Developing and Paying for Gene Therapies: How to Measure Value?

Boston, MA—May 24, 2017—The International Society for Pharmacoeconomics and Outcomes Research (ISPOR) hosted a session this morning at its 22nd Annual International Meeting in Boston, MA, USA that examined the issue of developing and paying for gene therapies.

The session, Developing and Paying for Gene Therapies: Can We Resolve the Conflicts? [IP18], was moderated by Bill Dreitlein, PharmD, Director of Pharmaceutical Policy, Institute for Clinical and Economic Review, Boston, MA, USA. Panelists included:

  • Adrian Towse, MA, MPhil, Director, Office of Health Economics, London, UK
  • John Watkins, PharmD, MPH, Pharmacy Manager, Formulary Development, Premera Blue Cross, Mountlake Terrace, WA, USA
  • Clark Paramore, MSPH, Head of Value Demonstration, BlueBird Bio, Cambridge, MA, USA

As gene therapies are projected to gain approvals in the United States this year, the issue of how to measure value for these innovative treatments has come front and center. Although gene therapies are potentially curative and target conditions with high unmet need, they are expected to have a high price once they come to market. This scenario is creating tension between manufactures seeking compensation for their innovation, payers struggling with affordability issues, and technology assessment organizations striving to measure value.

Bill Dreitlein, PharmD presented opinions from a stakeholder forum supported by the Institute for Clinical and Economic Review (ICER) and moderated the discussion. Clark Paramore, MSPH provided a manufacturer’s perspective, and John Watkins, PharmD, MPH offered a US payer’s viewpoint. Adrian Towse, MA, MPhil delivered a European outlook where some gene therapies are already licensed and where the National Institute for Health and Care Excellence (NICE) recently completed a “mock appraisal” of a regenerative medicine.

Additional information on the ISPOR 22nd Annual International Meeting can be found here. Released presentations from the conference can be found here. Interested parties can follow news at ISPOR’s press site and on social media using the conference hashtag #ISPORBoston.

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