BIOS 6321: Clinical trials and experimental design (Spring 2016)

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• Restricted access

Instructor

• Tatsuki Koyama, PhD, Associate professor of biostatistics
• tatsuki.koyama@vanderbilt.edu; PRB 582; 936.1232
• Office hours: 3-4 Thursday (11138A WEA)

Teaching Assistant

• Allison Hainline
• allison.e.hainline@vanderbilt.edu
• Office hours: 12-1 Monday

Schedule

• Lectures: Tuesday and Thursday 1:30-3:00, 2525 Conference Room #11105

Course description

This course covers the statistical aspects of study designs, monitoring and analysis. Emphasis is on studies of human subjects, i.e. clinical trials. Topics include: principles of measurement, selection of endpoints, bias, blinding, randomization and balance, blocking, study designs, sample size, interim monitoring of accumulating results, flexible and adaptive designs, sequential analysis and adjustment techniques, history of clinical trials, and ethics of animal and human subject experimentation.

Prerequisite

• At least one semester of graduate level statistics
  • Bios 6311 or equivalent
• Familiarity with R
  • Bios 6301

Recommended materials

The lectures will loosely follow "Introduction to Statistical Methods for Clinical Trials" by Thomas D. Cook and David L. DeMets.
Other good books: ''Clinical trials: a methodologic perspective (2nd ed)'' by Piantadosi S
''Fundamentals of clinical trials (4th ed)'' by Friedman LM, Furberg CD, DeMets DL

Assignments

There will be homework assignments throughout the semester. Collaboration with classmates is encouraged, but you need to submit homework individually.

Exams

The midterm and final exams will be take-home. You will have a weekend (Thursday - Tuesday) to work on them. You are to work by yourself; no discussion is allowed.

Grading

• Homework and project (and class participation): 30%
• Midterm Exam (Take home): 35%
• Final Exam (Take home): 35%

The final exam will focus on the post-midterm materials, however, key concepts covered before the midterm may (almost surely) also appear.

Tentative schedule

• Midterm exam: 2/25 Thu (due 3/1 Tue)
• Final exam: 4/21 Thu (due 4/26)

1. Introduction and topics in statistics. (1/12 Tue ~ 1/28 Tue): [Chapter 1]
   • Experiments and observational studies (1 lecture)
   • Clinical trial phases (1 lecture): [1.3]
   • Topics in hypothesis testing (2 lectures)
   • Bayesian approaches (1 lecture)
2. Some fundamental topics in experimental design (2/2 Thu ~ 2/11 Tue)
   • Randomization (2 lectures): [Chapter 5]
   • Sample size and power (2 lectures): [Chapter 4]
3. Early phase clinical trials (2/13 Thu ~ 2/25 Tue)
   • Phase I clinical trials (1 lecture): [3.1.1]
     • Class discussion (1 lecture)
   • Phase II clinical trials (2 lectures): [3.1.2]
4. Phase III clinical trials: [3.2]
   • Introduction and group sequential designs (2 lectures): [Chapter 10]
   • Adaptive and flexible designs (2 lectures): [10.4, 10.7, 3.5]
     • Guest lecturer (1 lecture)
   • Factorial designs (2 lectures): [3.2.2]
   • Crossover designs (2 lectures): [3.2.2]
5. Other topics
   • Fraud and misconduct
   • Quality of life: [Chapter 9]
   • Noninferiority designs: [3.3]