We are pleased to release a special issue devoted to the 2022 ASA Biopharmaceutical Section Regulatory-Industry Statistics Workshop (RISW). It is a great tradition now to offer you a special issue in Statistics in Biopharmaceutical Statistics (SBR) for the annual workshop from RISW 2022. We are extremely thankful to Estelle Russek-Cohen, Dejun Tang, and David Ohlssen as Guest Associate Editors as well as the Editor-in-Chief of SBR, Toshimitsu Hamasaki. The editorial team was just wonderful to work with throughout thisprocess.
This special issue includes nine articles that represent diverse topics in statistics presented at the workshop. These topics include dose optimization, group sequential trials, adaptive platform trials, and clinical trial endpoint research in the biopharmaceutical industry and regulatory science. Many of these articles pursue Bayesian framework as well as some comparisons of methodologies. We hope that you find these articles helpful, and that this also sheds light on the many topics covered more in-depth at the Regulatory-Industry Statistics Workshop, sponsored by the ASA Biopharmaceutical Section.
The theme of RISW 2022 was Statistics Post-Pandemic: Paving the Scientific Path to Treatments, Vaccines, and Diagnostics. This conference was a celebration of many things: connecting again in-person with colleagues and friends, inclusion from many different statistical sections of the ASA—including Medical Device and Diagnostics—as well as a diversity of topics. Last—but certainly not least—we got to celebrate in-person the 40th Anniversary of the ASA Biopharmaceutical Section. As the theme indicates, it takes all areas to provide the best healthcare—treatments, vaccines, and diagnostics. It was also very significant from the perspective that it was the first in-person conference for RISW since 2019, prior to the pandemic. Not only was it the first in-person conference in a few years, but it was also the first time that the workshop was held in Rockville, MD at the Bethesda North Marriott Hotel and Conference Center. The organizing committee was wonderful, diverse in experience and perspective, and was very open-minded and flexible when it came to planning an in-person conference, hybrid, or prepared to go completely virtual again.
Two plenary sessions were offered on the first day after short courses. “Real World Evidence: Paving the Way for Innovation” was introduced by Donna Rivera, Associate Director for pharmacoepidemiology at the US Food and Drug Administration (FDA) Oncology Center of Excellence. Donna discussed the integrated nature of epidemiology and statistics in design and conduct of studies using real-world data along the evidence generation continuum. Through examples, she illustrated the accomplishments and future possibilities when statisticians and epidemiologists work together. Following Donna was Nancy Dreyer, emeritus for IQVIA and adjunct professor of epidemiology at The University of North Carolina at Chapel Hill. Her topic was how real-world evidence (RWE) is advancing scientific research and conclusions in multiple areas of research. She discussed various studies using large data during the pandemic and how the data informed decisions. In particular, she discussed the design and the data behind the NBA bubble studies. Panel members were Ted Lystig and PallaviMishra-Kalyani.
The second plenary session was a townhall format on the “Impact of Pandemic, Technology, and other External Factors on Health Care Research.” Hong Lu from the Division of Biostatistics at CDRH discussed FDA’s considerations on validation of software as a medical device for diagnostic purposes. Andre Potter from Office of Biostatistics at CDER discussed FDA guidance on digital health technology (DHT) and analysis considerations for DHT-derived endpoints. CV Damaraju, Janssen R&D statistician, discussed the first clinical trial managed with technology and remote visits. Gail Kongable, Abbott Senior Manager and family nurse practitioner discussed modeling and simulation of nutrition on diabetes and blood glucoselevels.
The overall program included 10 short courses, 2 plenary sessions, 42 parallel sessions, 43 roundtable discussions, and 38 posters. It was the first time that round tables could be expanded given the extra room afforded by the location and hotel. The parallel sessions covered many topics, but the most represented estimands and missing data, RWE, and complex/innovative study design.
Planning this first in-person workshop at a new venue was a unique experience. The committee had to constantly be flexible, open-minded, and try to overcome fear of getting back together with so many people after over 2 years of mostly remote work by most people. We are greatly appreciative of the workshop’s Steering Committee (members and advisers), ASA meeting planner Kristin Mohebbi, the ASA Biopharmaceutical Section Executive Committee and 40th Anniversary Committee, the ASA Section on Medical Devices and Diagnostics, and all our sponsors. The co-chairs of the workshop did not author any of the articles presented in thisedition.
We hope that you enjoy this special edition of SBR and that it spurs you to think of additional areas of research in statistics that are further needed or that need to be discussed in the appropriate forums going ahead.