
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
Lego's $650 Pokémon set is already sold out as demand, preorders surge - 2
Putin, Netanyahu discuss Middle East in phone call, Kremlin says - 3
'All Her Fault' ending explained: The shocking conclusion to the psychological thriller inspired by true events - 4
Thousands of genomes reveal the wild wolf genes in most dogs’ DNA - 5
5 things for parents to know about changes to kids vaccine schedule
Striking American and European television Projects: A Survey
Interstellar comet 3I/ATLAS isn't an alien spacecraft, astronomers confirm. 'In the end, there were no surprises.'
Tech for Wellbeing: Applications and Devices for a Better You
Journeys That could only be described as epic: Delightful Voyage Lines All over the Planet
Figure out how to Keep up with Your Dental Inserts for Long haul Achievement
Tire Brands for Senior Drivers: Guaranteeing Security and Solace
Cruising Solo All over the Planet: An Excursion of Self-Disclosure
Did we start the fire? A 400,000-year-old hearth sparks new questions about human evolution
A red meat allergy from tick bites is spreading – and the lone star tick isn’t the only alpha-gal carrier to worry about












