Skip to main page content
U.S. flag

An official website of the United States government

Dot gov

The .gov means it’s official.
Federal government websites often end in .gov or .mil. Before sharing sensitive information, make sure you’re on a federal government site.

Https

The site is secure.
The https:// ensures that you are connecting to the official website and that any information you provide is encrypted and transmitted securely.

Access keys NCBI Homepage MyNCBI Homepage Main Content Main Navigation
. 2010 Jan 6:10:1.
doi: 10.1186/1471-2288-10-1.

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

Lehana Thabane  1 , Jinhui Ma, Rong Chu, Ji Cheng, Afisi Ismaila, Lorena P Rios, Reid Robson, Marroon Thabane, Lora Giangregorio, Charles H Goldsmith
Affiliations

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

Lehana Thabane et al. BMC Med Res Methodol. .

Erratum in

  • Correction to: A tutorial on pilot studies: the what, why and how.
    Thabane L, Ma J, Chu R, Cheng J, Ismaila A, Rios LP, Robson R, Thabane M, Giangregorio L, Goldsmith CH. Thabane L, et al. BMC Med Res Methodol. 2023 Mar 11;23(1):59. doi: 10.1186/s12874-023-01880-1. BMC Med Res Methodol. 2023. PMID: 36927764 Free PMC article. No abstract available.

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.

PubMed Disclaimer

References

    1. Waite M. Concise Oxford Thesaurus. 2. Oxford, England: Oxford University Press; 2002.
    1. Last JM, editor. A Dictionary of Epidemiology. 4. Oxford University Press; 2001.
    1. Everitt B. Medical Statistics from A to Z: A Guide for Clinicians and Medical Students. 2. Cambridge University Press: Cambridge; 2006.
    1. Tavel JA, Fosdick L. ESPRIT Vanguard Group. ESPRIT Executive Committee. Closeout of four phase II Vanguard trials and patient rollover into a large international phase III HIV clinical endpoint trial. Control Clin Trials. 2001;22:42–48. doi: 10.1016/S0197-2456(00)00114-8. - DOI - PubMed
    1. Arnold DM, Burns KE, Adhikari NK, Kho ME, Meade MO, Cook DJ. The design and interpretation of pilot trials in clinical research in critical care. Crit Care Med. 2009;37(Suppl 1):69–74. doi: 10.1097/CCM.0b013e3181920e33. - DOI - PubMed

MeSH terms