Claire L Chan1, Monica Taljaard2, Gillian A Lancaster3, Jamie C Brehaut4, Sandra M Eldridge1. 1. Centre for Clinical Trials and Methodology, Institute of Population Health Sciences, Queen Mary University of London, London, E1 2AB, UK. 2. Ottawa Hospital Research Institute, Clinical Epidemiology Program, Centre for Practice-Changing Research, The Ottawa Hospital, Ottawa, ON, K1H 8L6, Canada; School of Epidemiology and Public Health, University of Ottawa, Ottawa, Ontario, Canada. Electronic address: mtaljaard@ohri.ca. 3. Keele Clinical Trials Unit, School of Medicine, Keele University, Keele, Staffordshire, ST5 5BG, UK. 4. Ottawa Hospital Research Institute, Clinical Epidemiology Program, Centre for Practice-Changing Research, The Ottawa Hospital, Ottawa, ON, K1H 8L6, Canada; School of Epidemiology and Public Health, University of Ottawa, Ottawa, Ontario, Canada.
Abstract
BACKGROUND AND OBJECTIVE: Feasibility studies are increasingly being used to support the development of, and investigate uncertainties around, future large-scale trials. The future trial can be designed with either a pragmatic or explanatory mindset. Whereas pragmatic trials aim to inform the choice between different care options and thus, are designed to resemble conditions outside of a clinical trial environment, explanatory trials examine the benefit of a treatment under more controlled conditions. There is existing guidance for designing feasibility studies, but none that explicitly considers the goals of pragmatic designs. We aimed to identify unique areas of uncertainty that are relevant to planning a pragmatic trial. RESULTS: We identified ten relevant domains, partly based on the pragmatic-explanatory continuum indicator summary-2 (PRECIS-2) framework, and describe potential questions of uncertainty within each: intervention development, research ethics, participant identification and eligibility, recruitment of individuals, setting, organization, flexibility of delivery, flexibility of adherence, follow-up, and importance of primary outcome to patients and decision-makers. We present examples to illustrate how uncertainty in these domains might be addressed within a feasibility study. CONCLUSION: Researchers planning a feasibility study in advance of a pragmatic trial should consider feasibility objectives specifically relevant to areas of uncertainty for pragmatic trials.
BACKGROUND AND OBJECTIVE: Feasibility studies are increasingly being used to support the development of, and investigate uncertainties around, future large-scale trials. The future trial can be designed with either a pragmatic or explanatory mindset. Whereas pragmatic trials aim to inform the choice between different care options and thus, are designed to resemble conditions outside of a clinical trial environment, explanatory trials examine the benefit of a treatment under more controlled conditions. There is existing guidance for designing feasibility studies, but none that explicitly considers the goals of pragmatic designs. We aimed to identify unique areas of uncertainty that are relevant to planning a pragmatic trial. RESULTS: We identified ten relevant domains, partly based on the pragmatic-explanatory continuum indicator summary-2 (PRECIS-2) framework, and describe potential questions of uncertainty within each: intervention development, research ethics, participant identification and eligibility, recruitment of individuals, setting, organization, flexibility of delivery, flexibility of adherence, follow-up, and importance of primary outcome to patients and decision-makers. We present examples to illustrate how uncertainty in these domains might be addressed within a feasibility study. CONCLUSION: Researchers planning a feasibility study in advance of a pragmatic trial should consider feasibility objectives specifically relevant to areas of uncertainty for pragmatic trials.
Authors: Rafael Dal-Ré; Cristina Avendaño-Solà; Brigitte Bloechl-Daum; Anthonius de Boer; Stefan Eriksson; Uwe Fuhr; Søren Holm; Stefan K James; Robert J Mentz; Emilio Perucca; Frits R Rosendaal; Shaun Treweek Journal: BMJ Date: 2019-03-27
Authors: M W Skinner; C Chai-Adisaksopha; R Curtis; N Frick; M Nichol; D Noone; B O'Mahony; D Page; J S Stonebraker; A Iorio Journal: Pilot Feasibility Stud Date: 2018-02-27
Authors: Lauralyn McIntyre; Monica Taljaard; Tracy McArdle; Alison Fox-Robichaud; Shane W English; Claudio Martin; John Marshall; Kusum Menon; John Muscedere; Deborah J Cook; Charles Weijer; Raphael Saginur; Alies Maybee; Akshai Iyengar; Alan Forster; Ian D Graham; Steven Hawken; Colin McCartney; Andrew Je Seely; Ian G Stiell; Kednapa Thavorn; Dean A Fergusson Journal: BMJ Open Date: 2018-08-23 Impact factor: 2.692
Authors: Monica Taljaard; Charles Weijer; Jeremy M Grimshaw; Adnan Ali; Jamie C Brehaut; Marion K Campbell; Kelly Carroll; Sarah Edwards; Sandra Eldridge; Christopher B Forrest; Bruno Giraudeau; Cory E Goldstein; Ian D Graham; Karla Hemming; Spencer Phillips Hey; Austin R Horn; Vipul Jairath; Terry P Klassen; Alex John London; Susan Marlin; John C Marshall; Lauralyn McIntyre; Joanne E McKenzie; Stuart G Nicholls; P Alison Paprica; Merrick Zwarenstein; Dean A Fergusson Journal: Trials Date: 2018-09-27 Impact factor: 2.279
Authors: Rosa R Baier; Eric Jutkowitz; Susan L Mitchell; Ellen McCreedy; Vincent Mor Journal: BMC Med Res Methodol Date: 2019-07-18 Impact factor: 4.615