Statistical methods for handling compliance in randomised controlled trials of device interventions: a systematic review

TY - JOUR

T1 - Statistical methods for handling compliance in randomised controlled trials of device interventions: a systematic review

AU - Fiorentino, Francesca

AU - Nohpal de la Rosa, Consuelo

AU - Day, Emily

PY - 2022/9/29

Y1 - 2022/9/29

N2 - We aimed to review the extent to which analysis of randomised controlled trials (RCTs) of device interventions include methods to handle compliance to the study intervention as described in the protocol. We conducted a systematic review of the statistical methods used to handle compliance to a device intervention when estimating the effect of the device compared to another intervention in RCTs. We searched EMBASE, MEDLINE, PsychInfo and the Cochrane Central Register of Controlled Trials. We sought to evaluate what methods were used and how using these methods impacted the estimate of the effect size. 158 RCTs were identified for inclusion, of which only 21 (13%) described using a method to account for compliance to the device intervention, consisting of alternative analysis populations such as per-protocol, modified intention-to-treat or as-treated, alongside a primary ITT analysis. No causal inference methods were used. 14 (9%) studies included compliance as a factor in the analysis and investigated its effect on outcomes. Even though some studies consider methods to handle compliance, causal inference methods have not been well adopted in the analysis of device trials. Increased awareness of the applications of statistical methods to adjust for compliance is needed. [Abstract copyright: Copyright © 2022 The Author(s). Published by Elsevier Inc. All rights reserved.]

AB - We aimed to review the extent to which analysis of randomised controlled trials (RCTs) of device interventions include methods to handle compliance to the study intervention as described in the protocol. We conducted a systematic review of the statistical methods used to handle compliance to a device intervention when estimating the effect of the device compared to another intervention in RCTs. We searched EMBASE, MEDLINE, PsychInfo and the Cochrane Central Register of Controlled Trials. We sought to evaluate what methods were used and how using these methods impacted the estimate of the effect size. 158 RCTs were identified for inclusion, of which only 21 (13%) described using a method to account for compliance to the device intervention, consisting of alternative analysis populations such as per-protocol, modified intention-to-treat or as-treated, alongside a primary ITT analysis. No causal inference methods were used. 14 (9%) studies included compliance as a factor in the analysis and investigated its effect on outcomes. Even though some studies consider methods to handle compliance, causal inference methods have not been well adopted in the analysis of device trials. Increased awareness of the applications of statistical methods to adjust for compliance is needed. [Abstract copyright: Copyright © 2022 The Author(s). Published by Elsevier Inc. All rights reserved.]

KW - randomised controlled trials

KW - analysis

KW - adherence

KW - statistical methods

KW - device

KW - compliance

U2 - 10.1016/j.jclinepi.2022.09.015

DO - 10.1016/j.jclinepi.2022.09.015

M3 - Article

C2 - 36183902

SN - 1878-5921

JO - Journal of clinical epidemiology

JF - Journal of clinical epidemiology

M1 - S0895-4356(22)00236-0

ER -