between 2012 and 2022, 100% of all 36 monoclonal antibody and fusion protein marketing authorisation applications included a Phase III comparative efficacy study, yet in no case did the clinical data determine the regulatory decision. Thus, the most costly component of biosimilar development (EUR 19-26 million per study, representing 20-50% of total expenditure and adding 12-24 months to timelines), is likely to function as a de facto procedural requirement rather than a scientifically decisive evidentiary input. Indeed, there is increasing experience with tailored clinical efficacy and safety approaches for biosimilars at the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA). Based on EMA data, in 2025 more than 70% of adopted scientific advice procedures for biosimilars incorporated a tailored clinical efficacy and safety approach. In addition, among all biosimilar marketing authorisation applications submitted last year under Article 10(4) of Directive 2001/83/EC, approximately 8% proposed a tailored clinical efficacy and safety approach. While there is a gradual shift toward more tailored approaches by EMA 158 , a slow uptake of these changes risks putting Europe at a competitive disadvantage compared with other regions that operate under more agile frameworks. 

The EU’s regulatory framework will be marked by persistent fragmentation in the implementation of clinical trials across Member States. The EU’s share of global clinical trial initiations has halved, declining from 18% in 2013 to 9% in 2023. Without fundamental reforms, particularly in streamlining authorisation procedures and simplifying regulatory requirements, the EU risks a further decline in its clinical research competitiveness. Meanwhile, the more centralised markets of the US and China continue to outperform the EU, further consolidating their dominance. China’s share of global clinical trials, for instance, has surged from 8% to 29% over the last decade, while the US maintains its strong position. Current projections suggest that the EU’s growth in clinical trial activity will remain sluggish compared to these faster-expanding regions, risking a further relative decline in its standing as a key hub for clinical research.

However, this does not imply a decline in the absolute number of EEA trials as the Clinical Trial Regulation (fully applicable to all clinical trials since January 2025) is expected to generate a ‘learning effect’ as sponsors and national authorities gain experience with the Clinical Trials Information System (CTIS). This will lead to moderate improvements in median authorisation times and greater procedural stability over 2025-2030. Between 2025 and 2030, AI tools are projected to achieve 60-70% routine adoption, e.g. in trial design and conduct, and digital trial components are expected to become increasingly integrated into standard protocol design. However, national heterogeneity in ethics review, contracting, insurance, and site activation will persist. Clinical trial activity concentrates further in a few Member States, large firms widen their advantage over SMEs, and clinical translation of the EU's strong scientific output stagnates. SMEs and academic innovators increasingly rely on partnerships with larger companies, slowing EU-based evidence generation and eroding Europe's leadership in emerging technologies. In absolute terms, the number of EU clinical trials has remained broadly stable over the past decade at approximately ~2,400–2,500 trials per year, with no structural upward trend expected under the baseline 159 .

For SoHO, 50 national competent authorities will continue to oversee blood and tissue establishments (currently at 1,400 and 3,258, respectively), with recurring administrative costs to businesses estimated at EUR 4.14 million over ten years under the incoming risk-based authorisation model. While Regulation (EU) 2024/1938 introduces a more unified SoHO Preparation Authorisation (SPA) model, regulatory uncertainty will persist for novel, complex, and cross-framework SoHO innovations. Meanwhile, permitting for strategic health biotechnology projects is projected to remain fragmented and slow under the lack of EU-level priority-project recognition or fast-track governance mechanisms.

These burdens are further aggravated by the absence of structured innovation support. In the food and feed domain, over 90% of application dossiers assessed by EFSA in 2021-2023 contained administrative or scientific errors leading to the suspension of the legal deadlines for risk assessment (the so-called stop-the-clock procedure) resulting in delays in the overall authorisation process, which are likely to persist under the baseline scenario. For AI-enabled methodologies, EMA's Qualification Procedure for Novel Methodologies costs EUR 89,000 per request, takes 160-250 days, and produces confidential, non-reusable outcomes, meaning each new entrant faces identical navigation costs with no cumulative learning. The scale of regulatory demand on the EMA is itself accelerating. It received 635 scientific advice requests in 2024, a figure that grew 16% year-on-year in the first half of 2024 alone, this mismatch between the pace of technological innovation and the capacity of existing regulatory infrastructure to absorb it is expected to grow under the dynamic baseline.

Competitiveness and investment attractiveness

The capital base underpinning the biotech sector shows a pronounced investment gap. As presented under section 3.2.2, EU biotech venture capital stood at approximately EUR 4.8 billion across 243 rounds in 2024, roughly six times below US levels. The average EU late-stage financing round (approximately USD 49 million) is around half of the US equivalent, and the EIB estimates the structural annual investment gap at approximately EUR 40 billion. This capital shortfall is not merely quantitative; it is structurally embedded (see sections 3.1 and 3.2.2). Despite the existence of significant public financial engagement, this gap shows no prospect of autonomous closure under the baseline. The deep dependence on external capital, which weakens strategic autonomy over the direction and location of value creation, is expected to grow. The median time gap between funding rounds has increased from 17 to 20 months, with higher incidence of down rounds at Series C stage (growth stage), signalling that investor confidence in the EU’s late-stage pipeline remains fragile. Without sufficient public investment to bridge the late-stage capital gap and catalyse private co-investment, the structural investment deficit is projected to persist through 2038.

The exit asymmetry is equally stark and is expected to continue. Only 138 EU biotech healthcare companies completed an IPO compared to 451 in the US (over the period 2013-2023). In 2024, no European biotech raised more than EUR 58.1 million in IPO proceeds.

The commercial viability of biotechnology investments is further conditioned by the existing IP protection framework. Health biotech innovators reinvest a large portion of their revenue back into R&D. By ensuring a viable commercial timeframe, the SPC is one of the measures that enables innovative companies to re-invest their revenue in additional research and development, improving patient access to innovative therapies, and generating broader economic benefits. However, intellectual property, including SPC, is just one part of innovators’ investment decisions. Without policy change, innovators’ decisions on investment location continue to reflect expected lifecycle revenues under protection, alongside market size, access conditions, regulatory predictability and operational considerations. In the immediate baseline period to 2030, effective protection durations are expected to remain broadly stable, with the revised general pharmaceutical legislation not expected to produce observable effects within this period. In the longer term to 2038, effective regulatory protection outcomes are expected to become more heterogeneous across medical products, reflecting the continued shift towards biologics and advanced therapies with longer and more variable development timelines. The EU share of earliest biotech patent filings is projected to remain stable at low single-digit levels (around 3-5%), while global biotech patenting activity continues to expand.

This capital deficit compounds a broader erosion of the EU's global competitive position across several dimensions that collectively determine where biotechnology innovation is located, financed, and commercialised. The global clinical trials market is expected to grow at approximately 5.7% annually, compared with approximately 4.8% in Europe, indicating a gradual but persistent loss of relative competitiveness for the EU as a location for clinical research in the absence of improvements to the clinical trial ecosystem. In biosimilars, the EU’s first-mover advantage has declined. While 71% of biosimilars are approved in the EU before the US, there has been a rising share of biosimilars that get approved in the US first, or only received US approval, which indicates the growth of the US market. At the same time, the European biosimilar medicines sector makes a significant contribution to European gross domestic product, contributing EUR 25.6 billion in 2024 and representing approximately 6% of the total pharmaceutical industry contribution. 160  The number of the CHMP of EMA scientific opinions on biosimilars has itself increased markedly, from 8 in 2023 to 25 in 2024 and 40 in 2025, reflecting a growing pipeline that amplifies the stakes of regulatory competitiveness. Cumulative biosimilar authorisations are also projected to rise from 151 in 2025 to 250-350 by 2030 and 450-650 by 2038, with new reference product classes entering biosimilar development expanding from approximately 27 to 35-45 by 2030 and 55-75 by 2038. However, 79% of the approximately 100 biologics losing exclusivity by 2032 have no biosimilars in development, representing a ‘biosimilar void’ valued at approximately EUR 122 billion in global forecast sales at loss of exclusivity. This indicates that a substantial proportion of the projected market value will not materialise without a reduction in per-product development costs. 

The competitive erosion is reinforced by the fact that no single-entry point exists for health biotechnology innovators to navigate the regulatory and funding landscape. Investor-innovator connections will likely remain ad hoc and geographically concentrated in a few leading ecosystems. Disparities in innovation capacity and ecosystem maturity across Member States are expected to persist, with Central and Eastern European Member States lacking the cluster foundations that sustain leading ecosystems in some Member States. These fragmentation effects are compounding: without a coordinated ecosystem support mechanism, the navigational burden for SMEs is expected to increase further as new regulatory frameworks layer additional complexity and the pipeline of frontier biotechnologies expands. Meanwhile, other major jurisdictions are actively accelerating policy and investment efforts to strengthen their biotechnology ecosystems 161 , suggesting that, in the absence of intervention, the EU’s relative competitive position is likely to continue to deteriorate over time.

Innovation and research

The baseline reveals a structural and persistent translation deficit at the centre of the EU biotechnology innovation system. Under the baseline, the EU innovation system’s upstream scientific base will remain internationally competitive, while the downstream deficiency in infrastructure for converting that science into products, trials, and market-ready applications will grow.

One of the key innovation-side deficits is the absence of structured pathways for technologies that do not fit neatly within existing regulatory categories. Developers of novel health biotechnology products will continue to operate simultaneously across two to three regulatory frameworks, with no structured cross-framework anticipatory dialogue in place. Forecast analyses project that up to 30% of such products could be stalled by regulatory uncertainty between 2030 and 2038, even as the volume of novel, hybrid health technologies is expected to increase by approximately 20% between 2025 and 2030. In ATMPs, as the current definitions fail to capture emerging modalities, this is projected to widen progressively as in vivo genome editing and cell-device combinations advance over 2030-2038. Without clarification of tissue-engineered product and related definitions, more products are likely to require case-by-case classification by EMA and bespoke scientific advice from 2030 onwards, increasing regulatory workload and amplifying unpredictability for the SME dominated ATMP developer base (over 60% of ATMP developers are SMEs). For GMMs used outside food and feed, the EU's process-based framework has resulted in no market authorisations for deliberate release under Directive 2001/18/EC, effectively excluding the EU from the specific parts of global markets in biofertilisation, biocontrol, bioremediation, and bioleaching using GMMs. Taking into account GM and conventional products, these markets have a combined value exceeding EUR 29 billion and growth rates of 9-14% annually. These global GMM-relevant markets are projected to grow significantly under the baseline: biofertilisers from EUR 1.18 billion (2024) to EUR 2.42 billion by 2030; microbial biocontrol from EUR 5.55 billion (2025) to EUR 14.2 billion by 2032; bioremediation from EUR 14.1 billion (2024) to EUR 32.9 billion by 2033; and bioleaching from EUR 8.66 billion (2024) to EUR 18.2 billion by 2033. Under the baseline scenario, the EU's partial exclusion from these markets persists indefinitely, and European companies developing GMM products will increasingly serve non-EU markets or relocate R&D and commercialisation activities to jurisdictions such as the US, where a product-based regulatory approach has already enabled commercial deployment. Similarly, under the baseline, organ processing practices would be driven by local institutional arrangements rather than a coherent EU standard. Under the baseline, the share of EU transplantation programmes with active processing capability is projected to rise from an estimated 27% to approximately 35-38% by 2030 and 42-48% by 2035, before plateauing at approximately 48-55% by 2040. This plateau reflects the absence of an EU-level framework.

Regulatory sandboxes, which could provide structured testing environments for such innovations, are either absent or fragmented and underused across the relevant domains. No EU-level sandbox exists for veterinary innovation. Similarly, the EU food acquis does not provide for the operation of regulatory sandboxes; any existing food and feed sandboxes remain weakly connected to food law and unevenly available across Member States. The incoming SoHO risk-based authorisation model does not incorporate a sandbox mechanism under the baseline. In the SoHO domain specifically, expert estimates indicate that sandbox-eligible cases represent 5-10% of overall SoHO activities, translating to 90-250 eligible cases per annum; based on comparable sandbox acceptance rates observed in other regulatory domains (7-16%), between 6 and 40 sandboxes could be established per annum, illustrating both the latent demand for structured innovation pathways and the scale of innovation potential that remains unexploited under the current baseline.

