Short Courses*

MONDAY AFTERNOON, MAY 1 | 1:00 – 4:00 pm

Short Course

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


John L. Allinson, FIBMS, Head, Biomarker Strategy, Drug Development Services, LGC Group

Viswanath Devanarayan, Ph.D., Global Head & Senior Research Fellow, Exploratory Statistics & Bioinformatics, AbbVie, Inc.

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

MONDAY EVENING, MAY 1 | 5:00 – 8:00 pm

Dinner Workshop

SC2: Liquid Biopsy for Immuno-Oncology and Precision Medicine

Characterizing the Cancer Genome from the Circulation

Rebecca Leary, Ph.D., Senior Investigator, Next Generation Diagnostics, Oncology Research, Novartis Institutes for BioMedical Research

Circulating tumor DNA (ctDNA) provides an opportunity for non-invasive assessment of tumor genotype and may enable rational use of targeted and/or immune modulating therapies at multiple clinical milestones. Implementation of ctDNA-based assays across clinical and research settings highlights important assay characteristics and suggests future clinical applications.

The Prognostic Potential of Tumor-Derived Exosomes Isolated from Plasma of Patients with Cancer

Theresa L. Whiteside, Ph.D., Professor, Pathology, Immunology and Otolaryngology, University of Pittsburgh Cancer Institute

Plasma of cancer patients is enriched in exosomes, and a high or increasing total exosome content is associated with disease progression. Only some of these exosomes originate from tumor cells. Isolation by immunoaffinity-based capture and analysis of the cargo of tumor-derived exosomes allow for evaluations of their potential as biomarkers of prognosis, outcome or responses to therapy. In combination with capture and analysis of T-cell derived exosomes from the same plasma, it is possible to obtain information about the patient's immune dysfunction and to correlate immune and tumor biomarkers with clinical endpoints. Thus, plasma exosomes can be useful as biomarkers of tumor progression and immune response to the tumor.

Tumor-Specific and PD-L1 Subtype CTC Capture/Detection in Relevance of Clinical Utility

Shulin Li, Ph.D., WT & Louise Jarrett Moran Distinguished Chair & Professor, Pediatrics – Research, The University of Texas MD Anderson Cancer Center

CTCs or subtype of CTCs are associated with either overall or tumor progression-free survival. At least two independent CTC capture technologies have been approved by FDA for CTC capture and enumeration, but neither of these technologies are tumor-specific yet. In this presentation, the presenter will focus on the discussion of universal and tumor-specific CTC capture technology, and on the PD-L1 subtype of CTCs in clinical prognosis.


Dinner Short Course

SC3: Preparing for Companion Diagnostic Device Studies and Submissions to FDA


Kate Simon, Ph.D., Senior Consultant, Biologics Consulting

This course will provide an overview of how FDA regulates companion diagnostics devices from the perspective of a former FDA reviewer. Best practices for companion diagnostic device submissions will be covered, as well as avoiding common mistakes. This course will help audience members prepare for clinical validation studies and subsequent data submissions to FDA for companion diagnostic devices.
Topics covered will include:
1. The regulatory differences between diagnostics for in vitro Diagnostic use (IVD), Research Use Only (RUO), Investigational Use Only (IUO) and Laboratory Developed Tests (LDTs)
2. Regulatory requirements for companion diagnostic device development
3. Overview of analytical and clinical validation requirements for companion diagnostic devices
4. When and how to submit a Significant Risk Determination (SRD) and Investigational Device Exemption (IDE) to FDA for a clinical validation study of a companion diagnostic

TUESDAY EVENING, MAY 2 | 6:00 – 9:00 pm

Dinner Short Course

SC4: Next-Generation Sequencing as a Clinical Test


Seth Crosby, M.D., Director, Partnerships & Alliances, Washington University School of Medicine

Avni Santani, Ph.D., Director, Division of Genomic Diagnostics, Children’s Hospital of Philadelphia; Assistant Professor, Clinical Pathology, Perelman School of Medicine, University of Pennsylvania

