A tutorial on pilot studies: the what, why and how

Published on Dec 1, 2010in BMC Medical Research Methodology2.51
· DOI :10.1186/1471-2288-10-1
Lehana Thabane63
Estimated H-index: 63
(St. Joseph's Healthcare Hamilton),
Jinhui Ma15
Estimated H-index: 15
(St. Joseph's Healthcare Hamilton)
+ 7 AuthorsCharles H. Goldsmith72
Estimated H-index: 72
(St. Joseph's Healthcare Hamilton)
Pilot studies for phase III trials - which are comparative randomized trials designed to provide preliminary evidence on the clinical efficacy of a drug or intervention - are routinely performed in many clinical areas. Also commonly know as "feasibility" or "vanguard" studies, they are designed to assess the safety of treatment or interventions; to assess recruitment potential; to assess the feasibility of international collaboration or coordination for multicentre trials; to increase clinical experience with the study medication or intervention for the phase III trials. They are the best way to assess feasibility of a large, expensive full-scale study, and in fact are an almost essential pre-requisite. Conducting a pilot prior to the main study can enhance the likelihood of success of the main study and potentially help to avoid doomed main studies. The objective of this paper is to provide a detailed examination of the key aspects of pilot studies for phase III trials including: 1) the general reasons for conducting a pilot study; 2) the relationships between pilot studies, proof-of-concept studies, and adaptive designs; 3) the challenges of and misconceptions about pilot studies; 4) the criteria for evaluating the success of a pilot study; 5) frequently asked questions about pilot studies; 7) some ethical aspects related to pilot studies; and 8) some suggestions on how to report the results of pilot investigations using the CONSORT format.
  • References (27)
  • Citations (975)
Published on Feb 27, 2009in PLOS ONE2.78
Peter T. Choi15
Estimated H-index: 15
(UBC: University of British Columbia),
W. Scott Beattie39
Estimated H-index: 39
(UHN: University Health Network)
+ 2 AuthorsHomer Yang13
Estimated H-index: 13
(U of O: University of Ottawa)
Background Early randomized controlled trials have suggested that neuraxial blockade may reduce cardiorespiratory complications after non-cardiothoracic surgery, but recent larger trials have been inconclusive. We conducted a pilot study to assess the feasibility of conducting a large multicentre randomized controlled trial in Canada.
Published on Jan 1, 2009in Critical Care Medicine6.97
Donald M. Arnold37
Estimated H-index: 37
Karen E. A. Burns35
Estimated H-index: 35
+ 3 AuthorsDeborah J. Cook153
Estimated H-index: 153
Background: Pilot trials are important to ensure that large randomized trials are rigorous, feasible, and economically justifiable. The objective of this review is to highlight the importance of randomized pilot trials and to describe key features of their design and interpretation using examples from critical care. Methods: We searched MEDLINE (1997–2007) and contacted experts to identify pilot randomized trials to exemplify and summarize their key methodologic features including objectives, sa...
Published on Dec 1, 2008in Orphanet Journal of Rare Diseases3.69
Shein-Chung Chow31
Estimated H-index: 31
(Duke University),
Mark Chang9
Estimated H-index: 9
(Millennium Pharmaceuticals)
In recent years, the use of adaptive design methods in clinical research and development based on accrued data has become very popular due to its flexibility and efficiency. Based on adaptations applied, adaptive designs can be classified into three categories: prospective, concurrent (ad hoc), and retrospective adaptive designs. An adaptive design allows modifications made to trial and/or statistical procedures of ongoing clinical trials. However, it is a concern that the actual patient populat...
Published on Sep 29, 2008in BMJ27.60
Peter Craig14
Estimated H-index: 14
Paul Dieppe91
Estimated H-index: 91
(NOC: Nuffield Orthopaedic Centre)
+ 3 AuthorsMark Petticrew78
Estimated H-index: 78
(Lond: University of London)
Evaluating complex interventions is complicated. The Medical Research Council9s evaluation framework (2000) brought welcome clarity to the task. Now the council has updated its guidance
Published on Jul 3, 2008
Miquel Porta60
Estimated H-index: 60
(Autonomous University of Barcelona)
The new edition of this classic text remains the definitive dictionary in epidemiology. In fact, it is more than a dictionary, with some reviewers remarking that if they had to limit their professional library to one volume, this would be the book they would choose. In the complex field of epidemiology, the definition and concise explanation of terms is a key to understanding epidemiologic concepts, and the dictionary goes beyond simple definitions, as it place each term firmly and clearly in it...
