Ethical Implications of Patient Access Restrictions

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

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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 engages in a discussion with Nance and Ciafaloni regarding the ethical challenges posed by patient subgroup restrictions on orphan drugs. Nance highlights the difficulties providers face when forced to deny treatment based on rigid approval criteria, emphasizing the need for flexible communication channels with decision makers. Ciafaloni emphasizes the societal responsibility to ensure equitable access to revolutionary therapies like gene therapy, urging collaborative efforts among stakeholders. The dialogue underscores the importance of open communication, involving key opinion leaders, providers, and patient advocacy groups to strike a balance between cost control and patient access. As they navigate these ethical dilemmas, the consensus emerges that prioritizing collaboration over financial considerations is paramount in ensuring fair and equitable access to life-altering treatments.

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