Next-generation sequencing (NGS) is used widely in clinical research for the discovery of disease-associated genes, and the clinical community is beginning to embrace this technology for diagnostic testing. The rapid evolution of NGS technologies presents significant opportunities and challenges for researchers and clinicians for improving health outcomes, particularly with respect to an increased emphasis on personalized and preventive medicine. Adoption of NGS in the clinical laboratory setting requires the adoption of many processes and procedures, such as the analytic and clinical validation of the test, CLIA certification/CAP accreditation, standards for reference materials, availability for proficiency testing, and questions regarding reimbursement and informed consent. The success of NGS as a viable diagnostic modality depends on many branches of the health care community working together. This session will be informative and practical for the researcher and laboratorians who are considering launching NGS as a clinical test.


Dinner ThinkTank

SC5: PD-L1 Assays for Biomarkers and Companion Diagnostics

Developing an Immunohistochemistry Test for “Programmed Cell Death 1 Ligand” (PD-L1) as a Companion Diagnostic for Pembrolizumab

Kenneth Emancipator, M.D., Executive Medical Director and Head of Companion Diagnostics, Merck & Co.

The rapid clinical development of pembrolizumab required even more rapid development of a PD-L1 immunohistochemistry assay. The assay was developed initially to assess PD-L1 as a predictive biomarker, then to enrich clinical trials. Merck subsequently partnered with Dako/Agilent to develop the first FDA-approved companion diagnostic in cancer immunotherapy, which subsequently enabled pembrolizumab to become the first immunotherapy approved for first-line treatment of non-small cell lung cancer. 

Regulatory Update on PD-L1 Assays

Janaki Veeraraghavan, Ph.D., Biologist, Office of In Vitro Diagnostics & Radiological Health, CDRH, FDA

Identification and development of biomarkers, to predict response to checkpoint inhibitors/immune therapeutic agents and accurately identify patients that derive the most therapeutic benefit, presents significant challenges. The talk will focus on the regulatory considerations during the development of biomarkers as predictive or prognostic markers and detail the various regulatory pathways to bring a biomarker test for clearance or approval by the FDA.

PD-L1 as a Biomarker: Opportunities and Challenges

Kurt A. Schalper, M.D., Ph.D., Assistant Professor, Pathology and Medicine (Medical Oncology), Yale School of Medicine

Detection of PD-L1 protein in tumor samples using immunohistochemistry (IHC) is associated with increased benefit from PD-1 axis blockers. Different PD-L1 IHC assays have been approved by the FDA for clinical use, but their performance is limited. Here we will address key technical and biological factors affecting the PD-L1 IHC test and discuss possibilities to improve the biomarker performance using novel quantitative tools and alternative approaches.

Panel Discussion


Kenneth Emancipator, M.D., Executive Medical Director and Head of Companion Diagnostics, Merck & Co.


Jean-Marie Bruey, Ph.D., Companion Diagnostics Group Leader, Genentech

Morganna Freeman, D.O., Medical Oncologist, Immunotherapeutics, The Angeles Clinic and Research Institute

Arnold B. Gelb, M.D., MS, FASCP, FCAP, Senior Director, Global Clinical Biomarkers and Companion Diagnostics, EMD Serono

Kurt A. Schalper, M.D., Ph.D., Assistant Professor, Pathology and Medicine (Medical Oncology), Yale School of Medicine

Janaki Veeraraghavan, Ph.D., Biologist, Office of In Vitro Diagnostics & Radiological Health, CDRH, FDA

WEDNESDAY EVENING, MAY 3 | 6:15 – 9:15 pm

Dinner Executive ThinkTank

SC6: Complementary Diagnostics

Opportunities and Challenges in Developing and Commercializing Complementary Diagnostics

Peter Hoehn, JD, Global Business Leader, Janssen Diagnostics

Complementary diagnostics have recently been recognized by the FDA as its own diagnostic category. Distinct from traditional companion diagnostics, complementary diagnostics are broader in application and with significant opportunity to shape precision medicine. However, complementary diagnostics come with their own set of regulatory and commercial challenges. This presentation will provide an overview of the differences between companion and complementary diagnostics and will address the unique challenges of complementary diagnostics and offer potential solutions that both diagnostic companies and pharmaceutical companies should consider.