The innovation investment consequences of these constraints are illustrated in the biosimilars domain. Under the current framework, the sector’s contribution to European CTs for biosimilar medicine candidates amounts to EUR 4.9 billion. Under the baseline, the near-term biosimilar MAA volume is projected at 25-45 per year, with the lower bound reflecting persistence of the biosimilar void and the upper bound assuming gradual spontaneous adaptation; the medium-term range rises to 35-55 per year. However, without formalised regulatory streamlining, per-product development costs remain at EUR 90-280 million over 6-9 years, with the CES representing the single largest component (EUR 19-26 million per product, approximately 57% of total clinical development costs being clinical), constraining the commercial viability of biosimilar development for lower-value reference biologics

Public health and safety

Under the baseline, while the EU maintains among the highest regulatory safety standards globally, aspects of the current regulatory architecture - particularly complexity, fragmentation and procedural duplication - can constrain the pace at which patients, consumers, and citizens benefit from biotechnological innovation. This tension will continue to manifest across the following interconnected dimensions:

First, delayed and uneven patient access to biotechnology-derived treatments will continue. The most consequential public health gap in the baseline concerns the widening distance between the availability of advanced biotechnology therapies and the speed at which EU patients can access them. The EU’s share of global clinical trial initiations has halved from 18% in 2013 to 9% in 2023, with trial authorisation taking 100-120 days compared to approximately 30-60 days in the United States and China. For ATMPs, the current provisions of the CTR will continue to add up to 50 additional assessment days. In addition, foresight analyses projecting that up to 30% of novel health biotechnology products could be stalled by 2030-2038.

· Under the baseline, annual healthcare system savings from biosimilars are projected to rise from approximately EUR 13 billion in 2024 to EUR 16-22 billion per year by 2030 and EUR 22-35 billion per year by 2035-2038, but the upper bounds of these projections are constrained by the biosimilar void, meaning that the full savings potential depends on whether per-product development costs can be reduced sufficiently to make lower-value biologics commercially viable targets for biosimilar competition.

·In organ transplantation, the absence of a harmonised EU-level authorisation framework for organ processing means that advanced ex-vivo machine perfusion techniques diffuse unevenly, with no systematic mechanism for capturing processing-related safety data. Under the baseline, with a transplant growth trajectory of 1.54% CAGR annual transplant volumes are projected to rise to approximately 35,350 by 2030, 38,200 by 2035, and 41,200 by 2040. However, demand-side growth will outpace supply-side gains: waiting lists are expected to grow at approximately 0.5% per year to approximately 55,000-56,500 active patients by 2035 and 57,000-58,000 by 2040, meaning waiting-list mortality is projected to decline only marginally from approximately 3,366 deaths per year to approximately 3,100-3,200 by 2035 and 2,900-3,100 by 2040. The EU's dialysis-dependent population is projected to rise from approximately 310,000 to 330,000-340,000 by 2035 and 350,000-365,000 by 2040, with demand-side growth driven by population ageing, rising diabetes and hypertension prevalence, and widening dialysis initiation criteria outpacing the supply-side gains from transplant volume increases. The organ discard rate is projected to decline from approximately 12-13% to 10–11% by 2035 and 9-10.5% by 2040 under organic, centre-led adoption, but these gains will be concentrated in liver and lung organs in a small number of advanced Member States and will remain below the levels achievable with wider processing adoption.

Second, in the food and feed area, although safety standards will be maintained, innovation will remain constrained. EFSA's rigorous risk assessment framework continues to underpin world-leading food safety standards. In the area of nutrition, biotechnology can enhance nutritional properties. However, the current legislation faces limitations in addressing the scientific aspects given the limited mandate of EFSA to deliver scientific advice in relation to nutrition matters. This may become even more relevant in the future considering the expected increase in the prevalence of diet-related health issues in the coming years. Similarly, regarding the One Health risks from veterinary regulatory design, the dual-track system for GMO-containing VMPs generates an 'innovation tax' of 2-3 times longer approval periods without commensurate safety benefit: no GMO-containing VMP has ever received a negative opinion on its GMO component. The absence of a legal 'firewall' clarifying that animals treated with GMO-derived VMPs are not themselves classified as GMOs will continue to create commercial risk. It could deter farmer uptake of next-generation biotech vaccines, sustaining reliance on antimicrobials and impeding progress towards the EU's target of halving antimicrobial sales by 2030.

Third, the baseline presents a structural gap in the EU's capacity to detect and prevent biological threats. Wastewater-based surveillance beyond SARS-CoV-2 and influenza remains limited (only 11 Member States monitor antimicrobial resistance, six monitor emerging pathogens), with no agnostic detection layer for novel or engineered threats. Concurrently, synthetic nucleic acid screening remains voluntary and fragmented: only 69 of over 700 global custom synthesis providers are confirmed to screen. The RAND analysis estimates that the expected monetised annual loss due to biological threats across all event categories amounts to approximately EUR 184 billion for large-scale events (1% annual probability; EUR 19.6 trillion expected harm), supplemented by small-scale events (22% annual probability, EUR 72 million expected harm), agricultural events (4% probability, EUR 75.5 billion expected harm), and non-transmissible events (5–25% probability, EUR 121 million expected harm). For attacks specifically, the probability that they involve synthetic nucleic acid ordered from providers ranges from 30% to 70% depending on event type, while for accidents the corresponding probability ranges from 6% to 17%.

The biosecurity and biodefence baselines expose a separate category of public health vulnerability. In the domain of synthetic nucleic acid synthesis, the EU market (estimated at approximately EUR 850 million in 2024, representing approximately 0.2% of the European biotechnology market 162 ) is expected to grow at an annual rate of 10-20%, but competitiveness is shaped by the absence of a level playing field: companies that voluntarily screen their customers face competitive disadvantages against providers that do not screen, and as DNA synthesis prices continue to decline, the cost of screening represents an increasing share of the final order price. Without a change in biosecurity policy, the annual probability of a large-scale biological attack or accident is estimated at 1%, with expected economic harm to the EU of a large-scale event of ca. EUR 20 trillion. The risk is set to increase further as synthetic DNA and RNA continues to become cheaper and more widely available. Meanwhile, pathogen surveillance remains reliant on clinical surveillance networks and targeted wastewater-based monitoring for known agents, with no systematic agnostic detection capacity for novel or engineered biological threats. 

Environment and sustainability

The baseline reveals a tension at the intersection of the EU's biotechnology regulatory architecture and its environmental policy ambitions: while these frameworks uphold high environmental and safety standards, certain features – such elements of their process design and limited risk differentiation - can delay or constrain the deployment of biotechnology applications with significant potential to advance the environmental and sustainability objectives. This tension manifests most acutely across three intervention areas.

The most consequential environmental baseline deficit concerns GMMs. No GMM has been authorised for deliberate environmental release under Directive 2001/18/EC. As innovations utilising genetic engineering are not brought to the market in the EU, the EU is therefore entirely absent from this important segment of rapidly growing global markets in biofertilisation (EUR 1.18 billion in 2024, growing at 12.8% CAGR), microbial biocontrol (EUR 5.55 billion in 2025, 14.3% CAGR), bioremediation (EUR 14 billion in 2024, 9.9% CAGR), and bioleaching (EUR 8.65 billion in 2024, 8.9% CAGR), sectors with a combined global market exceeding EUR 29 billion and projected growth rates of 9-14% annually. In contrast, a product-based regulatory approach in the US has already enabled commercial deployment. Under the baseline scenario, this partial market exclusion persists indefinitely, and European companies developing GMM products will increasingly serve non-EU markets or relocate, while the environmental benefits of GMM-based microbial solutions for resource-efficient agriculture, pollution remediation, and sustainable extraction remain unrealised within the EU.

In the veterinary domain, the dual-track regulatory system for GMO-containing VMPs results in a regulatory friction that can slow the uptake of next-generation biotech vaccines in food-producing species, with potential ramifications for animal health, farmers and food security.

The food chain sandbox landscape is characterised by fragmentation and lack of coherence with Union law; only a few Member States operate sandbox-type mechanisms, none anchored in a harmonised EU framework under the General Food Law. As a result, findings and best practices cannot be systematically integrated into the evolution of the existing legal framework. This limits structured, pre-market testing of sustainable food production technologies, leaving innovation potential for the food system's environmental transition partly unexploited.

4Approach to the Biotech Act

4.1Objectives of the proposal

4.1.1General objectives

The general objective of the proposed Regulation and Directive is threefold: (i) to improve the functioning of the internal market by establishing a framework to strengthen the competitiveness of the health biotechnology sector, from research to production, (ii) to create the conditions for the development and timely placing on the single market, of biotechnology innovations, products and services, (iii) while safeguarding high standards for the protection of human health, animal health, patients and consumers, food and feed safety, the environment, and biosecurity.

4.1.2Specific objectives

This general objective translates into the following specific objectives, as presented in the proposed Regulation and Directive: (i) strengthen the biotechnology sector and reinforce the EU’s research, development and production capabilities; (ii) support funding of, investments in, and access to capital for, biotechnology companies and projects; (iii) improve the EU manufacturing capacity of, and expertise in biosimilars, including through international cooperation; (iv) facilitate the application of AI into the EU’s biotechnology and health technology manufacturing ecosystems and frameworks, in line with the AI Act 163 ; (v) ensure a legislative framework that encourages innovation and takes account of technological and scientific developments and progress; (vi) prevent the misuse of biotechnologies and strengthen biodefence capabilities and (vii) enable the effectiveness of the above specific objectives through a legislative framework conducive to the use of biotechnology innovations.

4.2Choice of the legal instruments and legal basis

4.2.1Choice of the legal instruments

A Regulation is the most suitable for measures requiring uniform application across the EU, such as those concerning recognition and support for heath biotechnology strategic projects and high impact health biotechnology strategic projects, and measures to boost EU biodefence and biosecurity and prevent biotechnology misuse. The Regulation is also justified to amend existing EU regulations in the area of health and food law. Its directly applicable nature ensures consistent implementation without national transposition. A Directive is the appropriate instrument for amendments to Directive 2001/18/EC and Directive 2010/53/EU.

4.2.2Legal basis

The appropriate legal bases are therefore as follows:

-Article 114 TFEU is the basis for the adoption of measures that increase harmonisation and remove fragmentation and, therefore, seek to create a level playing field within, and fully exploit the scale of the single market for biotechnology products and biomanufacturing. The proposal also seeks to achieve the objective of a high level of health and safety protection as per Article 114(3).

–Article 168(4) TFEU is the basis for the adoption of certain measures that contribute to achieve a high level of human health protection, i.e. standards of quality and safety of organs and SoHO, blood and blood derivatives; measures in the veterinary and phytosanitary fields, and standards of quality and safety for medicinal products and devices for medical use.

-Article 173(3) TFEU is the basis for measures in support of Member States’ action for the competitiveness of the EU’s industry, in particular the provisions regarding the EU Health Biotechnology Investment Pilot.

4.3Subsidiarity: necessity for EU action and EU added value 

Under the subsidiarity principle, the Union may act in areas of non-exclusive competence only where the objectives of the proposed action cannot be sufficiently achieved by the Member States.

4.3.1Necessity for EU action

The objectives of the proposed Biotech Act cannot be achieved by Member States acting alone, as the identified drivers are cross-border and systemic, affecting the functioning of the single market and the global competitiveness of EU companies. These challenges may differ in intensity across Member States and regions, but their nature is Union-wide. Access to finance is fragmented and EU biotechnology companies lack the capacity to access private finance at a competitive scale, including at later stages of development. European biotechnology clusters are dispersed and lack continental scale to compete globally. The development and deployment of AI in biotechnology remains limited, also due to the insufficient cross-border availability and sharing of relevant data.

While several Member States have taken action, these bottlenecks persist and improvements would remain slow and uneven. National measures alone cannot provide the scale, coordination and speed required, although in certain areas (e.g. access to finance, development of regional innovation ecosystems and clusters, and support to research infrastructures, etc.), Member States may still take complementary action alongside Union measures.

Important regulatory barriers stem from existing EU legislation, such as lack of legal clarity, complex or outdated rules, disproportionate administrative burden and additional national requirements. As these drivers originate in Union law, they cannot be effectively addressed by Member States acting individually. Union action is therefore necessary to facilitate innovation, enhance legal clarity and improve market access.

