Applying Value-Based Frameworks in ATTR-CM Management

In ‘Applying Value-Based Frameworks in ATTR-CM Management,’ our experts delve into how cost-effectiveness, budget impact, and real-world outcomes are being applied to evaluate ATTR-CM therapies when comparative trial data remains unavailable. Dr. Alexander identifies five key stakeholders in real-world evidence generation: payers, pharmaceutical companies, academic centers, community healthcare organizations, and patient advocacy groups, each playing a distinct role in developing meaningful data.

From his academic perspective, Dr. Alexander describes how Stanford works closely with its specialty pharmacy to track therapy utilization and link it to outcomes such as hospitalizations and mortality, with plans to integrate more patient-reported data over time. He emphasizes that meaningful real-world insights will require aggregation across multiple healthcare systems and geographic regions, and anticipates that collaborative data from these efforts will begin emerging within the next year or two. He also highlights the value of patient advocacy group surveys in capturing symptom burden before and after treatment initiation.

Dr. Alexander points to downstream cost implications as a particularly important area of measurement, citing data from the acoramidis trial showing a reduction in atrial fibrillation incidence, which carries meaningful economic consequences through reduced ablations, cardioversions, antiarrhythmic drug use, and related hospitalizations.

Dr. Haumschild reinforces these points, noting that real-world evidence should capture total cost of care rather than drug cost alone, and highlights the role of an amyloidosis clinical pharmacy specialist embedded within the cardiology clinic as a key contributor to data development, patient education, and pathway creation. Both panelists agree that a more complete evidence base will ultimately enable payers to better adopt and adapt to emerging outcomes data.

Our next episode, ‘Navigating Benefit Design and Utilization Management in ATTR-CM,’ further explores ATTR-CM, highlighting how clinical evidence and patient characteristics should inform benefit design, prior authorization criteria, step therapy, and duration of coverage for TTR stabilizers and silencers.