
By Michael Erman
NEW YORK (Reuters) -The U.S. Food and Drug Administration can approve new personalized treatments for rare and deadly genetic diseases based on data from a handful of patients, two of the agency's top officials said on Wednesday.
FDA Commissioner Marty Makary and Chief Medical and Scientific Officer Vinay Prasad said in an essay published in the New England Journal of Medicine that for certain conditions, companies could rely on appropriately designed studies with small sample sizes rather than randomized trials. They will rely on biological plausibility and clinical improvements in those early patients.
"Current regulations are onerous and unnecessarily demanding," Makary and Prasad wrote. "For patients and families, there is no time to wait."
The new "plausible-mechanism" pathway would allow the agency to grant marketing authorization after manufacturers demonstrate success with several consecutive patients.
Companies that receive these approvals will be required to collect real-world evidence to confirm efficacy continues and to look for safety issues that might arise.
The new approach will prioritize treatments for rare diseases that are fatal or cause severe childhood disability. Common diseases with unmet medical needs may also qualify.
While makers of cell and gene therapies are likely to be significant beneficiaries of the new approval process, Makary and Prasad said that other types of treatments could also receive licensure this way.
"The FDA will work as a partner and guide in ushering these therapies to market," the officials wrote.
(Reporting by Michael ErmanEditing by Bill Berkrot)
LATEST POSTS
- 1
RFK Jr. says he's following 'gold standard' science. Here's what to know - 2
Tire Brands for Senior Drivers: Guaranteeing Security and Solace - 3
Amplifying Cash The executives: The Upsides and downsides of Various Ledgers - 4
David Duchovny's new thriller has him stripping down at 65. But its chilling premise hits close to home. - 5
Tablets: Upgrade Your Understanding Experience
Top notch DSLR Cameras for Photography Devotees
Famous Restroom Beautifying Styles For 2024
Toddler diagnosed with cancer makes remarkable recovery after aggressive treatment
The 15 Most Rousing TED Chats on Self-awareness
Florida has quietly become America's execution capital
Slims down for Maintainable Weight reduction
Interstellar comet 3I/ATLAS will fly by Earth Friday. Here are the latest images
The many ways that baking is winter therapy. With a delicious ending
Scientists detect X-ray glow from interstellar comet 3I/ATLAS extending 250,000 miles into space













