
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
Poll: Most are satisfied with their health insurance, but a quarter report denials or delays - 2
Twelve injured near Beit Shemesh, reports of shrapnel impact in Eilat as Iran targets Israel - 3
10 Demonstrated Tips to Boost Your New Android Cell phone: A Thorough Aide - 4
Hamas propaganda expert explains Israel's internal conflicts influenced Hamas's Oct. 7 assault - 5
Man threatens attack on German high-speed train, injures several
Planet-eating stars hint at Earth's ultimate fate
Passenger Missing After Going Overboard Disney Cruise Ship
What’s the shadowy organisation taking Gaza Palestinians to South Africa?
Innovative Versatility: Examples of overcoming adversity from Entrepreneurs
Antimatter took to the road for the very first time. Here’s why it matters
Palestinians tell BBC they were sexually abused in Israeli prisons
'Crammed into a cell with vermin at New Year'
Instructions to Pick the Ideal Pre-assembled Home for Your Necessities
Minnesota jury says Johnson & Johnson owes $65.5 million to woman with cancer who used talcum powder













