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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Article Info
Publication History
Published online: April 27, 2021
Accepted:
December 15,
2020
Identification
Copyright
© 2021 ISPOR-The Professional Society for Health Economics and Outcomes Research. Published by Elsevier Inc.
