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Evaluating Policies of Expanding vs. Restricting First-Line Treatment Choices: A Cost-Effectiveness Analysis Framework.

  • Jiao, Boshen1
  • Carlson, Josh J2
  • Garrison, Louis P Jr2
  • Basu, Anirban2
  • 1 The Comparative Health Outcomes, Policy, and Economics (CHOICE) Institute, School of Pharmacy, University of Washington, Seattle, WA; Department of Global Health and Population, Havard T.H. Chan School of Public Health, Boston, MA. Electronic address: [email protected].
  • 2 The Comparative Health Outcomes, Policy, and Economics (CHOICE) Institute, School of Pharmacy, University of Washington, Seattle, WA.
Published Article
Value in Health
Publication Date
Jan 06, 2024
DOI: 10.1016/j.jval.2023.12.013
PMID: 38191022


Healthcare payers often implement coverage policies that restrict the utilization of costly new first-line treatments. Cost-effectiveness analysis (CEA) can be conducted to inform these decisions by comparing the new treatment to an existing one. However, this approach may overlook important factors such as treatment effect heterogeneity and endogenous treatment selection, policy implementation costs, and diverse patient preferences across multiple treatment options. We aimed to develop a comprehensive CEA framework that considers these real-world factors, facilitating the evaluation of alternative policies related to expanding or restricting first-line treatment choices. We introduced a metric of incremental cost-effectiveness ratio (ICER) that compares an expanded choice set (CS) including the new first-line treatment to a restricted CS excluding the new treatment. ICER(CS) accounts for treatment selection influenced by heterogeneous treatment effects and policy implementation costs. We examined a basic scenario with two standard first-line treatment choices and a more realistic scenario involving diverse preferences towards multiple choices. To illustrate the framework, we conducted a retrospective evaluation of including versus excluding abiraterone acetate plus prednisone (ADT+AAP) as a first-line treatment for metastatic hormone-sensitive prostate cancer. The traditional ICERs for ADT+AAP versus ADT alone and ADT+ docetaxel were $104,269 and $206,324/quality-adjusted life year (QALY), respectively. The ICER(CS) for comparing an expanded CS with ADT+AAP to a restricted CS without ADT+AAP was $123,179/QALY. The proposed framework provides decision-makers with policy-relevant tools, enabling them to assess the cost-effectiveness of alternative policies of expanding versus restricting patients' and physicians' first-line treatment choices. Copyright © 2024 International Society for Pharmacoeconomics and Outcomes Research, Inc. Published by Elsevier Inc. All rights reserved.

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