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Wednesday June 17, 2026 10:15am - 11:30am EDT
Component Type: Session
Level: Intermediate
CE: ACPE 1.25 Knowledge UAN: 0286-0000-26-609-L04-P; CME 1.25; RN 1.25

Senior FDA leaders will discuss evolving regulatory trends in rare disease development, including accelerated approval, innovative trial designs, patient engagement, and real-world evidence, followed by open Q&A with attendees.

Learning Objectives

Identify current FDA regulatory trends and scientific approaches shaping rare disease drug development; Apply FDA expectations around accelerated approval, surrogate endpoints, and innovative trial designs; Integrate patient voice and real-world evidence into development strategies to support benefit-risk assessment.

Chair

James Valentine, JD, MHS

Speakers
VK

Vijay Kumar

Acting Director OTP/CBER, FDA, United States
avatar for James Valentine

James Valentine

Director, Hyman, Phelps & McNamara, PC, United States
James Valentine advises medical product companies and patient advocacy organizations on regulatory strategy for the development and approval of drugs and biologics, with a focus on rare diseases. He has helped secure FDA approvals, and often works in areas without well-defined regulatory... Read More →
Wednesday June 17, 2026 10:15am - 11:30am EDT
201AB The Pennsylvania Convention Center 1101 Arch Street Philadelphia, PA 19107 USA
  08: RegPolicy-Strategy-GlobalCollaboration, Session |   02: ClinicalTrialOps-Innovation, Session

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