The Role of Hypothesis-Generating and Pilot Studies as Precursors to Hypothesis-Testing in Clinical Research

• Activity Number: 49
• Type: Invited
• Date/Time: Sunday, July 29, 2012 : 4:00 PM to 5:50 PM
• Sponsor: Statistics in Mental Health Research
• Author(s): Helena Chmura Kraemer*+
• Companies: Stanford University/University of Pittsburgh
• Keywords: Pilot study ; Hypothesis-testing ; Exploratory Studies

Abstract

Hypothesis-testing studies are the "gold standard" of clinical research, but the quality of such studies depends crucially on the "building blocks" of hypothesis-generating studies and pilot studies. The distinction among the three types of studies, however, is often obscured by both researchers and reviewers of research proposals or submitted papers. Such confusion wastes time, effort and funding, raises ethical concerns related to using human participants in research that is unlikely to provide new and useful information, and corrupts the research literature with false and confusing findings. We aim here to clarify the role of hypothesis-generating and pilot studies as essential precursors to hypothesis-testing studies. We describe each type of early-phase research and discuss common problems and misconceptions. For each approach, we propose a set of criteria that we encourage reviewers to use in evaluating proposals. Finally, we consider issues related to publication of the results of such studies.