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Rare but Real: Navigating Rare Disease Product Development and Market Access Challenges
Session Chair(s)
James Valentine, JD, MHS
Director
Hyman, Phelps & McNamara, PC, United States
Experts representing industry, non-profit, research, regulatory, and payor perspectives will explore key hurdles and solutions to rare disease treatment development, from recruitment and diagnosis to late-stage development and access after accelerated approval.
Learning Objective : Identify hurdles and solutions to clinical trial recruitment, including early diagnosis and newborn screening; Identify approaches to patient stratification, disease understanding, and novel biomarkers, while de-risking late-stage development in competitive and high-failure landscapes; Examine challenges of market access and pricing after accelerated approval, and gain insights into addressing payer-specific concerns to ensure equitable access.
Speaker(s)
Panelist
Anish Bhatnagar, MD
Soleno Therapeutics Inc, United States
Chief Executive Officer
Panelist
Philip (P.J.) Brooks, PhD
National Center for Advancing Translational Sciences (NCATS), NIH, United States
Deputy Director, Division of Rare Diseases Research Innovation
Panelist
Matthew Klein, MD, MS
PTC Theraputics, United States
CEO
Panelist
Kim Quaintance-Lunn
Alexion, United States
Vice President, Head of Regulatory Science and Execution
Jamie Sullivan, MPH
EveryLife Foundation for Rare Diseases, United States
Director of Public Policy
Panelist
Kerry Jo Lee, MD
FDA, United States
Assoc. Dir. for Rare Diseases; Program Manager, Accelerating Rare Disease Cures
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