It has been a busy year for rare disease developers, as several new guidance documents were released by the US Food and Drug Administration (FDA).
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, there have been quite a few drugs rejected by the FDA for a variety of reasons, including the agency’s disagreement with surrogate endpoints and trial design, both of which are features which the new guidance documents touch upon, signalling that the agency is open to the use of these, but only in certain scenarios.
Ahead of Rare Disease Day, which is celebrated on 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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