It’s been a busy year for rare disease developers, with the US Food and Drug Administration (FDA) releasing several new guidance documents.
These documents include the Rare Disease Evidence Pathway (RDEP), the Plausible Mechanism Pathway and the Innovative Designs for Clinical Trials of Cellular and Gene Therapy Products in Small Populations, as well as the renewal of the Rare Pediatric Disease Designation and Priority Review Voucher Programs.
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Despite these positive changes, several drugs have still been rejected by the FDA for reasons such as disagreements over surrogate endpoints and trial design – areas addressed in the new guidance, indicating the agency may accept them, but only in specific circumstances.
For Rare Disease Day, 28 February 2026, Abigail Beaney, editor of Clinical Trials Arena, spoke with Robert Barrie, editor of Pharmaceutical Technology and Frankie Fattorini, reporter for Pharmaceutical Technology and GlobalData Insights, about some of the recent developments in the space.
You can listen to the podcast here:
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