This Issue Panel will focus on current market access challenges with innovative cell and gene therapies in oncology and rare diseases and potential solutions for enabling patient access. It will address key access considerations in defining a compelling value proposition of a new therapy in diseases with low awareness as well as value-based pricing of one-time, high-cost “curative” therapies. In addition, it will highlight a prominent European HTA perspective on the valuation of cell and gene therapies.
To discuss the challenges of the standard value framework when applied to one-time “curative” therapies and potential solutions to address them
To outline current market access challenges with innovative therapies in oncology rare diseases and cell & gene therapies
To understand pragmatic challenges as well as opportunities in the health technology appraisal of oncology rare diseases and cell & gene therapies through the lens of German HTA
Yoojung Yang, PharmD, MS, Head of North America, HEOR, Vertex Pharmaceuticals
Lou Garrison, PhD, Professor Emeritus, The Comparative Health Outcomes, Policy, and Economics (CHOICE) Institute, University of Washington
Meriem Bouslouk-Marx, PhD, MSc International Health, G-BA Benefit Assessment Expert, AMNOG Orphan Drug Specialist, MBM Future Health
Samuel Ang, Global Access Lead, Takeda