Rare disease R&D investment is outpacing the broader biotech market, while the pipeline of innovative therapies continues to deepen. With enhanced sequencing and data infrastructure, Europe’s constraint is no longer scientific. Funding and regulatory complexity remain the core limitations to making rare disease therapies accessible to the patients who need them most. Dr. Daria Julkowska, Scientific Coordinator at ERDERA, mentioned, “Innovation that cannot reach patients is not innovation at all”, at the 13th European Conference on Rare Diseases and Orphan Products in Prague, Czech Republic, on 3–4 June 2026.
A large share of rare disease development still originates in non-profit and academic structures where the financial model and the cost of generating regulatory-accepted clinical evidence remain high. Financing pressure is pushing non-ultra-rare programs out of Europe, with the US being the first market to grant authorisation. Derisking assets to attract capital, rethinking funding models, and addressing the funding rather than the scientific barriers to drug repurposing were all flagged as priorities if Europe is to retain its pipeline. Evidence generation emerged as a challenge specific to small populations, where short trials and limited patient numbers constrain conventional data collection. One of the speakers stated that real-world evidence and patient-reported outcomes must carry greater weight, and that the immediate priority must be consolidating fragmented datasets and improving how data sharing is implemented.
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Europe’s framework is credited with rigorous risk assessment, but insufficient accountability and a tendency to weigh risk in isolation from benefit remain. The same therapies that have been approved in the US under accelerated pathways may have their access delayed within European markets, bringing out potential risks when dealing with pediatric conditions. Only a small number of conditional authorisations are withdrawn, favouring faster access and a more explicit patient voice in both approval and withdrawal decisions. The existing tension between precaution and timely access may define Europe’s rare diseases agenda.
