Course Detail

This course will provide students with a more in-depth understanding of clinical trial design, conduct and strategy for therapeutic products.  Clinical trial design elements will be examined in the context of their impact on clinical trial outcomes.  Emphasis will be placed upon trial designs that reflect the biological nature and mechanism of action of the therapeutic product being tested, rather than a cookbook approach.  Design elements related to small molecules, antibodies, therapeutic proteins, therapeutic vaccines, and cell and gene therapies will be discussed. Discussions will include operational issues impacting execution of clinical trials and why they are critical elements of successful clinical development programs.  Students will gain an understanding of the principles for use of particular biostatical testing procedures and in what context certain methods should be used.  There will be an emphasis on clinical development problem solving as students work on team projects and defend their own clinical development solutions in the context of product profiles, strategy and timelines.

Required Texts
Designing Clinical Research, By Hulley, Cummings, Browner, Grady, Hearst and Newman (Lippincott Williams and Wilkins, Publisher)
Fundamentals of Clinical Trials, By Friedman, Furberg and DeMets (Springer, Publisher)

• History of Clinical Trials  
• Phase 1, 2, and 3 study design
• Phase 4 and post-marketing study design
• Clinical Trial elements including objectives, eligibility criteria, treatment regimen, adverse event assessment and reporting, dose modification, efficacy response assessment, informed consent, data monitoring committees
• Operational issues including clinical site selection, interactions with Investigational Review Boards, data monitoring, clinical data management, case report forms
• Clinical trial study reports and analyses
• Basic biostatistics related to superiority, non-inferiority and equivalence study designs
• Clinical development program problem solving
• Clinical development strategy in a competitive landscape

After completing this course, students should be (1) conversant in clinical trial design elements including objectives, eligibility criteria, treatment regimen, adverse event assessment, dose modification, efficacy response assessment, informed consent, and data monitory committees, (2) knowledgeable in the critical operational issues that must be tracked for successful execution of clinical studies, (3) knowledgeable in the design principles for Phase 1, 2, 3 and 4 studies, (4) able to apply biostatical principles and know the appropriate settings in which to use particular biostatical test methods, (5) able to understand clinical trial analysis and reporting, (6) able to develop greater confidence in creating a clinical development strategy and (7) able to develop a compelling presentation to defend a particular clinical trial development strategy.

Classroom attendance and participation in course activities     20%
Written and/or oral assignments                                           20%
Team projects and presentations                                          30%
Final exam                                                                          30%