Design and analysis of pilot studies: recommendations for good practice

TLDR

Pilot studies are essential in health research but are often misused, mistreated, or misrepresented. This paper focuses on pilot studies designed to inform the planning of randomized controlled trials. The authors present a methodological framework, illustrated with literature examples, and discuss why a pilot study might be undertaken. A well‑conducted pilot study with clear aims and objectives, framed formally, promotes methodological rigor, scientific validity, and publishability, leads to higher‑quality RCTs, and prevents studies driven solely by limited patient numbers.

Abstract

Abstract Pilot studies play an important role in health research, but they can be misused, mistreated and misrepresented. In this paper we focus on pilot studies that are used specifically to plan a randomized controlled trial (RCT). Citing examples from the literature, we provide a methodological framework in which to work, and discuss reasons why a pilot study might be undertaken. A well‐conducted pilot study, giving a clear list of aims and objectives within a formal framework will encourage methodological rigour, ensure that the work is scientifically valid and publishable, and will lead to higher quality RCTs. It will also safeguard against pilot studies being conducted simply because of small numbers of available patients.