Pilot randomized controlled trials in the orthopaedic surgery literature: a systematic review

Nicole Simunovic,Sheila Sprague,Mohit Bhandari,Bijal Desai,Shivani Shah,Andrew Duong,Amr Saleh,Archita Srinath,Veeral Desai

doi:10.1186/s12891-018-2337-7

Abstract

BackgroundThe primary objective of this systematic review is to examine the characteristics of pilot randomized controlled trials (RCTs) in the orthopaedic surgery literature, including the proportion framed as feasibility trials and those that lead to definitive RCTs. This review aim to answer the question of whether pilot RCTs lead to definitive RCTs, whilst investigating the quality, feasibility and overall publication trends of orthopaedic pilot trials.MethodsPilot RCTs in the orthopaedic literature were identified from three electronic databases (EMBASE, MEDLINE, and Pubmed) searched from database inception to January 2018. Search criteria included the evaluation of at least one orthopaedic surgical intervention, research on humans, and publication in English. Two reviewers independently screened the pool of pilot trials, and conducted a search for corresponding definitive trials. Screened pilot RCTs were assessed for feasibility outcomes related to efficiency, cost, and/or timeliness of a large-scale clinical trial involving a surgical intervention. The quality of the pilot and definitive trials were assessed using the Checklist to Evaluate a Report of a Non-Pharmacological Trial (CLEAR NPT).ResultsThe initial search for pilot RCTs yielded 3857 titles, of which 49 articles were relevant for this review. 73.5% (36/49) of the orthopaedic pilot RCTs were framed as feasibility trials. Of these, 5 corresponding definitive trials (10.2%) were found, of which four were published and one ongoing. Based on author responses, the lack of a definitive RCT following the pilot trial was attributed to a lack of funding, inadequacies in recruitment, and belief that the pilot RCT sufficiently answered the research question.ConclusionsBased on this systematic review, most pilot RCTs were characterized as feasibility trials. However, the majority of published pilot RCTs did not lead to definitive trials. This discrepancy was mainly attributed to poor feasibility (e.g. poor recruitment) and lack of funding for an orthopaedic surgical definitive trial. In recent years this discrepancy may be due to researchers saving on time and cost by rolling their pilot patients into the definitive RCT rather than publish a separate pilot trial.

Highlights

The primary objective of this systematic review is to examine the characteristics of pilot randomized controlled trials (RCTs) in the orthopaedic surgery literature, including the proportion framed as feasibility trials and those that lead to definitive Randomized controlled trial (RCT)
For definitive RCTs, the time elapsed between the date of publication of the pilot and definitive trial and whether or not the sample size was calculated based on event rates from the pilot trial were determined
In order to distinguish between pilot trials created solely for investigating the efficacy of interventions compared to feasibility trials, specific reference to feasibility objectives were evaluated

Summary

Introduction

The primary objective of this systematic review is to examine the characteristics of pilot randomized controlled trials (RCTs) in the orthopaedic surgery literature, including the proportion framed as feasibility trials and those that lead to definitive RCTs. Definitive randomized controlled trials (RCTs) exist to demonstrate unmistakable evidence of a certain inventions benefit on a patient [1]. They are very impactful for clinical practice are typically expensive and time-consuming [2]. Given the resources and time, investigators often conduct pilot trials designed with an aim to demonstrate the feasibility of the larger-scale definitive trial [3]. Site or investigator level feasibility trials focus on identifying challenges and probable solutions with respect to the investigator and clinical aspects of the trial (drug dosages, actual study population, recruitment and follow-up, usage of assessment tools, etc.) [4]

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