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

被引：1689

作者：

Thabane, Lehana ^{[1
,2
]}

Ma, Jinhui ^{[1
,2
]}

Chu, Rong ^{[1
,2
]}

Cheng, Ji ^{[1
,2
]}

Ismaila, Afisi ^{[1
,3
]}

Rios, Lorena P. ^{[1
,2
]}

Robson, Reid ^{[3
]}

Thabane, Marroon ^{[1
,4
]}

Giangregorio, Lora ^{[5
]}

Goldsmith, Charles H. ^{[1
,2
]}

机构：

[1] McMaster Univ, Dept Clin Epidemiol & Biostat, Hamilton, ON, Canada

[2] St Josephs Healthcare Hamilton, Biostat Unit, Hamilton, ON, Canada

[3] GlaxoSmithKline Inc, Dept Med Affairs, Mississauga, ON, Canada

[4] McMaster Univ, Dept Med, Div Gastroenterol, Hamilton, ON, Canada

[5] Univ Waterloo, Dept Kinesiol, Waterloo, ON N2L 3G1, Canada

来源：

BMC MEDICAL RESEARCH METHODOLOGY | 2010年 / 10卷

关键词：

PROOF-OF-CONCEPT; SAMPLE-SIZE; TRIALS; RECOMMENDATIONS; DESIGN;

D O I：

10.1186/1471-2288-10-1

中图分类号：

R19 [保健组织与事业（卫生事业管理）];

学科分类号：

摘要：

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.

引用

页数：10

共 34 条

[1] STATISTICS NOTES - ABSENCE OF EVIDENCE IS NOT EVIDENCE OF ABSENCE [J].

ALTMAN, DG ;

BLAND, JM .

BRITISH MEDICAL JOURNAL, 1995, 311 (7003) :485-485

[2]

[Anonymous], International Ethical Guidelines for Research Involving Human Subjects

[3]

[Anonymous], DECLARATION HELSINKI

[4]

[Anonymous], 2001, SOCIAL RES UPDATE

[5]

[Anonymous], BMJ

[6]

[Anonymous], ICH HARM TRIP GUID G

[7] The design and interpretation of pilot trials in clinical research in critical care [J].

Arnold, Donald M. ;

Burns, Karen E. A. ;

Adhikari, Neill K. J. ;

Kho, Michelle E. ;

Meade, Maureen O. ;

Cook, Deborah J. .

CRITICAL CARE MEDICINE, 2009, 37 (01) :S69-S74

[8]

*BELM REP, RES ETH GUID

[9] Effects of Neuraxial Blockade May Be Difficult To Study Using Large Randomized Controlled Trials: The PeriOperative Epidural Trial (POET) Pilot Study [J].

Choi, Peter T. ;

Beattie, W. Scott ;

Bryson, Gregory L. ;

Paul, James E. ;

Yang, Homer .

PLOS ONE, 2009, 4 (02)

[10] Adaptive design methods in clinical trials - a review [J].

Chow, Shein-Chung ;

Chang, Mark .

ORPHANET JOURNAL OF RARE DISEASES, 2008, 3 (1)

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