4.3.2EU added value

The proposed actions focus on areas where there is a demonstrable value added in acting at EU-level due to the scale, speed and scope of the efforts needed.

A harmonised but simplified EU regulatory framework, supported by strengthened collaboration in selected policy areas (access to capital, AI and data) is expected to ensure patients, users and citizens across the EU can benefit from biotechnology innovations.

A large market with a streamlined, fit-for-purpose regulatory framework ensures a level playing field and reduces compliance costs in the single market, thereby supporting biotechnology market uptake. Coordinated EU action in the field of biotechnologies will generate economies of scale, reduce duplication of efforts, increase legal certainty for cross border entrepreneurs, and unlock investments, infrastructures and skills development that Member States alone could not achieve. Union-level coordinated action is expected to yield higher benefits than fragmented national measures.

Regulatory streamlining and clarification measures will reduce administrative burden, accelerate time-to-market and improve the functioning of the internal market, also by addressing the observed diverging national interpretations and additional requirements. The proposed Biotech Act also seeks to reinforce the EU’s strategic autonomy in a critical technological area while fostering adequate biosecurity safeguards and biodefence capabilities.

4.4Intervention logic of the proposal 

Figure 4. 164  

Figure 5 illustrates the overall architecture of the Biotech Act, highlighting the two complementary pillars of regulatory streamlining and facilitation to accelerate time-to-market and industrial enablers to strengthen the EU biotechnology ecosystem.

Figure 5. Biotech Act architecture

5Description of the proposed measures and analysis of their main impacts

The measures described below are outlined in the proposal for a Regulation and the proposal for a Directive 165 . They are presented in 17 packages of interventions, which fall under two categories: eight interventions related to regulatory streamlining and facilitation measures aiming at regulatory simplification (addressing drivers 1 and 3), and nine interventions linked to industrial enablers (addressing drivers 2 and 3). They are presented in more detail in Annex 7.

The summary assessment of measures 166  below includes tables capturing the key impacts across standard impact categories 167 . Where sufficiently robust evidence is available, key impacts are quantified and expressed using numerical estimates. Where quantification is not feasible, impacts are assessed qualitatively using a standardised rating scale 168 , indicating the direction and relative magnitude of effects. For certain measures, tables are not used where the nature of the impacts or the available metrics are better suited to alternative forms of presentation (e.g. narrative analysis or visual representations), to ensure a more accurate and meaningful representation of the expected effects.

5.1Interventions for regulatory simplification: measures and expected impacts

Chapter IX of the proposed Regulation and Directive include amendments to EU legislative frameworks in the areas of health, food and feed safety as well as GMMs, which aim to simplify existing procedures and accelerate time-to-market. These amendments are needed to ensure the effectiveness of the self-standing measures on industrial enablers laid down in the proposed Regulation.

5.1.1Intervention n°1: Regulatory facilitation for novel health biotechnology products

The Regulation proposal sets out the provision of regulatory assistance or support by the EU Health Biotechnology Support Network to developers. This targeted, early-stage support will be particularly valuable for SMEs, which often lack extensive in-house regulatory expertise. The support will consist of facilitating information on, and access to, applicable legislative frameworks, including procedures for seeking guidance on regulatory status, rules applicable to products combining different technologies or regulatory frameworks, and access to regulatory sandboxes.

Conduct of business: These tools are expected to generate positive effects, as the repository would provide guiding documented regulatory interpretations for similar products, enabling firms to benchmark approaches. Navigation assistance by the Support Network will deliver targeted, early-stage support particularly valuable for SMEs, which often lack extensive in-house regulatory expertise and often rely on costly external consultancy. The foresight panel for emerging innovation would increase the regulatory readiness of the legislative frameworks in the health area by allowing them to integrate approaches to new technologies in a timely way. The panel, offering a forum for structured dialogue across the frameworks, also aims to ensure a cross-framework perspective on emerging technologies, which is needed as innovation increasingly develops across the silos of the different frameworks. This would make the existing frameworks more conducive to processing products based on new biotechnologies.

Regulatory sandboxes offer a controlled environment where developers of truly disruptive innovations are supported by regulators to identify suitable authorisation routes. Evidence from analogous sandboxes already applicable in other sectors, notably the UK Financial Conduct Authority study 169 , suggests that sandbox participation can reduce time-to-market by 20-40%, increase funding probability by 15-50%, and improve firm survival and patenting by 20-30%, with benefit-to-cost ratios ranging from 3/1- 10/1 over three to five years.

The measures are also expected to have a positive impact on administrative costs for businesses (including SMEs), through reducing administrative burden and costs by eliminating duplicative work and regulatory unpredictability for businesses and innovators. The measures aim to avoid undue delays and inefficiencies in regulatory pathways at an early stage of development.

R&I: The measures are also expected to generate positive impacts. Clearer pathways for researchers would reduce the chilling effect that uncertainty currently has on frontier research, encouraging more high-risk, high-novelty projects being developed and retained in Europe. The repository and regulatory assistance would help research institutions and firms plan development activities with confidence, while the sandboxes would ensure that truly disruptive research in novel products is still accommodated by the regulatory system. The foresight panel aims to connect the research world, where innovation emerges, with regulators. This would allow to reflect collaboratively on how to process products based on new technologies in the best way. These measures combined would guide funding bodies and researchers toward promising areas likely to be facilitated in a system conducive to innovation, eventually unlocking the potential for increasing EU gross domestic expenditure on research and development and better translating scientific breakthroughs into clinical applications.

Competitiveness, trade and investment: The Biotech Act as a whole is expected to address structural weaknesses that currently deter investment and early-stage scaling in Europe. Greater regulatory clarity and predictability, through the repository and navigation assistance, should reduce perceived regulatory risk, making the EU more attractive for biotech investment. The sandbox might signal to global investors that tailored regulatory tools exist for disruptive innovations, providing viable pathways to market and reducing uncertainty. The foresight panel demonstrates that the EU actively incorporates emerging trends into regulatory planning, aligning long-term policy with cutting-edge innovation and supporting confidence that governance and rules will adapt to technological progress. These measures have the potential to reverse declining trends in EU biotech and partially close the investment and commercialisation gap with the US and China.

Functioning of the internal market: Its functioning is expected to improve through greater harmonisation and transparency, as the repository promotes convergence by making consistent regulatory interpretations visible across Member States and reducing divergent classifications that fragment the market and distort competition. Navigation assistance and structured guidance minimise interpretive disputes and streamline cross-border compliance. For public authorities, the Biotech Act is designed to improve efficiency and preparedness by reducing duplication through the shared repository reference base, streamlining pre-authorisation interactions through navigation assistance, and building anticipatory capacity through the sandbox and foresight panel. Although establishing the sandbox and operating the foresight panel require upfront investment, these costs are limited given the expected low number of such sandboxes, borne at EU-level with no additional burden on Member States, and are expected to be offset by efficiency gains, reduced uncertainty, and scale effects. For public health and safety, the combined regulatory tools aim to offer a more innovation-conducive regulatory system, bringing more breakthrough products with potential major patient benefit to the market through a more efficient and adapted regulatory process – without compromising safety standards.

Public authorities: The proposed measures will entail only limited costs, while delivering long-term efficiency gains, particularly through streamlined authorisation processes that reduce administrative workload. Regulatory assistance via the support network will leverage existing national structures, adopting an antenna approach to better coordinate and deploy assistance providers at the national level. The regulatory status repository, though requiring initial development, will build upon existing or planned resources—such as the SoHO compendium or the Medical Devices Manual on Borderline and Classification—or simply reference national publications, thereby minimising duplication and resubmissions by developers. This, in turn, will further alleviate the burden on public authorities by reducing uncertainty and improving procedural efficiency. The foresight panel, while incurring marginal costs for logistical support, staff allocation, and expert compensation, is designed to strengthen anticipatory governance, ensuring greater preparedness for emerging technological shifts and ultimately lowering the future regulatory burden for complex, innovative products. Though no data exists on the cost-benefit ratio of a regulatory sandbox for novel health biotech products, its use will be highly targeted, keeping expenses minimal. Importantly, investments to establish the sandbox and operate the foresight panel will be fully funded by the Commission, imposing no additional financial obligations on Member States.

On public health and safety, no impacts are expected as safety standards remain untouched by these provisions. 

 Table 1. Summary assessment of the effects due to the intervention n°1

5.1.2Intervention n°2: Targeted regulatory reform of the General Food Law 170

-the broadening of the scope of general pre-submission advice (currently limited to administrative and regulatory requirements) to cover scientific matters, such as study design and testing strategies, while merging it with the renewal-related advice into a single, unified and simplified procedure;

-a shortening to three months of the procedural delay for non-compliance with the study notification requirements at pre-submission phase;

-a targeted and limited revision of the Scientific Committee/Scientific Panel governance while maintaining the composition of the Scientific Committee;

-the expansion of EFSA mandate to provide scientific advice on all aspects of human nutrition.

Secondly, the proposal establishes a comprehensive regulatory sandboxes framework within Union food law, enabling Member States to create controlled environments for testing innovative technologies, products and substances at pre-market stage, data requirements and alternative regulatory requirements for food production, processing and distribution, feed produced for food-producing animals, food contact materials, and GMO-containing products. The proposal, however, does not allow the establishment of regulatory sandboxes for the following categories of products: (a) Novel foods: Experience has shown that certain types of novel foods involve highly innovative processes and complex scientific considerations, which require thorough oversight even at testing/experimental phase. This makes such foods less suited to the flexible, experimental nature of sandboxes. Furthermore, experience has shown that certain types of novel foods trigger ethical or cultural concerns among various consumer segments regarding their acceptability. Therefore, all aspects of novel foods in general are best addressed solely within the applicable rigorous regulatory framework established by Regulation (EU) 2015/2283 of the European Parliament and of the Council and should not be subject to regulatory sandboxes 171 ; (b) Recycled plastic food contact materials: As regards innovations concerning novel plastic recycling technologies for plastics intended to come into contact with food, chapter IV of Commission Regulation (EU) 2022/1616 already establishes a framework that is meant to encourage the development of such novel technologies without prior authorisation. To ensure uniform rules on the development of novel recycling technologies that safeguard the health of the consumers, it is appropriate to exclude the development of recycling technologies from the possible use of regulatory sandboxes and rely instead on the procedure established in chapter IV of Regulation (EU) 2022/1616; 172 and, (c) GMOs regulated under Part B of Directive 2001/18/EC: For certain GMOs legal pathways already exist to allow testing of innovations, such as under Part B of Directive 2001/18/EC on the deliberate release of genetically modified organisms (GMOs) for purposes other than placing on the market, and therefore there should not be a duplication of paths in the interest of legal certainty. For this reason, regulatory sandboxes should be restricted to products containing or consisting of GMOs subject to authorisation under Part C of Directive 2001/18/EC.

Regulatory sandboxes established in the context of Regulation (EC) No 178/2002 would not replace or circumvent existing pre-market authorisation procedures, as any product subject to pre-market authorisation – for example a new feed additive tested within a sandbox – would still need to undergo the full authorisation process before being placed on the EU market.

Conduct of business: The proposed extension of the scope of EFSA’s general pre-submission advice to cover also scientific aspects is expected to significantly improve application dossier quality, reduce validation timelines and limit ‘stop-the-clocks’ during the risk assessment phase, thereby contributing to shorten time-to-market. By enabling applicants, especially SMEs and first-time applicants, to clarify study design, endpoints and methodologies in advance, with EFSA staff and external experts, the measures should increase predictability without undermining the ‘non-committal’ character of the pre-submission advice and without altering the applicant’s burden of proof or EFSA’s independence. Shortening the procedural delay for non-compliance with study notification obligations from six to three months further reduces procedural delays at pre-submission phase. In parallel, the introduction of a framework for regulatory sandboxes allows companies to test innovative technologies, products and processes in controlled environments before pre-market authorisation procedures, helping them to understand data requirements and regulatory expectations while maintaining oversight and safety. Overall, the conduct of business along the food chain is expected to become more innovation-friendly and less encumbered by avoidable procedural obstacles.

Administrative costs on businesses (including SMEs): the reform is expected to reduce unnecessary burdens linked to incomplete or poor-quality application dossiers. The enlarged scope of pre-submission advice should help applicants tailor their data generation more precisely to EFSA’s requirements, avoiding mis-designed studies and decreasing the likelihood of repeated information requests and related delays. This is particularly important for SMEs and one-time applicants, for whom administrative and compliance costs weigh more heavily and who currently underuse existing advice instruments. By clarifying notification obligations and other procedural requirements at an early stage, the reform can streamline compliance and reduce the cumulative administrative effort over the life cycle of an application.

