Cost-Effectiveness of Tafamidis Therapy for Transthyretin Amyloid Cardiomyopathy

• DOI: 10.1161/circulationaha.119.045093
• Publication Date: 2020-02-12T10:00:20Z
• Authors: Dhruv S. Kazi; Brandon K. Bellows; Suzanne J. Baron; Changyu Shen; David J. Cohen; John A. Spertus; Robert W. Yeh; Suzanne V. Arnold; Brett W. Sperry; Mathew S. Maurer; Sanjiv J. Shah
• Keywords: Male; Amyloid Neuropathies, Familial; Benzoxazoles; Cost-Benefit Analysis; Quality of Life; Humans; Female; Cardiomyopathies; Aged; 3. Good health

Background: In patients with transthyretin amyloid cardiomyopathy, tafamidis reduces all-cause mortality and cardiovascular hospitalizations slows decline in quality of life compared placebo. May 2019, received expedited approval from the US Food Drug Administration as a breakthrough drug for rare disease. However, at $225 000 per year, it is most expensive ever launched United States, its long-term cost-effectiveness budget impact are uncertain. We therefore aimed to estimate potential effect on health care spending. Methods: developed Markov model wild-type or variant cardiomyopathy heart failure (mean age, 74.5 years) using inputs ATTR-ACT trial (Transthyretin Amyloidosis Cardiomyopathy Clinical Trial), published literature, review documents, healthcare claims, national survey data. no disease–specific treatment (“usual care”) therapy. The reproduced 30-month survival, life, hospitalization rates observed ATTR-ACT; future projections used parametric survival control arm, constant hazards reduction arm. discounted costs quality-adjusted life-years by 3% annually examined key parameter uncertainty deterministic probabilistic sensitivity analyses. main outcomes were lifetime incremental ratio annual impact, assessed sector perspective. This study was independent sponsor. Results: Compared usual care, projected add 1.29 (95% interval, 0.47–1.75) an cost $1 135 (872 000–1 377 000), resulting $880 (697 564 000) life-year gained. Assuming threshold $100 gained current price, cost-effective 0% 10 simulations. A 92.6% price $16 563 would be necessary make 000/quality-adjusted life-year. Results sensitive assumptions related effectiveness tafamidis. Treating all eligible States (n=120 estimated increase spending $32.3 billion. Conclusions: Treatment produce substantial clinical benefit but greatly exceed conventional thresholds list price. On basis recent experience high-cost medications, access uptake this effective therapy may limited unless there large costs.