Brussels, 25 April 2023 - On February 28th 2023, Rare Disease Day, EuropaBio's Small and Medium-sized Enterprises (SME) Platform hosted a session on rare diseases and invited European SMEs to share their experiences and discuss the current regulatory and incentives framework ahead of the revision of the pharmaceutical legislation and the legislations on orphan and paediatric medicines. Participants discussed Research and Development, Regulation, and the European Market and investment environment.

Science, Research and Development

Each rare disease affects a small number of people, which brings significant challenges in clinical trial design and recruitment, alongside the limited knowledge of the overall clinical evidence and disease progression process.

Participants discussed the importance of data sharing and patient registries to fill information gaps and the European Medicines Agency's essential support in protocol assistance and scientific advice.

Regulatory frameworks

Smaller companies discussed the concept of unmet medical needs and why it must stay broader, allowing to guide investment in areas with fewer therapeutic options and where scientific developments are more promising. Participants discussed some of the challenges and considerations for market launch in Europe and how they can't do it in all 27 Member States. Challenges include the lack of rare disease patients in some countries, country-specific requirements (e.g., the need for pharmacovigilance departments) and the lack of specialized care centres able to administer complex new treatments.

Market and investment environment

As national healthcare structures evolve, new technologies are adopted, public awareness increases, and information is improved, more rare disease patients will be diagnosed, creating additional financial pressure in an already underfunded sector.

Smaller companies face a challenging financial environment and struggle to raise investment from public and private partners to finance their products' research and development. They must have a predictable legal framework to make informed long-term decisions and be able to raise external capital to address the EU's market fragmentation.

Please consult our event report for our session's conclusion and SME recommendations.