Competitiveness, trade and investment flows: The targeted amendments are expected to strengthen the EU’s competitive position by making the regulatory environment more conducive to innovation while preserving high safety standards. Faster and more predictable risk assessment processes, fewer unnecessary administrative hurdles and reduced non-compliance penalties should lower the cost of bringing new products to market and make the EU a more attractive location for R&D and investment. By facilitating the development and approval of innovative products and related technologies, the reform supports the ability of EU firms to compete globally, leveraging the existing reputation of EU standards as a quality benchmark and reinforcing the EU’s capacity to shape international norms.

Functioning of the internal market and competition: the reform is not expected to materially alter the baseline. The measures would apply horizontally and uniformly to all operators and Member States, and do not change the core harmonised framework established by the General Food Law and sectoral legislation. The level playing field across the internal market, underpinned by common risk-analysis principles and centralised EFSA risk assessment, would therefore remain intact.

Innovation and research: The new legislative framework for regulatory sandboxes is expected to be potentially transformative. By allowing supervised experimentation with technologies, products and substances in real-world conditions before pre-market authorisation, sandboxes significantly reduce both technological and regulatory risks for innovators, particularly start-ups and SMEs. Shorter learning cycles, direct feedback on safety, usability and acceptance, and clearer insight into data and evidentiary expectations should accelerate development and derisk investment in more ambitious, less incremental innovations. Participation in regulatory sandboxes established by Member States in accordance with the proposed amendment of the General Good Law, with visible regulatory engagement, is also likely to signal credibility to investors and industrial partners, facilitating access to finance and strategic collaborations. Overall, the measures are expected to improve the innovation climate in the EU food and feed sector, while keeping full pre-market authorisation requirements in place for products entering the market.

Public authorities: Concerning public authorities, notably national competent bodies, as well as EFSA, the reform is expected to improve the efficiency and the quality of scientific and regulatory outputs. Enhanced pre-submission advice, including scientific aspects and delivered by EFSA staff and external experts who may also support subsequent risk assessment, should improve dossier quality, reduce the frequency and duration of stop-the-clock procedures, and allow better allocation of EFSA’s resources along the application process. Adjustments to EFSA’s scientific panel governance – having EFSA staff chair panels and the Scientific Committee – are expected to reinforce coherence, methodological consistency and timeliness across assessments. While the expanded advice function may require some additional staff and coordination efforts, these are expected to be offset by savings from fewer procedural delays and less remedial work on weak-quality dossiers. For national authorities, participation in regulatory sandboxes offers a vehicle for systematic regulatory learning, enabling them to test data requirements and alternative regulatory approaches in a controlled setting and to feed robust evidence into EU-level scientific guidance and regulatory standards.

Public health and safety: the targeted amendments are designed to preserve, and potentially enhance in the longer term, the high level of safety standards currently afforded by EU food law. The streamlining of EFSA procedures and the extension of pre-submission advice aim to ensure that risk assessments are supplied with more relevant, better structured data, thereby supporting robust scientific opinions within shorter timelines. The expanded mandate of EFSA on nutrition matters, enabling EFSA to provide advice on all nutrition matters aim at further equip EU regulators with the necessary scientific knowledge to address challenges relating with the increasing prevalence of diet-related health issues. The regulatory sandbox framework, under strict safeguards, is expected to function as a regulatory learning tool, generating richer real-world evidence on exposures, hazards, patterns of use and the effectiveness of risk-management options. This should enable more accurate risk characterisation, more proportionate and effective risk-management measures, and earlier detection of potential safety issues in a controlled environment. Provided that capacity is available to analyse and use sandbox generated data, the overall effect is expected to be at least the maintenance, and potentially the strengthening, of the EU’s high standards of public, animal and plant health protection and environmental safety.

Table 2. Summary assessment of the effects due to the intervention n°2

5.1.3Intervention n°3: Targeted regulatory reform of the Advanced Therapy Medicinal Products (ATMPs) framework 173

The proposed regulatory simplification and future-proofing of ATMPs includes two interconnected changes. First, it establishes a risk-proportionate approach for clinical trials involving ATMPs containing or consisting of GMOs. Under this approach sponsors would be exempted from submitting environmental risk assessments (ERAs) for products presenting no or negligible risks. Second, it aims at future-proofing the regulatory framework by amending the definition of tissue-engineered products to reflect technical and scientific advances, without extending the scope of the definition.

Expected impacts:

Overall, the reform is expected to strengthen the EU’s attractiveness for high-risk biotech investment and support the growth of a rapidly expanding ATMP sector across Member States. The proposed amendments are expected to contribute to the EU’s global competitiveness in biotechnology. Predominantly positive impacts are expected for businesses (in particular SMEs), research and innovation, the internal market, and public health.

Conduct of businesses, innovation and research: The introduction of a targeted exemption from ERA requirements during the CT application phase for clearly defined categories of low-risk investigational ATMPs directly addresses a key barrier to conducting clinical trials in Europe. Even where the recently agreed pharmaceutical legislation reform addresses the fragmentation of ERA requirements across Member States by centralising/harmonising the ERA procedure, requiring sponsors to obtain ERA approval prior to, or alongside, a CT authorisation, imposes a disproportionate regulatory burden at an early stage of development where the risk profile of the product is already well-characterised and low. This deters sponsors –particularly SMEs – from choosing the EU as a trial location, diverting early-stage innovation to jurisdictions with more streamlined processes. This competitive disadvantage is evident when comparing with the US: the Food and Drug Administration grants an exclusion from ERA for the vast majority of gene therapy Investigational New Drud (IND) applications at the clinical trial stage. That regulatory efficiency accelerates development timelines and explains why a significant share of ATMP clinical trials are conducted in the US rather than in the EU. While an ERA will still be required at the marketing authorisation stage, removing this requirement during clinical development removes a critical friction point. This provision is expected to attract clinical research on certain categories of ATMP in the EU even if it is not possible to quantify the precise impact at this stage.

An empowerment which would allow the Commission to amend the ATMP Regulation via delegated acts, in order to amend the definition of tissue engineered products will future‑proof the ATMP framework by allowing to take into consideration technical and scientific advancements in the field of ATMPs. The need for such adaptations is underscored by concrete cases. For example, an acellular tissue-engineered vascular graft intended for urgent revascularisation in severe arterial injury has faced 18 years of classification uncertainty in the EU. Its acellular nature excluded it from the existing tissue-engineered product (TEP) definition. Meanwhile, the FDA approved the same product, demonstrating how regulatory rigidity can drive innovation away from Europe.

Both measures are expected to lower operational costs by removing non‑value‑adding regulatory steps, potentially removing a burden of 0.15-0.3 FTE-years per clinical trial application, and reducing uncertainty. Together, they speed the transition from research to clinical development, particularly for SMEs, while making the EU a more competitive and predictable environment for ATMP innovation.

Functioning of the internal market and competitiveness: The impact of streamlining Regulation (EU) No 536/2014, in conjunction with modifications to ERA requirements for investigational GMO-ATMPs and amendments to ATMP definitions, is expected to be profound and multifaceted concerning internal market dynamics and EU competitiveness. By exempting certain investigational ATMPs containing GMOs from full ERA submissions, this change not only facilitates faster progression from trial initiation to product development, but also significantly influences sponsors’ decisions to conduct clinical trials in Europe. Consequently, the EU could emerge as a leading destination for biotech investment, accelerating the development of innovative therapies. Future-proofing ATMP definitions allows EU to enhance its scientific and technological leadership in biotechnology and regenerative medicine, while further cementing its reputation as a regulator pioneer. The reform is also expected to attract more high-risk capital, support the growth of more than 580 ATMP companies across over 20 Member States, and help maintain EU competitiveness.

Public health and safety: The Biotech Act targets procedural simplification rather than substantive relaxation of safety standards. The ERA exemptions only apply to ATMPs which fall into clearly defined categories with no or negligible risks. Safety oversight is maintained through the review of sponsor declarations within the clinical trial assessment process, comprehensive risk–benefit evaluation and long-term follow-up requirements.

By reducing unnecessary procedural delays and shortening assessment timelines, the measures are expected to facilitate earlier initiation of CTs and potentially earlier access for EU patients to safe, effective and often transformative advanced therapies.

Furthermore, the ability to adapt ATMP definitions to scientific progress helps reduce the risk of regulatory blind spots for emerging technologies, ensuring that innovative therapies remain subject to the most appropriate regulatory oversight.

Table 3. Summary assessment of the effects due to the intervention n°3

5.1.4Intervention n°4: Targeted regulatory reform of clinical trials 174  

The amendments to the CTR aim to shorten authorisation timelines, strengthen collaboration between Member States, and enhance regulatory efficiency without compromising safety, quality, or ethical standards.

The proposed amendments articulate around five pillars:

(I)Faster authorisation procedures

(II)More predictable and efficient authorisation procedures

The amendments also encourage participation of vulnerable populations in clinical trials, by carefully weighing the potential risks and benefits of their exclusion versus inclusion in trials. Additionally, women who become pregnant or start breast-feeding during their trial participation will not be automatically excluded from the trial. Their continued participation will instead be evaluated by assessing the risks and benefits involved.

(III)Context-specific clinical trial authorisation procedures

(IV)Innovation and Harmonised Data Governance

(V)Governance and Enforcement

The policy measures outlined above aim to make the EU more attractive to sponsors, by reducing administrative burden and shorten time to market, thereby increasing the number of clinical trials conducted within the EU. The impact of the regulatory changes is assessed both quantitatively and qualitatively through a two-step procedure (Figure 6). First, the expected effect on the total number of EU-based clinical trials resulting from the simplification and acceleration of the authorisation process and conduct of clinical trials is examined. Second, the broader economic implications and public health benefits arising from the increased number of clinical trials conducted across Member States are assessed.

Figure 6. Schematic illustration of the assessment of possible impacts of the proposed regulatory changes to the Clinical Trials Regulation

Step 1 - Expected impact on the number of clinicals run in the EU

Faster, more efficient and better coordinated procedures, as well as the uptake of AI and digital tools are expected to increase the number of clinical trials conducted in the EU/EEA 178 , 179  and encourage participation by a wider set of Member States. This can reduce current concentration in a few jurisdictions, level the playing field for SMEs and support a more integrated EU clinical research market.

According to one survey with sponsors 180 , the anticipated increase of clinical trial authorisations for selected individual policy changes lies between 5% and 28%, and by 32% for a bundled sub-set of policy measures included in the proposed Biotech Act 181 . Another study, based on limited empirical evidence, suggests that the proposed revision of the CTR could increase the number of clinical trials by on average of 10% 182 . In addition, sponsors believe that the regulatory change will foster multinational trials in the EU with a broader geographic scope (see Figure 7).

To quantify the economic and public health impacts of the regulatory change, we assume that the number of clinical trials will increase by 10% to 30%. These estimates draw on two separate studies 183 . The lower bound reflects the average projected increase in clinical trials of 10%. This estimate also aligns with the ACT EU target of having additional 500 multinational clinical trials over the next five years 184 . Meanwhile, the upper bound is derived from survey responses collected from sponsors as part of the second study 185 . The range was selected to reflect the differing findings of the two studies, while accounting for the respective strengths and limitations of each. The wide disparity in these estimates highlights the challenges in assessing the reform’s impact on clinical trial activity, given data limitations and inherent uncertainties about future developments.

Figure 7: Survey replies from sponsors to the following question: “To what extent would each of the following policies expand the geographic scope of multinational clinical trials in the EU, by increasing the number of participating Member States in a clinical trial in the EU?” 186

Step 2 – Economic and Public Health Impact

The subsequent section assesses the expected impact of the increase in the number of authorised clinical trials on economic and public health outcomes. Economic impacts are evaluated along the following dimensions: competitiveness, gross-value added (GVA), work productivity, employment, R&D spillovers, a simplified cost-benefit analysis for sponsors, and relevant EU and national actors involved. The public health impact is measured by the expected change in number of enrolled participants and the composition of the participant populations.

