Evidence map›Paper›PMID 28470760›Full record

ArticleStatistics in medicine2017

Economic evaluation of factorial randomised controlled trials: challenges, methods and recommendations.

Helen Dakin, Alastair Gray

Abstract read
In one paragraph

Article in Statistics in medicine, 2017. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 20 papers, 1 of them a synthesis that pooled it.

0numbers the graph read from it
0cells of the map it votes in
20citing papers in PubMed, 1 pooled it
–field-weighted citation impact
1 · What the graph read from it

What it found

Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.

The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.

2 · The registry

The trial behind it

Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.

Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.

3 · Its place in the literature

Who cites it

20 citing papers in PubMed, 1 synthesis or guideline pooled it.

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  18. Decision Making for Healthcare Resource Allocation: Joint v. Separate Decisions on Interacting Interventions.Medical decision making : an international journal of the Society for Medical Decision Making · 2018
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4 · The record

Corrections and comments

PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.

5 · Who and what money

Authors and funding

2 authors.

Helen DakinNuffield Department of Population Health, University of Oxford, U.K.ORCID http://orcid.org/0000-0003-3255-748X
Alastair GrayNuffield Department of Population Health, University of Oxford, U.K.ORCID http://orcid.org/0000-0003-0239-7278

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

Increasing numbers of economic evaluations are conducted alongside randomised controlled trials. Such studies include factorial trials, which randomise patients to different levels of two or more factors and can therefore evaluate the effect of multiple treatments alone and in combination. Factorial trials can provide increased statistical power or assess interactions between treatments, but raise additional challenges for trial-based economic evaluations: interactions may occur more commonly for costs and quality-adjusted life-years (QALYs) than for clinical endpoints; economic endpoints raise challenges for transformation and regression analysis; and both factors must be considered simultaneously to assess which treatment combination represents best value for money. This article aims to examine issues associated with factorial trials that include assessment of costs and/or cost-effectiveness, describe the methods that can be used to analyse such studies and make recommendations for health economists, statisticians and trialists. A hypothetical worked example is used to illustrate the challenges and demonstrate ways in which economic evaluations of factorial trials may be conducted, and how these methods affect the results and conclusions. Ignoring interactions introduces bias that could result in adopting a treatment that does not make best use of healthcare resources, while considering all interactions avoids bias but reduces statistical power. We also introduce the concept of the opportunity cost of ignoring interactions as a measure of the bias introduced by not taking account of all interactions. We conclude by offering recommendations for planning, analysing and reporting economic evaluations based on factorial trials, taking increased analysis costs into account. © 2017 The Authors. Statistics in Medicine published by John Wiley & Sons Ltd.

Indexed as

Antihypertensive AgentsBiasBiostatisticsComputer SimulationCost-Benefit AnalysisHumansHypertensionModels, StatisticalQuality-Adjusted Life YearsRandomized Controlled Trials as TopicRegression AnalysisAntihypertensive Agentscost-utility analysisfactorial designguidelinesrandomised controlled trialtrial-based economic evaluation

Identifiers

PMID28470760
PMCPMC5599939

What OpenQuestion holds

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Registered trials

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Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.