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

For students only

• Restricted access

Instructor

• Tatsuki Koyama, PhD, Associate professor of biostatistics
• tatsuki.koyama@vanderbilt.edu; PRB 582; 936.1232
• Office hours: Thursdays 12:30-1:30 Cancer Division office at 2525.

Teaching Assistant

• JoAnn Alvarez, MA, Biostatistician III
• Office hours: Tuesdays 3:00-4:00 Cancer Division office at 2525
• joann.alvarez@vanderbilt.edu

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

-- TatsukiKoyama - 06 Jan 2013