(a) Economic impacts

Survey responses from sponsors and Member State representatives 187 , including national competent authorities, ethics committees and ministries, suggest that a subset of policy measures introduced in amendments to the CTR are expected to strengthen the EU’s competitiveness (see Figure 8).

Figure 8: Survey replies to the following question “To what extent would the policies increase/decrease the competitiveness of the EU compared to the baseline in terms of, e.g. R&D expenditures in the pharmaceutical sector including revenue for healthcare providers, number of marketing authorisations and patents granted in the EU/EEA, Gross Value Added in the pharmaceutical sector? 188

The economic impact for commercially-sponsored clinical trials is likely to differ from that of non-commercial/academic ones due to systematic differences in their characteristics. In particular, 71.5% of applications submitted by commercial sponsors involve multinational trials, whereas 85.6% of applications submitted by academic sponsors relate to mono-national trials. 189 Academic sponsors are also more likely to conduct late-phase clinical trials, particularly post-marketing authorisation trials 190 , which may entail different cost structures 191 . These differences suggest that the economic impacts of commercial and non-commercial clinical trials should be assessed separately.

Between January 2022 and January 2025, commercial trials accounted for 53.1% of all clinical trials authorised in the EU. Figure 9 illustrates the incremental benefit for an increase in clinical trials across three economic dimensions, i.e. GVA, R&D spill-over and work productivity 192 assuming an increase of 10% or 30% in commercial clinical trials as a result of the legislative changes proposed in the amendments to the CTR. A 10% increase in clinical trials is estimated to generate approximately EUR 3.6 billion in economic gains and create roughly 16,500 additional jobs across the EU, while a 30% increase could result in approximately EUR 10.7 billion in economic gains and 49,500 additional jobs 193 .

Figure 9. Economic impacts assuming a 10% and 30% increase in commercial clinical trials 194  

The value of non-commercial trials on the economy has been recently demonstrated for some regions and countries, such as the UK 195 . Due to a lack of sufficiently robust and comparable data for the EU, this analysis does not attempt to quantify the economic impacts of the proposed regulatory changes for non-commercial trials. Therefore, the estimated economic impacts presented for commercial trials should be considered a lower bound estimate of the overall economic gains associated with the increase in the number of authorised cinical trials.

The proposed regulatory changes are expected to encourage more non-commercial trials, particularly minimal-intervention and low-intervention trials, by simplifying authorisation requirements and streamlining the conduct of trials typically conducted by academic sponsors 196 . Survey replies support the assumption that these amendments would reduce administrative barriers and make academic research easier, including increased availability of data from comparative trial  197 or the Health Technology Assessment (HTA) procedures. The primary advantages extend beyond the authorisation stage, encompassing risk-proportionate safety reporting, more targeted monitoring, and flexible models for the supply and distribution of investigational medicinal products.

To assess the efficiency of the amendments proposed to the Clinical Trial Regulation, a simplified cost-benefit analysis has been carried out as part of two supporting studies. These analyses provide a broad indication of the range of expected cost savings based on different methodologies.

One study evaluates the impacts using estimates from sponsors and public authorities, collected through targeted surveys, on expected changes in costs and the number of clinical trials 198 . Based on these estimates annual cost savings for setting up and the conduct of trials are approximately EUR 1.24 billion for a subset of policy measures included in the Commission proposal. However, it should be noted that response rates for some survey questions have been low (N<10), which may limit the representativeness of conclusions on cost patterns. The policy measures assessed are largely aligned with, but not identical to, those in the Commission’s proposal. Additionally, not all the proposed changes are captured therein, and those are expected to have a positive effect on clinical trials in Europe. Important policy changes, such as the introduction of a single assessment procedure for combined studies, the integration of artificial intelligence, and new structures for governance and enforcement measures, are not part of the assessment as the survey was designed prior to the finalisation of the proposed amendments. Therefore, the calculations can be used as suggestive evidence rather than precise estimates of the cost savings.

The assessment of a second study 199  considers the total set of measures included in the Commission proposal. It estimates direct annual cost-savings for sponsors of approximately EUR 1.5 billion to EUR 3.1 billion through more effective administration and faster authorisation procedures and indirect annual savings of equal amounts through an accelerated conduct of clinical trials. The calculations are based on the assumptions that the amendments reduce the total costs of a clinical trial by 5% due to more effective administration, a clinical trial runs on average 2 to 2.5 years, the average costs of a clinical trial amount EUR 30-50 million and the number of clinical trials remains unchanged. It is to be noted that the limited data availability on the average costs of a clinical trial may substantially impact the estimated cost reductions due to the proposed regulatory changes.

(b) Expected Impact on Public Health

Clinical trials provide early access to innovative treatments for European patients, up to five to ten years before the commercial launch. In the case of rare diseases, clinical trials often present the only available treatment option for patients 200 . In this context, a crucial indicator is not only the number of clinical trials conducted but also the number of participants enrolled in those trials. 

Between 2023 and 2025, the estimated average annual number of newly enrolled participants was 163,643 for commercial trials and 341,632 for non-commercial trials 201 . Assuming a 10% to 30% increase in the number of authorised clinical trials, with the median number of participants per trial remaining constant, this would result in an additional 7,847 202  to 23,540 newly enrolled participants included in commercial trials and 10,381 to 31,145 in non-commercial trials per year.

The increase in the number of participants is expected to contribute to improved patient health outcomes and quality of life, while at the same time generating more robust evidence to inform Europe’s treatment guidelines and HTA decisions, and potentially facilitating faster market authorisation of the medical products under investigation.

In addition to increasing access to innovative and improved treatment options through clinical trials, the new provisions should also support efforts to reduce health inequalities. For example, pregnant and breastfeeding women would no longer be automatically excluded from trial participation, as is the case under the current regulatory framework. Instead, their participation would be evaluated based on the associated risks and benefits. Furthermore, the inclusion of vulnerable populations, such as minors and pregnant or breastfeeding women, is encouraged, provided that the potential benefits of participation are carefully weighed against the associated risks.

Finally, the anticipated increase in clinical trial activity is expected to foster science, innovation, and skill development. Conducting clinical trials generates knowledge, products, and technologies that stimulate further innovation, drive economic activity, and contribute to the development of a more highly skilled workforce across the sector. These spillover benefits from R&D extend beyond the returns captured by private investors, creating broader societal gains. The associated economic impact of these spillover effects for commercial trials is quantified and presented by the value of the R&D spillover in Figure 9.

Table 4. Summary assessment of the effects due to the policy measure

5.1.5Intervention n°5: Targeted regulatory reform of Veterinary Medicinal Products (VMPs) 203

The measure related to the VMPs containing or consisting of GMOs delivers procedural simplification (1-3 month CT application saving; cumulative EUR 5-14.5 million in CTA administrative savings; EUR 560,000 in marketing authorisation dossier savings over the period 2026-2040) and cross-Member States harmonisation, with a high-value, zero-cost legal clarification on GMO status of treated animals eliminating a latent risk. While standard marketing authorisation timelines are unaffected, the benefit materialises under accelerated/emergency pathways (estimated at 30-60 days for 1-3 products/year). While no measurable increase of marketed products is expected, the measure contributes to preventing the progressive offshoring of the innovation process to other jurisdictions where next generation veterinary biotechnology would be developed and trialled.

The proposed SPC extension for VMPs is a low to medium impact signalling instrument. No near-term commercial benefit is expected due to the virtual non-existence of veterinary biosimilar competition and the fact that existing data protection periods already outlast patent/SPC terms. Therefore, the impact of this measure is not appropriately captured through quantification of direct commercial or cost effects, because the primary function of the SPC extension is not to deliver measurable near-term revenue protection but to serve three signalling and future-proofing purposes:

1.Policy signalling: The measure communicates EU institutional commitment to veterinary biotechnology innovation addressing zoonotic diseases, potentially influencing long-term R&D allocation decisions at the margin.

2.Future-proofing: Should veterinary biosimilar competition eventually materialise at scale (plausibly from the mid-2030s onward), the mechanism would already be in place, avoiding the need for reactive legislative intervention.

3.EU manufacturing incentive: The requirement that at least one manufacturing step be performed in the Union introduces an explicit industrial policy lever.

There is no centralised EU database publicly listing how many VMPs have obtained SPCs, and national patent office data are not often broken down by human vs. veterinary use. However, based on data provided by the EMA, out of the 28 biotechnology VMPs for zoonotic diseases approved between 2010 and 2025, only 8 fulfilled all eligibility criteria for the SPC extension. On average, this is 1 product for each 2 years. A forward-looking estimate would foresee an average of 3 eligible products per year over 2026-2040, based on the retrospective eligibility analysis (8 out of 28 over 16 years ≈ 2/year) and adjusted upward modestly to reflect the growing share of biotech vaccines in the pipeline. The targeted SPC extension is designed to be cost-neutral for the EMA. Given the narrow scope of the measure, the EMA is expected to handle a very low volume of applications.

The possibility of using a regulatory sandbox for animal health innovation provides a structured framework to facilitate the development and marketing or use of products, methods or technologies that are not regulated under EU legislation. Expected utilisation is modest (∼1 sandbox per five years; 2-3 established over 2026-2040). Its primary value is qualitative: pathway creation, evidence generation for adaptive regulation, and investment signalling. The proposed measure creates new EMA/Commission responsibilities (application assessment, implementing acts, reporting).

Finally, the reduction of burden for the handling of variations not requiring assessment delivers the most quantifiable and immediately realised benefits with a central estimate of EUR 22.5 million present-value cumulative staff cost savings (2025-2040) and 45-55% reduction in submission events. This will result in approximately EUR 2.5 million/year in fee savings for applicants. The removal of the confirmatory step reduces the national competent authorities workload. It creates a one-off IT cost for EMA of EUR 150,000-300,000. The measure is a purely administrative reform and has no impact on safety or efficacy. The proposed measure has received positive feedback from industry.

Table 5. Summary assessment of the effects due to the intervention n°5

5.1.6Intervention n°6: Targeted regulatory reform of the substances of human origin (SoHO) framework 204

First, the SoHO regulatory status consultation procedures is streamlined by empowering the Commission to adopt implementing acts establishing binding time limits for regulatory status consultation processes within the SoHO framework.

Second, the Regulation proposal establishes a regulatory sandbox framework for SoHO, enabling Member States to create time-limited controlled environments for developing and testing innovative SoHO products, services, processes, or substances that cannot yet fully comply with standard regulatory requirements.

The new regulatory sandbox framework allows a Member State (or several jointly) to set up a regulatory sandbox upon a substantiated request from a SoHO entity, providing a time-limited controlled environment to facilitate the development and testing of innovative SoHO products, services, processes or substances under the supervision of one or more competent authorities. Two eligibility conditions should be met: (a) the innovation is expected to contribute distinctively to SoHO safety, quality, including effectiveness, or to significantly improve patient access to treatment; and (b) applying the SoHO Regulation’s requirements would impede or significantly delay development due to scientific or regulatory challenges inherent to the innovation.

The regulatory sandbox is an evidence-generating mechanism: it should enable assessment in a real-world environment under strict regulatory supervision, producing the data needed to demonstrate safety and quality, including effectiveness, in view of subsequent distribution decisions. Derogations from the SoHO Regulation may be included, but only if clearly described, strictly necessary, and justified in a sandbox plan. Derogations may take the form of adapted, enhanced, waived or deferred requirements. Sandboxes cannot derogate from the SoHO Regulation’s standards on voluntary and unpaid donation.

Operationally, activities must follow a sandbox plan developed by the SoHO competent authorities, informed by data and consultations with the innovation’s developers, and specifying participants and roles; which SoHO requirements cannot be complied with and the resulting derogations and adaptations; risk-mitigation measures; the sandbox duration; and the monitoring framework. The competent authorities must consult the SoHo Coordination Board (SCB) where appropriate, and the SCB is tasked with supporting and fostering a common approach. The SCB may also collaborate with the Foresight Panel for Emerging Health Innovation established by the Biotech Act, linking sandbox practice to upstream horizon-scanning and cross-framework learning.

The proposed amendments create a structured route for SoHO innovations, which cannot yet be developed in full compliance with the SoHO Regulation, or if compliance would impede or significantly delay development due to inherent scientific or regulatory challenges. The proposed amendments also formalise cross-authority collaboration and information exchange, which improve transparency and comparability across Member States while retaining core safeguards.

