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

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. Department of Health Services, University of Washington, Seattle, USA. Hutchinson Institute for Cancer Outcomes Research and Division of Public Health Sciences, Fred Hutchinson Cancer Research Center, Seattle, USA. Department of Pediatrics, University of Washington, Seattle, USA. Clinical Research Division, Fred Hutchinson Cancer Research Center, Seattle, USA. National Heart, Lung and Blood Institute, National Institutes of Health, Bethesda, USA. The Emmes Company, LLC, Rockville, USA. Department of Pharmacy, University of Washington, Seattle, USA. 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. bdevine@uw.edu. Department of Health Services, University of Washington, Seattle, USA. bdevine@uw.edu.

PharmacoEconomics. 2021
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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.
Study details
Study Design : Economic Study
Language : eng
Credits : Bibliographic data from MEDLINE®/PubMed®, a database of the U.S. National Library of Medicine