3 March 2017 - Paper presents recommendations for improving evidence generation and assessment methods for gene therapies; also analyses the options for innovative payment mechanisms that can balance clinical value with affordability.

The Institute for Clinical and Economic Review (ICER) has released a new white paper: Gene Therapy: Understanding the Science, Assessing the Evidence, and Paying for Value. 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. 

The paper 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 amortising over multiple years payment for one-time curative treatments.

Read ICER White Paper