Statistical Issues in Design, Monitoring, and Analysis of Randomized Controlled Trials

BIOSTAT 226 Winter 2019 (1 unit)
Course Director: Joan Hilton, ScD, MPH
Department of Epidemiology & Biostatistics


This course covers current issues in the design and analysis of randomized clinical trials (RCTs). It is part of the Biostatistical Methods in Clinical Research series, continuing on from Biostatistical Methods IV (BIOSTAT 210), covering additional biostatistical methods and allowing more in-depth exploration of the topics of Biostatistical Methods II (BIOSTAT 208) and Biostatistical Methods III (BIOSTAT 209).

In the healthcare setting, RCTs are experiments in which investigators randomly assign eligible patients into intervention groups, and then compare the outcomes of the groups to determine the relative effect of the intervention. The primary outcome variable in such trials is typically a measure of ultimate clinical status, such as disease progression. In this course, we will examine study design and analytic methods that have been developed to ensure the validity of the results from RCTs.

By the end of the course, students will have:

  • a working knowledge of a variety of classical and adaptive study designs for clinical trials;
  • understood the methods behind interim monitoring of Phase III trials;
  • be able to design a superiority or noninferiority trial for binary responses; and
  • understood concepts behind Bayesian designs and analyses.

Designing Clinical Research (EPI 202), Biostatistical Methods I (BIOSTAT 200), Biostatistical Methods II (BIOSTAT 208) and Biostatistical Methods III (BIOSTAT 209). Exceptions to these prerequisites may be made with the consent of the Course Director, space permitting.

Course Director:

Joan Hilton, ScD, MPH
Phone: 415-514-8029


Course content will be delivered through weekly interactive lectures. These will take place on Tuesdays from 10:30 AM to 12:00 PM, Feb. 12 to Mar. 19.

All course materials and handouts will be posted on the course's online syllabus.


Each lecture will be accompanied by homework, assigned on the day of the lecture. Homework assignments are due prior to the following lecture and should be submitted by e-mail to the Course Director.

Grades will be based on 5 homework assignments (75%) and final exam (25%).

Students not in full-year TICR Programs who satisfactorily pass all course requirements will, upon request, receive a Certificate of Course Completion.

UCSF Graduate Division Policy on Disabilities


This course is sponsored by the Training in Clinical Research (TICR) Program, and space is limited. Preference is given to UCSF-affiliated personnel. We regret that auditing is not permitted.

To apply for this course, please fill out and submit the application below. Please see our fees page for cost information. The deadline for application is December 21, 2018. Only one application needs to be completed for all courses desired during the quarter.

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