The U.S. Food and Drug Administration today issued draft guidance for sponsors seeking approval for targeted individualized therapies by generating substantial evidence of effectiveness and safety when randomized controlled trials are not feasible due to small patient populations.
FDA News Releases published a clinical update in Research Highlights on 23 Feb 2026. The item focuses on FDA Launches Framework for Accelerating Development of Individualized Therapies for Ultra-Rare Diseases. Open the detail page to review the full original feed content.