Enhancing Collaboration for Patient-Centered Care in Rare Diseases

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

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

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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 comprehensive dialogue addressing the importance of collaboration in health care decision-making. Pak emphasizes the need for inclusive discussions involving researchers, clinicians, payers, and manufacturers to navigate the complexities of treatment access and affordability. Ciafaloni highlights the remarkable advancements in gene therapies and the imperative of data sharing to drive continued progress. Chen underscores the significance of identifying the right patients and fostering cooperation to enhance access and quality of care. Nance expresses gratitude for platforms facilitating such discussions and emphasizes the vital role of communication in navigating rapid changes in health care. The panelists collectively stress the importance of equitable access, efficacy, and collaboration in driving patient-centered care forward. Haumschild concludes by acknowledging the audience’s role in benefiting from and contributing to these essential discussions.

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