US Edition
FDA proposes new treatment approval pathway for ultrarare diseases
The FDA's draft guidance aims to facilitate approval and commercialization of personalized genetic therapies tested in few patients, addressing rare diseases often overlooked by industry.
- On Monday, the FDA proposed a draft pathway to authorize and potentially commercialize bespoke experimental treatments, with a 60-day public comment period.
- Because many rare conditions affect tiny populations, randomized trials are impractical and drugmakers often lack incentive, while compassionate use programs have been cumbersome and barred commercial profit.
- Called the 'plausible mechanism' approach, the protocol requires companies to justify why randomized trials are not feasible and mandates real-world evidence collection for genome-editing therapies and RNA-based drugs.
- Senior FDA officials said recent changes, including Monday's pathway, do not set new standards, but critics and observers note procedural concerns and say commercializing treatments could alter drugmakers' incentives.
- Last year, researchers at Children's Hospital of Philadelphia and the University of Pennsylvania used CRISPR gene-editing to treat a baby, showing individualized medicine's promise and potential acceleration.
37 Articles
37 Articles
US FDA proposes framework to speed rare disease gene therapy approvals
The U.S. Food and Drug Administration proposed on Monday a new framework to speed approvals of personalized treatments for rare and life-threatening genetic diseases, allowing drugmakers to rely on small, well-controlled studies when traditional trials are not possible.
FDA moves to facilitate personalized therapies for rare diseases
The Food and Drug Administration is making it possible for pharmaceutical companies to produce bespoke medicines for individual patients, an effort to revolutionize the standard of care for rare diseases. Trump administration officials announced on Monday new draft guidance from the FDA that would update approval standards for personalized genetic medicines, creating a new approval pathway for therapies targeted to patient groups too small for …
FDA proposes new treatment approval pathway for ultrarare diseases
The Food and Drug Administration (FDA) announced a new proposal Monday for flexible drug approval pathway treatments addressing ultrarare diseases. The FDA unveiled draft guidance on a proposed regulatory pathway for individualized therapies, treatments for rare conditions that affect a very small population, with the proposal specifically focusing on genome editing and RNA-based therapies. The…
Associated Press News
FDA proposes new system for approving customized drugs and therapies for rare diseases
The Food and Drug Administration is proposing a system for approving customized drugs and medical treatments for patients with rare or hard-to-treat diseases.
FDA Launches Framework for Accelerating Development of Individualized Therapies for Ultra-Rare Diseases
FDA NEWS RELEASE 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 ge…
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