A Tutorial on Pilot Studies: the What, Why and How

Overview

Journal BMC Med Res Methodol

Publisher Biomed Central

Specialties General Medicine
Health Services

Date 2010 Jan 8

PMID 20053272

Citations 1111

Authors

Lehana Thabane

Jinhui Ma

Rong Chu

Ji Cheng

Afisi Ismaila

Lorena P Rios

Reid Robson

Marroon Thabane

Lora Giangregorio

Charles H Goldsmith

Affiliations

Soon will be listed here.

Abstract

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.

Citing Articles

Optimizing sample size for supervised machine learning with bulk transcriptomic sequencing: a learning curve approach.

Qi Y, Wang X, Qin L Brief Bioinform. 2025; 26(2).

PMID: 40072846 PMC: 11899567. DOI: 10.1093/bib/bbaf097.

Co-designed neck exercise (EPIC-Neck) vs usual exercise care for people with chronic non-specific neck pain: protocol for a randomised feasibility study with process evaluation.

Price J, Rushton A, Ives N, Jolly K, Parmar P, Greaves C Pilot Feasibility Stud. 2025; 11(1):26.

PMID: 40057809 PMC: 11889939. DOI: 10.1186/s40814-025-01608-6.

A feasibility study of the internet-based intervention "Strategies for Empowering activities in Everyday life" (SEE 1.0) applied for people with stroke.

Larsson-Lund M, Barcheus I, Ranner M, Vikman I, Jacobsson L, Lexell E BMC Health Serv Res. 2025; 25(1):330.

PMID: 40033363 PMC: 11877923. DOI: 10.1186/s12913-025-12456-8.

Overcoming barriers to implementation of patient engagement in clinical trials: feasibility testing of an embedded study.

Castonguay G, Bedard S, Dubois A, Lessard E, Rivard L, Rouly G Res Involv Engagem. 2025; 11(1):15.

PMID: 40012063 PMC: 11866851. DOI: 10.1186/s40900-025-00689-0.

Examining the Implementation of the Italian Version of the Teen Online Problem-Solving Program Coupled With Remote Psychological Support: Protocol for a Randomized Controlled Trial.

Corti C, Papini M, Strazzer S, Borgatti R, Romaniello R, Poggi G JMIR Res Protoc. 2025; 14:e64178.

PMID: 39984161 PMC: 11890147. DOI: 10.2196/64178.

References

. ICH harmonized tripartite guideline: Guideline for Good Clinical Practice. J Postgrad Med. 2001; 47(1):45-50. View

Gould A . Planning and revising the sample size for a trial. Stat Med. 1995; 14(9-10):1039-51; discussion 1053-5. DOI: 10.1002/sim.4780140922. View

Yin Y . Sample size calculation for a proof of concept study. J Biopharm Stat. 2002; 12(2):267-76. DOI: 10.1081/bip-120015748. View

Craig P, Dieppe P, Macintyre S, Michie S, Nazareth I, Petticrew M . Developing and evaluating complex interventions: the new Medical Research Council guidance. BMJ. 2008; 337:a1655. PMC: 2769032. DOI: 10.1136/bmj.a1655. View

Wittes J, Brittain E . The role of internal pilot studies in increasing the efficiency of clinical trials. Stat Med. 1990; 9(1-2):65-71; discussion 71-2. DOI: 10.1002/sim.4780090113. View

Altman D, Bland J . Absence of evidence is not evidence of absence. BMJ. 1995; 311(7003):485. PMC: 2550545. DOI: 10.1136/bmj.311.7003.485. View

Kraemer H, Mintz J, Noda A, Tinklenberg J, Yesavage J . Caution regarding the use of pilot studies to guide power calculations for study proposals. Arch Gen Psychiatry. 2006; 63(5):484-9. DOI: 10.1001/archpsyc.63.5.484. View

Gould A . Timing of futility analyses for 'proof of concept' trials. Stat Med. 2005; 24(12):1815-35. DOI: 10.1002/sim.2087. View

Cook D, Rocker G, Meade M, Guyatt G, Geerts W, Anderson D . Prophylaxis of Thromboembolism in Critical Care (PROTECT) Trial: a pilot study. J Crit Care. 2005; 20(4):364-72. DOI: 10.1016/j.jcrc.2005.09.010. View

10.

Tavel J, Fosdick L . Closeout of four phase II Vanguard trials and patient rollover into a large international phase III HIV clinical endpoint trial. Control Clin Trials. 2001; 22(1):42-8. DOI: 10.1016/s0197-2456(00)00114-8. View

11.

Coffey C, Muller K . Properties of internal pilots with the univariate approach to repeated measures. Stat Med. 2003; 22(15):2469-85. DOI: 10.1002/sim.1466. View

12.

Arnold D, Burns K, Adhikari N, Kho M, Meade M, Cook D . The design and interpretation of pilot trials in clinical research in critical care. Crit Care Med. 2008; 37(1 Suppl):S69-74. DOI: 10.1097/CCM.0b013e3181920e33. View

13.

van Teijlingen E, RENNIE A, Hundley V, GRAHAM W . The importance of conducting and reporting pilot studies: the example of the Scottish Births Survey. J Adv Nurs. 2001; 34(3):289-95. DOI: 10.1046/j.1365-2648.2001.01757.x. View

14.

. International ethical guidelines for biomedical research involving human subjects. Bull Med Ethics. 2004; (182):17-23. View

15.

Fardon T, Haggart K, Lee D, Lipworth B . A proof of concept study to evaluate stepping down the dose of fluticasone in combination with salmeterol and tiotropium in severe persistent asthma. Respir Med. 2006; 101(6):1218-28. DOI: 10.1016/j.rmed.2006.11.001. View

16.

Lancaster G, Dodd S, Williamson P . Design and analysis of pilot studies: recommendations for good practice. J Eval Clin Pract. 2004; 10(2):307-12. DOI: 10.1111/j..2002.384.doc.x. View

17.

Moher D, Schulz K, Altman D . The CONSORT statement: revised recommendations for improving the quality of reports of parallel-group randomized trials. J Am Podiatr Med Assoc. 2001; 91(8):437-42. DOI: 10.7547/87507315-91-8-437. View

18.

Halpern S, Karlawish J, Berlin J . The continuing unethical conduct of underpowered clinical trials. JAMA. 2002; 288(3):358-62. DOI: 10.1001/jama.288.3.358. View

19.

Chow S, Chang M . Adaptive design methods in clinical trials - a review. Orphanet J Rare Dis. 2008; 3:11. PMC: 2422839. DOI: 10.1186/1750-1172-3-11. View

20.

Choi P, Beattie W, Bryson G, Paul J, Yang H . Effects of neuraxial blockade may be difficult to study using large randomized controlled trials: the PeriOperative Epidural Trial (POET) Pilot Study. PLoS One. 2009; 4(2):e4644. PMC: 2645707. DOI: 10.1371/journal.pone.0004644. View