1. COURSE PROPOSAL: METHODS IN CLINICAL CANCER RESEARCH Primary Instructor: Elizabeth Garrett-Mayer, PhD Professor of Biostatistics, Dept. of Public Health Sciences Director of Biostatistics, Hollings Cancer Center

2. Context: K-12 award Paul Calabresi Clinical and Translational Oncology Training Program Goal: To train successful patient-oriented clinical and laboratory-based investigators who can work effectively with each other in a collaborative, translational research setting Program learning objectives: Researchers will be able to independently design, implement and manage all types of clinical trials with an emphasis on translation of laboratory findings into clinical applications, as well as facilitating laboratory studies aimed at examining the basic mechanisms of poorly understood clinical phenomena.

3. Didactic Training Fall Year 1Spring Year 1Summer Year 1Fall Year 2 Clinical Biostatistics Core Clinical Research Training Industry/Regulatory Grants Overview Clinical Epidemiology Methods in Clinical Cancer Research Critical ReviewLeadership EthicsCancer Immunology Principles of Cancer Biology (PhD)/Multi. Disc. Clinical Oncology (MD) Methods and Outcomes in Cancer Pop’n Sciences Principles of Targeted Drug Discovery/ Translational ’Omics MSCR core coursesK-12 courses

4. Methods in Clinical Cancer Research Modeled largely after the “Vail” course which is funded by NCI, AACR, ASCO and industry and started in 1996 to train clinical researchers in oncology. Occurs every summer in Vail, CO. The workshop itself is one week long with didactic lectures daily (4-8 lectures per day). Proposed curriculum includes greater detail on trial design. Required for K-12 scholars, but open to all students at MUSC.

5. Course Objectives At the end of the course, students should be able to: Understand the key components required for designing, activating and implementing a cancer clinical trial. Write a proposal for a cancer clinical trial, including objectives, endpoints, trial design, patient population selection, and have some understanding of the required sample size and analytic techniques used to analyze the data at the end of the trial. Effectively review and critique clinical trial protocols and published cancer clinical trials research.

6. Course Content Topics Number of lectures Cumulative hours Instructor Trial Designs and Analysis Phase I34.5 EGM, Britten Phase II39 EGM Phase III313.5 EGM Prevention and Control115 Alberg Observational studies116.5 Wallace Endpoint selection118 EGM Power calculations119.5 EGM Correlative studies121 EGM Imaging in clinical trials122.5 Ravenel Trials in specific treatment modalities124 Warren Quality of life and other patient reported outcomes125.5 Sterba Informed Consent127 Stuart/Britten/Lilly Protocol Review and IRB process128.5 Warren/EGM Data collection and privacy130 EGM Trial monitoring and investigator responsibilities131.5 Britten Grants and mentoring133 Warren Disparities research134.5 Ford Protocol review meetings237.5 NA Journal club discussion**545 EGM TOTAL3045

7. Evaluation Students will be graded based on the following components where each assignment is given numeric score, according to the Merit Grades for the MUSC grading system. Written reviews of protocols, given as assignments. There will be 3-4 protocols assigned and the review will be structured with particular questions about appropriateness of study design, clarity of the study aims, incorporation of early stopping rules in the trial design, etc. (45% of grade) Oral presentation of journal article presenting results of a cancer clinical trial. The article will be selected by the student and Dr. Garrett-Mayer. The student will present to the class an overall summary of the trial and provide a critique of the methods employed. (25% of grade) Submitted LOI: The LOI will be submitted twice. First, a draft will be submitted about two-thirds through the course. Dr. Garrett-Mayer will provide feedback. This first draft will constitute 15% of the total grade. The final LOI will be submitted as the ‘final’ and will also count for 15% of the course grade. Total: 30% of grade The three components will be combined (using the percentages indicated) to arrive at a final merit grade for the course.

8. Other details Prerequisites: Consent of the instructor Textbooks: None required. Suggested texts for reference: Clinical Trials: A Methodologic Perspective (Piantadosi) Oncology Clinical Trials (Kelly & Halabi) Principles of Anti-Cancer Drug Development (Hidalgo, Eckhardt, Garrett-Mayer, Clendennin)