The cohort multiple randomized controlled trial design: a valid and efficient alternative to pragmatic trials?
Randomized controlled trials (RCTs)-the gold standard for evaluating the effects of medical interventions-are notoriously challenging in terms of logistics, planning and costs. The cohort multiple randomized controlled trial approach is designed to facilitate randomized trials for pragmatic evaluation of (new) interventions and is a promising variation from conventional pragmatic RCTs. In this paper, we evaluate methodological challenges of conducting an RCT within a cohort. We argue that equally valid results can be obtained from trials conducted within cohorts as from pragmatic RCTs. However, whether this design is more efficient compared with conducting a pragmatic RCT depends on the amount and nature of non-compliance in the intervention arm.
Department of Radiation Oncology.
Joanne M
JM
van der Velden
Department of Radiation Oncology.|Julius Center for Health Sciences and Primary Care, University Medical Center Utrecht, Utrecht, The Netherlands.
Helena M
HM
Verkooijen
Department of Radiation Oncology.|Julius Center for Health Sciences and Primary Care, University Medical Center Utrecht, Utrecht, The Netherlands.
Danny A
DA
Young-Afat
Department of Radiation Oncology.
Johannes Pm
JP
Burbach
Department of Radiation Oncology.
Marco
M
van Vulpen
School for Health and Related Research, University of Sheffield, Sheffield, UK.|School of Healthcare, University of Leeds, Leeds, UK.
Clare
C
Relton
Julius Center for Health Sciences and Primary Care, University Medical Center Utrecht, Utrecht, The Netherlands.
Carla H
CH
van Gils
Julius Center for Health Sciences and Primary Care, University Medical Center Utrecht, Utrecht, The Netherlands.
Anne M
AM
May
Julius Center for Health Sciences and Primary Care, University Medical Center Utrecht, Utrecht, The Netherlands.
Rolf Hh
RH
Groenwold
D015331:Cohort Studies; D006801:Humans; D010349:Patient Compliance; D064792:Pragmatic Clinical Trials as Topic; D016032:Randomized Controlled Trials as Topic / Q000379:methods / Q000592:standards*; D012107:Research Design
Pragmatic randomized controlled trials; cohort multiple randomized controlled trial; efficiency; instrumental variable analysis; validity