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How much should we–and can we–pay for gene therapies? Find out at ISPOR 2019

The 2019 International Society for Pharmacoeconomics and Outcomes Research (ISPOR) Annual Meeting has kicked off in New Orleans. One interesting Panel that I am moderating is titled “How much should we – and can we- pay for gene therapies?” I am serving as the moderator and the panelists on this issue panel include:

  • Anupam B. Jena, MD, PhD. Ruth L. Newhouse Associate Professor, Department of Health Care Policy, Harvard Medical School.
  • Jeremy Schafer, PharmD, MBA. Senior Vice President, Precision for Value.
  • Ramesh Arjunji, PhD. Senior Director, Blobal HEOR & RWE, AveXis.

The topic is described as follows.

Issue: As the first gene therapies enter the market, they offer the potential to
stop the progression of previously incurable diseases, such as leukodystrophies
and sickle cell disease. At the same time, high pricing and uncertain longterm
benefits have caused insurers and policymakers to question the value of these
therapies. Current prices of launched treatments range from over $300,000 USD
to $850,000 USD per treated patient, with future gene therapies expected to cost
in the millions. Even if value can be established at these price points, financing
these treatments poses significant challenges to insurers. Affordability, future
innovation, and patient access depend on the identification of pricing and
payment mechanisms for gene therapies that meet the diverse needs of
patients, insurers, manufacturers, and others.

OVERVIEW: This panel will debate the standards by which gene therapies should be priced and will explore opportunities for novel pricing and payment mechanisms. Jason Shafrin will moderate the panel and will provide an overview of the current and future landscape for gene therapies, framing key questions: What role should cost-effectiveness analyses play in determining appropriate price points? What approaches should be used to account for the uncertainty in valuing expected long-term benefits? How should insurers bear the costs of
treatments that generate long-term benefits to patients and potentially to different, future insurers? Anupam Jena will review the role of innovative pricing mechanisms and budget thresholds from an academic economist’s perspective. Jeremy Schafer, as head of a payer advisor team and as a former payer, will reflect on the real-world budget constraints of insurers and the need for protections to ensure against continued health care cost growth. Ramesh Arjunji will represent the manufacturer’s perspective and will discuss the unique
considerations for rare disease innovation and the impact on patients of access

The panel takes place at 5pm in Room 278-282. I hope to see you there!

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