LAUSR

This special issue is dedicated to Duke-Industry Statistics Symposium (DISS) 2021, which was organized by the Department of Biostatistics and Bioinformatics, Duke University School of Medicine and co-sponsored by Amgen, ASA-NC Chapter, Boehringer-Ingelheim, Genentech, ICSA, IQVIA, Janssen, Journal of Biopharmaceutical Statistics, Lilly, Merck, Otsuka, Parexel, PPD, SAS, UCB, and ViiV. The theme is “Emerging Clinical Initiatives in Pharmaceutical Development: Methodology and Regulatory Perspectives”. Development in pharmaceutical industry is constantly evolving from the methodology and regulatory perspectives. Such development accelerates in these days because of great availability of big data and the inception of the 21 Century Cures Act (U.S. House of Representatives 2015), which stimulates the growing popularity in use of data from sources outside traditional clinical trials. It becomes possible that information such as those from disease or patient registries, claims data, historical trials, and digital health, can be leveraged to generate evidence to inform a regulatory decision-making. Consequently, statistical approaches for observational studies and big data become popular in pharmaceutical development. Methods that can be readily applicable in the regulatory setting are in great need. These methods often utilize techniques in fields such as propensity scores methodology, Bayesian statistics, and machine learning theory. Meanwhile, innovative designs and statistical methods for traditional clinical trials continue to be developed. Novel or complex clinical trial designs, such as various types of adaptive designs, are in need to fit for different situations. In addition, Bayesian inference has continued to make its influence in the pharmaceutical development. It is a great era for statisticians in pharmaceutical industry to develop innovative methods. Duke-Industry Statistics Symposium (DISS) 2021 was originally planned as an in-person meeting for 2020. Due to the pandemic, it was postponed to 2021 and was also converted to virtual format. It was successfully held in April 2021 under the leadership of Prof Xiaofei Wang and the Organizing Committee. DISS 2021 attracted more than 230 attendees with a fully packed agenda which includes 6 short courses, 25 parallel sessions, a virtual career fair, and poster sessions. Two keynote speeches were given by Dr ShaAvhrée Buckman-Garner from the FDA and Prof David Page, Chair from the Department of the Biostatistics & Bioinformatics which is also the host department of DISS. In the keynote speech delivered by Dr Buckman-Garner, Director – Office of Translational Sciences (OTS) Center for Drug Evaluation and Research at the FDA, she highlighted her perspectives on the progress, challenge, and opportunity. She described the OTS strategy via four goals through optimizing, enhancing/enabling, empowering, and connecting. New Drug Modernization Efforts were highlighted with six strategic objectives: scientific leadership, integrated assessment, benefit-risk monitoring, managing talent, operating excellence, and knowledge management. She also discussed a few challenges including but not limited to the need to 1) ensure that policies keep pace with technological