Drug repurposing expands the therapeutic applications of existing medications, while repositioning examines drugs that never reached market. In neurodegeneration, the approach is described as a pragmatic shortcut into mid- to late-stage trials.
The FDA will drop its longtime requirement for two rigorous studies to approve new drugs, moving to a default position of requiring only one study. Commissioner Marty Makary says the change reflects modern scientific advances and aims to accelerate drug availability without compromising safety. The shift follows decades of increasing flexibility for rare and fatal diseases, with about 60% of first-of-a-kind drugs already approved based on single studies in recent years.
The FDA will now require only one adequate and well-controlled clinical trial, combined with confirmatory evidence, to approve new drugs, ending its longtime two-study standard dating back to the 1960s.
| NCT ID | Title | Status | Phase |
|---|---|---|---|
| NCT06619925 |
The Role of After-school Exercise Services in Promoting Myopia and Physical and Mental Health in Children |
NOT_YET_RECRUITING | |
| NCT05612230 |
A Lifecourse Map of Diseases in China |
UNKNOWN |
