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Cost-effectiveness and affordability of novel cardiovascular therapies: what physicians need to know
  1. Enrico Giuseppe Ferro1,2,
  2. Chia-Liang Liu1,3,
  3. Dhruv S Kazi1,2
  1. 1Richard A. and Susan F. Smith Center for Outcomes Research in Cardiology, Beth Israel Deaconess Medical Center, Boston, Massachusetts, USA
  2. 2Harvard Medical School, Boston, Massachusetts, USA
  3. 3Harvard T.H. Chan School of Public Health, Boston, Massachusetts, USA
  1. Correspondence to Dr Dhruv S Kazi, Beth Israel Deaconess Medical Center, Boston, MA 02215, USA; dkazi{at}bidmc.harvard.edu

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Cardiovascular clinicians are increasingly expected not only to optimise their patients’ health outcomes, but also to control costs and ensure the judicious use of healthcare resources. Clinicians must therefore have a rigorous understanding of cost-effectiveness in order to determine whether the increased effectiveness or safety of novel therapies justifies their higher costs, compared with usual care. These economic considerations are becoming increasingly salient as newly approved cardiovascular drugs enter the market at eye-wateringly high costs.1

Terms like cost-effectiveness, affordability and budget impact are sometimes erroneously used interchangeably, yet they can have profoundly different implications for patients, providers and payors. In this paper, we offer a foundational understanding of cost-effectiveness analysis (CEA) through three case studies of novel cardiovascular medications: tafamidis, proprotein convertase/subtilisin type 9 inhibitors (PCSK9i) and direct oral anticoagulants (DOACs). By helping readers confidently and correctly interpret CEAs, we hope to ensure that novel and effective cardiovascular therapies reach the patients most likely to benefit from them.

Case study 1: is tafamidis cost-effective?

The cost-effectiveness of a novel therapy is estimated by comparing the cost and benefit of a new therapy with the best available alternative (often the pre-existing standard of care), to generate a ratio known as the incremental cost-effectiveness ratio (ICER) (figure 1).2 If the ICER is equal to or lower than the society’s willingness-to-pay threshold, the new therapy is considered cost-effective in the population studied, that is, the health benefits are assumed to justify the additional costs. If the ICER exceeds the threshold, the therapy is not considered cost-effective. Willingness-to-pay levels vary among countries based on their economic development and political priorities. A consensus statement among US cardiovascular professional societies suggested that therapies with ICERs below $50 000/ quality-adjusted life-year (QALY) …

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Footnotes

  • Twitter @enricoferromd

  • Contributors All authors contributed to the drafting of the manuscript.

  • Funding This study was funded by Richard A and Susan F Smith Center for Outcomes Research in Cardiology at Beth Israel Deaconess Medical Center, Boston, MA, Institutional Funds.

  • Competing interests None declared.

  • Patient and public involvement Patients and/or the public were not involved in the design, or conduct, or reporting, or dissemination plans of this research.

  • Provenance and peer review Commissioned; internally peer reviewed.

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