June 2 (Reuters) – The U.S. Food and Drug Administration on Tuesday proposed allowing makers of cell and gene therapies targeting rare and life-threatening diseases to use existing scientific knowledge to expedite development.
Here are a few details:
• The agency in its draft guidance said the recommendations outline how developers can leverage chemistry, manufacturing and controls data, and nonclinical and clinical prior knowledge from other relevant products.
• The information that could be used includes medical or scientific information that is generally accepted by experts as well as knowledge gained from developing and manufacturing similar products and processes, the FDA said.
• This prior knowledge could help streamline submissions and subsequent review, and to expedite product development.
• The FDA had proposed a new framework earlier this year to accelerate approvals of personalized treatments for rare genetic diseases, allowing drugmakers to rely on small, well-controlled studies when traditional trials are not possible.
• The agency had faced intense scrutiny under former Commissioner Marty Makary, who stepped down last month after a string of controversial rare-disease treatment decisions and public clashes with drugmakers.
• A permanent replacement has not yet been announced, and Deputy Commissioner for Food Kyle Diamantas is leading the agency in an acting capacity.
(Reporting by Mariam Sunny in Bengaluru; Editing by Sahal Muhammed)
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