Gene Therapy: Understanding the Science, Assessing the Evidence, and Paying for Value 

This white paper was developed following a December 2016 meeting of ICER’s membership program, during which leaders from 20 payer and manufacturer organizations convened to share their perspectives on the future of gene therapy. The paper explores the scientific frontier being opened up by emerging gene therapies, with the potential for curing or substantially improving the lives of individuals who have had limited treatment options in the past. 

We also addresses the challenges associated with generating the evidence needed to make robust assessments of safety, effectiveness, and value of these treatments.  In particular, the paper explores the need to consider new mechanisms for pricing, payment, and delivery, including an analysis of different options for amortizing over multiple years payment for one-time curative treatments.

Date of review: December 2016

For questions or additional information, please contact info@icer.org.