
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
The most effective method to Boost Benefits in Gold Speculation: Master Techniques and Tips - 2
Four Dead in Last Month From Animal Attacks in Nepal - 3
South Africa collects record $117B tax haul - 4
Mossad unveils network of Hamas terror infrastructure across Europe - 5
Live long and loiter: Why NASA's ESCAPADE probes will wait a year in space before heading to Mars
8 Espresso Bean Starting points All over the Planet
Find the Standards of Viable Refereeing: Settling Debates with Strategy
Illumina unveils dataset to speed up AI-powered drug discovery
French rapper Gims placed under investigation for 'aggravated money laundering'
First Houthi launch toward Israel since war began triggers alerts across the Negev
Doctors seek to understand why quitting antidepressants causes withdrawal for some
Some super-smart dogs can pick up new words just by eavesdropping
Overseeing Individual budgets Successfully
An Extended period of Voyaging Carefully: the World with Reason













