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The Use of Cost-Effectiveness Analysis in Sickle Cell Disease: A Critical Review of the Literature

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Abstract

Novel interventions for sickle cell disease (SCD) bring hope to patients, yet concern about the associated economic costs exists. Cost-effectiveness analysis (CEA) uses standardized methods, with robust underpinnings in health economics, to estimate the value of these interventions compared with usual care. However, because of the complexity and lifetime trajectory of SCD, CEAs are challenging to conduct. The objectives of this rapid review were to summarize the main characteristics, components, and results of published CEAs of existing interventions for SCD, identify research gaps, and provide directions for future analyses. We identified records through searches of bibliographic databases, from reference lists of relevant review articles, and through consultation with experts. A total of 13 CEAs met our inclusion criteria and were qualitatively synthesized. These evaluated blood transfusions (n = 2), hematopoietic stem cell transplantation (n = 1), pharmaceuticals (n = 2), hypothetical cell or genetic therapy (n = 1), screening programs (n = 4), and interventions for SCD treatment complications (n = 3). A limited number of potential SCD and treatment complications were evaluated. No study adopted a societal perspective in the base case, six studies examined lifetime cost-effectiveness, seven studies employed a Markov or discrete-event simulation model, and eight studies used an outcome metric that captured both quality and length of life. To better compare the value of emerging and current therapies, future CEAs should adopt a societal perspective incorporating both medical and nonmedical costs, comprehensively model SCD complexity using robust health economic simulation models over the patient’s entire lifespan, and capture the intervention’s effect on both survival and quality of life.

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Acknowledgements

The authors gratefully acknowledge the following collaborators: N. DiFronzo (National Heart, Lung, and Blood Institute); C. Henry, K. Johnson, D. Louden, A. Morgan, J. Rich (University of Washington); and W. Wright (Fred Hutchinson Cancer Research Center). The authors also appreciate the valuable insights and suggestions provided by the members of the Clinical and Economic Analysis Initiative Expert Panel.

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Authors and Affiliations

  1. The Comparative Health Outcomes, Policy, and Economics (CHOICE) Institute, University of Washington, 1959 NE Pacific Street, H-375T, Box 357630, Seattle, WA, 98195-7630, USA

    Boshen Jiao, Anirban Basu, Scott Ramsey & Beth Devine

  2. Department of Health Services, University of Washington, Seattle, USA

    Anirban Basu & Beth Devine

  3. Hutchinson Institute for Cancer Outcomes Research and Division of Public Health Sciences, Fred Hutchinson Cancer Research Center, Seattle, USA

    Joshua Roth & Scott Ramsey

  4. Department of Pediatrics, University of Washington, Seattle, USA

    M. Bender

  5. Clinical Research Division, Fred Hutchinson Cancer Research Center, Seattle, USA

    M. Bender

  6. National Heart, Lung and Blood Institute, National Institutes of Health, Bethesda, USA

    Ilsa Rovira

  7. The Emmes Company, LLC, Rockville, USA

    Traci Clemons

  8. Department of Pharmacy, University of Washington, Seattle, USA

    Dalyna Quach

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  1. Boshen Jiao
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Correspondence to Beth Devine.

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Funding

This research was, in part, funded by the National Institutes of Health (NIH) Agreement OTA OT3HL152448, OT3HL151434. The views and conclusions contained in this document are those of the authors and should not be interpreted as representing the official policies, either expressed or implied, of the NIH.

Conflict of interest

Boshen Jiao, Anirban Basu, Joshua Roth, M. Bender, Ilsa Rovira, Traci Clemons, Dalyna Quach, Scott Ramsey, and Beth Devine have no conflicts of interest that are directly relevant to the content of this article.

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Author contributions

BJ, AB, JR, MB, TC, IR, and BD designed research. BJ and BD performed research. BJ and DQ collected data. BJ, AB, JR, MB, DQ, SR, and BD analyzed and interpreted data. BJ wrote the first draft of the manuscript. BJ, AB, JR, MB, DQ, SR, TC, IR, and BD reviewed and revised the manuscript. All the authors approved the final version for submission.

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Jiao, B., Basu, A., Roth, J. et al. The Use of Cost-Effectiveness Analysis in Sickle Cell Disease: A Critical Review of the Literature. PharmacoEconomics 39, 1225–1241 (2021). https://doi.org/10.1007/s40273-021-01072-z

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