Diagnostics: What's in a Label?

Marielena Mata, Ph.D., Program Director, Precision Medicine & Companion Diagnostics, GlaxoSmithKline

Supporting Therapeutic Outcomes: Complementary Diagnostics in Immuno-Oncology

George A. Green IV, Ph.D., Group Director, Pharmacodiagnostic Center of Excellence, Bristol-Myers Squibb

Unlike drugs targeting metabolic pathways that can be assessed using mutational analysis, many novel drugs currently in development target complex systems that cannot be clearly bifurcated using a single biomarker. In immuno-oncology, biomarkers or panels may provide distinct, additive, or overlapping information about drug efficacy. Frequently, these results may be informative to patient outcomes, but not definitive enough to require their use in patient treatment decisions. These situations have led to the development of complementary diagnostics, which are strongly tied to clinical evidence for drug efficacy, but are not a part of the treatment indication. This discussion will review the case for complementary diagnostics, provide working examples, and make comparisons between the development and execution of companion diagnostic and of complementary diagnostic programs.

Companion vs. Complementary from Clinical and Regulatory Perspectives

Abdel B. Halim, Pharm.D., Ph.D., DABCC-CC, DABCC-MD, DABCC-Tox, Vice President, Translational Medicine, Biomarkers & Diagnostics, Celldex Therapeutics

The relatively recent recognition of a couple of initially-thought companion diagnostic tests as complementary tests by FDA for PD-L1 raised a lot of discussions about CDx from different perspectives. In this presentation, differences between companion and complementary Dx from strategic, clinical, operational, regulatory and possible reimbursement aspects will be highlighted. Also, the presentation will touch on possible biological and technical aspects which may impact the whole precision medicine initiative.

Complementary vs. Companion Diagnostics: Two Sides of the Same Coin?

Victoria H. Brophy, Ph.D., Director, Genomics & Oncology Research, Roche Molecular Systems, Inc.

Complementary diagnostics are optional aids in therapy decision making while companion diagnostics are required for safe and effective drug use. While complementary diagnostics themselves are not a new idea, co-development of the complementary diagnostic with the drug is a more recent strategy. From an IVD perspective, is validation of a complementary diagnostic different from that of a companion diagnostic?


Dinner Short Course

SC7: Immune Monitoring in Cancer


Sacha Gnjatic, Ph.D., Associate Professor, Tisch Cancer Institute, Hematology/Oncology, Immunology, Icahn School of Medicine at Mount Sinai

Immunomonitoring aims to define qualitatively and quantitatively immune responses in patients, to establish correlates with clinical course of disease, and to help identify prognostic, predictive, or pharmacodynamic biomarkers as well as potential novel therapeutic targets. The course will first define the scope of assays covered by immune monitoring, which encompass various disciplines from pathology to immunology via proteomics and genomics. A focus on the types of assays depending on material available will be discussed, from either the cancer tissue site or from peripheral blood. Defining specificity of immune responses against cancer will also be addressed, as it is critical to properly address mechanisms of antitumor responses, either spontaneously occurring or as a result of treatment. Finally, examples of these approaches will be shown when applied to immunotherapeutic strategies, from cancer vaccines to checkpoint blockade.

Topics covered will include:
1. What is immune monitoring in cancer?
2. What assays can be used to measure tumor immunity?
3. Tissue vs. periphery, advantages/disadvantages
4. Antigen specificity, the key to relevance for adaptive immunity
5. Defining immunocompetence in cancer
6. Measuring immune changes during cancer immunotherapy

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