Cost-Effectiveness Analysis Alongside Clinical Trials II—An ISPOR Good Research Practices Task Force Report

Ramsey, Scott D.; Willke, Richard J.; Glick, Henry A.; Reed, Shelby D.; Augustovski, Federico; Jönsson, Bengt; Briggs, Andrew; Sullivan, Sean D.

doi:10.1016/j.jval.2015.02.001

Cited by 622 publications

(637 citation statements)

References 154 publications

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“…None of the interactions were statistically significant ( p ≥ 0.172); given that most two‐arm trials are not powered to detect significant differences in mean costs or NMB 4, 62 and the variance around the interaction term is four‐fold higher than that for main effects 17, 23, this finding is likely to be common among economic evaluations of factorial trials.…”

Section: Methods For the Worked Examplementioning

confidence: 95%

“…Second, guidelines for economic evaluation recommend presenting uncertainty using cost‐effectiveness acceptability curves (CEACs) and the value of information 4. For two‐arm trials, CEACs can be based on one‐sided p ‐values from regression 35, while both the value of information and CEACs can be estimated from the mean incremental NMB and its standard error (SE) by assuming a normal distribution 40, 41, 42.…”

Section: Challenges For Economic Evaluation Of Factorial Trialsmentioning

confidence: 99%

“…should be considered at the start of the study. Although it may not be appropriate to exclude interactions from the analysis solely on the basis of prior beliefs, a priori considerations may identify components of NMB that need to be analysed separately (see recommendation 7).Any economic evaluation of factorial trials should follow best practice guidelines for trial‐based economic evaluation 4, 5, 65 and take account of the distribution of costs and benefits, missing data, censoring and correlations between costs and benefits.A clear decision rule, or combination of rules, determining the situations where interactions will be included in the base case analysis should be pre‐specified to avoid data dredging and bias. For clinical endpoints, a two‐stage testing approach is generally used, whereby interactions are only included in the main analysis if they are statistically significant in an initial test 16, 17, 21, 67; this approach increases statistical power but introduces a small amount of bias, which may be acceptable for analyses driven by statistical significance.…”

Section: Recommendationsmentioning

confidence: 99%

“…Any economic evaluation of factorial trials should follow best practice guidelines for trial‐based economic evaluation 4, 5, 65 and take account of the distribution of costs and benefits, missing data, censoring and correlations between costs and benefits.…”

Section: Recommendationsmentioning

confidence: 99%

“…There is an extensive literature on methods for trial‐based economic evaluation 4, 5, but currently little guidance on how such methods should be applied to different types of trial. In particular, no research has yet assessed the best methods for conducting economic evaluations alongside factorial trials, 1

One of the authors (HD) searched Medline via PubMed for (factorial[Title]) AND (‘economic evaluation’ OR ‘cost‐effectiveness’), searched past papers presented to the Health Economists' Study Group (HESG, http://www.hesg.org.uk) for the term ‘factorial’ and searched the British Library Electronic Theses Online Service (EThOS, http://ethos.bl.uk) for the terms ‘factorial’ and ‘cost’ in combination.

…”

Section: Introductionmentioning

confidence: 99%

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

Dakin

Gray

2017

Statistics in Medicine

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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.

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Section: Methods For the Worked Examplementioning

confidence: 95%

Section: Challenges For Economic Evaluation Of Factorial Trialsmentioning

confidence: 99%

Section: Recommendationsmentioning

confidence: 99%

Section: Recommendationsmentioning

confidence: 99%

…”

Section: Introductionmentioning

confidence: 99%

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

Dakin

Gray

2017

Statistics in Medicine

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Effect of a Community Health Worker–Led Multicomponent Intervention on Blood Pressure Control in Low-Income Patients in Argentina

