
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
Have gravitational waves provided the first hint of primordial black holes born during the Big Bang? - 2
2024 Moving Styles for Kitchen Redesigns - 3
‘Ukrainian housewives’ and Skyranger delays – German defense poster child Rheinmetall is in hot water - 4
6 Fun Urban areas For Seniors To Travel - 5
How C-reactive protein outpaced ‘bad’ cholesterol as leading heart disease risk marker
What's inside Mexico's Popocatépetl? Scientists obtain first 3D images of the whole volcano
Internet goes (cocoa) nuts: The funniest reactions to 12 tonne theft of KitKat bars
Emotional wellness Matters: My Fight with Tension
Which European countries have mandatory or voluntary military service
Figure out How to Introduce Sunlight powered chargers on Your Rooftop securely
Gaza receiving over 70,000 cubic meters of water per day, COGAT claims
5 Side interests That Work on Psychological wellness
The Way to Fruitful Weight reduction: Individual Wellbeing Excursions
Ultra-Orthodox protests erupt across Israel on haredi IDF enlistment day













