Coverage of Gene Therapies: Balancing Cost, Clinical Impact, and Durability of Effect

EP: 1.Overview of Gene Therapy Approaches and Impacts

EP: 2.Key Factors in Gene Vector Selection

EP: 3.Viral Vectors in Gene Therapy

EP: 4.Navigating Gene Therapy Eligibility: The Role of Genetic Testing

EP: 5.Evaluating Transformative Gene Therapies

EP: 6.Shifting the Treatment Paradigm in Spinal Muscular Atrophy

EP: 7.Developments in Muscular Dystrophy Gene Therapy

EP: 8.Review of Clinical Evidence for Delandistrogene Moxeparvovec for DMD

EP: 9.Driving Clinical Insights: Harnessing Functional Assessments in DMD Research

EP: 10.Duchenne Muscular Dystrophy Treatment Landscape Review

EP: 11.Strategic Insights Into Evolving Gene Therapy Trends

EP: 12.Sustainable Gene Therapy Coverage Approaches

EP: 13.Interpreting Clinical Data to Guide Coverage Decisions for Rare Disease Therapies

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EP: 14.Coverage of Gene Therapies: Balancing Cost, Clinical Impact, and Durability of Effect

EP: 15.Innovative Payment Models for Gene Therapies

EP: 16.Ethical Implications of Patient Access Restrictions

EP: 17.Enhancing Collaboration for Patient-Centered Care in Rare Diseases

This is a video synopsis/summary of a Peer Exchange involving Ryan Haumschild, PharmD, MS, MBA; Jessica Nance, MD, MS; Kimberly C. Chen, DO, MSHLM; Emma Ciafaloni, MD, FAAN; and Mary Pak, MD, FACP.

Haumschild facilitates a conversation on the complexities of coverage determinations for non-oncology gene therapies with Chen and Ciafaloni. Chen highlights the challenge of balancing long-term cost savings with high initial prices, emphasizing collaboration with manufacturers and providers to develop alternative payment models. They discuss the impact of treatment durability on coverage decisions, cautioning against labeling therapies as cures and stressing the importance of long-term data collection to inform policy decisions. Ciafaloni underscores the need for nuanced evaluation of treatment durability and emphasizes the importance of involving disease experts in policy development to ensure patient-centric decisions. The discussion highlights the evolving understanding of treatment effectiveness and the importance of data-driven, patient-focused coverage determinations in the rapidly advancing field of gene therapy. Haumschild emphasizes the need for comprehensive, data-driven approaches to patient management, acknowledging the challenges of balancing treatment accessibility with cost considerations in care of rare diseases.

Video synopsis is AI-generated and reviewed by AJMC^® editorial staff.