短期課程

September 6 - Afternoon (1-4 pm)

(SC1) Fit-for-Purpose Biomarker Assay Development and Validation

This tutorial will provide recommendations on the fit-for-purpose best practices in the development and validation of biomarker assay for exploratory or advanced biomarker applications. Strategies for different applications at various phases of biomarker development will be described.

• Nomenclature, Types of Biomarker Methods/Assays, Method Development and Validation Roadmap
• Pre-Analytical and Bioanalytical Elements
• Calibration Curve Model Selection, Evaluation and Weighting
• Method Feasibility and Optimization
• Special Considerations for Multiplex Assays and Cross-Validation of Assays

Course Instructors:

John L. Allinson, FIBMS, Vice President, Biomarker Laboratory Services, ICON Development Solutions

Viswanath Devanarayan, Ph.D., Director, Exploratory Statistics, Abbott Laboratories

(SC2) Assay Development and Validation of Pharmacodynamic Biomarkers in Circulating Tumor Cells

This course will describe characterization and detection of CTCs and implementation of CTC biomarker assays, including instrumentation, data analysis and reporting, troubleshooting, and quality control. The issues of fit-for-purpose method development and validation for measurement of CTC biomarkers in animal studies and clinical patient samples in drug development will be addressed. The challenges in procedure standardization, quality control, assay reference, and assay transfer to multiple testing sites to support clinical trials will also be discussed.

• Application of CTC Biomarker Assays in Clinical Drug Development
• Detection Systems for CTC Biomarkers
• Key Elements in Method Development of CTC Biomarkers
• Method Feasibility and Optimization of CTC Biomarker Assays
• Fitness for Purpose: CTC Biomarker Assay Validation

Course Instructor:

Lihua Wang, M.D., Ph.D., Senior Scientist, Pharmacodynamic Assay Development and Implementation Section, SAIC-Frederick, National Cancer Institute

(SC3) Novel Cancer Biomarkers

Genomic biomarkers are rapidly being embraced as a highly accurate means of detecting cancer and predicting tumor response to treatment, thereby improving patient outcomes. Blood-based tests for diseases such as colorectal cancer and non-invasive strategies for detecting biomarkers in the skin are being developed to improve screening compliance in the general population. Advances in cancer biomarker detection will be discussed, including:

• Non-Invasive Strategies for Identifying Biomarkers in the Skin
• Biomarkers for Prediction of Tumor Sensitivity to Therapies Targeting Cell Surface Death Receptors
• Detection of Colorectal Cancer using the Septin9 DNA Methylation Biomarker
• Enabling Personalized Cancer Care

Course Instructors:

Shannon R. Payne, Ph.D., Medical Science Liaison, Medical Affairs, Epigenomics, Inc.

William Wachsman, M.D., Ph.D., Associate Professor, Medicine, Hematology-Oncology and Moores Cancer Center, University of California, San Diego School of Medicine; Staff Physician, VA San Diego Healthcare System

Baolin Zhang, Ph.D., Principal Investigator and Product Quality Reviewer, Division of Therapeutic Proteins, Office of Biotechnology Products, Center for Drug Evaluation and Research, U.S. Food and Drug Administration

Philip M. Arlen, M.D., President and CEO, Neogenix Oncology

(SC4) Adaptive Clinical Trials: Strategies and Tools

There is great potential for clinical trials designed with adaptive features to result in more efficient decision making within a drug development program. However, clinical trials with adaptive features are more complex to implement than traditional designs such as fixed-sample or group sequential.  In this course, we will share our experience in AD trial design and implementations as well as provide an overview of software available for various steps of adaptive clinical trials.

Part I. Goals of Phase 2 designs
• Role of adaptive randomization
- to treat patients better
- for statistical efficiency
- to focus/refocus on multiple questions simultaneously
• Role of adaptive sample size
• Basing adaptations on predictive probabilities of Phase 3 success
• Available software

Part II. Group Sequential Methods for Clinical Trial Design
• Introduction to Group Sequential Tests (GSTs)
• Benefits of early stopping offered by GSTs
• Error spending designs
• Application of GSTs to survival data
• Information monitoring
• Overview of software for implementing GSTs

Part III. Practical Considerations and Strategies for Adaptive Design Trial Implementations
• Randomization and clinical supply strategies
• IVRS vendor capability assessment and quality control
• Enrollment management and patient enrollment modeling and simulation,
• Data quality and interim analysis planning,
• Use of DMC.

Instructors (respectively):
Jason Connor, Ph.D., Statistical Scientist, Berry Consultants
Christopher Jennison, Professor of Statistics, University of Bath, UK
Weili He, Ph.D., Associate Director, Clinical Biostatistics, Merck Sharp & Dohme Corp.

September 6 - Dinner (6:00-9:00 PM)

(SC5) Biomarker Qualification and Validation

As biomarkers play an increasingly important role in drug development, preventive medicine and medical diagnostics, quality assurance and assay validation become essential procedures in efficient biomarker development. Methods for discovering and validating biomarkers will be discussed, as well as accelerating biomarker discovery through visual analytics, and developing molecular tools for defining the lethal phenotype of prostate cancer.

Course Instructors:

Jake Y. Chen, Ph.D., Assistant Professor of Informatics and Computer Science, Indiana University School of Informatics; Founding Director, Indiana Center for Systems Biology and Personalized Medicine

Robert H. Getzenberg, Ph.D., The Donald S. Coffey Professor and Director of Urology Research, The Brady Urological Institute, Professor of Oncology and Pharmacology and Molecular Sciences, The Johns Hopkins University School of Medicine

September 8 - Dinner (6:30-9:00 PM)

(SC6) Integration and Analysis of Heterogeneous Translational Data

Complex translational data sets span heterogeneous data sources such as longitudinal clinical records, sample annotation spreadsheets, and results from a variety of assays performed on disparate instrumentation platforms. To realize the true value of this data, we must integrate it into a rich analytical environment where we can apply platform-agnostic data analysis approaches. In this course we will:

• Introduce the core concepts of data warehousing
• Apply these concepts to the integration and analysis of translational data
• Illustrate the use of open source business intelligence tools in this domain
• Discuss deploying these tools on cloud servers
• Provide hands-on opportunities to use web based tools to explore and analyze translational data sets

Course Instructors:

Janet Siebert, President, CytoAnalytics

Wade T. Rogers, Ph.D., Director, Computational Biology and Research Informatics, University of Pennsylvania

(SC7) Planning Biomarker Development: Aligning Strategy, Science, and Technology

The development of biomarkers as decision tools in drug development is a relatively new process. While new technologies have greatly accelerated the rate of discovery of biomarkers, their efficient integration in the drug development process often presents a significant challenge. A pragmatic approach to planning, organizing, and prioritizing biomarker development will be discussed including:

• Biomarker development core principles and terminology
• The FDA Target Product Profile Guidance as a model for developing biomarker objectives
• Time and resource planning in function of biomarker classes
• Parallel processes of method validation and biomarker qualification

Course Instructor:

Thierry Sornasse, Ph.D., Consultant, Integrated Biomarker Strategies