Webinar recorded on August 13, 2026                             Pricing: Free for Members and Non-Members

Please note that this session is not accredited for continuing pharmacy education credit.

Program Description

Barth syndrome is an ultra-rare, X-linked mitochondrial disease affecting roughly 150 individuals in the United States, yet it carries a heavy clinical burden marked by progressive cardiomyopathy, skeletal myopathy, profound fatigue, and high early mortality. Conditions this rare pose distinct challenges for payers, who must weigh coverage decisions for therapies studied in necessarily small populations using trial designs that depart from traditional randomized controlled models. This webinar brings together a clinical genetics expert and a managed care medical director to examine the disease, its mitochondrial mechanism, and the evidence supporting elamipretide, alongside a candid discussion of the coverage and access questions ultra-rare therapies raise—from label interpretation and natural history comparators to value-based contracting and the broader question of how innovation in ultra-rare disease is funded. Participants will leave with a clearer understanding of how clinical realities in rare disease intersect with coverage policy, and where collaboration among manufacturers, clinicians, and payers can improve diagnosis, equitable access, and patient care.