Rare Pediatric Disease Designation Requests Skyrocket Ahead Of Voucher Program’s Sunset
US FDA received 110 designation requests in July, a high mark that reflects the 60 days needed to review such requests ahead of the 30 September statutory expiration; agency is allowing email submission of RPD and orphan drug designation requests during the COVID-19 pandemic and expects to roll out an online portal for orphan designation requests later this year.
You may also be interested in...
Congress should give FDA more flexibility to administer orphan designations, such as the ability to revoke designations later if the disease prevalence grows beyond the rare disease threshold.
US incentive program would gain only four more years, despite calls for a permanent extension.
Chair of the House Energy and Commerce Committee raises questions about the priority review voucher program’s value, suggesting another limited extension may be upcoming.