• June 3-6, 2024
  • San Diego Convention Center, California

Sessions

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Changing Clinical Trial Designs in the IRA Environment and Impacts on Stakeholders in Rare and Ultra-Rare Disease Space

Wednesday, June 05, 2024
Breakout Session
Orphan and Rare Disease
26A
Rare and ultra-rare disease settings present numerous challenges for clinical R&D and regulatory review due to small heterogeneous populations, recruitment difficulties, geographical barriers, scarcity of natural history studies to inform endpoint selection and clinical trial duration, and ethical challenges related to randomization of patients to placebo arms, to name a few. This panel will discuss various approaches to clinical trial design, including digitalization, shared single placebo arm, incorporating external control and longitudinal measures, AI implementation, as well as understanding regulatory and policy environment, incentives to payers, value to patients and their caregivers, and successful execution of novel clinical design programs (CDPs) and analytical approaches.
Moderator
Mariia Salova, MD, MS
Consultant II
Avalere
Speakers
Amy House
Head of Portfolio Strategy
Beacon Therapeutics
Primal Kaur, MD, MBA
Vice President, Global Development
Amgen
Matthew Klein, MD, MS, FACS
Chief Executive Officer and President
PTC Therapeutics
Mike Stodghill
Patient Perspective
Mackenzie's Mission
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