Sustainable Gene Therapy Coverage Approaches

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

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EP: 12.Sustainable Gene Therapy Coverage Approaches

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

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 moderates a discussion on managed care challenges in gene therapy adoption with Chen, focusing on the implications of a growing pipeline of gene therapies for rare genetic diseases. Chen highlights the urgency of providing access to life-saving therapies for patients with limited treatment options while navigating cost-containment measures. The discussion delves into the importance of robust clinical evidence in informing coverage decisions, stressing the need for established outcome measures and longitudinal data to assess treatment efficacy and quality-of-life improvements. Nance emphasizes the critical role of physicians in advocating for patients and conveying the profound impact of gene therapies on disease progression and patient outcomes. The panelists underscore the complexities of integrating gene therapies into health care systems, urging collaboration among stakeholders to develop sustainable coverage models that balance cost-effectiveness with patient access. Haumschild concludes by acknowledging the transformative potential of gene therapies and the ongoing efforts to overcome barriers to their widespread adoption in clinical practice.

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