US EditionFDA 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The FDA's draft guidance aims to facilitate approval and commercialization of personalized genetic therapies tested in few patients, addressing rare diseases often overlooked by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.
83 Articles
83 Articles
FDA Proposes New Approval Pathway for Rare Disease Gene Therapy
(MedPage Today) -- The FDA on Monday unveiled draft guidance for a new "plausible mechanism" approval pathway for ultra-rare disease treatments. "What is a 'plausible mechanism' pathway? It's common sense," FDA Commissioner Marty Makary, MD, MPH...
FDA Unveils Plan for Quickening Development of Personalized Therapies
U.S. regulators on Feb. 23 formally unveiled a plan for speeding up the process of bringing drugs aimed at rare diseases to market. The Food and Drug Administration (FDA) in draft guidance stated that it is proposing to make it easier to secure approval of genome editing and ribonucleic acid-based therapies for rare genetic disorders, or conditions that affect fewer than 200,000 people in the country. Because of the limited number of patients, i…
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.
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