Value and Values in Payment for Gene Therapies

The ELSI Friday Forum was held on May 12, 2023.

Advances in gene therapies for monogenic disorders have brought hope of curative treatment. Since the Food and Drug Administration first approved a gene therapy in 2017, the pipeline has steadily increased such that 20 new gene therapies are expected to be approved each year starting soon. Health system decision makers around the world are facing complex issues related to value, affordability, and equity posed by the high upfront cost of these new therapies. Traditional health economics approaches to assess value are intricately linked to values, the normative aspects of resource allocation decisions. Building upon discussions in the CERA-funded Value Ethics Working Group, this panel presentation will provide an overview of pragmatic and ethical aspects of health technology assessment (HTA) and payment models for gene therapies from an international perspective. 

• Renske M.T. ten Ham, PhD, PharmD, MSc (The University Medical Center Utrecht)
• R. Brett McQueen, PhD (University of Colorado Anschutz Medical Campus)
• Moderated by Hadley S. Smith, PhD, MPSA (Harvard Medical School, Harvard Pilgrim Health Care Institute)