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 TopicsSorted ascending Note
16 4/25/17   Final exam due
  4/20/17 13  
  2/23/17 -- -- -- -- No class
9 3/7/17 -- -- -- -- Spring break
  3/9/17 -- -- -- -- Spring break
  3/30/17 -- -- -- -- No Class
  4/27/17 -- -- -- --  
17 5/2/17 -- -- -- -- Finals week
  5/4/17 -- -- -- -- Finals week
14 4/11/17 Adaptive designs  
  4/13/17 Adaptive designs  
  1/19/17 Bayesian analysis
Randomization
 
11 3/21/17 Crossover design  
  3/23/17 Crossover designs  
13 4/4/17 Dr. Lorraine Ware  
  4/6/17 Dr. Rafe Donahue  
1 1/10/17 Experiments and observational studies  
10 3/14/17 Factorial design  
15 4/18/17 Fraud and misconduct  
8 2/28/17 Group sequential design Midterm exam due
  3/2/17 Group sequential design  
12 3/28/17 Meta analysis  
7 2/21/17 Non-inferiority  
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  
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
 
  1/12/17 Topics in hypothesis testing  
2 1/17/17 Topics in hypothesis testing
Clinical trial phases
Bayesian analysis
 
  3/16/17 Topics in regression analysis  
Edit | Attach | Print version | History: r17 | r16 < r15 < r14 < r13 | Backlinks | View wiki text | Edit WikiText | More topic actions...
Topic revision: r15 - 27 Mar 2017, TatsukiKoyama
 

This site is powered by FoswikiCopyright © 2013-2022 by the contributing authors. All material on this collaboration platform is the property of the contributing authors.
Ideas, requests, problems regarding Vanderbilt Biostatistics Wiki? Send feedback