BIOS 321: Clinical trials and experimental design (Spring 2014)

For students only

Instructor

Teaching Assistant

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, masking, randomization and balance, blocking, study designs, sample size projections, study conduct, interim monitoring of accumulating results, flexible and adaptive designs, sequential analysis, analysis principles, adjustment techniques, compliance, data and safety monitoring boards (DSMB), Institutional Review Boards (IRB), the ethics of animal and human subject experimentation, history of clinical trials, and the Belmont report.

Prerequisite
Bios 311 or equivalent
Bios 301 or equivalent (familiarity with R)
Familiarity with R: If you did not take Bios 301, check with the instructor.

Textbook and other recommended materials
Required book: ''Clinical trials: a methodologic perspective (2nd ed)'' by Piantadosi S
Recommended book: ''Fundamentals of clinical trials (4th ed)'' by Friedman LM, Furberg CD, DeMets DL

Assignments
There will be homework assignments throughout the semester. You are welcome to discuss the homework assignments with classmates, but you need to submit homework individually.

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

Week Session Date Topic Reading (PS) Other reading Homework/Exam
1 1 1/7 (Tu) Experimental studies and observational studies      
  2 1/9 (Th) Clinical trial terminologies and clinical trial phases 2.1 - 2.2, 6.1 - 6.3    
2 3 1/14 (Tu) Topics in hypothesis testing 5.1 - 5.2    
  4 1/16 (Th) Bayesian approaches 5.3 - 5.6    
3 5 1/21 (Tu) schedule adjustment      
  6 1/23 (Th) Randomization and masking/blinding 13.1 - 13.9    
4 7 1/28 (Tu) Randomization in clinical trials      
  8 1/30 (Th) Sample size and power 1 11.1 - 11.5    
5 9 2/4 (Tu) Sample size and power 2 11.6 - 11.7    
  10 2/6 (Th) Phase I clinical trials 1 10.1 - 10.3    
6 11 2/11 (Tu) Phase I clinical trials 2 10.4 - 10.7    
  12 2/13 (Th) ECMO and TGN1412      
7 - 2/18 (Tu) No class Brad Calin short course      
  13 2/20 (Th) Phase II clinical trial     Midterm exam due 2/25
8 14 2/25 (Tu) Guest Ben Saville, PhD    
  15 2/27 (Th)        
9 - 3/4 (Tu) No class Spring break      
  - 3/6 (Th) No class Spring break      
      Schedule adjustment Topics: Phase III clinical trials (group sequential and adaptive designs); Meta-analysis; IRB; Misconduct and fraud;    
10 16 3/11 (Tu) Phase II clinical trial      
  17 3/13 (Th) Phase III clinical trial      
11 18 3/18 (Tu) Group Sequential Designs      
  - 3/20 (Th) No class      
12 19 3/25 (Tu) Phase III clinical trial      
  20 3/27 (Th) Phase III clinical trials      
13 21 4/1 (Tu) Factorial designs 19.1 - 19.7    
  22 4/3 (Th) Guest Rafe Donahue, PhD    
14 23 4/6 (Tu) Factorial designs      
  24 4/10 (Th) Crossover designs 20.1 - 20.5    
15 25 4/15 (Tu) Crossover designs      
  26 4/17 (Th) Fraud and Misconduct     Final exam due 4/22
16 27 4/22 (Tu)        

-- TatsukiKoyama - 06 Jan 2013
Topic revision: r12 - 25 Aug 2015, RobertGreevy
 

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