
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 Alter Your Savvy Bed for A definitive Rest Insight - 2
The Solution to Defeating Tarrying: Systems for Expanded Efficiency - 3
What's going on with Katseye? The Manon Bannerman hiatus drama, explained. - 4
Step by step instructions to Explore Assessment Ramifications of Disc Rates - 5
The most effective method to Pick A Trade-in vehicle Stage
German petrol stations hike prices as once-a-day rule takes effect
This St Nick Truly Can Advise How To Drink And Hack Your Headache
Turning into a Sharp Financial backer: Individual budget Wins
Spanish bishops and government sign deal for compensation of church sexual abuse victims
Pick Your Favored kind of sandwich
Palestinians tell BBC they were sexually abused in Israeli prisons
Alice Wong, founder of the Disability Visibility Project, dies at 51
Why is Jerome Powell being investigated? Making sense of the DOJ's probe into the Federal Reserve chair.
No respite for German economy as experts slash forecast over Iran war













