
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
10 Hints and Deceives to Expand Cell Phone Information Use: Capitalize on Your Information - 2
Every year, she thanks the trooper for the arrest that led to her sobriety - 3
Virtual Domains d: A Survey of \Inundation and Ongoing interaction Mechanics\ Computer game - 4
Chinese astronauts’ return to Earth delayed over fears spaceship damaged by debris - 5
Instructions to Grasp the Innovation Behind 5G Pinnacles\
A Gastronomic Experience in Healthy Enjoyments: A Survey of \Nutritious and Tasty\ Solid Cooking Recipe Book
FDA claims on COVID-19 vaccine safety are unsupported by reliable data – and could severely hinder vaccine access
Allow Innovative Progressions To have a Massive Effect
Exploring ways to reduce the impact of space junk on Earth
Toyota Motor Europe to roll out smart EV charging through new partnerships
This Week In Space podcast: Episode 186 — Snow on the Moon?
Islamic State group militants claim capture and execution of a Nigerian brigadier general
Israel explores creation of int'l force with Greece, Cyprus to deter Turkey
Spanish police and soldiers track boars, reinforce farm security amid swine fever outbreak












