
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
EPA watchdog finds nation’s most contaminated sites are vulnerable to flooding, wildfires - 2
Boats escort freed whale away from shallow waters off German coast - 3
Knesset FADC extends emergency draft for 280,000 IDF reservists until January 1 - 4
Turning into a Sharp Financial backer: Individual budget Wins - 5
Watch SpaceX launch 119 payloads to orbit from California early on March 30
Giude to Best Web based Learning Stage
Productive CRM Programming for Client Relationship The executives
RSF attack on Sudan’s South Kordofan kills at least 14, including children
'Euphoria' Season 3 trailer includes Eric Dane's final appearance, Jacob Elordi and Sydney Sweeney at the altar and Zendaya's 'Breaking Bad' era
Mom finds out she has cancer after noticing something was off while breastfeeding
Figure out How to Take part in Open Conversations Around 5G Pinnacles
Getting through a Lifelong Change: Individual Examples of overcoming adversity
Higher cost, worse coverage: Affordable Care Act enrollees say expiring subsidies will hit them hard
Study casts doubt on potential for life on Jupiter's moon Europa













