ISSUE: Most payers worldwide are grappling with the challenge of paying for very expensive but effective and potentially curative gene and other advanced therapy medicinal products (ATMPs). An exponential growth in the availability of new gene therapies and ATMPS is expected in the coming decade. Finding solutions on how societies can finance expensive but effective, potentially curative medicinal products in ways that enhance welfare is pressing. There are fundamental disagreements among stakeholders on these issues. In this Issues Panel, panelists will debate whether we can find common ground?

OVERVIEW: Dr. Campbell will moderate a session with Panelists discussing the issues and solutions to this problem. Dr. Steuten will provide an overview of recent and recommended changes to HTA methodologies and evidence-generation activities in nine European countries, plus Canada and Australia, that would enable the full value of ATMPs and gene therapies to be captured in HTA. She will illustrate these activities using examples of best practices internationally. Dr. Briggs will consider the evaluation of ATMPs within a fixed-threshold system such as the UK, including issues of budget impact and possible risk-sharing arrangements. Dr. Basu will discuss the role of budget impact in assessing value-based prices for gene therapies, present a case analysis of pricing gene therapy for sickle cell disease in the US, and demonstrate what different US state Medicaid programs can do to absorb the initial budget impact.