The need to balance merits and limitations from different disciplines when considering the stepped wedge cluster randomized trial design

BMC Medical Research Methodology

Hendrik Koffijberg²  Johannes B. Reitsma⁴  Johannes J. M. van Delden³  Karel G. M. Moons⁴  Rieke van der Graaf³  Ingeborg van der Tweel¹  Esther de Hoop¹ 

[1]Department of Biostatistics and Research Support, University Medical Center Utrecht, Julius Center for Health Sciences and Primary Care, Utrecht 3508, GA, The Netherlands

[2]Department of Health Technology Assessment, University Medical Center Utrecht, Julius Center for Health Sciences and Primary Care, Utrecht 3508, GA, The Netherlands

[3]Department of Medical Humanities, University Medical Center Utrecht, Julius Center for Health Sciences and Primary Care, Utrecht 3508, GA, The Netherlands

[4]Department of Epidemiology, University Medical Center Utrecht, Julius Center for Health Sciences and Primary Care, Utrecht 3508, GA, The Netherlands

关键词: Biostatistics;    Research ethics;    Health economics;    Cluster randomized trial;    Stepped wedge design;    Epidemiologic research design;   

Others  :  1230332
DOI  :  10.1186/s12874-015-0090-2

 received in 2015-05-04, accepted in 2015-10-19,  发布年份 2015

【 摘 要 】

Background

Various papers have addressed pros and cons of the stepped wedge cluster randomized trial design (SWD). However, some issues have not or only limitedly been addressed. Our aim was to provide a comprehensive overview of all merits and limitations of the SWD to assist researchers, reviewers and medical ethics committees when deciding on the appropriateness of the SWD for a particular study.

Methods

We performed an initial search to identify articles with a methodological focus on the SWD, and categorized and discussed all reported advantages and disadvantages of the SWD. Additional aspects were identified during multidisciplinary meetings in which ethicists, biostatisticians, clinical epidemiologists and health economists participated. All aspects of the SWD were compared to the parallel group cluster randomized design. We categorized the merits and limitations of the SWD to distinct phases in the design and conduct of such studies, highlighting that their impact may vary depending on the context of the study or that benefits may be offset by drawbacks across study phases. Furthermore, a real-life illustration is provided.

Results

New aspects are identified within all disciplines. Examples of newly identified aspects of an SWD are: the possibility to measure a treatment effect in each cluster to examine the (in)consistency in effects across clusters, the detrimental effect of lower than expected inclusion rates, deviation from the ordinary informed consent process and the question whether studies using the SWD are likely to have sufficient social value. Discussions are provided on e.g. clinical equipoise, social value, health economical decision making, number of study arms, and interim analyses.

Conclusions

Deciding on the use of the SWD involves aspects and considerations from different disciplines not all of which have been discussed before. Pros and cons of this design should be balanced in comparison to other feasible design options as to choose the optimal design for a particular intervention study.

【 授权许可】

   
2015 de Hoop et al.

【 图 表 】

Fig. 1.

Fig. 1.

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