Panelists:
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
Abstract: 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 in the near future, 20 new gene therapies are expected to be approved each year. 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 economic value are intricately linked to values, the normative aspects of resource allocation decisions. This panel presentation will provide an overview of pragmatic and ethical aspects of health technology assessment and payment models for gene therapies from an international perspective.
ELSI Friday Forum sponsored by NHGRI
Location and Address
Online