A SoHO preparation resulting from an innovation developed as part of a regulatory sandbox may be distributed for human application only where authorised in accordance with Article 38(1) of Regulation (EU) 2024/1938. The purpose of the regulatory sandbox is therefore to make authorisation feasible by enabling supervised testing and evidence generation in novel or complex cases where applying the current regulation’s requirements would impede or significantly delay development. These are, for example, new technologies for which there are currently no fitting technical safety and quality requirements by the Expert Bodies (EDQM, ECDC); new technologies that are only partially covered by SoHO rules.

Sandbox-eligible cases would be a minority of cases which could not be authorised under the SoHo Preparation Authorisations (SPA) model established in the 2024 SoHO Regulation, but would bring in high extra value. Expert interviews suggested that SoHO products eligible for the sandbox route likely represent 5–10% of overall SoHO activities, i.e. 90-250 eligible cases per year 205 . Sandbox use cases demonstrate acceptance rate of between 7-16% of sandbox applications, demonstrating great interest from developers in the sandbox instrument but limited capacity of the regulators to establish sandboxes 206 . Considering the high resource requirements of a regulatory sandbox, we can further assume that only a share of eligible cases will be pursued. If all eligible cases apply for the sandbox tool, between 6 and 40 regulatory sandboxes can be established pear year.

Expected impacts:

Since the regulatory sandboxes are a first-of-its-kind instrument, the initial take-up could be slow, also as, in the first years, most innovations are expected to be well regulated under the new model for SPA in the 2024 SoHO regulation.

Conduct of business: Regulatory sandboxes offer a pathway for addressing complex cases that would otherwise not be pursued. We estimate a slowly growing interest in the regulatory sandbox from SoHO entities, leading to regulatory sandbox requests, particularly in strong innovation nations and for SoHO innovations where no safety and quality requirements are prepared by the expert bodies.

Administrative costs on businesses, including SMEs: Regulatory sandbox is expected to have similar costs to high-risk authorisations (between EUR 1,200 and EUR 6,000 per patient) due to the need to involve a certain number of data subjects and a thorough assessment process 207 . Therefore, while there would not be any extra cost for high-risk cases, if low- or medium-risk cases are pursued using the sandbox route, the direct cost of each case will increase. We assume that half of the cases directed to the sandbox route will be high risk cases and half of the cases will be medium risk cases. Under the low estimate, the additional cost of regulatory sandbox route for SoHO entities will be EUR 718,500 (under the assumption that six sandboxes are established per year) and EUR 4,790,000 (under the assumption that 40 sandboxed are established per year).

As a positive secondary effect this would allow SoHO entities to engage with authorities early in an environment that tolerates iterative learning, prototyping, and co-development of evidence strategies, thereby lowering the volume of redundant work and iterative back-and-forth that currently inflates administrative burdens.

Competitiveness, trade and investment flows: By providing a flexible, but coordinated and proportionate pathway for novel SoHO activities, regulatory sandboxes will reduce uncertainty and transaction costs for innovations. This could improve EU attractiveness for certain R&I projects. Particularly strong competitiveness effects can be expected in the forerunner countries with strong innovation potential. Such effects will come from two sources. First, projects that are authorised thanks to sandbox evidence, which would not be pursued otherwise.

Second, under the assumption that sandbox route allows for a responsible approach with limited or more flexible types of appropriate evidence, and for less authorisations to be obtained, innovative SoHO activities will become more attractive for SoHO entities.

Functioning of the internal market and competition: The regulatory sandboxes might have an indirect effect if the outcomes obtained in one Member State are reused in other Member States when assessing similar innovations. This can in particular be expected given the central role of the SoHO Coordination Board in supporting and monitoring sandboxes, including joint sandboxes undertaken by two or more Member States. The overall approach of open access innovation in the largely public SoHO sector will also contribute to a fast and harmonized roll-out of novel technologies.

Innovation and research: The regulatory sandboxes provide a pathway to authorisation and a signal to the R&I stakeholders. As the immediate response to the sandbox measure, we can therefore expect to see an increase in the actor activity in this segment. These changes in risk and incentives and the reduction of the administrative burden could attract more public entities into innovation and stimulate innovation activity in novel, borderline cases and cases requiring cross-framework coordination.

Innovation in SoHO is continuous, often incremental and largely driven by activity in public organisations, with knowledge diffusion through publications and professional societies rather than patents 208 . In leading Member States, major public hospitals treat research as a regular part of their activity and are linked to universities and research institutes. This creates a favourable ecosystem for SoHO innovation, which is nevertheless dependent on the availability of public and private research grants. However, high administrative costs deter these actors from innovative and novel SoHO activities.

Controlled testing environments created through the regulatory sandboxes would enable experimentation under protection from disqualification due to a mismatch with the current framework. By allowing for adapted standards of regulatory evidence generation, regulatory sandbox pathway can enable innovative research that might otherwise be abandoned for fear of regulatory rejection. Therefore, these proposed amendments support the ‘throughput’ phase of the innovation process. Innovation and research impacts at Member State level are expected to be positive mainly via risk reduction, faster evidence generation, increase in academic research, and regulator capability-building, but uneven across Member States depending on capacity and ecosystem maturity.

Regulatory sandboxes could be expected to shorten the path from innovation to safe clinical use, especially where the baseline suffers from uncertain borderlines and divergent evidence thresholds. In the financial technology area, firms that went through the regulatory sandboxes had, on average, a 40% shorter time from engagement with the regulator to market authorisation than similar firms using the standard process. In SoHO, therefore, gains can also be expected. However, no baseline data is available for quantification as these gains would be realised in the cases that otherwise would not be pursued due to difficulty gathering evidence for authorisation.

Public authorities: The addition of the sandbox instrument is expected to expand the mandate and increase the workload of public authorities in the short and medium term for the benefit of significant cost reduction in the longer term due to achieved system efficiency.

To quantify the operational costs of the regulatory sandboxes on public authorities, person-day cost of the sandbox should be compared with person-day cost of the SPA model, taking into account both the costs of learning by doing and the cost reduction expected in the medium term due to cross-Member State learning leading to improved efficiency of operations when novel SoHO activities are concerned. Furthermore, OECD guidance indicates that investments are needed for regulators in terms of time, expertise, and organisational capacity when instituting a sandbox instrument.

These costs would be borne primarily by national competent authorities but also by the Commission due to (i) the role in supporting the regulatory sandbox activities and hosting the EU SoHO Platform, and (ii) the engagement of the SCB as a key operational and consensus-building mechanism. Experts interviewed for this study assessed the role of the EU-level guidance as high for organisation of SoHO activities on the Member State level 209 . Commission-defined principles and formal mandates are essential to stimulate cross-body collaboration among relevant public authorities within Member States and to harmonise common national procedures for multi-framework cases.

We calculate the cost of regulatory sandbox for public authorities as follows: 1) assessing request (no change per risk category), 2) workload of regulatory sandbox (50 person-days) and 3) assessment of clinical evidence (reduced by 40%). This brings the workload on a medium-risk case to 75-94 person-days per case (EUR 22,575 – EUR 28,294); and on the high-risk case to 98-149 person-days per case (EUR 29,498 – EUR 44,849).

Public health and safety: The sandbox instrument grants earlier access to therapies for patients involved in the clinical evidence collection, before the activity is approved. As illustrated above, regulatory sandbox has similar evidence requirements as a high-risk authorisation process. In total, regulatory sandboxes are expected to involve 450-3,000 patients p/a. The impact of such access could be significant in the case when sandboxes provide evidence generation for SoHO activities that would otherwise not be feasible to authorise, for example, in the cases of rare diseases or niche treatments. Follow-up monitoring and assessment should consider whether these patients would have been served at all if the sandbox route did not exist, since authorisations for sandbox-eligible cases would be very difficult to obtain ordinarily. A key condition of sandbox eligibility is a significant expected public benefit, which further implies that sandboxes will address the needs that would otherwise not be met.

Longer-term effects are expected to arise due to institutionalisation of cross-Member State learning and publication of sandbox reports on the EU SoHO Platform. The proposal constrains derogations to standard SoHO procedures to what is strictly necessary and requires explicit risk mitigation and monitoring and therefore disallows derogations on voluntary or unpaid donation standards.

Table 6. Summary assessment of the effects due to the intervention n°6

5.1.7Intervention n°7: Targeted regulatory reform of the Directive on the deliberate release into the environment of GMOs 210  

The proposal introduces specific provisions in Part C of Directive 2001/18/EC addressing the placing on the market of GMMs as or in products other than food and feed with the aim of creating a tailored, more efficient and streamlined regulatory framework for the placing on the market of GMMs as or in products, while maintaining a high safety level for human and animal health and the environment. The proposed provisions relate to the risk assessment, to the validity of the consent granted for their placing on the market, and to detection methods applicable to all GMMs. In addition, the proposal introduces the concept of low-risk GMMs and a specific set of rules for adapted information requirements and a streamlined authorisation procedure for this subgroup of GMMs to be detailed by way of Commission delegated and implementing acts. The requirement for post-market environmental monitoring of low-risk GMMs is equally adapted in the proposal, providing the possibility to a notifier, based on certain conditions, to propose to omit post-market environmental monitoring.

Expected impacts:

Conduct of business: The proposed regulatory framework for GMMs as or in products other than food and feed is expected to improve some framework conditions for companies being active in the GMM field. By shifting towards more product‑specific risk assessment criteria, introducing specific modalities for detection methods, granting unlimited validity for market consents and creating an accelerated pathway for low‑risk GMMs, where certain data requirements would be waived in consideration of an existing Qualified Presumption of Safety delivered by EFSA, the intervention should shorten time‑to‑market and reduce procedural rigidity. This is expected to pave the way for GMM-related consents for placing on the EU market of GMMs as or in products. Earlier market entry would mitigate income losses from regulatory delays and improve the business case for investment, particularly for SMEs and start‑ups that are more sensitive to regulatory timelines and uncertainty.

Administrative costs on businesses (including SMEs): The impacts are expected to be very positive. While quantitative estimates are lacking for non‑food GMMs, a qualitative assessment indicates that a more product‑focused risk assessment and a dedicated low‑risk GMM pathway should substantially reduce the burden associated with preparing extensive dossiers under the current GMO regime. Streamlined, more predictable procedures, the unlimited validity of consents and the possibility to waive post‑market environmental monitoring where justified would lower recurring costs, especially for GMMs considered to be low risk. This is expected to make compliance more manageable for resource‑constrained SMEs and start‑ups, to stimulate exploration of novel products and partnerships between large firms and smaller innovators, and to reduce the incentive to relocate development activities to jurisdictions such as the US or China with more flexible GMM frameworks.

Competitiveness, trade and investment flows: The new regulatory framework is expected to act as an enabler and accelerator. By reducing regulatory costs and uncertainty and clarifying a route to market for GMMs, the intervention could render more projects investable, thereby supporting an expansion of the innovation pipeline in sectors such as biofertilisation, biocontrol, bioremediation, and bioleaching. Over time, this is expected to translate into a larger portfolio of authorised GMM products and more robust EU‑based value chains in these areas. The more predictable pathway from laboratory to market would likely attract additional venture capital and corporate investment and may encourage non‑EU companies with established GMM products to enter the EU market. The number and resilience of biotech start‑ups and SMEs operating in GMM‑related domains is likewise expected to increase, as founders and investors gain confidence that commercialisation in Europe can realistically be achieved within acceptable timeframes. However, all these effects would be gradual and path-dependent, unfolding over several years and dependent on how the regulatory scrutiny process works in practice.

Functioning of the internal market and competition: There are no adverse impacts expected. The new regulatory framework for GMMs modifies a horizontal framework and will apply across all Member States and to all economic operators, so no distortions of intra‑EU trade or competition between Member States are anticipated. The analysis notes that competition and displacement may arise between GMM‑based products and their non‑GM counterparts; however, in the absence of concrete evidence and given that many firms are likely to offer both types of products, no material changes in internal market functioning or competition patterns are expected as a direct consequence of the new GMM framework.

