
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
A 3-limbed Kemp's ridley sea turtle is now being tracked at sea by satellite - 2
UAE recalls some Nestle infant formula products, Qatar warns consumers - 3
IDF strikes Hamas terror cell operating near Israeli troops in northern Gaza - 4
Air New Zealand cuts flights and hikes fares as fuel prices surge - 5
Sunken warship found off Danish coast after 225 years in ‘remarkable’ discovery
Remote Headphones: Improve Your Sound Insight
McDonald's is bringing two 'KPop Demon Hunters' meals to McDonald's. Here's what they include and when they launch.
This star-forming galaxy is blowing out powerful winds topping 2 million mph
21 Incredibly Interesting Contemplations To Observe Consistently
ByHeart baby formula from all lots may be contaminated with botulism bacteria, tests show
Italian authorities detain civilian rescue ship, German NGO says
Step by step instructions to Buy a Jeep Wrangler on a Senior's Spending plan
Honda’s Biggest Flex Isn’t Its Superbikes, It’s Selling 500K Bikes In One Month
Best Amusement Park in Asia: Which One Is a Must-Visit













