Push To Create Ultra-Rare Orphan Drug Designation Finds Resistance At US FDA

Agency officials don’t think a new definition or incentives will fix the issues related to development in rare diseases with only a handful of patients, as advocates continue pushing the idea.

roadblock
Without FDA support, passing legislation to create ultra-orphan incentives will become more difficult. • Source: Shutterstock

Efforts to create an ultra-rare disease designation and related incentives seem to have hit a roadblock at the US Food and Drug Administration.

More from Rare Diseases

More from Pink Sheet

Trump’s Tariffs Will Lead To ‘Instability’ And ‘Less Investment’ In UK

 

While the pharma industry appears to be exempt from US tariffs imposed by President Trump, a member of the UK House of Lords says the details are unknown and warned that uncertainty “leads to less investment” in business as a whole.

Industry Benefits As EU CTIS Hits Global Data Sharing Milestone

 

The EU Clinical Trials Information System has achieved primary registry designation in the International Clinical Trials Registry Platform in a move that is expected to reduce regulatory burden for companies and help them lower compliance costs by aligning with publication requirements in medical journals.

UK Health Secretary Admits VPAG ‘More Expensive’ Than Expected & Needs To Be ‘Resolved’

 

The UK government has listened to industry concerns about high clawback rates under the voluntary scheme and will review it in June in a bid to resolve the issue and “move on to bigger and more important things,” health secretary Wes Streeting says.