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

from ISPOR News & Press Releases at on April 30, 2019 at 08:20AM

Opportunities Are Potentially Being Missed to Improve Healthcare Decision Making

Lawrenceville, NJ, USA—April 30, 2019—Value in
, 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

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

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.

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.

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

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



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
(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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