Abstract
BackgroundA randomized, controlled trial, intended to include 460 patients, is currently studying peroperative goal-directed hemodynamic treatment (GDHT) of aged hip-fracture patients. Interim efficacy analysis performed on the first 100 patients was statistically uncertain; thus, the trial is continuing in accordance with the trial protocol. This raised the present investigation’s main question: Is it reasonable to continue to fund the trial to decrease uncertainty? To answer this question, a previously developed probabilistic cost-effectiveness model was used. That model depicts (1) a choice between routine fluid treatment and GDHT, given uncertainty of current evidence and (2) the monetary value of further data collection to decrease uncertainty. This monetary value, that is, the expected value of perfect information (EVPI), could be used to compare future research costs. Thus, the primary aim of the present investigation was to analyze EVPI of an ongoing trial with interim efficacy observed.MethodsA previously developed probabilistic decision analytic cost-effectiveness model was employed to compare the routine fluid treatment to GDHT. Results from the interim analysis, published trials, the meta-analysis, and the registry data were used as model inputs. EVPI was predicted using (1) combined uncertainty of model inputs; (2) threshold value of society’s willingness to pay for one, quality-adjusted life-year; and (3) estimated number of future patients exposed to choice between GDHT and routine fluid treatment during the expected lifetime of GDHT.ResultsIf a decision to use GDHT were based on cost-effectiveness, then the decision would have a substantial degree of uncertainty. Assuming a 5-year lifetime of GDHT in clinical practice, the number of patients who would be subject to future decisions was 30,400. EVPI per patient would be €204 at a €20,000 threshold value of society’s willingness to pay for one quality-adjusted life-year. Given a future population of 30,400 individuals, total EVPI would be €6.19 million.ConclusionsIf future trial costs are below EVPI, further data collection is potentially cost-effective. When applying a cost-effectiveness model, statements such as ‘further research is needed’ are replaced with ‘further research is cost-effective and ‘further funding of a trial is justified’.Trial registrationClinicalTrials.gov NCT01141894
Highlights
A randomized, controlled trial, intended to include 460 patients, is currently studying peroperative goal-directed hemodynamic treatment (GDHT) of aged hip-fracture patients
When the primary outcome’s statistical uncertainty, as revealed by interim analysis, indicates need for further data collection, researchers may ask the question: Is it reasonable to continue to fund the trial to decrease uncertainty? Increasingly, resource allocation in health care considers the principles of cost-effectiveness in different jurisdictions; cost-effectiveness has become a key criterion for decision makers when deciding which health-care interventions should be made available in collectively funded health-care systems [3]
This section describes (1) the clinical trial’s design and efficacy data extracted from the interim analysis, (2) the decision-analytic model and model inputs used in our analyses, and (3) the expected value of perfect information (EVPI) estimation method
Summary
A randomized, controlled trial, intended to include 460 patients, is currently studying peroperative goal-directed hemodynamic treatment (GDHT) of aged hip-fracture patients. That model depicts (1) a choice between routine fluid treatment and GDHT, given uncertainty of current evidence and (2) the monetary value of further data collection to decrease uncertainty This monetary value, that is, the expected value of perfect information (EVPI), could be used to compare future research costs. Extending this rationale to clinical trials, after interim analysis, a trial should be continued when the costs of undertaking additional research are less than its benefits, in terms of reducing uncertainty This article describes such an interim analysis of a randomized controlled clinical trial of aged hip-fracture patients (ClinicalTrials.gov NCT01141894) and the estimation of the value of extending the trial on the basis of value of information analysis. Our present investigation’s primary and secondary aims were to (1) analyze expected value of perfect information (EVPI) when studying GDHT, and (2) provide decision support material when stakeholders must determine if it is reasonable to fund further data collection after the interim analysis
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