Tuesday, Apr 04, 2023
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Tuesday, Apr 04, 2023
11:45 AM - 1:00 PM EDT
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Market Access of Rare Diseases - Learning from Current Experience in Oncology and Gene Therapies and Future Perspective
Parallel Session - Issue Panel
Overview
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.
Learning Objectives
- 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
Speaker(s):
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
Location: Flicker 3, Fontainebleau Miami Beach
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Location: Flicker 3, Fontainebleau Miami Beach
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2023040411:4513:00 003
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TUE, APR 4
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