Tuesday, April 29  

5:00-6:00 pm Conference Pre-Registration

6:00-9:00 Dinner Course*

Exosomes and Microvesicles as Cancer Biomarkers
(*Separate registration required)

Wednesday, April 30

7:30-8:30 am Conference Registration and Morning Coffee

8:30-8:40 Welcome Remarks from Conference Director

Julia Boguslavsky, Executive Director, Conferences, Cambridge Healthtech Institute

Translation from Biomarker Assay to Companion Diagnostic 

8:40-8:45 Chairperson’s Opening Remarks

8:45-9:10 Transforming a Research Assay to a Companion Diagnostic

Ron Mazumder, Ph.D., MBA, Global Head, R&D and Operations, Janssen Diagnostics, Janssen Pharmaceutical Companies of Johnson & Johnson

Developing a predictive biomarker on a diagnostic platform often involves converting an assay which uses research-grade reagents and a research platform onto a diagnostic instrument which can be commercialized globally with GMP-manufactured and validated reagents. Furthermore, design verification under Quality Systems Regulations, clinical validation and clinical reproducibility must be completed during the drug development process. I will highlight considerations from case studies to explore each of these topics.

9:10-9:35 Tracking Value Creation across Diverse Biomarker Studies and Platforms in Pharmaceutical Development

MichaelBurczynskiMichael Burczynski, Ph.D., Executive Director, Biomarker Technologies, Discovery Medicine and Clinical Pharmacology, Bristol-Myers Squibb

An ever-growing list of biomarkers (and diagnostics assays) is frequently analyzed across many stages of drug development. One of the more vexing challenges facing pharmaceutical companies today is determining how to ensure that biomarkers are both judiciously implemented, yet also maximally utilized, to answer questions in translational research. For various reasons (cost, limited resources, shortened cycle times) it is no longer possible or acceptable to simply run multiple biomarker assays in clinical studies for exploratory purposes or to inform future therapeutic strategies. Biomarker assays (irrespective of platform) need to answer extremely well defined questions in drug development, and should employ associated metrics that can be objectively evaluated after study conclusion to determine whether the biomarker studies generated sufficient value to the program or therapeutic area in question. The present talk will focus on these principles and describe a general approach to 1) defining the potential value of biomarker assays prior to clinical study execution; 2) tracking biomarker assays and programs in clinical trials at a large pharma company via a specifically designed database strategy; and 3) objectively assessing the impact of biomarker assays following clinical study conclusion.

9:35-10:00 Successful Implementation of Global Biomarker Strategies Requires Laser Focus on Preanalytical Processes  

Marisa Dolled-Filhart, Ph.D., Associate Director, Pathology and Companion Diagnostics, Merck  

Generation of quality biomarker data begins with the development of an accurate and precise method. However, this is simply not sufficient. Studies show that the activities preceding sample analysis, the preanalytical activities, are as important, if not more important than method performance. Many reports cite significant assay variability being attributed to what happens prior to the sample arriving at the bioanalytical lab. Paying close attention to how the sample is collected, processed and shipped will ultimately determine your success.

10:00-10:30 Networking Coffee Break

10:30-10:55 Talk Title to be Announced

AndrewSchadeAndrew Schade, M.D., Ph.D., Senior Director, Diagnostics and Experimental Pathology, Tailored Therapeutics, Eli Lilly and Company

10:55-11:20 Companion Diagnostics: How Personal Does It Have to Get?

Daniel Edelman, Ph.D., Facility Head, Clinical Molecular Profiling Core, National Cancer Institute, NIH

Companion diagnostics are becoming an important and critical tool for clinicians in the treatment of patients suffering from diseases for which individualized target therapy is available. The FDA has approved 19 laboratory tests (as of December 2013) to aid in this improved treatment of patients. Yet, their impact on public health could depend on how personal they have to get. Also, laboratory-developed tests (LDTs) may provide a greater benefit in some circumstances. In this talk I will compare and contrast these and other relevant issues.

11:20-11:50 Sponsored Presentation

JermeyBridgeCookJeremy Bridge-Cook, Senior Vice President, Research and Development, Luminex Corporation

The development of biomarkers from discovery to clinical implementation as a Companion Diagnostics is a process which is inherently unpredictable; no two development pathways are alike. Given this unpredictability, it is desirable to develop biomarkers using a platform which provides flexibility, but which also reduces complexity as much as possible. The xMAP ® platform has been used extensively at all stages of biomarker development. The speaker will highlight some examples of discovery, validation and clinical implementation of biomarkers on xMAP ®.

