
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
The Iconic, Instantly Recognizable Plastic Chair That's Known All Around The World - 2
Between 600 to 800 aid trucks entering Gaza daily since start of ceasefire, COGAT confirms - 3
Artemis II astronauts will see parts of the moon no human has before. Here’s how - 4
Tesla plans to expand production at German car plant - 5
6 Famous Urban communities for Shopping on the planet
Did Japan’s PM Actually Back the Memecoin Bearing Her Name?
Tuesday, April 7. Russia’s War On Ukraine: News And Information From Ukraine
Brazil expands pesticide packaging reverse logistics
Pulsars to the extreme: Spinning dead stars found blasting radio signals from the 'edge of their magnetic reach'
Divorce filings, feuds and legal trouble: The 'Mormon Wives' drama keeps piling up
Instructions to Upgrade the Proficiency of Your Sunlight powered chargers
I visited every country by 25. Antarctica showed me how much I still hadn't seen.
Florida has quietly become America's execution capital
The Extraordinary Excursion of Dental Embed Innovation