Published on Jun 1, 2007in Respiratory Medicine3.24
Thomas C. Fardon20
Estimated H-index: 20
(Dund.: University of Dundee),
K. Haggart14
Estimated H-index: 14
(Dund.: University of Dundee)
+ 1 AuthorsBrian J. Lipworth57
Estimated H-index: 57
(Dund.: University of Dundee)
Summary We conducted a double blind, randomised, placebo-controlled, crossover study evaluating the effects of halving inhaled steroid dosage plus salmeterol, or salmeterol and tiotropium. Eighteen life-long non-smoking severe asthmatics [mean FEV 1 1.49 l (51%)] were run-in for 4 weeks on HFA-fluticasone propionate 1000 μg daily, and were subsequently randomised to 4 weeks of either (a) HFA-fluticasone propionate 500 μg BD/salmeterol 100 μg BD/HFA-tiotropium bromide18 μg od; or (b) fluticasone ...
Published on May 1, 2006in Archives of General Psychiatry
Helena C. Kraemer117
Estimated H-index: 117
(Stanford University),
Jim Mintz61
Estimated H-index: 61
(UCLA: University of California, Los Angeles)
+ 2 AuthorsJerome A. Yesavage61
Estimated H-index: 61
Clinical researchers often propose (or review committees demand) pilot studies to determine whether a study is worth performing and to guide power calculations. The most likely outcomes are that (1) studies worth performing are aborted and (2) studies that are not aborted are underpowered. There are many excellent reasons for performing pilot studies. The argument herein is not meant to discourage clinical researchers from performing pilot studies (or review committees from requiring them) but s...
Published on Dec 1, 2005in Journal of Critical Care2.78
Deborah J. Cook153
Estimated H-index: 153
(McMaster University),
Graeme Rocker43
Estimated H-index: 43
(Dal: Dalhousie University)
+ 22 AuthorsJamie Cooper12
Estimated H-index: 12
(Monash University)
Abstract Purpose There is no randomized trial comparing low–molecular weight heparin (LMWH) and unfractionated heparin (UFH) for thromboprophylaxis in medical-surgical ICU patients. The primary objective of this randomized pilot study on LMWH vs UFH was to assess the feasibility of conducting a large randomized trial with respect to timely enrollment and blinded study drug administration, practicality of twice-weekly lower limb ultrasounds to screen for deep venous thrombosis, LMWH bioaccumulati...
Published on Jun 30, 2005in Statistics in Medicine1.85
A. Lawrence Gould26
Estimated H-index: 26
(USMA: United States Military Academy)
SUMMARY 'Proof of concept' (PoC) trials often are carried out to determine if a treatment is biologically active or inactive. If the predictive probability of rejecting a null hypothesis of no treatment eect on completion of the trial calculated using information accumulated before trial completion is suciently low, then the trial could be terminated and resources redirected more productively. If the predictive probability is high, some time might be gained by proceeding with further steps in dr...
Cited By975
Published on Jun 6, 2018in Journal of Maternal-fetal & Neonatal Medicine1.57
Sarah D. McDonald30
Estimated H-index: 30
(McMaster University),
Kristen Viaje (McMaster University)+ 7 AuthorsLehana Thabane63
Estimated H-index: 63
(St. Joseph's Healthcare Hamilton)
AbstractObjectives: Obese women are at increased risks for complications during pregnancy, birth, and in their infants. Although guidelines have been established for the clinical care of obese pregnant women, management is sometimes suboptimal. Our goal was to determine the feasibility of implementing and testing a clinical carepath for obese pregnant women compared to standard care, in a pilot cluster randomized controlled trial (RCT).Methods: A pragmatic pilot cluster RCT was conducted, random...
Published on Feb 13, 2019in Pilot and Feasibility Studies
Rumana Newlands5
Estimated H-index: 5
Maria Ntessalen + 5 AuthorsLeone Christina Agnese Craig4
Estimated H-index: 4
Being overweight or obese following breast cancer diagnosis can increase cancer recurrence and mortality, so effective interventions for weight loss in this group could enhance survival. A pilot randomised controlled trial was conducted to assess whether a weight loss programme comprising generic Weight Watchers® referral offered to women treated for breast cancer with or without additional breast cancer-tailored dietetic support is feasible and shows promise for improving weight and quality of ...
