The keynote speaker, Andrea Cook, PhD, is a senior investigator at Kaiser Permanente Washington Health Research Institute and an affiliate associate professor of biostatistics at the University of Washington. Cook is lead biostatistician for the Biostatistics and Study Design Core of the National Institutes of Health (NIH) Collaboratory, which facilitates the implementation of pragmatic clinical trials. She shared her experiences working on pragmatic clinical trials and related resources, such as the Living Textbook.
Amita Manatunga, PhD, professor of biostatistics at Emory University, presented the first case study, which aims to investigate the effectiveness of advanced care planning for end-stage renal disease (ESRD). The patient and family-centered care planning, specifically "Sharing Patient's Illness Representation to Increase Trust" (SPIRIT), intervention study brought to light new statistical considerations for Manatunga to investigate. The SPIRIT study presented was based on ongoing work with Mi-Kyung Song, PhD, professor of nursing at Emory University.
Kevin Dobbin, PhD, associate professor of biostatistics and BERD co-director at the University of Georgia, wrapped up the presentations for the day by focusing on a particular type of pragmatic trial, stepped wedge designs (SWD). He summarized the advantages and disadvantages of SWD and provided statistical advice for those considering to use SWD.
The Research Forum ended with time for participants to ask questions for the experts in the room. This discussion was moderated by Mohamed Mubasher, PhD, professor and senior biostatistician at Morehouse School of Medicine. Some ideas from this discussion included: 1) emphasis that randomized clinical trials are still the gold standard for research, but pragmatic trials can be used to address feasibility; 2) the importance of recording data and regular communication among researchers during the conduct of a pragmatic trial, to maintain study integrity; and 3) encouragement for methodological research in the area of pragmatic clinical trials, despite recent advances, particularly regarding unbiased designs, sample size determination for longitudinal outcomes, and interim analyses.