Rare Disease Moonshot’s contribution to the Consultation on the EU Biotech Act
The Rare Disease Moonshot welcomes the European Commission’s ambition to develop a comprehensive EU Biotech Act. This initiative represents a timely opportunity to unlock the full potential of biotechnology as a strategic driver of health innovation, economic resilience, and scientific leadership in Europe. With biotechnology at the forefront of medical breakthroughs, green and digital transitions, and industrial competitiveness, the EU Biotech Act must aim to define a bold, forward-looking framework that empowers Europe’s biotech community to deliver transformative impact.
The Rare Disease Moonshot is a multi-stakeholder initiative that brings together public and private actors in rare diseases, an area where biotechnology offers unparalleled promise. Rare diseases affect 30 million people in the EU, and over 80% have a genetic origin. Biotech innovation, including gene therapies, biologics, and molecular diagnostics, is central to delivering solutions. Yet, Europe’s biotech potential remains underleveraged due to regulatory complexity, fragmented support structures, and limited pathways for scale-up and deployment.
The EU Biotech Act should be designed not as a patchwork of corrections, but as a forward-looking framework with structural enablers. It should reduce administrative barriers, simplify regulatory pathways, improve the interface between research and application, and ensure sustainable funding and production environments. Importantly, it must support innovation (and innovators) from end to end: from discovery to clinical validation, to manufacturing and patient access—particularly in areas of high societal need.
To ensure the EU Biotech Act delivers on its full potential, we recommend the following priority actions, aligned with the needs of the biotech ecosystem:
- Define a forward-looking EU Biotech Act anchored in strategic enablers. The Act should go beyond legislative adjustments to establish a coherent framework that proactively supports biotechnology development, deployment, and scale-up across sectors.
- Simplify and modernise regulatory pathways for biotech innovation. This includes flexible approval mechanisms for ATMPs and complex biologics, regulatory sandboxes, harmonised guidance documents, and coordinated scientific advice to accelerate clinical development and authorisation.
- Equip biotech innovators with hands-on, integrated support mechanisms. The EU should strengthen the visibility and functionality of the Biotech and Biomanufacturing Hub, establish a Biotech Innovation Interface, and provide advisory, legal, regulatory, and acceleration services tailored to SMEs and start-ups.
- Enable deployment through investment in biotech infrastructure and manufacturing. EU funding should prioritise the development of regional biotech clusters to secure industrial resilience and strategic autonomy.
- Introduce a dedicated European Biotech Innovation Fund. Designed to complement existing instruments like the Innovation Fund and EIC Accelerator, it should provide financing to support early-stage and translational biotech, especially in high-need, low-return areas like rare diseases.
- Align innovation incentives with market access through coordinated push and pull tools to reward biotech solutions for their societal value and patient impact.
Many much-needed changes can be achieved through ongoing simplification, cutting red tape, smart implementation of existing legislation, and piloting new funding instruments that could be fully deployed under the next funding framework.