Optimizing Access and Closing Data Gaps in ATTR-CM Management

In the final episode, ‘Optimizing Access and Closing Data Gaps in ATTR-CM Management,’ the panelists explore how payers can balance fair patient access with sustainable budget management through thoughtful utilization management strategies. Dr. Alexander emphasizes that linking prescription approvals to real-world healthcare utilization data, such as cardiovascular intervention rates and hospitalization trends, can provide payers with meaningful post-approval benchmarks for evaluating therapy impact.

Dr. Alexander also highlights misdiagnosis as an underrecognized driver of inappropriate utilization, noting that he deprescribes ATTR therapies roughly half a dozen times per year due to false positive or misinterpreted technetium scans. He stresses the importance of adhering to American Society of Nuclear Cardiology guidelines, including the use of SPECT imaging rather than planar-only scans, and ensuring that monoclonal gammopathy workup has been completed and, where necessary, confirmatory biopsy performed. He frames prior authorization documentation as a valuable tool for catching these diagnostic errors before therapy initiation.

On the question of timely access, Dr. Alexander advocates for a collaborative model between amyloid centers of excellence and community practices, with telemedicine serving as a key enabler. He describes how telemedicine at Stanford has allowed his team to partner with providers hundreds of miles away, expediting diagnostic interpretation, therapy initiation, and genetic counseling for hereditary ATTR subtype stratification, all without requiring patients to travel.

Looking ahead, both panelists identify critical data gaps, particularly around treatment response measurement tools and optimal management strategies for patients with more advanced disease. Dr. Alexander draws a parallel to LDL monitoring in statin therapy, expressing the need for similarly precise biomarkers to guide decisions around treatment continuation, switching, or escalation to combination therapy. Dr. Haumschild reinforces this with a multiple myeloma analogy, noting that measurable treatment response benchmarks could help payers and providers make more informed, cost-effective escalation decisions.

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