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.
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Influence of expedited review pathways and regulatory incentives is on display in 2020’s novel approvals, which have a composition reassuringly consistent with recent years.