Building A Competitive, Patient‑First Future For Rare Diseases In Europe

By Rachel Smith, Therapeutic Area Lead, Rare and Genetic Diseases

As Europe faces intensifying global competition for life sciences innovation, industry leaders, regulators, and patient organizations gathered at the EURORDIS Round Table of Companies to examine how the region can remain competitive while improving access for people living with rare diseases.

This article explores key insights from the discussion, highlighting the need to align regulatory reform with patient‑centered outcomes rather than treating competitiveness and access as opposing forces. Participants emphasized the EU pharmaceutical reform and proposed Biotech Act as a once‑in‑a‑generation opportunity to streamline clinical trials, reduce fragmentation across Member States, and accelerate rare disease innovation—without compromising safety or scientific rigor.

Central to success is meaningful, early patient involvement, proportionate evidence requirements, and lifecycle alignment across regulatory approval, health technology assessment, and reimbursement. The article concludes that by embedding patient partnership and regulatory agility into a more integrated European ecosystem, Europe can strengthen its leadership in rare disease development and deliver faster, more equitable access to life‑changing therapies