2019 Short Courses

1. An Introduction to Biomarkers for Statisticians – A Brief Guide to Utilizing the Most Appropriate Analytical Tools


  • Ena Bromley, BioStat Solutions, Inc.
  • Lin Li, BioStat Solutions, Inc.


The development in molecular biology and translation research has led us into the era of precision medicine.? Biomarkers are playing an increasingly important role in clinical research and drug development.? You may have heard some of these terms – APOE-ε4, PAM50, PD-L1, CD4, or HDL – all of these are biomarkers.? Then, what are biomarkers?? Why do they matter?? Which analytical tools are most appropriate in the design, analysis, and interpretation of biomarker data?

In this short course, biostatisticians and data scientists will be introduced to several common types of biomarkers, such as DNA, RNA, and proteins.? Topics that will be covered include familiarization with basic terminology, definitions, data structure, and statistical considerations around data quality control.? Attendees will also be introduced to predictive biomarkers, prognostic biomarkers, and pharmacodynamic biomarkers among others through illustration and visualization.? Typical biomarker analyses will be demonstrated through applications of commonly used statistical tools and methods.

Audience: Statisticians, data scientists

Required: Basic understanding of biology and statistics

2. Accelerating Drug Discovery Through Precision Medicine and Innovative Designs – Concepts, Rationales, and Case Studies


  • Sandeep Menon, Pfizer Inc. / Boston University / Tuffs University School of Medicine
  • Weidong Zhang, Pfizer Inc.


Precision medicine has paved the way for a new era of delivering tailored treatment options to patients according to their biological profiles. Advancement of the biotechnologies such as next generation sequencing (NGS) technology and other omics technologies have enabled us to interrogate a patient?s many molecular biomarkers and associate them with disease and drug responses.? In addition, incorporation of biomarker information in the innovative clinical trial design has presented drug developers unprecedented opportunities to bring successful drugs to patients in need.

The first part of this course will focus on the concepts of precision medicine, biomarker discovery, and their applications in clinical trials.? Comprehensive review of omics data and major technologies will be presented.? Statistical considerations and challenges, such as data normalization, dimension reduction, biomarker threshold development, and using biomarkers for decision making in clinical development, will be discussed in detail.

The second part of this course will focus on the strategy of the study design that is important to assessment of biomarker performance and reliability and will eventually lead to regulatory acceptance.? A general overview of the concept and statistical methodologies and designs related to precision medicine will be presented.? Specifically, we will discuss various designs including adaptive designs available at our disposal and its merits and limitations.

Audience: Statisticians, data scientists

Required: Basic understanding of biology, drug development , and/or clinical trials.

Category: Uncategorized

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