BIOS 6321: Clinical trials and experimental design (Spring 2018)

For students only


Teaching Assistant

  • Lectures: Tuesday and Thursday 1:30-3:00, 2525 Conference Room #11105
  • Midterm exam: 2/27/18 (due)
  • Final exam: 4/24/18 (due)

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.


If you are not a graduate student in the Department of Biostatistics, please email me to confirm you have sufficient math background to take this course.

  • At least one semester of graduate level statistics
  • Calculus (3+ semesters)
  • Familiarity with R

Recommended materials

The lectures will loosely follow "Statistical Design and Analysis of Clinical Trials: Principles and Methods" by Weichung Joe Shih and Joseph Aisner.
Other good books: "Introduction to Statistical Methods for Clinical Trials" by Thomas D. Cook and David L. DeMets; ''Fundamentals of clinical trials (4th ed)'' by Friedman LM, Furberg CD, DeMets DL


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


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.

  • 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.


  1. Introduction and topics in statistics
    • Experiments and observational studies (1 lecture)
    • Clinical trial phases (1 lecture)
    • Topics in hypothesis testing (2 lectures)
    • Bayesian approaches (1 lecture)
  2. Some fundamental topics in experimental design
    • Randomization (2 lectures)
    • Sample size and power (2 lectures)
  3. Early phase clinical trials
    • Phase I clinical trials (1 lecture)
      • Class discussion (1 lecture)
    • Phase II clinical trials (2 lectures)
  4. Phase III clinical trials
    • Introduction and group sequential designs (2 lectures)
    • Adaptive and flexible designs (2 lectures)
      • Guest lecturer (1 lecture)
    • Factorial designs (2 lectures)
    • Crossover designs (2 lectures)
  5. Other topics
    • Fraud and misconduct
    • Quality of life
    • Noninferiority designs

Week Date Topics Note
1 1/9/18 Experiments and observational studies  
  1/11/18 Topics in hypothesis testing  
2 1/16/18 Snow day!! No class  
  1/18/18 Sick day!! No class  
3 1/23/18 No class (Department event) Homework 1 due
  1/25/18 Topics in hypothesis testing
Clinical trial phases
4 1/30/18 Bayesian analysis  
  2/1/18 Randomization  
5 2/6/18 Randomization  
  2/8/18 Sample size and power Homework 2 due
6 2/13/18 Special lecture -Jonathan Chipman  
  2/15/18 Sample size and power Homework 3 due
7 2/20/18 Sample size and power / Phase I clinical trials
  2/22/18 Phase I clinical trials Homework 4 due
8 2/27/18 Phase I clinical trials Midterm Exam due
  3/1/18 Phase I clinical trials / intro to Phase II clinical trials  
9 3/6/18 -- -- -- -- Spring break
  3/8/18 -- -- -- -- Spring break
10 3/13/18 Phase II clinical trials  
  3/15/18 Phase II clinical trials  
11 3/20/18 Noninferiority hypothesis
Homework 5 due
  3/22/18 Phase III clinical trials / Group sequential designs  
12 3/27/18 Group sequential designs Homework 6 due
  3/29/18 Adaptive designs  
13 4/3/18 Adaptive designs Homework 7 due
  4/5/18 Factorial designs  
14 4/10/18 Crossover designs  
  4/12/18 Crossover designs Homework 8 due
15 4/17/18 Guest lecturer (Dr. Lorraine Ware)  
  4/19/18 Meta analysis Homework 9 due
16 4/24/18   Final exam due
17 5/1/18    

Topic revision: r19 - 23 Apr 2018, TatsukiKoyama

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