- 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
- 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
Disability rights activist and author Alice Wong dies at 51 - 2
Lula’s former human rights minister formally accused of sexual misconduct - 3
Palestinians reel under winter rains as Israel blocks Gaza shelter supplies - 4
ByHeart sued over recalled formula by parents of infants sickened with botulism - 5
Putin, Netanyahu discuss Middle East in phone call, Kremlin says
Instructions to Guarantee Kids Foster Solid Dental Propensities
All that You Really want to Be familiar with Dental Inserts Facilities
Instructions to Figure out the Various Phases of Cellular breakdown in the lungs
Building Tough Connections: Individual Bits of knowledge on Association
Tracking down the Right Equilibrium: Charges versus Personal Costs in Senior Protection.
Figure out How to Explore Your Direction to the Best Dental Embed Trained professional: A Far reaching Manual
Medicine doesn’t just have ‘conscientious objectors’ − there are ‘conscientious providers,’ too
Picking the Right Doctor prescribed Medication Inclusion in Senior Protection.
Vote in favor of Your Number one Smartwatch: Exactness and Style Matter












