From Research to Clinic: Addressing Ethical and Policy Challenges Facing the Gene Therapy Ecosystem

PROJECT NARRATIVE The translational gene therapy ecosystem faces several interrelated challenges: 1) obtaining sufficient evidence of safety/efficacy from clinical trials to justify regulatory approval of new gene therapies to treat inherited diseases, various cancers, and other complex diseases; 2) establishing cost and payer coverage policies so patients have access to affordable treatments; and 3) generating sufficient safety and effectiveness evidence of approved gene therapies when used in the clinical setting. The project will develop recommendations and issue/policy briefs to help stakeholders in the translational gene therapy ecosystem in their deliberations and decision-making about addressing gene therapy challenges and associated ethical and policy issues and to identify ethical and policy issues within the translational gene therapy ecosystem that warrant additional normative and empirical investigation by various stakeholders.