
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
Creative Style: 10 Architects Reclassifying the Business - 2
The most effective method to Offset Album Rates with Liquidity Needs - 3
37 Things Just Individuals Experiencing childhood during the 80s Will Comprehend - 4
4 African Vacationer Locations - 5
Several killed in Ukraine and Russia after cross-border attacks
Scientists have found an alarming environmental impact of vast data centers
Witness the elegance of the cosmic butterfly in a remarkable telescope photo
Which restaurants and fast food chains will be open on Christmas Eve and Christmas Day?
'The Boys' Season 5 premiere: How to watch for less, what to know about the final series and more
Apollo vs. Artemis: What to know about NASA's return to the moon
Ober Gabelhorn glacier reveals remains of man missing for over three decades
Nearly half of reindeer have been wiped out and armadillos are in Iowa. Here’s how animals are weathering warming holidays
Steven Spielberg's 'Disclosure Day' trailer drops: What we know about the alien movie
Mexican Woman Accused of Assaulting Partner With Belt After He Refused Sex, Police Say












