Statistical Methodologies for Modern Clinical Studies

Code: MA781 | L-T-P-C: 3-0-0-6

Probability Theory (MA683) or equivalent

Preamble / Objectives:

Statistical methods play an essential role in designing and analysing modern clinical studies. Multi-stage clinical trials in personalized medicine require advanced statistical methodologies. This course will enable PhD students to learn existing and new methodologies that are used in advanced clinical studies with real examples. The course is designed to prepare Ph.D. students to develop new statistical methodologies for advanced clinical studies/trials.

Course Content/ Syllabus:

Overview of clinical trials: design and analysis of phase-I dose-finding trials, overview of phase-II and phase-III trials. Regulatory affairs: data monitoring and interim analysis. Statistical methods to design and analysis: non-inferiority and equivalence trials, factorial and cross-over trials, cluster randomized trials and stepped wedge trials, sequential, group-sequential and adaptive trials, seamless phase II/III trials. Pharmacovigilance: phase IV trials including spontaneous reporting system. Dynamic treatment regimens: sequential multiple assignment randomized trials (SMART) designs.

Texts:

1. Steven Piantadosi. Clinical Trial: A methodological Perspective. Second Edition, WILEY, 2005.
2. Frideman, Furberg, DeMets, Reboussin and Granger. Fundamental of Clinical Trials. Fifth Edition, Springer, 2015.
3. Cook TD and DeMets DL. Introduction to Statistical Methods for Clinical Trials. First Edition, Chapman & Hall/CRC, Boca Raton, FL,  2008.
4. Chakraborty B and Moodie EEM. Statistical Methods for Dynamic Treatment Regimes: Reinforcement Learning, Causal Inference, and Personalized Medicine. First Edition,  Springer, New York, 2013.