Addressing The Rare Disease Challenge Via Patient Engagement

 
• By Ayisha Sharma

Following the FDA’s release of draft guidance to enhance patient voice, In Vivo spoke with Ipsen’s rare disease regulation expert on creating a more patient-centric R&D and approval pathway, from modified endpoints to trial decentralization and beyond.    

list of ticked boxes on blackboard
Industry Should Treat Patient Voice As More Than A Tick-Box Exercise • Source: Shutterstock
Key Takeaways:
  • Expert comments from Martine Zimmermann, Ipsen’s head of regulatory and R&D quality, on improving the development and approval process for rare disease therapies, particularly in the pediatric setting.

It has been 40 years since the US passed the Orphan Drug Act 1983, which introduced financial incentives for the biopharma sector to develop treatments for rare diseases, from longer market exclusivity to R&D tax credits. While hundreds of therapies have since been approved, most rare diseases still lack treatment options.

The therapeutic space has presented a unique challenge due to the complex underlying biology and lack of understanding of the...

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