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 Objectives
Identify hurdles and solutions to clinical trial recruitment, incl. early diagnosis and newborn screening; Explore 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.
Wednesday June 18, 2025 8:30am - 9:30am EDT
TBDWalter E. Washington Convention Center 801 Allen Y. Lew Place, NW Washington, DC 20001-3614 USA
