University of Pittsburgh, Pittsburgh, Pennsylvania, USA.
Clin Transl Sci. 2011 Oct;4(5):332-7. doi: 10.1111/j.1752-8062.2011.00347.x.
Advances in clinical and translation science are facilitated by building on prior knowledge gained through experimentation and observation. In the context of drug development, preclinical studies are followed by a progression of phase I through phase IV clinical trials. At each step, the study design and statistical strategies are framed around research questions that are prerequisites for the next phase. In other types of biomedical research, pilot studies are used for gathering preliminary support for the next research step. However, the phrase "pilot study" is liberally applied to projects with little or no funding, characteristic of studies with poorly developed research proposals, and usually conducted with no detailed thought of the subsequent study. In this article, we present a rigorous definition of a pilot study, offer recommendations for the design, analysis and sample size justification of pilot studies in clinical and translational research, and emphasize the important role that well-designed pilot studies play in the advancement of science and scientific careers.
临床和转化科学的进展得益于通过实验和观察获得的先前知识的积累。在药物开发的背景下，临床前研究之后是 I 期到 IV 期临床试验的逐步推进。在每个阶段，研究设计和统计策略都围绕着研究问题展开，这些问题是下一阶段的前提条件。在其他类型的生物医学研究中，初步研究用于为下一步研究提供初步支持。然而，“初步研究”一词被广泛用于几乎没有或没有资金的项目，这些项目的特点是研究提案制定不充分，通常在没有后续研究详细思考的情况下进行。在本文中，我们提出了一个严格的初步研究定义，为临床和转化研究中的初步研究设计、分析和样本量论证提供了建议，并强调了精心设计的初步研究在推进科学和科学事业中的重要作用。
