The FDA has released draft guidance outlining an approval pathway for individualized therapies aimed at ultra-rare diseases, building on a framework first outlined by agency leaders in November.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 proposed pathway, dubbed the “Plausible Mechanism Framework,” would allow sponsors to pursue approval for gene-editing and RNA-based treatments when traditional randomized trials are not feasible because of extremely small patient populations, according to a Feb. 23 announcement from the agency. Under the framework, therapies must demonstrate that they address a disease’s underlying cause and show strong results from small, well-characterized studies.
“This guidance is a critical step to tailor our regulatory approach to patients with ultra-rare conditions,” FDA Commissioner Marty Makary, MD, said.
The framework was released jointly by the Center for Biologics Evaluation and Research and the Center for Drug Evaluation and Research.
Vinay Prasad, MD, director of the Center for Biologics Evaluation and Research, described the approach as “a revolutionary advance in regulatory science.” Tracy Høeg, MD, PhD, acting director of the Center for Drug Evaluation and Research, said the pathway is expected to spur industry investment in individualized drug development.
HHS Secretary Robert F. Kennedy Jr. said the initiative delivers on a promise to help accelerate cures for families affected by rare conditions. The FDA will accept public comments on the draft guidance for 60 days.
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