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

Ryan Haumschild, PharmD, MS, MBA, leads a discussion on factors that influence coverage of non-oncology gene therapies, exploring the balance between long-term cost savings and high initial prices, patient selection, and the durability of effect for these therapies.

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.

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