Report: Health Economics Outcomes are Rarely Considered in Adaptive Clinical Trial Designs

Opportunities Are Potentially Being Missed to Improve Healthcare Decision Making

Lawrenceville, NJ, USA—April 30, 2019—Value in Health, the official journal of ISPOR—the professional society for health economics and outcomes research, announced today the publication of new research showing that consideration is rarely given to the health economic analysis of adaptive clinical trials. As a result, opportunities to use health economic outcomes in the design and analysis of adaptive trials are being missed. The paper, “A Review of Clinical Trials With an Adaptive Design and Health Economic Analysis,” was published in the April 2019 issue of Value in Health

Both health economics and adaptive designs aim to enable efficient and accurate decision making. Health economic analysis facilitates the comparison of the costs and benefits of alternative technologies to determine which is the most cost-effective. Adaptive designs use data collected as a trial progresses to inform modifications to the trial without compromising the validity or integrity of the study. However, while adaptive designs have the potential to benefit patients and healthcare providers and are becoming a popular alternative to the traditional fixed sample size trial in appropriate clinical circumstances, they could impact accuracy by introducing bias into health economic analyses. It has been unclear, historically, whether the methods are typically considered together in the design, analysis, and reporting of trials.

To establish how often and effectively health economic outcomes are used in the design, analysis, and reporting of clinical trials that have adaptive designs, the authors assessed the results of such trials registered and published up to August 2016. A total of 553 articles were identified. Of these, 37 trials met the inclusion criteria and were subject to full data extraction.

In the review of these 37 clinical trials, 3 used health economic outcomes in the design, and none seemed to appropriately adjust the health economic outcomes, where thought to be required, to account for biases introduced by the adaptive study design. Of the 19 trials with results, 1 used health economic outcomes at the interim analysis. The reporting of health economic results was suboptimal for 17 of the 19 trials with published results.

“Using health economics and adaptive designs together could help us make healthcare decisions more efficiently and save scarce resources,” said author Laura Flight, School of Health and Related Research (ScHARR), University of Sheffield, Sheffield, England, United Kingdom. “Before this approach is used in practice it is important to have a clear understanding of how adaptive designs and health economics are used together in the design, analysis, and reporting of clinical trials. Our review has shown that further work is needed to establish whether adaptive designs and health economic analyses can be used together to increase the efficiency of health technology assessments without compromising accuracy.”

The study was funded by the National Institute for Health Research Doctoral Research Fellowship Programme (Grant Reference Number DRF-2015-08-013). The views expressed are those of the authors and not necessarily those of the NIHR or the Department of Health and Social Care.



ISPOR, the professional society for health economics and outcomes research (HEOR), is an international, multistakeholder, nonprofit dedicated to advancing HEOR excellence to improve decision making for health globally. The Society is the leading source for scientific conferences, peer-reviewed and MEDLINE®-indexed publications, good practices guidance, education, collaboration, and tools/resources in the field.

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Value in Health (ISSN 1098-3015) is an international, indexed journal that publishes original research and health policy articles that advance the field of health economics and outcomes research to help healthcare leaders make evidence-based decisions. The journal’s 2017 impact factor score is 5.494. Value in Health is ranked 3rd among 94 journals in healthcare sciences and services, 3rd among 79 journals in health policy and services, and 6th among 353 journals in economics. Value in Health is a monthly publication that circulates to more than 10,000 readers around the world.

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