
Navigating Benefit Design and Utilization Management in ATTR-CM
Experts weigh prior authorization, step therapy, and 12‑month coverage to balance access and cost for high‑value long‑term therapies.
Episodes in this series

This episode, titled ‘Navigating Benefit Design and Utilization Management in ATTR-CM,’ features the experts examining how payers should structure utilization management policies for ATTR-CM disease-modifying therapies. Dr. Alexander begins by reiterating the importance of patient-centered therapy selection, then addresses step therapy, expressing personal opposition to it in the current landscape given the absence of strong evidence-based rationale for designating one therapy as a default over another. He notes that tafamidis, as the first approved agent, might seem like a natural starting point, but that clinical data does not definitively support a step-through approach.
On duration of coverage, Dr. Alexander emphasizes that these are long-term medications without precise short-term tools for measuring treatment response or disease progression, making shorter reauthorization intervals of six months clinically impractical. He advocates for reauthorization periods of at least 12 months to allow for meaningful clinical assessment.
Dr. Alexander also highlights prior authorization complexity as a structural barrier to community adoption, noting that practices without dedicated specialty pharmacy resources often face delays or fail to get patients on therapy altogether, whereas large academic centers have the infrastructure to navigate the process more effectively. He calls for clearer communication from payers about required documentation to improve community-level access.
Dr. Haumschild adds that prior authorization serves a legitimate stewardship function for health plans working to sustain benefits, and that requiring documentation of diagnostic workup and adherence to inclusion and exclusion criteria ensures appropriate use. He suggests that when appropriateness is clearly established upfront, payers can move away from burdensome short reauthorization cycles and extend coverage to 12-month intervals, creating a more sustainable and accessible framework for both patients and providers.
In the final episode of this series, ‘Optimizing Access and Closing Data Gaps in ATTR-CM Management,’ the panelists conclude their conversation on ATTR-CM by examining how payers can implement utilization management strategies that maintain fair patient access while managing budget impact, the role of telemedicine in expanding specialist reach, and the critical data gaps that need to be addressed to strengthen both clinical and coverage decision-making.



