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The number needed to treat and relevant between-trial comparisons of competing interventions.

ABSTRACT: The number needed to treat (NNT) is considered an intuitive as well as popular effect measure. The aims of this review were to 1) explain why we cannot compare trial-specific NNT estimates for the competing treatments evaluated in different randomized controlled trials (RCTs) and 2) outline the principles of how relative treatment effects of different trials can be compared and results can be presented as NNT, without violating the principles of valid between-trial comparisons. Our premise is that ratio measures for relative treatment effects of response outcomes are less prone to effect modification than absolute difference measures of response outcomes. Accordingly, any between-trial comparisons of the efficacy of competing interventions using the study-specific ORs are less likely to be invalid or biased than comparisons based on the study-specific NNT estimates. However, treatment-specific ORs obtained from a meta-analysis or taken directly from an individual study can be transformed into consistent treatment-specific NNT estimates that allow for credible comparisons of treatments when these ratio measures are applied to the same reference response estimate. The theoretical discussion is illustrated with a relevant indirect comparison of biologics for the treatment of ulcerative colitis. Between-trial comparisons directly based on the NNT of individual trials may result in erroneous conclusions and should be avoided. Treatment-specific NNT estimates need to be based on the same probability of response with the common reference treatment against which the interventions are compared.

SUBMITTER: Jansen JP

PROVIDER: S-EPMC6298880 | biostudies-literature | 2018

REPOSITORIES: biostudies-literature

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The number needed to treat and relevant between-trial comparisons of competing interventions.

Jansen Jeroen P JP Khalid Javaria Mona JM Smyth Michael D MD Patel Haridarshan H

ClinicoEconomics and outcomes research : CEOR 20181214

The number needed to treat (NNT) is considered an intuitive as well as popular effect measure. The aims of this review were to 1) explain why we cannot compare trial-specific NNT estimates for the competing treatments evaluated in different randomized controlled trials (RCTs) and 2) outline the principles of how relative treatment effects of different trials can be compared and results can be presented as NNT, without violating the principles of valid between-trial comparisons. Our premise is th ...[more]

PMID: 30588048

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Project description:Objective:Schizophrenia clinical trials commonly measure observed changes in Positive and Negative Syndrome Scale (PANSS) total score. However, it is more intuitive to think of response vs nonresponse, a binary outcome. Assessing binary outcomes enables calculation of number needed to treat (NNT) for therapeutic outcomes, number needed to harm (NNH) for adverse outcomes, and likelihood to be helped or harmed (LHH) to demonstrate benefit/risk tradeoffs. Here, NNT, NNH, and LHH were used to evaluate the clinical usefulness of aripiprazole lauroxil in patients with an acute schizophrenia exacerbation. Methods:Categorical efficacy and tolerability data were taken from the pivotal Phase 3 trial evaluating aripiprazole lauroxil for treatment of an acute exacerbation of schizophrenia. NNT and NNH values, with 95% CIs, were calculated in this post hoc analysis. Results:Using the intent-to-treat population for the pooled doses of aripiprazole lauroxil (441 mg [n=196] and 882 mg [n=204] q4w), responder rates (?30% improvement from baseline PANSS total score) were 35.3% for aripiprazole lauroxil arms vs 18.4% for placebo (n=196), yielding a NNT of 6 (95% CI: 5-11). Discontinuation rates due to adverse events (AEs) were higher among patients randomized to placebo than to either aripiprazole lauroxil dose. Akathisia was the only AE with an incidence ?5% in each aripiprazole lauroxil group and at least twice that of placebo (11.6%, 11.5%, and 4.3% of the patients receiving aripiprazole lauroxil 441 mg, 882 mg, and placebo, respectively), producing a NNH of 14 (95% CI: 9-33) for pooled aripiprazole lauroxil doses vs placebo. Calculating LHH for therapeutic response vs akathisia, aripiprazole lauroxil was 2.3 times more likely to result in a therapeutic response than an incident of akathisia. Conclusion:Using metrics of NNT, NNH, and LHH, aripiprazole lauroxil was an efficacious and well-tolerated intervention in a pivotal study in patients with an acute schizophrenia exacerbation.

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The number needed to treat and relevant between-trial comparisons of competing interventions.

Publications

The number needed to treat and relevant between-trial comparisons of competing interventions.

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