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FDA Launches Framework for Accelerating Development of Individualized Therapies for Ultra-Rare Diseases | FDA

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The .gov means it’s official.Federal government websites often end in .gov or .mil. Before sharing sensitive information, make sure you're on a federal government site. The site is secure. The https:// ensures that you are connecting to the official website and that any information you provide is encrypted and transmitted securely. FDA NEWS RELEASE More Press Announcements The U.S. Food and Drug Administration today issued draft guidance for sponsors seeking approval for targeted individualized therapies by generating substantial evidence of effectiveness and safety when randomized controlled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. The draft guidance, issued by the Center for Biologics Evaluation and Research and Center for Drug Evaluation and Research, specifically discusses genome editing and RNA-based therapies such as antisense oligonucleotides but leaves open the potential that this framework may apply to additional tailored therapeutics provided they directly a

More Press Announcements For Immediate Release: February 23, 2026 The U.S. Food and Drug Administration today issued draft guidance for sponsors seeking approval for targeted individualized therapies by generating substantial evidence of effectiveness and safety when randomized controlled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. The draft guidance, issued by the Center for Biologics Evaluation and Research and Center for Drug Evaluation and Research, specifically discusses genome editing and RNA-based therapies such as antisense oligonucleotides but leaves open the potential t

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