Irazola

Mills

et al. 2017

JAMA

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174

182

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IMPORTANCE Despite extensive knowledge of hypertension treatment, the prevalence of uncontrolled hypertension is high and increasing in low-and middle-income countries.OBJECTIVE To test whether a community health worker-led multicomponent intervention would improve blood pressure (BP) control among low-income patients with hypertension.DESIGN, SETTING, AND PARTICIPANTS A cluster randomized trial was conducted in 18 centers for primary health care within a national public system providing free medications and health care to uninsured patients in Argentina. A total of 1432 low-income adult patients with uncontrolled hypertension were recruited between June 2013 and April 2015 and followed up to October 2016.INTERVENTIONS Nine centers (743 patients) were randomized to the multicomponent intervention, which included a community health worker-led home intervention (health coaching, home BP monitoring, and BP audit and feedback), a physician intervention, and a text-messaging intervention over 18 months. Nine centers (689 patients) were randomized to usual care. MAIN OUTCOMES AND MEASURESThe coprimary outcomes were the differences in systolic and diastolic BP changes from baseline to the end of follow-up of patients with hypertension. Secondary outcomes included the proportion of patients with controlled hypertension (BP <140/90 mm Hg). Three BP measurements were obtained at each of 2 baseline and 2 termination visits using a standard protocol, the means of which were used for analyses. RESULTSOf 1432 participants (mean age, 55.8 years [SD, 13.3]; 772 women [53.0%]), 1357 (94.8%) completed the trial. Baseline mean systolic BP was 151.7 mm Hg for the intervention group and 149.8 mm Hg for the usual care group; the mean diastolic BP was 92.2 mm Hg for the intervention group and 90.1 mm Hg for the usual care group. Systolic BP reduction from baseline to month 18 was 19.3 mm Hg (95% CI, 17.9-20.8 mm Hg) for the intervention group and 12.7 mm Hg (95% CI, 11.3-14.2 mm Hg) for the usual care group; the difference in the reduction was 6.6 mm Hg (95% CI, 4.6-8.6; P < .001). Diastolic BP decreased by 12.2 mm Hg (95% CI, 11.2-13.2 mm Hg) in the intervention group and 6.9 mm Hg (95% CI, 5.9-7.8 mm Hg) in the control group; the difference in the reduction was 5.4 mm Hg (95% CI, 4.0-6.8 mm Hg; P < .001). The proportion of patients with controlled hypertension increased from 17.0% at baseline to 72.9% at 18 months in the intervention group and from 17.6% to 52.2% in the usual care group; the difference in the increase was 20.6% (95% CI, 15.4%-25.9%; P < .001). No adverse events were reported.CONCLUSIONS AND RELEVANCE Low-income patients in Argentina with uncontrolled hypertension who participated in a community health worker-led multicomponent intervention experienced a greater decrease in systolic and diastolic BP than did patients who received usual care over 18 months. Further research is needed to assess generalizability and cost-effectiveness of this intervention and to understand which components may have contributed most to th...

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Cost-effectiveness of Mifepristone Pretreatment for the Medical Management of Nonviable Early Pregnancy

et al. 2020

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IMPORTANCE Early pregnancy loss (EPL) is the most common complication of pregnancy. A multicenter randomized clinical trial compared 2 strategies for medical management and found that mifepristone pretreatment is 25% more effective than the standard of care, misoprostol alone. The cost of mifepristone may be a barrier to implementation of the regimen. OBJECTIVE To assess the cost-effectiveness of medical management of EPL with mifepristone pretreatment plus misoprostol vs misoprostol alone in the United States. DESIGN, SETTING, AND PARTICIPANTS This preplanned. prospective economic evaluation was performed concurrently with a randomized clinical trial in 3 US sites from May 1, 2014, through April 30, 2017. Participants included 300 women with anembryonic gestation or embryonic or fetal demise. Cost-effectiveness was computed from the health care sector and societal perspectives, with a 30-day time horizon. Data were analyzed from July 1, 2018, to July 3, 2019. INTERVENTIONS Mifepristone pretreatment plus misoprostol administration vs misoprostol alone. MAIN OUTCOMES AND MEASURES Costs in 2018 US dollars, effectiveness in quality-adjusted lifeyears (QALYs), and treatment efficacy. Incremental cost-effectiveness ratios (ICERs) of mifepristone and misoprostol vs misoprostol alone were calculated, and cost-effectiveness acceptability curves were generated. RESULTS Among the 300 women included in the randomized clinical trial (mean [SD] age, 30.4

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Cost-Effectiveness Analysis Alongside Clinical Trials II—An ISPOR Good Research Practices Task Force Report

Cited by 622 publications

References 154 publications

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

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

Effect of a Community Health Worker–Led Multicomponent Intervention on Blood Pressure Control in Low-Income Patients in Argentina

Cost-effectiveness of Mifepristone Pretreatment for the Medical Management of Nonviable Early Pregnancy

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