AFFORDABILITY OF NOVEL DISEASE-MODIFYING THERAPIES: TAFAMIDIS AND ATTR-CM
Author(s)
John Hsu, MD1, Mary Price, MA2, Jason H Wasfy, MD3, Joy Shi, PhD2, Vicki Fung, PhD2, David Cheng, PhD4, Justin Grodin, MD5, Mathew S. Maurer, MD6, Mike Chernew, PhD7, Joseph Newhouse, PhD7;
1Mongan Institute, Clinical Economics and Policy Analysis Group, Director, Boston, MA, USA, 2Mongan Institute, Clinical Economics and Policy Analysis Group, Boston, MA, USA, 3MGH/HMS, Boston, MA, USA, 4MGH, Boston, MA, USA, 5UTSW, Dallas, TX, USA, 6Columbia University, New York, NY, USA, 7Harvard University, Boston, MA, USA
1Mongan Institute, Clinical Economics and Policy Analysis Group, Director, Boston, MA, USA, 2Mongan Institute, Clinical Economics and Policy Analysis Group, Boston, MA, USA, 3MGH/HMS, Boston, MA, USA, 4MGH, Boston, MA, USA, 5UTSW, Dallas, TX, USA, 6Columbia University, New York, NY, USA, 7Harvard University, Boston, MA, USA
OBJECTIVES: Disease-modifying therapies, e.g., tafamidis, can reduce mortality but may be very expensive even for Medicare beneficiaries. We examined the association between Medicare Part D coverage generosity on tafamidis initiation and adherence among beneficiaries newly diagnosed with transthyretin amyloid cardiomyopathy (ATTR-CM) between 2017-2023 (US tafamidis approval was in May 2019).
METHODS: Using 100% traditional fee-for-service Medicare (TM) data, we identified beneficiaries with a new ATTR-CM diagnosis, the month of tafamidis initiation, then examined therapy adherence by coverage generosity. We defined adherence as having adequate drug supply for over 80% of days covered. We used logistic and linear regression models to compare 2023 initiation and adherence among subjects with standard versus employer-supplement insurance (enhanced) coverage, adjusting for individual characteristics. During the study period, the standard Part D coverage had no annual OOP spending limit.
RESULTS: Among 24,885,802 TM beneficiaries, 50% had standard and 13% enhanced coverage; most newly diagnosed patients (e.g., 88.9% with standard and 80.2% with enhanced coverage in 2023) did not start therapy. Mean tafamidis OOP spending for a full-year supply was $16,601 (standard Part D), and $1,377 (enhanced). After adjustment, newly diagnosed beneficiaries with enhanced vs. standard coverage were more likely to start therapy, e.g., OR=2.02 in 2023, 95%CI:1.72-2.36; adjusted adherence was substantially lower among beneficiaries with standard compared with enhanced coverage (e.g., OR=0.26 in 2023, 95%CI: 0.17-0.39; 94% adherence among those with enhanced coverage).
CONCLUSIONS: Beneficiaries with standard Part D benefits were less likely to be treated after their ATTR-CM diagnosis or stay adherent to therapy, than those with more generous benefits. By 2025, Part D standard benefits have improved with a beneficiary OOP maximum ($2,000/year) for on-formulary drugs, but remain less generous than under ESI coverage. Drug affordability in the Medicare program could remain a barrier for high-cost disease-modifying therapies.
METHODS: Using 100% traditional fee-for-service Medicare (TM) data, we identified beneficiaries with a new ATTR-CM diagnosis, the month of tafamidis initiation, then examined therapy adherence by coverage generosity. We defined adherence as having adequate drug supply for over 80% of days covered. We used logistic and linear regression models to compare 2023 initiation and adherence among subjects with standard versus employer-supplement insurance (enhanced) coverage, adjusting for individual characteristics. During the study period, the standard Part D coverage had no annual OOP spending limit.
RESULTS: Among 24,885,802 TM beneficiaries, 50% had standard and 13% enhanced coverage; most newly diagnosed patients (e.g., 88.9% with standard and 80.2% with enhanced coverage in 2023) did not start therapy. Mean tafamidis OOP spending for a full-year supply was $16,601 (standard Part D), and $1,377 (enhanced). After adjustment, newly diagnosed beneficiaries with enhanced vs. standard coverage were more likely to start therapy, e.g., OR=2.02 in 2023, 95%CI:1.72-2.36; adjusted adherence was substantially lower among beneficiaries with standard compared with enhanced coverage (e.g., OR=0.26 in 2023, 95%CI: 0.17-0.39; 94% adherence among those with enhanced coverage).
CONCLUSIONS: Beneficiaries with standard Part D benefits were less likely to be treated after their ATTR-CM diagnosis or stay adherent to therapy, than those with more generous benefits. By 2025, Part D standard benefits have improved with a beneficiary OOP maximum ($2,000/year) for on-formulary drugs, but remain less generous than under ESI coverage. Drug affordability in the Medicare program could remain a barrier for high-cost disease-modifying therapies.
Conference/Value in Health Info
2026-05, ISPOR 2026, Philadelphia, PA, USA
Value in Health, Volume 29, Issue S6
Code
CO161
Topic
Clinical Outcomes
Disease
SDC: Cardiovascular Disorders (including MI, Stroke, Circulatory), SDC: Rare & Orphan Diseases, STA: Biologics & Biosimilars