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Surgery
Original research
How do pilot and feasibility studies inform randomised placebo-controlled trials in surgery? A systematic review
  1. Sian Cousins1,
  2. Alexander Gormley2,
  3. Katy Chalmers1,
  4. Marion K Campbell3,
  5. David J Beard4,
  6. Natalie S Blencowe1,
  7. Jane M Blazeby1
  1. 1Surgical Innovation theme, Bristol National Institute for Health and Care Research (NIHR) Biomedical Research Centre; Royal College of Surgeons of England (RCSEng) Bristol Surgical Trials Centre, Centre for Surgical Research, Population Health Sciences, Bristol Medical School, University of Bristol, Bristol, UK
  2. 2Bristol Dental School, University of Bristol, Bristol, UK
  3. 3Royal College of Surgeons of England, Aberdeen Surgical Trials Centre; Health Services Research Unit, University of Aberdeen, Aberdeen, UK
  4. 4Nuffield Department of Orthopaedics, Rheumatology and Musculoskeletal Sciences; RCSEng Surgical Intervention Trials Unit; NIHR Oxford Biomedical Research Centre, University of Oxford, Oxford, UK
  1. Correspondence to Dr Sian Cousins; sian.cousins{at}bristol.ac.uk

Abstract

Introduction Randomised controlled trials (RCTs) with a placebo comparator are considered the gold standard study design when evaluating healthcare interventions. These are challenging to design and deliver in surgery. Guidance recommends pilot and feasibility work to optimise main trial design and conduct; however, the extent to which this occurs in surgery is unknown.

Method A systematic review identified randomised placebo-controlled surgical trials. Articles published from database inception to 31 December 2020 were retrieved from Ovid-MEDLINE, Ovid-EMBASE and CENTRAL electronic databases, hand-searching and expert knowledge. Pilot/feasibility work conducted prior to the RCTs was then identified from examining citations and reference lists. Where studies explicitly stated their intent to inform the design and/or conduct of the future main placebo-controlled surgical trial, they were included. Publication type, clinical area, treatment intervention, number of centres, sample size, comparators, aims and text about the invasive placebo intervention were extracted.

Results From 131 placebo surgical RCTs included in the systematic review, 47 potentially eligible pilot/feasibility studies were identified. Of these, four were included as true pilot/feasibility work. Three were original articles, one a conference abstract; three were conducted in orthopaedic surgery and one in oral and maxillofacial surgery. All four included pilot RCTs, with an invasive surgical placebo intervention, randomising 9–49 participants in 1 or 2 centres. They explored the acceptability of recruitment and the invasive placebo intervention to patients and trial personnel, and whether blinding was possible. One study examined the characteristics of the proposed invasive placebo intervention using in-depth interviews.

Conclusion Published studies reporting feasibility/pilot work undertaken to inform main placebo surgical trials are scarce. In view of the difficulties of undertaking placebo surgical trials, it is recommended that pilot/feasibility studies are conducted, and more are reported to share key findings and optimise the design of main RCTs.

PROSPERO registration number CRD42021287371.

  • SURGERY
  • Clinical Trial
  • Feasibility Studies

Data availability statement

All data relevant to the study are included in the article or uploaded as supplementary information.

https://creativecommons.org/licenses/by/4.0/

This is an open access article distributed in accordance with the Creative Commons Attribution 4.0 Unported (CC BY 4.0) license, which permits others to copy, redistribute, remix, transform and build upon this work for any purpose, provided the original work is properly cited, a link to the licence is given, and indication of whether changes were made. See: https://creativecommons.org/licenses/by/4.0/.

https://doi.org/10.1136/bmjopen-2022-071094

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    Data availability statement

    All data relevant to the study are included in the article or uploaded as supplementary information.

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    Footnotes

    • Twitter @Gormley, @NatalieBlencowe

    • Contributors SC: conceptualisation; methodology; formal analysis; supervision; writing – original draft. AG and KC: formal analysis; writing – review and editing. MKC, DJB and NSB: conceptualisation; methodology; writing – review and editing. JMB: funding acquisition; conceptualisation; metholodology; formal analysis; supervision; writing – review and editing; SC is the guarantor.

    • Funding This work was supported by the National Institute for Health and Care Research Biomedical Research Centre at University Hospitals Bristol and Weston NHS Foundation Trust and the University of Bristol (BRC-1215-20011).

    • Competing interests None declared.

    • Patient and public involvement Patients and/or the public were not involved in the design, or conduct, or reporting, or dissemination plans of this research.

    • Provenance and peer review Not commissioned; externally peer reviewed.

    • Supplemental material This content has been supplied by the author(s). It has not been vetted by BMJ Publishing Group Limited (BMJ) and may not have been peer-reviewed. Any opinions or recommendations discussed are solely those of the author(s) and are not endorsed by BMJ. BMJ disclaims all liability and responsibility arising from any reliance placed on the content. Where the content includes any translated material, BMJ does not warrant the accuracy and reliability of the translations (including but not limited to local regulations, clinical guidelines, terminology, drug names and drug dosages), and is not responsible for any error and/or omissions arising from translation and adaptation or otherwise.