Innovative Therapies, Unequal Access: Bridging the gap for rare disease treatments. The added value of the ERN-EuroBloodNet

Speakers: Mariangela Pellegrini

At the 12^th edition of the European Conference on Rare Diseases & Orphan Products (ECRD) coordinated by EURORDIS and co-organized by Orphanet (15 & 16 May 2024 in Brussels)  Mariangela Pellegrini, representing ERN-EuroBloodNet, shed light on the growing disparities in access to innovative therapies for rare disease patients across Europe, underlining the need of bridging the gap between market access and effective patient access to treatments.

Using CAR T-cell therapy and a PNH study in the EU as examples, Mariangela Pellegrini illustrated how these obstacles impact real-world access to life-saving treatments.

Indeed, despite advancements in medical research, patients often face significant barriers to accessing cutting-edge treatments. The challenges stem from: disparities in healthcare infrastructure across EU countries, complex reimbursement processes that hinder timely access and affordability that concerns extending beyond drug prices to include preclinical costs, market authorization, and patient care.

To move from discussion to implementation, Mariangela Pellegrini emphasized the need for data-driven decision-making, collaborative research efforts, and strategic partnerships between EU countries. Initiatives like ERN-EuroBloodNet registries can play a key role in gathering real-world evidence to support fairer health policies.