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

Published May 24, 2017
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.

###

  ABOUT ISPOR The International Society for Pharmacoeconomics and Outcomes Research (ISPOR) is a nonprofit, international, educational and scientific organization that promotes health economics and outcomes research excellence to improve decision making for health globally. Web: www.ispor.org | LinkedIn: http://bit.ly/ISPOR-LIn | Twitter: http://bit.ly/ISPOR-T (@ISPORorg) | YouTube: http://bit.ly/ISPOR-YT | Facebook: http://bit.ly/ISPOR-FB

Related Stories

ISPOR Europe 2026 Plenaries Announced

Sep 30, 2026

ISPOR—The Professional Society for Health Economics and Outcomes Research (HEOR) announced details for its ISPOR Europe 2026 conference. The conference is the leading global event in Europe for health economics and outcomes research (HEOR) and is scheduled for 8-11 November in Vienna, Austria with the theme, “Shaping the Future of Access Through Science and Policy.”

ISPOR and ISPE Publish Joint Real-World Evidence Transparency Position Statement

Sep 29, 2026

Transparency in research enables the public and decision makers to trust the science that underpins real-world evidence (RWE). ISPOR—The Professional Society for Health Economics and Outcomes Research and the International Society for Pharmacoepidemiology (ISPE) have published a joint RWE Transparency Position Statement.

New Study Sheds Light on QALY and Disability Debate

Sep 28, 2026

Value in Health, the official journal of ISPOR—The Professional Society for Health Economics and Outcomes Research, announced today the publication of a study showing no evidence that the use of quality-adjusted life years (QALYs) inherently disadvantages people with disabilities.
Your browser is out-of-date

ISPOR recommends that you update your browser for more security, speed and the best experience on ispor.org. Update my browser now

×