
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
NAFFIC and Aware to Launch First China-Europe Digital Product Passport - 2
This Week In Space podcast: Episode 187 — An Inspired Enterprise - 3
Merck urges science-led US vaccine schedule after CDC trims childhood vaccine list - 4
Tech Patterns 2023: 12 Advancements to Keep an eye Out For - 5
Kate Hudson, 46, says she doesn't need long workouts to feel good
5 Great Crossover Vehicles For Eco-friendliness In 2024
As juries turn against social media for harming kids, Big Tech's invincibility starts to show cracks
How to watch ‘The Traitors’ U.K. Season 4 premiere today from the U.S.
Lego's $650 Pokémon set is already sold out as demand, preorders surge
Which Instax Camera Would it be a good idea for you to Purchase?
Israeli girl suffers cardiac arrest during sirens in Safed, hospitalized in serious condition
Manual for Big name Work out schedules
From Amateur to Master: My Involvement in Photography
Figure out How to Augment Your Rooftop Substitution Speculation












