Remdesivir’s Path To Orphan Designation Could Be Exploited By Others, Experts Warn
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.
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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.
The EU Orphan Regulation and the Paediatric Regulation have helped to increase the number of products developed for rare diseases and children, but they both have some shortcomings that need to be addressed, according to an evaluation of the legislation carried out by the EU authorities. The European pharmaceutical industry has warned against opening up the legislation, saying it is “critical to maintain a stable and predictable incentives framework."
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.