EPTRI has submitted its feedback to the European Commission’s proposal for the EU Biotech Act. Its message is that paediatric research should be recognised as a strategic component of European biotechnology policy.
EPTRI supports the proposed Biotech Act as an opportunity to strengthen Europe’s biotechnology ecosystem and to turn scientific excellence into innovative healthcare. It sees the Act as a way to reinforce both European competitiveness and equitable access to innovation, provided it addresses the needs of all patient populations.
A gap in the current draft
From a paediatric research perspective, the current draft does not yet fully recognise children’s health as a strategic priority for biotechnology innovation. EPTRI notes that children appear mainly as a vulnerable population, or in connection with Paediatric Investigation Plans (PIPs). They are not treated as a population with distinct biological and therapeutic needs that require dedicated research and innovation.
This matters because biological development, disease progression and treatment responses in children differ substantially from those in adults. Without explicit objectives and incentives, advances in biotechnology may not translate into health benefits for children, and access to innovative therapies may be delayed.
Advanced Therapy Medicinal Products (ATMPs) illustrate the point. Many rare genetic diseases manifest early in life, and timely intervention can prevent irreversible disease progression and improve lifelong health outcomes.
EPTRI’s key recommendations
- An explicit paediatric research and innovation objective. Paediatric research should be considered along the entire innovation pathway: discovery research, first-in-child development, regulatory evaluation, manufacturing, clinical implementation and long-term follow-up. It should not rely only on existing PIPs.
- Support for rare and ultra-rare diseases. This means innovative funding models, support for academic and not-for-profit developers and SMEs, and proportionate regulatory pathways for highly personalised therapies, including platform technologies and N-of-1 approaches.
- Stronger collaboration across the ecosystem. EPTRI calls for closer links between European Reference Networks (ERNs), paediatric research infrastructures such as EPTRI, European paediatric clinical trial and specialty networks, specialised centres, patient registries and natural history platforms.
- Paediatric expertise in Advanced Therapy Centres of Excellence. These centres should include dedicated paediatric expertise, specialised children’s hospitals, newborn screening pathways, paediatric ethics and long-term follow-up, so that care is continuous from research through to clinical implementation.
- Stronger links between biotech innovation and child health. The Act should connect biotechnology with newborn screening, genomic diagnostics, cross-border access to specialised expertise, personalised medicine and prevention. This includes next-generation vaccines for healthy childhood to be translated into healthy adults.
Clinical trials: a welcome step, with conditions
EPTRI welcomes the proposed amendments to the Clinical Trials Regulation, including faster multinational authorisations, e-Consent and simplified procedures for minimal-intervention trials. It stresses that the benefits for paediatric research depend on how the changes are implemented. They should be applied consistently across Member States and preserve paediatric scientific and ethical expertise. They should involve children and families meaningfully and promote digital inclusion. EPTRI also asks for clear EU guidance on interpreting “minimal risk” in paediatric research.