ISSUE: GTx represents a new era of medicine, offering the potential for transformational benefits for patients, health systems and society. Health Technology Assessment (HTA) of GTx can be challenging for a variety of reasons, including clinical evidence generation; uncertainty; assessment of cost and affordability; and narrow value perspectives such as limitations of traditional quality of life measures.

The main objective of the panel will be to reconcile the various challenges facing value assessment for GTx, and put forward evidence-based recommendations to overcome these barriers. The panel will underscore the perspectives and priorities of patients, health economists, and industry, while highlighting policy and methodological changes that need to be made to current HTA practice and payer decision-making.

OVERVIEW: The moderator (Adrian Towse) will briefly introduce the topic, and summarize the findings of a literature review and expert roundtable programme. He will then pose key questions to the presenters, who will each speak for approximately 15 minutes, providing their diverse perspectives on the issues and key considerations.

Louis Garrison, Professor Emeritus, University of Washington will set out the economic arguments including the success points and sticking points on HTA reform for gene therapies in different countries. Bhash Parasuraman, Vice President, Pfizer will comment from an industry perspective, highlighting the urgent need for policy change to facilitate timely access to innovative gene therapies. Mark Skinner, President/CEO, Institute for Policy Advancement, Ltd. who has previously led the World Federation of Hemophilia and National Hemophilia Foundation will provide a patient perspective.

The panel will discuss which areas of HTA are in need of change and debate possible solutions such as outcomes-based pricing and other innovative payment schemes.