Short Courses*

Sunday, June 10, 5:00-8:30 pm

Dinner Short Course

SC1: Fit-for-Purpose Biomarker Assay Development and Validation

Instructors:

John L. Allinson, FIBMS, Vice President, Biomarker Services, Biologics Development Services, Tampa Bay

Viswanath Devanarayan, PhD, Global Head of Statistics & Data Sciences, Charles River Laboratories

This tutorial will provide recommendations on the “fit-for-purpose” best practices in the development and validation of biomarker assays for exploratory or advanced biomarker applications. Strategies for different applications at various phases of biomarker development will be described. Key elements in the method of development and validation will be illustrated with examples, including reference to standard material, sample stability and collection integrity, validation and QC samples, validity of reference standards, calibration curve fitting methods, method optimization and feasibility studies. Special challenges in protein biomarker assays will be discussed, including strategies for moving from biomarker panels in the exploratory phase to the few markers chosen to support clinical trials, cross-validation of biomarker assays, etc.

Outline:

1. Introduction: Nomenclature, types of biomarker methods/assays, method development and validation road-map, fundamental validity, similarity and differences from PK assays and diagnostic applications

2. Pre-analytical and bioanalytical elements: Target range, standards, validation and QC samples, stability, matrix effect, specificity and relative selectivity

3. Calibration curve model selection, evaluation and weighting

4. Method feasibility and optimization with precision profiles

5. Evaluation of some pre-study validation characteristics such as precision, bias, sensitivity and quantification limits

6. Use of sample controls for in-study performance monitoring and conformance testing among laboratories

7. Special considerations for multiplex assays, cross-validation of assays, etc.

8. Method comparisons

Tuesday, June 12, 1:15-5:00 pm

Executive ThinkTank

SC2: Digital Biomarker Implementation Strategies

Discussion Leader:

Daniel Karlin, MD, MS, Head, Clinical, Informatics & Regulatory Strategy, Digital Medicine and the Pfizer Innovation Research Lab

Panelists:

Michael J. Benecky, PhD, Senior Director, Global Regulatory Affairs, Precision and Digital Medicine, R&D Chief Regulatory Office, GlaxoSmithKline

Peter Bergethon, MD, Vice President, Quantitative Medicine and Clinical Technologies, Biogen

Yu-Feng Yvonne Chan, MD, PhD, Associate Professor, Genetics and Genomics Sciences & Emerging Medicine; Director, Center for Digital Health, Icahn Institute for Genomics and Multiscale Biology, Icahn School of Medicine at Mount Sinai

Christian Gossens, PhD, MBA, Global Head, Early Development Workflows, pRED Informatics, Roche Pharmaceutical Research and Early Development

Daniel Grant, PhD, Director, Early Development Lead, Novartis

Gabriel Vargas, MD, PhD, Executive Medical Director, Digital Health & Neuroscience Therapeutic Area Head, Early Development, Amgen

1:15-1:45 Introductions and Lunch Provided

 1:45 Roundtable: Choosing Digital Endpoints in Clinical Trials

• How are digital biomarkers different from traditional biomarkers?
• What clinical evidence is required for using digital biomarkers as clinical outcome measure in clinical trials?
• What is the value proposition for digital endpoints?
• How to progress digital endpoints beyond the exploratory phase
• How to deal with clinical meaningfulness and regulatory acceptability of digital biomarker data
• How to choose and validate a digital biomarker for a new application when there is no current biomarker for reference
• Where to prioritize investment for optimum clinical trial process
• What are the emerging applications for digital biomarkers?

3:00 Networking Refreshment Break

 3:45 Roundtable: Technology Advances in Biosensors, Wearables and mHealth for Clinical Applications

• How to validate digital biomarkers
• What would be sufficient qualification for regulatory acceptance?
• What evidence is needed for market access and payer acceptance?
• What are the challenges and opportunities for using wearables, biosensors and smartphones in clinical development?
• Can technology and analytics keep up with endpoint development?
• What data sharing and data standards are needed to enable progression?
• Is there a consortium validation model for digital biomarkers?
• Where will the technology and implementation stand in 5 years?

5:00 Close of ThinkTank

Tuesday, June 12, 6:30-9:00 pm

Dinner Short Course

SC3: Multiplexed Analysis of Tumor Tissues Using Spatially Resolved and Quantitative Platforms

Instructor:

Kurt A. Schalper, MD, PhD, Assistant Professor, Pathology and Medicine (Medical Oncology), Yale School of Medicine; Director, Translational Immuno-Oncology Laboratory, Yale Cancer Center

In this course we will discuss novel strategies to simultaneously analyze multiple biomolecules/analytes such as protein and mRNA targets in tumor tissues using diverse platforms including multispectral fluorescence, mass spectrometry imaging and barcoding-based signal detection systems. We will review the technical principles, practical aspects, determinants for optimal performance and possible uses in oncology research.

Outline:

1. Introduction: Current state of tissue biomarker analysis, major clinical/research uses. Value of spatial context and quantitative output

2. Platforms for multiplexed/quantitative measurement of tissue biomarkers

3. Principles and practical considerations of multiplexed tissue analysis

4. Sample preparation, assay validation strategies and reproducibility assessment

5. Use of multiplexed analysis for biomarker assessment

*Separate Registration Required