ISSUE: Gene therapies are likely to reach the United States in 2017. Potentially curative in nature and targeting conditions with high unmet need, gene therapies are expected to have a high price. This creates a tension between manufacturers seeking compensation for their innovation, payers struggling with affordability, and technology assessment organizations wrestling with how to measure value. Bill Dreitlein will moderate and briefly present opinions from an ICER supported stakeholder forum. Clark Paramore will provide a manufacturer's perspective. John Watkins will provide a US payer's perspective. Adrian Towse will provide a European perspective, where some gene therapies are already licensed and where NICE recently completed a “mock appraisal” of a regenerative medicine. OVERVIEW: Gene therapies repair, dis-activate or replace dysfunctional genes that cause disease. They offer the promise of large health gains and lifelong curative benefits for patients with rare and debilitating conditions. They also create conflicts between manufacturers, payers, and policy makers. Manufacturers innovate the science, bear the high cost and risk of development, and must navigate regulation that is necessarily different to that for drugs. Thus, manufacturers want to be compensated accordingly. Payers question the durability of therapeutic effect and worry that the high costs of gene therapies will pose major affordability challenges if paid for using traditional methods. This raises concerns about the sustainability of this model of innovation for health systems. Furthermore, technology assessment groups will be asked to consider a value proposition based upon lifetime benefit, supported by data from clinical trials of much shorter duration. This session will debate the issues contributing to the tension around gene therapies. Panelists will represent the perspectives of their respective industries and comment on possible solutions including: use of real-world evidence, creative financing models, centers of excellence, and new value assessment frameworks.