FDA's Revolutionary Pathway for Rare Disease Therapies
Published on: Nov. 13, 2025, 3:31 a.m. | Source: Devdiscourse
The FDA plans to approve groundbreaking personalized treatments for rare genetic diseases using data from few patients. This new 'plausible-mechanism' pathway relies on designing studies with small samples and prioritizes fatal or severe conditions. Companies will initially gain approval but must gather real-world evidence to confirm ongoing efficacy.
