<p>Definitive randomized controlled trials (RCTs) are the cornerstone of evidence-based medicine but can be complicated, protracted, and expensive. Given the challenges of large-scale trials, pilot trials serve as a crucial initial step, allowing for refinement and validation before embarking on the definitive RCT.<span><sup>1</sup></span> They are a crucial element of good study design and, while conducting a pilot RCT does not guarantee success of the definitive RCT, it increases the likelihood of successful trial completion.<span><sup>2</sup></span> More than US$100 billion is invested annually in biomedical research but often this research is conducted wastefully from poor study design and/or study procedures.<span><sup>3</sup></span> Conducting a well-designed pilot RCT before launching an expensive, time-consuming definitive trial can minimize research waste and improve study conduct.</p><p>Small RCTs cannot be branded pilot or feasibility trials to justify a small sample size. Pilot RCTs have a very specific purpose and inform future trial conduct.<span><sup>4</sup></span> Indeed, research models, including the Canadian Critical Care Trials Group programmatic model, the UK Medical Research Council, and the Australian Clinical Trials Alliance, highlight the importance of pilot RCTs as an integral and necessary step in interventional clinical research (Figure 1). Early piloting of research methods and interventions is important in evaluating feasibility and acceptability before the definitive RCT.</p><p>The importance of pilot trials has been acknowledged for decades<span><sup>5</sup></span> with trial methods evolving over time. It is within this context that we will discuss pilot RCTs used to inform larger definitive RCTs. We will situate pilot trial methods within a larger research framework and propose important concepts in design and reporting.</p><p>The terms “pilot” and “feasibility” trial are used interchangeably by some, but others purport that each type of trial has unique characteristics and therefore define them separately. Whitehead et al.<span><sup>4</sup></span> proposed that pilot trials are a type of feasibility trial with some distinguishing elements: (i) stricter methodology (closely following the definitive study design); (ii) intended to lead to further work; (iii) a smaller version of the larger study; and (iv) focuses on trial processes. This delineation suggests that a pilot RCT is a specific subset of feasibility trial. Henceforth, we adopt the term “pilot.”</p><p>Pilot RCTs allow researchers to test and establish feasibility of the study protocol, study processes, data collection, and intervention fidelity and acceptability.<span><sup>2, 4, 6</sup></span> Table 1 details trial elements tested by a pilot RCT.</p><p>An important indicator of trial feasibility is the ability to recruit the required numbers of participants, using inclusion/exclusion criteria, from the sample population. Recruitment to RCTs can be challe
{"title":"Methodological progress note: Pilot randomized controlled trials","authors":"Amanda Corley RN PhD, Nicole Marsh RN PhD, Samantha Keogh RN PhD","doi":"10.1002/jhm.13376","DOIUrl":"10.1002/jhm.13376","url":null,"abstract":"<p>Definitive randomized controlled trials (RCTs) are the cornerstone of evidence-based medicine but can be complicated, protracted, and expensive. Given the challenges of large-scale trials, pilot trials serve as a crucial initial step, allowing for refinement and validation before embarking on the definitive RCT.<span><sup>1</sup></span> They are a crucial element of good study design and, while conducting a pilot RCT does not guarantee success of the definitive RCT, it increases the likelihood of successful trial completion.<span><sup>2</sup></span> More than US$100 billion is invested annually in biomedical research but often this research is conducted wastefully from poor study design and/or study procedures.<span><sup>3</sup></span> Conducting a well-designed pilot RCT before launching an expensive, time-consuming definitive trial can minimize research waste and improve study conduct.</p><p>Small RCTs cannot be branded pilot or feasibility trials to justify a small sample size. Pilot RCTs have a very specific purpose and inform future trial conduct.<span><sup>4</sup></span> Indeed, research models, including the Canadian Critical Care Trials Group programmatic model, the UK Medical Research Council, and the Australian Clinical Trials Alliance, highlight the importance of pilot RCTs as an integral and necessary step in interventional clinical research (Figure 1). Early piloting of research methods and interventions is important in evaluating feasibility and acceptability before the definitive RCT.</p><p>The importance of pilot trials has been acknowledged for decades<span><sup>5</sup></span> with trial methods evolving over time. It is within this context that we will discuss pilot RCTs used to inform larger definitive RCTs. We will situate pilot trial methods within a larger research framework and propose important concepts in design and reporting.</p><p>The terms “pilot” and “feasibility” trial are used interchangeably by some, but others purport that each type of trial has unique characteristics and therefore define them separately. Whitehead et al.<span><sup>4</sup></span> proposed that pilot trials are a type of feasibility trial with some distinguishing elements: (i) stricter methodology (closely following the definitive study design); (ii) intended to lead to further work; (iii) a smaller version of the larger study; and (iv) focuses on trial processes. This delineation suggests that a pilot RCT is a specific subset of feasibility trial. Henceforth, we adopt the term “pilot.”</p><p>Pilot RCTs allow researchers to test and establish feasibility of the study protocol, study processes, data collection, and intervention fidelity and acceptability.<span><sup>2, 4, 6</sup></span> Table 1 details trial elements tested by a pilot RCT.</p><p>An important indicator of trial feasibility is the ability to recruit the required numbers of participants, using inclusion/exclusion criteria, from the sample population. Recruitment to RCTs can be challe","PeriodicalId":15883,"journal":{"name":"Journal of hospital medicine","volume":"19 9","pages":"821-826"},"PeriodicalIF":2.4,"publicationDate":"2024-04-24","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://onlinelibrary.wiley.com/doi/epdf/10.1002/jhm.13376","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140659039","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}