
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
'Everyone in this prison has had family killed or shot' - 2
Move. Cheer. Dance. Do the wave. How to tap into the collective joy of 'we mode' - 3
Twins were the norm for our ancient primate ancestors − one baby at a time had evolutionary advantages - 4
What to know about Jack Dorsey's new Vine revival, DiVine - 5
Illumina unveils dataset to speed up AI-powered drug discovery
Volunteers aiding humpback whale stranded in Baltic get death threats
WATCH: IDF strikes, dismantles missile launchers in southern Lebanon
How to watch the ‘Wicked: One Wonderful Night’ special — now streaming
'Euphoria' releases Season 3 photos with Zendaya, Sydney Sweeney, Jacob Elordi and others: See them
Bird flu poses risk of pandemic worse than COVID, France's Institut Pasteur says
Japan deploys the military to counter a surge in bear attacks
At least 11 killed in South Africa mass shooting
Manual for Tracking down the Mysterious Cascades in China
Instructions to Pick the Right Dental Expert for Teeth Substitution











