- CONTACT US
- AFS
- Business
- Bussiness
- Car
- Career
- Celebrity
- Digital Products
- Education
- Entertainment
- Fashion
- Film
- Food
- Fun
- Games
- General Health
- Health
- Health Awareness
- Healthy
- Healthy Lifestyle
- History Facts
- Household Appliances
- Internet
- Investment
- Law
- Lifestyle
- Loans&Mortgages
- Luxury Life Style
- movie
- Music
- Nature
- News
- Opinion
- Pet
- Plant
- Politics
- Recommends
- Science
- Self-care
- services
- Smart Phone
- Sports
- Style
- Technology
- tire
- Travel
- US
- World
- エンタメ
- スポーツ
- 科学
- 経済

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
Last Christmas, 3 million viewers watched a Chiefs love story — will Bills fans fall just as hard this year? - 2
'Seditious behavior': Trump accuses Democrats who made video reminding the military not to follow illegal orders of a crime — but is it? - 3
Winona Ryder didn't take the 'Stranger Things' plot lightly. How 'otherworldly' grief and a kidnapping in her hometown informed her character. - 4
Jamaica reports deadly leptospirosis outbreak after Hurricane Melissa - 5
Eurovision Song Contest changes voting rules after controversial allegations against Israel
A definitive Manual for Smart dieting on a Tight spending plan
Step by step instructions to Guarantee the Life span of Your Dental Inserts: Support and Care Guide
Changes to CDC website spark debate over autism and vaccine misinformation
The Magnificence of Do-It-Yourself Skincare: Regular Recipes and Tips
Experience Arranging: Planning for Epic Excursions
My Excursion to a Better Way of life: Health Experiences
Instructions to Floss Appropriately and Forestall Gum Sickness
Washington resident contracts bird flu, first human case in U.S. since February
Medicine doesn’t just have ‘conscientious objectors’ − there are ‘conscientious providers,’ too













