CLTR900: Analytic Approaches for Regulatory Science

Course Director:

Erin Wilhelm, MPH
Executive Director
Center of Excellence in Regulatory Science and Innovation (CERSI)
Program for Regulatory Science and Medicine (PRSM)
Instructor, Department of Pharmacology & Physiology

Course Description: Regulatory science analytic approaches will provide students with an introduction to the principles within patient-reported outcomes, scientific methods to gather and validate information through systematic reviews, and the use of bioinformatics. These techniques synthesize quantitative research by systematically searching literature or datasets for areas in which sufficient (and sufficiently comparable) data has accumulated.

This course will introduce students to systematic reviews and meta-analysis methods, patient-reported outcomes, and biomedical informatics as analytic tools and approaches used in regulatory science, clinical effectiveness research, and translational research. Students learn how to use a variety of methods for identifying, evaluating, and synthesizing information from randomized controlled trials and observational studies, assessing the strength of the evidence, and translating the results into regulatory decisions, as well as policy and practice guidelines. Concepts are introduced and illustrated through analysis of drug and vaccine safety and efficacy studies as well as other public health and medical case studies.

The use of health data mining techniques to explore data and identify sources of variation among studies is emphasized, as is the use of meta-analysis to identify future research questions. Patients-reported outcomes and the growing need for biomedical information technology tools, advanced biostatistics methods and health data min¬ing techniques are emphasized throughout the course and by utilizing relevant real-life examples. Students will be expected to carry out a real research project as described below. Results will be presented and discussed in class; select students will be invited to present to a public audience including interested researchers from Georgetown University, FDA, and MedStar Health Research Institute.

Prerequisite: Introduction to Biostatistics in Clinical Research (CLTR501) or equivalent

Learning Objectives:

  • Apply introductory statistical concepts to address issues of regulatory science. Statistical concepts may include:
    • Identify a research question
    • Conceptualize hypotheses
    • Identify sources of data
    • Use appropriate study designs
    • Determine the appropriate analytical methods
    • Draw valid and meaningful conclusions
    • Conduct a systematic review to identify a research question of interest.
  • Identify the sources of data necessary to address the question and determine the level of review required by the IRB.
    • Using existing data (e.g., EHR, publicly available databases such as NHANES, etc.)
    • Collecting new data (power calculation is necessary)
  • Apply bioinformatics techniques (e.g., Explorys) to improve the utilization of health records to conduct health services research, such as Patient Reported Outcomes.