Innovation and research: The proposed intervention is projected to have a positive and potentially substantial effect. By reducing administrative barriers, shortening authorisation timelines and providing regulatory clarity, the framework could stimulate R&D investment by universities, research organisations and companies. This is particularly important in the EU context, where excellent early‑stage biotech research has historically struggled to translate into scaled‑up applications and competitive firms. A more innovation‑friendly regulatory environment is expected to improve the EU’s relative position vis‑à‑vis other major innovation regions such as the US and China, increase private funding for GMM‑related research, and help retain and attract talent in this domain. Over the longer term, the biotechnology innovation system around GMMs could therefore become more dynamic, better leveraging Europe’s strong scientific base.

Public authorities and their administrative workload: The effects of the new regulatory framework for GMMs are difficult to assess. The average effort for national competent authorities per GMO authorisation of a non‑food/non‑feed GMM would fall. However, the extent to which burden will be reduced will fall in a wide range, resulting from the fact that the adaptation of risk assessment (applicable to all GMMs, and expected to go further for low-risk GMMs) will be case-by-case and may lead to considerable variability of data requirements. In the most conservative scenario, risk assessment would require roughly the same amount of data as today, and only a small fraction of products would qualify for the low‑risk procedure (interviews suggested that national authorities may be cautious in designating GMMs as low‑risk, based on a precautionary approach and no practical experience with non‑medical GMM releases), translating in very limited gains for competent authorities. In less conservative scenarios, the tailoring of risk assessment to GMMs is expected to deliver efficiency gains for national authorities. In addition, the harmonisation of criteria for low-risk products will allow the effective use of that procedure, delivering further efficiency gains and reduction of administrative burden. Further savings, in the phase following the initial authorisation, would result from the unlimited authorisation validity and waivers of post‑market environmental monitoring. These were not quantified and would accrue mainly at national level. At EU-level, the mechanisms regarding EFSA’s role in resolving disagreements between Member States is not modified, and the considerations above apply in those instances where -following a disagreement- EFSA would need to conduct an assessment. Both at national and EU-level, if application numbers rise significantly under the new framework, total administrative effort may increase or decrease depending on how many of these applications would have occurred under the existing regime and on the effective use of the low‑risk pathway.

Public health and safety: The proposed measures are designed to maintain the high protection standards of the current EU GMO regime while making their implementation more proportionate to the specific characteristics of GMMs. The intervention does not lower safety standards or abandon mandatory risk assessment but rather tailors information requirements and allows for more flexible approaches where risks are low and well understood. This is expected to reduce unnecessary regulatory burdens without compromising the high level of protection of human and animal health and the environment.

At the same time, by enabling the deployment of beneficial GMM applications that are currently discouraged or displaced to other regions, the new framework could help realise environmental and sustainability benefits, for instance by supporting more resource‑efficient agriculture, cleaner industrial processes or enhanced bioremediation. In doing so, it may contribute to broader EU policy objectives such as the European Green Deal and the 2030 Agenda for Sustainable Development, while preserving robust safeguards for health and the environment.

Table 7. Summary assessment of the effects due to the intervention n°7

5.1.8Intervention n°8: Targeted regulatory reform of the Directive on human organs intended for transplantation 211

The proposal amends Directive 2010/53/EU to reflect technological developments in organ preservation and processing and to provide EU-wide legal clarity and standards for innovative practices which aim to improve organ quality and expand transplantation opportunities.

First, the scope of the Directive is extended to explicitly include the processing of organs prior to transplantation, with a definition of “processing” being introduced by the proposal, encompassing any operation involving the handling of organs performed to maintain or improve the functional status of an organ prior to transplantation.

Second, a specific authorisation regime for organ processing per processing technology and per transplantation centre is established. A transplantation centre is to submit an application for authorisation to the competent authorities including a benefit-risk assessment where scientific evidence and clinical data are available. In the absence of such evidence and data, e.g. when no other EU transplantation centre has been authorised to apply the same organ processing technology, a clinical outcome monitoring plan must be submitted for approval. A clinical outcome monitoring plan must also be submitted for approval where the benefit-risk assessment identifies a significant risk. The extend of the clinical monitoring will be proportionate to the risks identified. Detailed rules on the authorisation procedure are to be established through Commission implementing acts and guidance on the benefit risk-assessment is to be established by the competent authorities.

Transplantation centres must request the written agreement of competent authorities prior to making significant changes to authorised processing steps. The competent authorities have the prerogative to suspend an authorisation where there is reasonable ground to suspect non-compliance. A central EU list of authorised processing techniques or with an approved clinical outcome plan is to be established by the Commission. This will serve as reference point to simplify later authorisations of similar processing technologies used in other EU transplant centres, and hence to facilitate the roll-out and uptake of the same processing technology for other centres and authorities.

Third, the proposal clarifies coordination with other Union health legislation. Where organ processing involves medicinal products, medical devices or SoHO preparations, national competent authorities must verify compliance under the relevant Union frameworks and cooperate with the corresponding national competent authorities under Directive 2001/83/EC, Regulation (EC) No 726/2004, Regulation (EU) 2017/745 and Regulation (EU) 2024/1938 to ensure coherent and more efficient oversight.

These amendments aim to provide EU-wide and cross-sector legal certainty, support innovation in transplantation, and ensure high standards of quality and safety while facilitating timely clinical uptake of advanced processing technologies.

Expected impacts:

Measure 1 – the enlarged scope is textually modest but legally consequential as the foundational enabler of Measures 2 and 3. On its own, however, it generates no direct operational requirements, administrative obligations or supervisory mandates. Its independent effect operates through a single channel: the provision of legal certainty by formally recognising organ processing as a regulated activity within the EU legislative framework. This resolves the legal ambiguity identified in the problem definition and sends a regulatory signal to transplantation centres and technology developers, but the substantive operational, administrative, and health impacts materialise only through Measures 2 and 3.

Measure 2 – on organ processing authorisation regime - is crucial and derives in nearly all quantified impacts for this intervention area.

The conduct of business is assessed as moderate positive: the framework relates to the operational model of transplantation centres by introducing a prior authorisation obligation per centre and per processing technology. However, the net effect is positive because the certainty channel (removing the legal ambiguity that functions as a structural drag on adoption) outweighs the compliance channel, yielding an estimated 70-110 additional programmes with active processing capability and approximately 1,435 additional transplants per year by 2035.

Administrative costs on businesses are assessed as marginal negative: the per-application cost is estimated at EUR 11,500-16,750 (Track 1/Track 2), with a cumulative EU burden of approximately EUR 2.5 million over 2029-2035, costs that are real but proportionate and partially offset by replacing fragmented and duplicative baseline compliance arrangements. Moreover, only the first centre in the EU has to complete the full pathway at the full cost, since subsequent centres can apply a shortened pathway by using evidence from the central list. This leads to significant administrative savings for the overall EU transplant community. Further, transitional measures are envisaged to avoid the authorisation of practices already in place, so that these are not to be re-authorized.

Competitiveness, trade and investment flows are marginally positive: the transparent authorisation pathway reduces the regulatory risk premium for private investment in organ processing technologies, but the effect is indirect given the niche character of the market. The internal market benefits from standardised authorisation procedures that create mutual trust for cross-border exchange of processed organs, with an estimated 120-250 additional cross-border exchanges per year by 2035, while the published list of authorised operations generates de facto EU-wide convergence of practice.

Innovation and research are moderate positive: the dual-track system provides a structured pathway for next-generation technologies (Track 2 conditional authorisation), an estimated 15-36 active monitoring plans generate structured EU-level evidence, and the “Processing” data field in the Annex creates the taxonomic infrastructure to disaggregate processing-attributable outcomes.

Public authorities face a marginal negative impact: the transition-phase assessment cost is approximately EUR 850,000 over three years across the EU, declining to approximately EUR 60,000-85,000 per year in steady state; the net incremental cost is moderated by the replacement of ad hoc national arrangements but represents a genuine, if manageable, expansion of the supervisory mandate.

Public health and safety is the strongest effect, assessed as significant positive: 1,435 additional transplants per year by 2035; 570-945 fewer organ discards; 57-72 waiting-list deaths averted annually; approximately EUR 158 m in cumulative dialysis cost savings over 2028–2035; and a structural improvement in safety oversight through the mandatory benefit-risk assessment and clinical-outcome monitoring mechanisms.

Measure 3 addresses the multi-regulatory-domain nature of organ processing, which require competent authorities to verify that products used in processing (medical devices, medicinal products, SoHO preparations) are duly authorised under their respective EU frameworks, and mandate cross-authority collaboration for the exchange of clinical outcome data. Its impact profile is narrower and more targeted than Measure 2.

The conduct of business is marginal positive: the cross-framework verification adds a structured step to the centre’s processing workflow but also brings EU harmonization and reduces the ad hoc legal uncertainty that centres currently face regarding the permissibility of specific product combinations.

Administrative costs are marginal negative: the verification dossier imposes a modest additional documentation requirement, functionally embedded within the Measure 2 application process. However, there is a significant simplification in administration by other centres, aiming to implement an organ processing step already authorized in other EU transplant centres, facilitating uptake of novel processes across the EU.

Competitiveness, trade and investment flows are marginal positive: the clearer multi-domain regulatory landscape marginally improves investment predictability for manufacturers of perfusates, gene therapy vectors, and other products used across regulatory frameworks.

The internal market is the principal beneficiary of this measure: the mandatory cross-authority coordination directly addresses the regulatory silo problem identified in Section 3.2.3 and creates legally binding cooperation obligations that facilitate the acceptance of processed organs involving multi-domain products across Member State borders.

Innovation and research are marginal positive: the mandated exchange of clinical outcome data between regulatory frameworks generates research-valuable evidence on multi-domain processing outcomes, contributing to a more coherent EU-level evidence ecosystem.

Public authorities face marginal negative costs from establishing inter-authority communication protocols, though Member States with existing cooperation infrastructure will face limited marginal effort.

Public health and safety benefits indirectly but materially: the cross-framework verification ensures that all products used in processing meet their respective EU safety standards, and the mandated data exchange improves pharmacovigilance and device vigilance for processing-related adverse events, closing a surveillance gap that currently exists between regulatory silos.

Table 8. Summary assessment of the effects due to the intervention n°8

5.2Interventions on industrial enablers: measures and expected impacts

5.2.1Interventions n°9, 10 and 11: Strategic projects, high-impact projects and ecosystem support framework

The proposed Regulation establishes a comprehensive framework to structure and coordinate the selection, development and scaling-up of strategic health biotechnology capacities and projects, encompassing:

(I)the recognition and the support of health biotechnology strategic projects (Intervention n°9),

(II)a targeted EU-level framework for high-impact strategic projects (Intervention n°10) 212 , and

(III)an ecosystem support framework (Intervention n°11).

Together, these measures aim to boost the EU’s competitiveness and resilience in health biotechnology, by strengthening biomanufacturing capacity, value chains, research and technology infrastructures in the sector, accelerating innovation and technology deployment and addressing ecosystem fragmentation through coordinated support mechanisms.

Strategic projects (recognised by Member States) would benefit from administrative support (including single points of contact at Member State level), accelerated procedures (including for permitting, subject to a maximum ten-month deadline, as well as for dispute resolution and litigation), a public interest status and measures to facilitate access to funding at national and EU-level. Provisions also proposed support to pro-competitive collaboration between projects, networks and clusters. High-impact projects (recognised at EU level) represent a limited flagship portfolio of systemic importance, including biotechnology development accelerators and centres of excellence for advanced therapies. These projects benefit from similar support but with priority status, including an accelerated eight -months permitting timeline and stronger signalling for Union financial support.

These project-level measures are underpinned by ecosystem support instruments:

(I)a strategic mapping of the EU biotechnology ecosystem to identify capacities, gaps and investment needs;

(II)an EU health biotechnology support network with national and regional antennas to assist innovators in navigating regulatory and funding pathways and accessing scaling and networking opportunities; and

(III)a European Health Biotechnology Steering Group to coordinate Member States and the Commission, facilitate exchange of best practices and ensure effective implementation for strategic and high impact health biotechnology projects.

The EU Health Biotechnology Support Network is not intended to duplicate existing EU-level SME support structures, such as the Enterprise Europe Network or future horizontal instruments under the European Competitiveness Fund. Rather, it operates as a sector-specific coordination and support layer, building on existing structures where appropriate, and providing health biotechnology-specific navigation, ecosystem coordination and cross-border integration functions. In particular, while existing networks may support firms on general business, innovation or internationalisation aspects, they are not designed to provide structured support for navigating complex, multi-framework health biotechnology regulatory pathways. The design of the Support Network is therefore intended to remain compatible with and complementary to future EU-level SME support architectures, including the envisaged EU for Business Network, notably by leveraging existing capacities while ensuring the availability of specialised expertise.

