
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
Health insurance premiums rose nearly 3x the rate of worker earnings over the past 25 years - 2
The Solution to Flexibility: Developing Internal Fortitude Notwithstanding Misfortune - 3
How to identify animal tracks, burrows and other signs of wildlife in your neighborhood - 4
The Extraordinary Excursion of Dental Embed Innovation - 5
Factbox-Artemis II crew includes first woman, Black astronaut and Canadian ever flown to moon
Flu cases skyrocket in US. See cases, where people got sick.
The most effective method to Move toward Compensation Conversations for Cutting edge Practice Enrolled Attendants
Getting ready for a Mechanized World: 10 Positions That computer based intelligence Could Dominate
Brazil's ex-president Bolsonaro operated on for hernia
Manual for Instructive Application for Youngsters
Savvy Tips for Seniors Hyundai IONIQ EV
6 Tire Brands Reasonable for Seniors
New hybrid mpox strain discovered in UK after US reports local spread
EU-funded BioSupPack project turns brewery waste into bioplastics













