
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
Why home maintenance deserves a spot in the annual health and budget plans - 2
Excelling at Discussion: Genuine Examples of overcoming adversity - 3
Watching ‘Home Alone’ with the kids this holiday season? Brace yourself for '6-7.' - 4
Russia Establishing Long-Range Drone Bases In Belarus, Warns Ukraine - 5
Iran war fuels fears of new inflation wave among German consumers
Iranian missile hit on Ne'ot Hovav factory leads to fear of chemical leakage
A24's 'Backrooms' trailer shows endless fluorescent-lit spaces and terrifying mannequins melting into the floor
Discovery of ancient pleasure boat reveals Egypt's maritime history
Step into Nature: A Survey of \Handling Trails with Solace\ Climbing Shoes
6 Top of the line Lodgings All over The Planet, Which One Do You Concur With
One lightly wounded after Iranian missile barrage targets northern Israel
Lebanon’s Hezbollah and Yemen’s Houthis join Iran in strike on Israel
The Solution to Defeating Tarrying: Systems for Expanded Efficiency
Nations for Rock Climbing













