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Noninferiority and equivalence tests in sequential, multiple assignment, randomized trials (SMARTs).

ABSTRACT: Adaptive interventions (AIs) are increasingly popular in the behavioral sciences. An AI is a sequence of decision rules that specify for whom and under what conditions different intervention options should be offered, in order to address the changing needs of individuals as they progress over time. The sequential, multiple assignment, randomized trial (SMART) is a novel trial design that was developed to aid in empirically constructing effective AIs. The sequential randomizations in a SMART often yield multiple AIs that are embedded in the trial by design. Many SMARTs are motivated by scientific questions pertaining to the comparison of such embedded AIs. Existing data analytic methods and sample size planning resources for SMARTs are suitable only for superiority testing, namely for testing whether one embedded AI yields better primary outcomes on average than another. This calls for noninferiority/equivalence testing methods, because AIs are often motivated by the need to deliver support/care in a less costly or less burdensome manner, while still yielding benefits that are equivalent or noninferior to those produced by a more costly/burdensome standard of care. Here, we develop data-analytic methods and sample-size formulas for SMARTs testing the noninferiority or equivalence of one AI over another. Sample size and power considerations are discussed with supporting simulations, and online resources for sample size planning are provided. A simulated data analysis shows how to test noninferiority and equivalence hypotheses with SMART data. For illustration, we use an example from a SMART in the area of health psychology aiming to develop an AI for promoting weight loss among overweight/obese adults. (PsycINFO Database Record (c) 2020 APA, all rights reserved).

SUBMITTER: Ghosh P

PROVIDER: S-EPMC7061067 | biostudies-literature |

REPOSITORIES: biostudies-literature

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Project description:BACKGROUND: Standards for reporting clinical trials have improved the transparency of patient-important research. The Consolidated Standards of Reporting Trials (CONSORT) published an extension to address noninferiority and equivalence trials. We aimed to determine the reporting quality of prostaglandin noninferiority and equivalence trials in the treatment of glaucoma. METHODS: We searched, independently and in duplicate, 6 electronic databases for eligible trials evaluating prostaglandins. We abstracted data on reporting of methodological criteria, including reporting of per-protocol [PP] and intention-to-treat [ITT] analysis, sample size estimation with margins, type of statistical analysis conducted, efficacy summaries, and use of hyperemia measures. RESULTS: Trials involving the four major prostaglandin groups (latanoprost, travoprost, bimatoprost, unoprostone) were analyzed. We included 36 noninferiority and 11 equivalence trials. Seventeen out of the included 47 trials (36%, 95% Confidence Intervals [CI]: 24-51) were crossover designs. Only 3 studies (6%, 95% CI: 2-17) reported a presented results of both ITT and PP populations. Twelve studies (26%, 95% CI: 15-39) presented only ITT results but mentioned that PP population had similar results. Thirteen trials (28%, 95% CI: 17-42) presented only PP results with no mention of ITT population results while 17 studies (36%, 95% CI: 24-51) presented only ITT results with no mention of PP population results. Thirty-four (72%, 95% CI: 58-83) of studies adequately described their margin of noninferiority/equivalence. Sequence generation was reported in 22/47 trials (47%, 95% CI: 33-61). Allocation concealment was reported in only 10/47 (21%, 95% CI: 12-35) of the trials. Thirty-five studies (74%, 95% CI: 60-85) employed masking of at least two groups, 4/47 (9%, 95% CI: 3-20) masked only patients and 8/47 (17%, 95% CI: 9-30) were open label studies. Eight (17%, 95% CI: 9-30) of the 47 trials employed a combined test of noninferiority and superiority. We also found 6 differing methods of evaluating hyperemia. CONCLUSION: The quality of reporting noninferiority/equivalency trials in the field of glaucoma is markedly heterogeneous. The adoption of the extended CONSORT statement by journals will potentially improve the transparency of this field.

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Noninferiority and equivalence tests in sequential, multiple assignment, randomized trials (SMARTs).

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