Cost-Effectiveness of Transthyretin Stabilizing Agents for the Treatment of Transthyretin Amyloid Cardiomyopathy (ATTR-CM)
Author(s)
Kanya K. Shah, MBA, MS, PharmD1, Sodam Kim, PharmD1, Marina Richardson, PhD, MSc2, WOOJUNG LEE, BS, PharmD, PhD2, Michael T. Kim, PharmD1, Bertha De Los Santos, MS, PharmD1, Jason H Wasfy, MD, MPhil3, Dmitriy Nikitin, MSPH2, David M Rind, MD, MSc2, Daniel Touchette, MA, PharmD1, Aaron N. Winn, MPP, PhD1;
1University of Illinois Chicago, Department of Pharmacy Systems, Outcomes, and Policy, Chicago, IL, USA, 2Institute for Clinical and Economic Review, Boston, MA, USA, 3Mass General Brigham and Harvard Medical School, Boston, MA, USA
1University of Illinois Chicago, Department of Pharmacy Systems, Outcomes, and Policy, Chicago, IL, USA, 2Institute for Clinical and Economic Review, Boston, MA, USA, 3Mass General Brigham and Harvard Medical School, Boston, MA, USA
OBJECTIVES: Tafamidis and acoramidis are transthyretin stabilizing agents (TTR-SA) indicated for transthyretin amyloid cardiomyopathy (ATTR-CM). We assessed the cost-effectiveness of TTR-SAs added to supportive care (SC) compared to SC alone in managing ATTR-CM from the US healthcare-sector perspective.
METHODS: Based on New York Heart Association (NYHA) Functional Classes I-IV, a Markov model simulated ATTR-CM disease progression with and without TTR-SAs over a lifetime horizon with 6-month cycles and 3% discount rate for health outcomes and costs. Cardiovascular-related hospitalizations were incorporated as transient events. Transition probabilities were sourced from a French Health Technology assessment of tafamidis. Mortality was modeled using US lifetables, published NYHA class-specific mortality hazards, and clinical trial survival. The annual medication cost was $163,000, estimated by discounting the acoramidis wholesale acquisition price by 27.5%. Outcomes were total life years (LYs), quality-adjusted life years (QALYs), equal value life years (evLYs), costs, and years in NYHA Class I/II. Sensitivity and scenario analyses were conducted.
RESULTS: Compared to SC alone, TTR-SAs plus SC had an incremental total cost of $663,000, an additional 1.4 LYs, 0.9 QALYs, 1.2 evLYs, and 0.9 years in NYHA Class I/II. Incremental cost-effectiveness ratios (ICERs) were $740,000 per QALY gained, $479,000 per LY gained, $532,000 per evLY gained, and $635,000 per additional year in NYHA Class I/II. At the non-discounted $225,000 annual drug price, an 88.8% discount would be required to achieve an ICER of $150,000 per QALY gained. Results were sensitive to NYHA-state utility, mortality hazards, cohort age, hospitalization costs, and NYHA Class IV hospitalization disutility inputs. In all probabilistic sensitivity analyses iterations, TTR-SAs were not cost-effective at common thresholds ($50,000-$200,000 per QALY).
CONCLUSIONS: Treatment with TTR-SAs plus SC led to improved health outcomes at higher costs compared to SC alone. At the assumed price, TTR-SAs would require a substantial discount to achieve commonly used cost-effectiveness thresholds in the US.
METHODS: Based on New York Heart Association (NYHA) Functional Classes I-IV, a Markov model simulated ATTR-CM disease progression with and without TTR-SAs over a lifetime horizon with 6-month cycles and 3% discount rate for health outcomes and costs. Cardiovascular-related hospitalizations were incorporated as transient events. Transition probabilities were sourced from a French Health Technology assessment of tafamidis. Mortality was modeled using US lifetables, published NYHA class-specific mortality hazards, and clinical trial survival. The annual medication cost was $163,000, estimated by discounting the acoramidis wholesale acquisition price by 27.5%. Outcomes were total life years (LYs), quality-adjusted life years (QALYs), equal value life years (evLYs), costs, and years in NYHA Class I/II. Sensitivity and scenario analyses were conducted.
RESULTS: Compared to SC alone, TTR-SAs plus SC had an incremental total cost of $663,000, an additional 1.4 LYs, 0.9 QALYs, 1.2 evLYs, and 0.9 years in NYHA Class I/II. Incremental cost-effectiveness ratios (ICERs) were $740,000 per QALY gained, $479,000 per LY gained, $532,000 per evLY gained, and $635,000 per additional year in NYHA Class I/II. At the non-discounted $225,000 annual drug price, an 88.8% discount would be required to achieve an ICER of $150,000 per QALY gained. Results were sensitive to NYHA-state utility, mortality hazards, cohort age, hospitalization costs, and NYHA Class IV hospitalization disutility inputs. In all probabilistic sensitivity analyses iterations, TTR-SAs were not cost-effective at common thresholds ($50,000-$200,000 per QALY).
CONCLUSIONS: Treatment with TTR-SAs plus SC led to improved health outcomes at higher costs compared to SC alone. At the assumed price, TTR-SAs would require a substantial discount to achieve commonly used cost-effectiveness thresholds in the US.
Conference/Value in Health Info
2025-05, ISPOR 2025, Montréal, Quebec, CA
Value in Health, Volume 28, Issue S1
Code
P39
Topic
Economic Evaluation
Disease
No Additional Disease & Conditions/Specialized Treatment Areas, SDC: Cardiovascular Disorders (including MI, Stroke, Circulatory), SDC: Rare & Orphan Diseases