
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
Chris Noth responds to backlash after seemingly shading 'Sex and the City' costar Sarah Jessica Parker: 'It is not news' - 2
Scientists uncover an ant assassination scheme that helps a parasitic queen rise to power - 3
How did I get my own unique set of fingerprints? - 4
What you need to know about flu treatments as cases spike across the US - 5
Roche breast cancer pill cuts risk of disease recurrence by 30% in trial
Lower-cost space missions like NASA's ESCAPADE are starting to deliver exciting science – but at a price in risk and trade‑offs
Family-Accommodating Snow Sports Experiences
Solid Living Tips: Experiences from a Wellness Fan
Somalia set for 'historic' first offshore oil drilling
4 astronauts depart ISS, leaving behind just 3 crewmates to staff the orbiting lab
12 Once-In-A-Lifetime Travel Experiences To Add to Your Bucket List
Ukraine: Russians abduct 50 Ukrainians from border village in Sumy
6 Vehicle Rental Administrations: Pick Your Ideal Ride
Raw oysters linked to ongoing salmonella outbreak infecting 64 across 22 states: CDC













