The Clinical Trials Research Pillar in the Division of Biostatistics is at the forefront of innovative clinical trial design and execution, with faculty leading data coordinating center grants across various areas of medicine. The group collaborates with the institution’s Center for Multisite Clinical Studies to transform ideas into robust multicenter clinical trial proposals. Research areas include platform trial design, monitoring, and analysis, with a focus on the utility of shared control groups, and Bayesian methods for interim monitoring and effect estimation. Faculty also advance strategies for incorporating novel biomarkers, develop methodologies for cluster-randomized and stepped-wedge trials in implementation science, and refine risk-based monitoring procedures and tools.

Other key areas of focus include meta-analysis, interim monitoring of ongoing randomized trials, and drug efficacy and safety trials supporting FDA New Drug Applications (NDAs). The team also specializes in designing trials for assessing cancer immunotherapies and developing approaches for N-of-1 trials and the aggregation of their data. Together, these efforts reflect the group’s commitment to advancing clinical trial methodologies to improve patient outcomes and inform regulatory decision-making.

Lead Faculty

Andrea B. Troxel, ScD
Director, Division of Biostatistics
Professor

Chang Yu, PhD
Professor

Faculty Members

Rebecca Anthopolos. DrPH
Assistant Professor

Hayley Belli, PhD
Assistant Professor

Charles Cleland, PhD
Associate Professor

Ivan L. Diaz, PhD
Associate Professor

Judith D. Goldberg, ScD
Professor

Keith S. Goldfeld, DrPH
Associate Professor

Erinn M. Hade, PhD
Associate Director, Division of Biostatistics
Associate Professor

Jiyuan Hu, PhD
Assistant Professor

Nicholas Illenberger, PhD
Assistant Professor

Sharon Meropol, MD, PhD
Research Assistant Professor

Michele Santacatterina, PhD
Assistant Professor

Our Methodology Research

• Troxel AB, Hade EM. The Registry-Based Randomized Trial: A Pragmatic Study Design. NEJM Evidence, 2024. DOI: 10.1056/EVIDe2300310.
• Park H, Tarpey T, Liu M, Goldfeld K, Wu Y, Wu D, Li Y, Zhang J, Ganguly D, Ray Y, Paul SR, Bhattacharia P, Belov A, Huang Y, Villa C, Forshee R, Verdun NC, Yoon H, Agarwal A, Simonoivich VA, Scibona P, Pratx LB, Belloso W, Avendaño-Sola C, Bar KJ, Duarte RF, Hsue PY, Luetkemeyer AF, Meyfroidt G, Nicola AM, Mukherjee A, Ortigoz MB, Pirofski L, Rijnders BJA, Troxel A, Antman EA, Petkova E. Development and validation of a treatment benefit index to identify who may benefit from convalescent plasma among patients hospitalized with COVID-19. JAMA Network Open 5(1): e2147375, 2022.
• Anthopolos R, Maron DJ, Bangalore S, Reynolds HR, Xu Y, O’Brien SM, Troxel AB, Mavromichalis S, Chang M, Contreras A, Hochman JS. ISCHEMIA-EXTEND Studies: Rationale and Design. American Heart Journal 254: 228-33, 2022.
• Yu C, Huang X, Nian H, He P. A weighted log-rank test and associated effect estimator for cancer trials with delayed treatment effect. Pharmaceutical Statistics 2021, 20(3):528-550.

Our Collaborative Research

• Ortigoza MB, Yoon H, Goldfeld KS, Troxel AB, Daily JP, Wu Y, Yi L, Wu D, Cobb GF, Baptiste G, O’Keeffe M, Corpuz MO, Ostrosky-Zeichner L, Amin A, Zacharioudakis, IM, Jayaweera, DT, Wu Y, Philley JV, Devine MS, Desruisseaux, MS, Santin AD, Anjan S, Matthew R, Patel B, Nigo M, Upadhyay R, Kupferman T, Dentino AN, Nanchal R, Merlo CA, Hager DN, Chandran K, Lai JR, Rivera J, Bikash CR, Lasso G, Hilbert TP, Paroder M, Ascencio AA, Liu M, Petkova E, Bragat A, Shker R, McPherson DD, Sacco RL, Keller MJ, Grudzen CR, Hochman JS, Pirofski L. the CONTAIN COVID-19 Consortium for the CONTAIN COVID-19 Study Group. Efficacy and safety of COVID-19 convalescent plasma in hospitalized patients: A randomized clinical trial. JAMA Internal Medicine 182(2): 115-26. Doi:10.1001/jamainternmed.2021.6850, 2022.
• Hochman JS, Anthopolos R, Reynolds HR, Bangalore S, Xu Y, O’Brien SM, Mavromichalis S, Chang M, Contreras A, Rosenberg Y, Kirby R, Bhargava B, Senior R, Banfield A, Goodman SG, Lopes RD, Pracon R, Lopez-Sendon JL, Maggioni AP, Newman JD, Berger JS, Sidhu MS, White HD, Troxel AB, Harrington RA, Boden WE, Stone GW, Mark DB, Spertus JA, Maron DJ. Survival after invasive or conservative management of stable coronary disease. Circulation 147: 8-19, 2023.
• Rummo PE, Roberto CA, Thorpe LE, Troxel AB, Elbel B. Effect of financial incentives and default options on food choices of adults with low income in online retail settings: A randomized clinical trial. JAMA Network Open 6(3): e232371, 2023.