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Evaluation ViewAttendees 24

Skill Building

Importance of Building Relationships with Health Authorities: Case Study for NDA Review of Orphan Product

Thursday, October 5, 2023
2:00 PM – 3:00 PM EDT
Location/Room: 510B
Advanced Level
Description: A case study in building relationships with health authorities over the course of years assisted the review of a complex NDA leading to approval for a rare disease. The session will dive into details of the Priority review of an NDA including; a Refusal to File, use of a novel electronic diary used for the primary outcome, FDA queries, generation of a REMS, PDUFA clock extension and FDA inspections that needed the Sponsor and FDA to work together during COVID to complete the review of the NDA. Participants will be able to discuss an example of "partnering" with health authorities. We always hear this but we rarely discuss examples of how this "partnering" early on in development comes full circle leading to a great outcome for patients with this rare disease. In addition, participants can use this information to strategically plan for multiple realistic scenarios during an NDA Priority review.

Learning Objectives:

  • Upon completion, participants will be to utilize learnings from the discussion to assist with their negotiations with FDA.
  • Upon completion, participants will be to generate better regulatory strategies as they move into pre-NDA and NDA interactions with the FDA.
  • Upon completion, participants will be to describe the experience and precedence of how the Sponsor overcame novel challenges with getting an NDA approved.