
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
Seven deaths possibly linked to malfunctioning glucose monitors - 2
Vote in favor of your Number one kind of juice - 3
What’s the shadowy organisation taking Gaza Palestinians to South Africa? - 4
Most loved Road Food: Which One Prevails upon You? - 5
‘We are the alternative’: Anti-Hamas Gaza militia tells BBC group is receiving international support
Step by step instructions to Pick A Keep money with High Fixed Store Loan costs
Germany and trade unions kick off tough public-sector wage talks
The 15 Most Rousing TED Chats on Self-awareness
My Excursion to a Better Way of life: Health Experiences
Hezbollah sees potential win as Israel backs down from disarmament goal
Cyber Monday 2025 streaming deal: Get $42 off six months of Apple TV
The most effective method to Execute a Lung-Solid Eating routine After a Cellular breakdown in the lungs Finding
Man triggers smoke bomb during failed crypto robbery
Why are malnutrition deaths soaring in America?