Expected impacts:

Conduct of business and administrative costs: Taken together, the measures are expected to generate significant positive impacts on conduct of business and administrative costs for firms, with positive but more incremental contributions to competitiveness, internal market functioning, innovation and, indirectly, public health and safety. Impacts on public authorities are expected to be front-loaded but moderate overall due to reliance on existing structures. The principal transmission channels operate at two complementary levels. At project level, impacts arise from reduced execution risk through more predictable and coordinated administrative handling, including time-bound permitting (ten months for strategic projects, eight months for high-impact projects), structured single points of contact model and facilitated access to finance. At ecosystem level, impacts arise from reduced transaction and navigation costs, improved access to investors and infrastructures, and strengthened cross-border collaboration through network and cluster effects.

Uptake is a key uncertainty across all components, therefore the scale of impacts should be interpreted as proportional to the number and maturity of projects applying for recognition and to the utilisation of ecosystem support services. This reflects the voluntary nature of the mechanism and the fact that promoters will weigh the administrative effort of application against the expected benefits of more predictable administrative pathway and improved access to support, consistent with observed dynamics in comparable strategic-project regimes 213 .

To illustrate the potential order of magnitude, three uptake scenarios can be defined for each of the project categories. As a calibration point, other EU-level priority-project frameworks 214 have operated at volumes ranging from several dozen to over one hundred projects, providing a useful order-of-magnitude reference (rather than a direct comparator) for the potential scale of project pipelines under coordinated policy frameworks. Against this backdrop, an indicative range for health biotechnology strategic projects could be framed as: (i) low uptake: around 25-30 recognised projects by 2038, (ii) medium uptake: around 60-70 projects, and (iii) high uptake: around 100-120 projects. For high-impact projects, reflecting a limited flagship portfolio, ranges would be: low uptake (six to –eight projects), medium uptake (12–15), and high uptake (20–25).

Conduct of business: Significant positive impacts are expected through greater predictability, shorter effective administrative lead times for strategic and high-impact projects, alongside reduced coordination frictions. Greater clarity and time-bound procedures are expected to improve planning horizons, milestone sequencing and time-to-revenue, reduce volatility in project schedules and strengthen firms’ capacity to align procurement, construction and staffing decisions with a clearer administrative pathway. Delay and uncertainty remain economically material for capital-intensive investments, with an indicative benchmark suggesting that regulatory delays of more than 12 months beyond initial projections have been associated with an average 34% increase in total development costs, alongside a high incidence of additional funding rounds and some programme abandonment.

For high-impact projects, these effects are reinforced through access to promoter-led shared late-stage translation infrastructures with cross-border relevance, supporting more efficient development and scale-up. Impacts can be evidenced through utilisation indicators such as the number of firms served, including SMEs, the number of service engagements (e.g. testing, validation and small-batch GMP runs), and utilisation rates, reflecting improved coordination and access to critical capabilities.

Ecosystem support measures contribute indirectly by improving visibility of strategic projects, facilitating collaboration and enhancing firms’ ability to navigate the EU regulatory and funding landscape. While these effects are not directly attributable in isolation, they reinforce the overall improvement in business conditions generated by the project-level measures. Overall, once implementation stabilises, a significant share of recognised projects is expected to complete permit granting within the applicable timelines.

Administrative costs on businesses, including SMEs: Significant positive impacts on administrative costs are expected, driven by reduced transaction costs, simplified administrative interactions and more predictable, time-bound procedures. For strategic and high-impact projects, reductions arise from fewer parallel interfaces, fewer iterative information exchanges and greater reuse of existing evidence, supported by single points of contact and structured administrative facilitation. These effects are expected to lower compliance costs and administrative burden, with avoided delay costs for capital-intensive investments constituting the main economic benefit.

For high-impact projects, impacts are more targeted, reflecting prioritised handling and faster decision-making for a limited number of catalytic projects, reducing schedule uncertainty rather than generating system-wide burden reductions. Ecosystem support measures further reduce navigation and search costs, particularly for SMEs and start-ups, through a single-entry point and improved preparedness for regulatory interactions. Overall, impacts are expected to be proportionately stronger for SMEs lacking in-house administrative capacity.

Competitiveness, trade and investment flows: Positive impacts on competitiveness, trade and investment flows are expected through improved bankability, stronger investment signalling and reduced time-to-market. For strategic and high-impact projects, more predictable delivery and prioritised handling are expected to lower risk premia and improve conversion from planning to financial close, supporting higher investment mobilisation and leverage, particularly for mature and bankable projects. High-impact projects are expected to generate stronger effects per project due to their scale, visibility and systemic relevance.

Ecosystem support measures reinforce these effects by improving access to investors through structured matchmaking and by reducing delays linked to incomplete or immature submissions, thereby shortening time-to-market 215 . Overall investment mobilisation will depend on project maturity and uptake, but the combined framework is expected to strengthen investment attractiveness and facilitate greater mobilisation of both public and private capital in the Union health biotechnology sector. Existing Union financing instruments can also amplify these effects through leverage: where support is backed by an EU budget guarantee, this can reduce risk for implementing partners and crowd in additional public and private investment beyond the direct Union contribution.

Internal market: Positive impacts on the functioning of the internal market are expected through a more visible and comparable pipeline of strategic projects and reduced information asymmetries for promoters, authorities and investors. Cross-border recognition for projects located in two or more Member States is expected to reduce duplicative processes and improve consistency of treatment, supporting more contestable investment conditions across Member States, while take-up patterns will remain sensitive to underlying administrative capacity and clustering effects. Internal market effects are expected to strengthen as uptake increases and the pipeline becomes sufficiently broad and geographically distributed to improve comparability and transparency across Member States.

These effects are reinforced by improved cross-border accessibility of infrastructures and shared capabilities in the context of high-impact strategic projects, facilitating resource pooling and multi-country collaboration. The Support Network is expected to reduce fragmentation of support services by providing comparable 216 antennas across Member States. Cluster networks further contribute by structuring cross-border ecosystems, enabling access to biomanufacturing and research infrastructures and supporting the formation of cross-border consortia 217 . 

Innovation and research: Impacts are expected through faster and more efficient translation of research and innovation into industrial deployment, supported by strategic projects and reinforced by a limited number of high-impact catalytic assets addressing late-stage bottlenecks. Strategic projects contribute through strengthening biomanufacturing infrastructures and innovation assets, while high-impact projects further enhance the number and speed of projects progressing through late-stage development, particularly for advanced therapies and other complex applications.

These effects are complemented by ecosystem measures, notably cluster networks and support structures that strengthen research-industry linkages and facilitate cross-border collaboration, thereby enhancing firm-level innovation capacity and access to shared infrastructures. Overall, innovation impacts are expected to scale with uptake and portfolio composition, with the main effect being improved translation and deployment rather than system-wide shifts in innovation performance.

Public authorities: They are expected to be front-loaded mainly due to process design, workload reallocation and coordination needs to operate single points of contact and provide structured support, as well as participation in governance and coordination structures. In many Member States, single points of contact may be established by designating or adapting existing administrative coordination mechanisms rather than creating entirely new structures, which may reduce upfront costs. Over time, efficiency gains are expected as procedures standardise, and administrative experience accumulates. Incremental impacts attributable specifically to the high-impact designation are expected to be limited and concentrated in selection, monitoring, coordination and reporting for a limited flagship portfolio, without duplicating cost drivers associated with general permitting operations and routine administrative facilitation. Administrative workload will scale with the number of recognised projects and monitoring cycles required.

The intervention also introduces additional obligations for Member States, including participation in the Steering Group, data provision for ecosystem mapping and facilitation of support network activities, but these are expected to build on existing structures, resulting in marginal set-up costs. Comparable initiatives (NZIA, CRMA) estimate 1–2 FTEs for one-stop shop functions and around two FTEs for governance participation. These initial obligations are expected to yield efficiency gains over time, as Member States gain access to a systematic evidence base and coordination forum that should improve national decision-making and reduce duplicative investments. At EU-level, technical assistance appropriations of approximately EUR 2.6 million annually (EUR 18.4 million over the MFF period) cover relevant coordination functions.

Public health and safety: Impacts are expected to be positive but indirect, mediated by the realised project portfolio and the extent to which faster delivery translates into earlier EU-based capacity for strategic products and a contribution to supply resilience outcomes. The scale of both administrative workload and resilience-relevant capacity effects is expected to increase with uptake, subject to project mix and implementation performance. For high-impact projects, the core impact pathway will be potentially faster, safer and more efficient development of innovative therapies, notably advanced therapies, supported by improved access to specialised testing, validation, small-batch GMP and quality control services and, where relevant, links to data and digital infrastructures.

Impacts are expected to materialise over the medium term and remain conditional on uptake by developers and manufacturers, as well as on broader regulatory, reimbursement and health system conditions affecting downstream patient access. Ecosystem support measures are not expected to have direct effects but may indirectly reinforce these outcomes by facilitating collaboration and improving system preparedness.

The tables below provide an illustrative, scenario-based quantification 218 of the expected impacts by 2038 under three uptake cases for the recognition and support framework for strategic health biotechnology projects (Table 9) and for high impact health biotechnology projects (Table 10) for each impact category. Impacts should be interpreted as orders of magnitude and as broadly proportional to the number, maturity and operationalisation of recognised projects over the period.

Table 9. Illustrative uptake scenarios and order-of-magnitude impacts for strategic health biotechnology projects

Table 10. Illustrative uptake scenarios and order-of-magnitude impacts for high impact strategic health biotechnology projects

5.2.2Intervention n°12: Biosimilars competitiveness framework

The proposal includes two measures focusing on biosimilars:

First, it proposes the development and update of EMA guidelines on tailored and risk-proportionate regulatory approaches for biosimilar development, reflecting manufacturing and analytical testing advances. Most importantly, the guidance will set out the criteria and provide for the potential reduction of clinical data requirements for biosimilar development and approval without affecting quality, safety and efficacy. The revision would be guided by the assessment and conclusions of the reflection paper on tailored clinical approach in biosimilar development by EMA and the CHMP Biosimilar Medicinal Products Working Party (BMWP), published on 27 March 2026 219 . The reflection paper concludes that “a tailored approach for clinical development of biosimilar candidates is possible. Comparative Efficacy Studies (CES) are no longer expected to be required for approval of biosimilars that can be thoroughly characterised using state-of-the-art analytical methods and have demonstrated similarity in physicochemical and functional properties”. Comparative clinical pharmacokinetics/pharmacodynamics (PK/PD) studies can in this context provide supportive safety and immunogenicity data. Over the past three years, applications concerning monoclonal antibodies and fusion proteins have represented more than 90% of all biosimilar marketing authorisation applications (see Annex 5 - additional background on the sector and problem definition). The proposed measure is therefore expected to have a broad practical impact, as it applies to the majority of current and future biosimilar development programmes. 

Second, it proposes the establishment of a framework for the recognition and the support of health biotechnology strategic projects focused on biosimilar research, development, manufacturing and marketing authorisation. The support package outlined in intervention n°9 applies to these projects. Furthermore, the proposed measure promotes international cooperation between economic operators and biotechnology clusters active in the area of biosimilars, including in the context of health biotechnology strategic projects.

The two measures are complementary and the intervention logic goes as follows: The EMA guidelines are expected to reduce the evidentiary burden and associated cost and time of biosimilar development, thereby lowering barriers to market entry. Simultaneously, biosimilar strategic project recognition aims to provide administrative, financial, and permitting support for biosimilar manufacturing and development projects within the EU. Furthermore, eligibility criteria for biosimilar strategic projects enable projects developing analytical methodologies that reduce clinical data needs to qualify for support under the measure, creating a feedback loop between regulatory modernisation and industrial investment.

Expected impacts of measure 1: update and development of EMA guidelines

Conduct of business: If, following EMA guidelines, CES requirement was waived for biosimilar mAb products where analytical similarity is comprehensively demonstrated, developers could reduce direct Phase III clinical trial costs by an estimated EUR 19–26 million per product 220 , with a broader range of EUR 14–46 million across all trial types 221 . Clinical development accounts for approximately 57% of total biosimilar development costs 222 , with the CES representing the single largest component.