BIOS 6321: Clinical trials and experimental design (Spring 2017)

For students only

Instructor

Teaching Assistant

Schedule
  • Lectures: Tuesday and Thursday 1:30-3:00, 2525 Conference Room #11105
  • Midterm exam: 2/28/17 (due)
  • Final exam: 4/25/17 (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.

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

Recommended materials
The lectures will loosely follow "Introduction to Statistical Methods for Clinical Trials" by Thomas D. Cook and David L. DeMets.
Other good book: ''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.

Topics

  1. Introduction and topics in statistics
    • 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
    • Randomization (2 lectures)
    • Sample size and power (2 lectures)
  3. Early phase clinical trials
    • 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)
    • 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
    • Noninferiority designs: [3.3]

Week Date Topics Note
1 1/10/17 Experiments and observational studies  
  1/12/17 Topics in hypothesis testing  
2 1/17/17 Topics in hypothesis testing
Clinical trial phases
Bayesian analysis
 
  1/19/17 Bayesian analysis
Randomization
 
3 1/24/17 Randomization  
  1/26/17 Randomization
Sample size and power
 
4 1/31/17 Sample size and power  
  2/2/17 Sample size and power
Phase I clinical trials
 
5 2/7/17 Phase I clinical trials  
  2/9/17 Phase I clinical trials  
6 2/14/17 Phase II clinical trials  
  2/16/17 Phase II clinical trials  
7 2/21/17 Non-inferiority  
  2/23/17 -- -- -- -- No class
8 2/28/17 Group sequential design Midterm exam due
  3/2/17 Group sequential design  
9 3/7/17 -- -- -- -- Spring break
  3/9/17 -- -- -- -- Spring break
10 3/14/17 Factorial design  
  3/16/17 Topics in regression analysis  
11 3/21/17 Crossover design  
  3/23/17 Crossover designs  
12 3/28/17 Meta analysis  
  3/30/17 -- -- -- -- No Class
13 4/4/17 Dr. Lorraine Ware  
  4/6/17 Dr. Rafe Donahue  
14 4/11/17 Treatment Effect Monitoring  
  4/13/17 Adaptive designs  
15 4/18/17 Adaptive designs  
  4/20/17 Fraud and misconduct  
16 4/25/17   Final exam due
  4/27/17 -- -- -- --  
17 5/2/17 -- -- -- -- Finals week
  5/4/17 -- -- -- -- Finals week
Topic revision: r17 - 16 Apr 2017, TatsukiKoyama
 

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