Methods Seminar: Pilot Studies versus Feasibility Studies to inform Future RCTs

There is growing consensus in the biostatistical community that pilot studies being done in preparation for RCTs should not be used to estimate effect sizes. If this is true, what is the purpose of a pilot study, and how is a pilot study different from a feasibility study? And how should grant applicants approach these issues strategically, given that some study sections still expect a pilot study to estimate an effect size? We will cover all of these issues and more related to pilot study design in this 1-hour moderated panel with statisticians and health services researchers with extensive expertise writing and reviewing pilot study grant proposals. Panelist Speakers: David Elashoff PhD, Professor of Medicine, Biostatistics and Computational Medicine, Director of UCLA CTSI Biostatistics, Epidemiology and Research Design (BERD) program. Sitaram Vangala, Principal Statistician, UCLA CTSI BERD Kenrik Duru MD, Professor of Medicine and Co-PI, UCLA Resource Center for Minority Aging Research/Center for Healthcare of Minority Elders (RCMAR/CHIME) Moderator: Catherine Sarkisian MD, Professor of Medicine and Co-Director of UCLA CTSI Special Populations Core References: • Tseng CH, Sim D, Sample size planning for pilot studies. arXiv preprint arXiv:2105.05483, 2021 • Westlund E, Stuart EA. The nonuse, misuse, and proper use of pilot studies in experimental evaluation research. American Journal of Evaluation. 2017 Jun;38(2):246-61. • Thabane L, Ma J, Chu R et al. A tutorial on pilot studies: the what, why and how. BMC medical research methodology 2010; 10(1): • Lancaster GA, Dodd S and Williamson PR. Design and analysis of pilot studies: recommendations for goodpractice. Journal of evaluation in clinical practice 2004; 10(2): 307–312. • Kistin C and Silverstein M. Pilot studies: a critical but potentially misused component of interventional research. Jama 2015; 314(15): 1561–1562. • https://statmodeling.stat.columbia.ed...