SC-07 - Cancer Pharmacoepidemiology and Genomics: What You Should Know in the Era of PrecisionMedicine

1. Course Aim

This course, co-sponsored by the “Molecular Epidemiology, Biomarkers, and Pharmacogenomics” and “Cancer” SIGs, will focus on the essentials every pharmacoepidemiologist should know about study design, databases and real-world applications of cancer personalized medicine including precision medicine, companion diagnostics, biomarkers and the biomarker development pipeline, diagnosis and treatment coding, drug and cancer risk associations and linking databases with genomic information.

2. Requisites Statement

Entry level. Attendees should have basic knowledge of pharmacogenomics.

3. Course Objectives

-To acquire an overview of genomics in cancer research, and how epidemiologists and real-world studies make contributions.
-To attain a better understand of methodological issues related to pharmacoepidemiology studies of cancer risk.
-To improve an understanding of cancer diagnosis and treatment coding in administrative claims, EHR and clinico-genomic databases.
-To understand better target therapy for application in pharmacoepidemiology cancer research designs and oncology drug development, including the use of biomarkers and companion diagnostics.
-To understand the benefits and challenges of pharmacoepidemiology study designs in oncology research and their impact on clinical practice applications.

4. Syllabus Outline

Introduction, Format and Objectives of the Course
o Through a series of case study-based lectures and a panel discussion this course will enhance your understanding of the important role pharmacoepidemiologist’s play in the design, analysis, evaluation, database utilization and interpretation of
o medication exposure and cancer risk
o oncology drug development,
o real-world evidence studies cancer drug effectiveness, and
o real-world evidence studies of adverse events
o Speakers will start with a case study, and weave their talk through the case study, using the study to introduce key challenges and potential solutions and may include alternative approaches, and individualization based on characteristics of the clinical situation.

A. Designing and Analysing Pharmacoepidemiology Studies in Cancer Research [Kelly Filipski]- 30 min
• Will present an introduction to cancer genomics - using both somatic and germline genomic data as examples

B. Clinical Evidence Generation in Oncology: Real World Data Designs and Approaches [Donna Rivera]- 30 minutes
o Will provide an overview of the current RWE Guidances as well as foundational approaches to study design and selection of RWD sources (administrative claims, EHRs, registiries) in oncology, as well as the limitations of various sources. Additionally, the discussion will include novel designs for clinical evidence generation with examples.

C. Cancer Pharmacoepidemiology and Pharmacogenomics in Oncology Drug Development [Wei Zhou and Leah Sansbury]- 30 minutes
• Will present a case study of the development of an anti-cancer agent.
o Examples of a case study may include: a PD-1/PD-L1 drug (such as Keytruda) or an anti-cancer targeted therapy or immunotherapy (such as a anti- B-Cell Maturation Antigen) or another example chosen by the speakers.
• Using this case example, we will discuss the epidemiologist’s contribution in different phases oncology drug development and includes important study design and evaluation concepts during the oncology drug pipleline with special consideration of pharmacogenomics and target therapy.

BREAK and Networking (30 minutes)

D. Designing Cancer Pharmacoepidemiology-Pharmacogenomics Studies to study Adverse Events [Bruce Carleton]-30 minutes
• Will present a case study for the design, analysis and interpretation of an adverse event for an anti-cancer agent.
o Case study will focus on vincristine neurotoxicity.
• Using this case example, we will provide considerations to how to efficiently approach the study of cancer therapy-induced adverse events with a particular focus on genomics study design, conduct and analysis to be derived from pharmacoepidemiological data and DNA-based genomics analyses. Techniques to use include genome-wide association studies, exome sequencing and whole genome sequencing based on validated phenotypes. Validation methods other than independent replication are discussed and why replication studies are inherently problematic. This example will help illustrate how the pharmacoepidemiological study of adverse events and pharmacogenomics can help improve the safety of cancer therapy.

E. Real-World Study Design and Analysis of Cancer Drug Effectiveness [Geoff Liu]- 30 minutes
• Will present a case study for the design, analysis and interpretation of the effectiveness of a currently approved anti-cancer agent in different populations or for additional indications.
o Examples of a case study may include: a PD-1/PD-L1 drug or another anti-cancer targeted or immunotherapy or another example chosen by the speakers.
• Using this case example, we will provide considerations to how to efficiently approach a real-world evidence study of an approved cancer therapy for effectiveness in different populations, sub-populations and additional clinical indications. There will be a particular focus on design, conduct and analysis, and discuss unique aspects of somatic cancer pharmacogenomics, focusing on somatic biomarkers including mutations, gene or protein expressions.

F. Interactive session – [All Faculty] 30 minutes
An opportunity to develop an approach to the design of cancer pharmacoepidemiology and genomics studies

G. Panel Discussion [Kelly Filipski, Moderator] – 30 minutes
a. Five-minute summary of the main points of the course; emerging topics (Andrew Freedman)
b. Focus some questions on AI
c. Get audience involved