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October 20, 2015

Pilot Studies: A Critical but Potentially Misused Component of Interventional Research

Author Affiliations
  • 1Department of Pediatrics, Boston Medical Center, Boston, Massachusetts
  • 2Boston University School of Medicine, Boston, Massachusetts
JAMA. 2015;314(15):1561-1562. doi:10.1001/jama.2015.10962

Pilot intervention studies, commonly defined as small studies carried out in preparation for larger investigations,1 are essential precursors to high-quality clinical trials. Although not all trials are preceded by pilot investigations, when they are, pilot studies inform how subsequent trials are conducted and have an important role in controlling the pipeline of intervention development and dissemination by influencing which large-scale trials are ultimately carried out and which are never brought to fruition. However, pilot studies have often involved reporting inconsistencies, misapplication of research techniques, and misinterpretation of results, with significant implications for intervention research.1-3 These suboptimal research practices likely emanate from the understandable but methodologically unsound view of pilot studies as mechanisms to obtain preliminary answers to primary research questions.1-3 The risks of this approach may include unjustified and potentially misleading conclusions in the short term as well as misinformed decisions about how—and even whether—to proceed to more definitive studies.