The FDA has released draft guidance outlining an approval pathway for individualized therapies aimed at ultra-rare diseases, building on a framework first outlined by agency leaders in November. The ...
The free virtual series extends Clinical Pathways Congress with timely insights into evidence-based pathways, value assessment, and access. By extending Clinical Pathways Congress throughout the year, ...
FDA proposes using a well-supported mechanistic rationale plus natural history comparators to generate substantial evidence when randomized trials are infeasible for ultra-rare populations.
Plastic pollution demands faster results. This blog calls for creative, effective pathways alongside multilateral talks to deliver real change.
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