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Accounting for Heterogeneity in Resource Allocation Decisions: Methods and Practice in UK Cancer Technology Appraisals

Published:April 27, 2021DOI:https://doi.org/10.1016/j.jval.2020.12.022

      Highlights

      • The availability of novel, more efficacious cancer therapies is increasing, resulting in significant treatment effect heterogeneity and complicated treatment and disease pathways. Technology appraisals (TAs) evaluate clinical and economic evidence to inform reimbursement decisions and resource allocation. Through critical appraisal of UK cancer TAs, we identify areas where considerations of heterogeneity can be improved. We focus on 3 cancer sites: colorectal, lung, and ovarian cancer, encompassing variation in screening, diagnostic and treatments pathways.
      • All TAs in this review used decision analytic modeling. The majority used partitioned survival models and evaluated aggregate outcomes of clinical trial populations. Only 2 models explicitly considered real-world patient heterogeneity in disease progression estimates. Moreover, predetermined subgroup analyses contained within the clinical studies that informed the TAs were rarely exploited in economic analyses.
      • This review highlights a paucity of information relating to the assessment of heterogeneity in colorectal, lung, and ovarian cancer TAs. We conclude that future cancer TAs should consider more flexible modeling approaches and apply real-world data to explore heterogeneity within their economic analyses, especially if the complexity of treatment and disease pathways is to be reflected.

      Abstract

      Objectives

      The availability of novel, more efficacious and expensive cancer therapies is increasing, resulting in significant treatment effect heterogeneity and complicated treatment and disease pathways. The aim of this study is to review the extent to which UK cancer technology appraisals (TAs) consider the impact of patient and treatment effect heterogeneity.

      Methods

      A systematic search of National Institute for Health and Care Excellence TAs of colorectal, lung and ovarian cancer was undertaken for the period up to April 2020. For each TA, the pivotal clinical studies and economic evaluations were reviewed for considerations of patient and treatment effect heterogeneity. The study critically reviews the use of subgroup analysis and real-world translation in economic evaluations, alongside specific attributes of the economic modeling framework.

      Results

      The search identified 49 TAs including 49 economic models. In total, 804 subgroup analyses were reported across 69 clinical studies. The most common stratification factors were age, gender, and Eastern Cooperative Oncology Group performance score, with 15% (119 of 804) of analyses demonstrating significantly different clinical outcomes to the main population; economic subgroup analyses were undertaken in only 17 TAs. All economic models were cohort-level with the majority described as partitioned survival models (39) or Markov/semi-Markov models. The impact of real-world heterogeneity on disease progression estimates was only explored in 2 models.

      Conclusion

      The ability of current modeling approaches to capture patient and treatment effect heterogeneity is constrained by their limited flexibility and simplistic nature. This study highlights a need for the use of more sophisticated modeling methods that enable greater consideration of real-world heterogeneity.

      Keywords

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