PDUFA VII: US FDA Will Offer Additional Meetings To Boost Rare Disease Endpoint Development
If they reach a public disclosure agreement, a handful of sponsors will be admitted to a new pilot program that allows four meetings with the FDA on rare disease endpoint issues.
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Intra-agency group would be tasked with ensuring consistent use of the expedited pathway across the FDA, with duties potentially including development of best practices, product review team training, and advising on withdrawals, according to the Senate's bipartisan user fee legislation.
‘Umbrella’ program is intended to ensure all rare disease and related programs at CDER are working together, which seems similar to the rare disease center of excellence concept that stakeholders want to coordinate activities across the FDA.
Moderna has adopted more inclusionary enrollment criteria and real-time epidemiology monitoring for its RSV vaccine program, while Pfizer is expanding its internal ‘Lightspeed’ approach. US FDA is considering how to extend the fast, frequent and informal interactions.