Published on Dec 1, 2019in Trials1.98
Lehana Thabane63
Estimated H-index: 63
(McMaster University),
Linda Cambon4
Estimated H-index: 4
(University of Bordeaux)
+ 7 AuthorsFrançois Alla46
Estimated H-index: 46
(University of Bordeaux)
Background An international workshop on population health intervention research (PHIR) was organized to foster exchanges between experts from different disciplines and different fields.
Published on Jan 10, 2019in BMC Medical Research Methodology2.51
Caroline Kristunas2
Estimated H-index: 2
(University of Leicester),
Karla Hemming25
Estimated H-index: 25
(University of Birmingham)
+ 2 AuthorsLaura J. Gray60
Estimated H-index: 60
(University of Leicester)
Background Stepped-wedge cluster randomised trials (SW-CRTs) are a pragmatic trial design, providing an unprecedented opportunity to increase the robustness of evidence underpinning implementation and quality improvement interventions. Given the complexity of the SW-CRT, the likelihood of trials not delivering on their objectives will be mitigated if a feasibility study precedes the definitive trial. It is not currently known if feasibility studies are being conducted for SW-CRTs nor what the ob...
Published on Jan 14, 2019in Health and Quality of Life Outcomes2.32
F. Ranjandish (Tabriz University of Medical Sciences), Hassan Mahmoodi1
Estimated H-index: 1
(Tabriz University of Medical Sciences)
+ 0 AuthorsA. Shaghaghi (Tabriz University of Medical Sciences)
Background Cardiovascular diseases (CVD) as a most frequent and costly NCDs account for about 17.3 million annual deaths worldwide. About 80% of these deaths are taking place in low and middle income countries (LMIC). The survivors may experience severe disabling consequences with extensive impacts on their quality of life. The HeartQoL is a relatively new scale to measure health-related quality of life in CVD patients and was validated for use in other languages. Main aim of the present study w...
Published on Apr 25, 2019in Trials1.98
Emma R. Lawlor2
Estimated H-index: 2
('QUB': Queen's University Belfast),
Margaret Cupples24
Estimated H-index: 24
('QUB': Queen's University Belfast)
+ 1 AuthorsMark Tully21
Estimated H-index: 21
('QUB': Queen's University Belfast)
Background Insufficient physical activity (PA) is a major public health issue. Whilst PA is an important contributor to disease prevention, engagement in PA decreases with age, particularly among women in socio-economically disadvantaged areas. Research using existing support networks to engage ‘hard to reach’ populations in PA interventions is sparse. We developed and tested the feasibility of a PA-promoting intervention for older women within existing community groups in socio-economically dis...
Published on 2019in Trials1.98
Jonathan Koffman22
Estimated H-index: 22
('KCL': King's College London),
E Yorganci ('KCL': King's College London)+ 7 AuthorsL Sampson (UCL: University College London)
Background : The AMBER (Assessment; Management; Best practice; Engagement; Recovery uncertain) care bundle is a complex intervention used in UK hospitals to support patients with uncertain recovery. However, it has yet to be evaluated in a randomised controlled trial (RCT) to identify potential benefits or harms. Aim : To investigate the feasibility of a cluster RCT of the AMBER care bundle. Methods : Mixed-method, feasibility cluster RCT. Prospective, quantitative data collected from patients (...
Published on Jan 5, 2019in Pilot and Feasibility Studies
Claire Carswell , Joanne Reid10
Estimated H-index: 10
+ 2 AuthorsHelen Noble14
Estimated H-index: 14
End-stage kidney disease is a life-changing illness. Many patients require haemodialysis, a treatment that impacts profoundly on quality of life and mental health. Arts-based interventions have been used in other healthcare settings to improve mental health and quality of life; therefore, they may help address the impact of haemodialysis by improving these outcomes. However, there is a lack of evidence assessing their effectiveness in this population and few randomised controlled trials (RCTs) e...
Published on Dec 1, 2019in BMC Neurology2.23
Tamis W. Pin2
Estimated H-index: 2
(PolyU: Hong Kong Polytechnic University),
Penelope B. Butler3
Estimated H-index: 3
(MMU: Manchester Metropolitan University),
Sheila Purves1
Estimated H-index: 1
(PolyU: Hong Kong Polytechnic University)
Background This pilot study was to examine the feasibility and tolerance of whole body vibration therapy (WBVT) for children and adults with moderate severity of cerebral palsy (CP) being graded as levels III or IV on the Gross Motor Function Classification Scale (GMFCS).