
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)
最近の投稿
- 1
Thousands of Walgreens nasal spray bottles recalled. See which ones. - 2
Survey: Canteen Cups With Great Warm Protection Impact - 3
She was the supermodel dubbed 'The Face' in the '80s. Joining OnlyFans in her 60s taught her a lot. - 4
Blood pressure drug recalled for possible cross-contamination - 5
Steinmeier honours Italian 'guest workers' who rebuilt German economy
Best Veggie lover Dinner: What's Your Plant-Based Pick?
FDA updates risk classification for voluntary shredded cheese recall
Farmers call for French blockades over cow disease cull
Trump says Venezuela will start 'turning over' oil to the U.S. Is that the reason he toppled Maduro — or is it something else?
German politician urges more face-to-face interaction in digital age
7 Logically Demonstrated Techniques for Better Rest
Seven deaths possibly linked to malfunctioning glucose monitors
Some are walking out. Some are shouting. Some are oblivious. How kids are reacting to THAT 'Wicked: For Good' scene
People can't get enough of this couple's Hallmark movie reviews. They don't know the painful backstory.