CyVek11:50-12:05 pm CyPlex: A Transformational Immunoassay Technology

Rajiv Pande, Ph.D., Vice President, Scientific Affairs, CyVek Inc.

CyPlex Systems is a novel quantitative immunoassay platform that integrates a disposable microfluidic cartridge with a fully automated desktop analyzer. Multiple samples can be loaded onto a single CyPlex cartridge, and multiple analytes per sample can be quantified simultaneously, within an hour. CyPlex cartridges combine a unique solid phase approach (glass nanoreactors)with microfluidics toprovide high quality multi-analyte results without the typical multiplexing compromises. CyPlex assays showexcellent robustness and are extremely easy to perform.

Meso Scale12:05-12:50 Luncheon Presentation: Validated Multiplexed Cytokine Assays: A New Standard for Immunoassays

Pankaj Oberoi, Ph.D., Vice President, Commercial Assays, Meso Scale Discovery

Inconsistencies between biomarker studies can be attributed to variability between assay kit lots. Even CE‐marked kits may lack reproducibility because the CE mark is self‐regulated. With the use of well‐characterized, purified reagents and highly optimized assays, MSD’s V‐PLEX™ product portfolio demonstrates consistent and robust immunoassays. MSD has developed a 30‐plex human biomarker assay that is analytically validated and shows excellent lot‐to‐lot reproducibility and superior sensitivity, precision, and accuracy.   



Commercialization Strategies for Companion Diagnostics  


1:25-1:30 Chairperson’s Opening Remarks

Cecilia Schott, Pharm.D., MBA, Head, Personalized Healthcare, Corporate Business Development, AstraZeneca

1:30-1:55 Commercializing Companion Diagnostics: The Long Road Ahead

Jeffrey Emch, Director, Global Diagnostics Marketing, Immunotherapeutics,GlaxoSmithKline

Commercialization of a companion diagnostic is an oxymoron with a business model that does not easily meld with the pharmaceutical business model. In this case, many sales channels and established processes available to a pharmaceutical company are not there in the commercialization of a diagnostic. Hence, be prepared to pay for some or all of the efforts to promote access to the test. In this talk, we will discuss some of the key considerations in defining the commercialization path.

1:55-2:20 Talk Title to be Announced

CeciliaSchottCecilia Schott, Pharm.D., MBA, Head, Personalized Healthcare, Corporate Business Development, AstraZeneca

2:20-2:45 Companion Versus Complementary Diagnostics: Economic, Regulatory, and Strategic Considerations  

Christopher-Paul Milne, DVM, MPH, JD, Assistant Research Professor, Director, Research, Center for the Study of Drug Development, Tufts University Medical School

Companion diagnostics, as defined by FDA, are devices that provide information for the safe and effective use of a corresponding therapeutic product, typically linked to a specific drug within its approved labeling. Complementary diagnostics are tests that can improve disease management, early diagnosis, patient risk stratification, and drug monitoring related to associated therapeutics, but don’t require a regulatory link to a specific therapeutic at the time of development. Companion diagnostics can be considered a subset of complementary diagnostics, while complementary tests may mature into companion diagnostics. This presentation will discuss the pros and cons of the companion versus complementary approach from the perspective of both the diagnostic and therapeutic developer and with examples from several therapeutic areas.

2:45-3:45 Refreshment Break in the Exhibit Hall with Poster Viewing


Technology Showcase: Molecular Diagnostics   


3:45-4:15 Proteomics-Assisted Discovery of a Host Response Plasma Biomarker Panel to Accurately Diagnose Persistent Coughers with Active TB Disease

Rushdy Ahmad, Ph.D., Research Scientist, The Broad Institute of Harvard and MIT

A simple to use, robust and accurate blood-based rapid diagnostic test is needed for tuberculosis control and prevention. Each year approximately 9 million people are infected with TB. The lack of a rapid TB diagnostic test contributes to nearly 1.5 million deaths every year. TB is one of the top killers of women worldwide, resulting in half a million deaths annually. At the Broad Institute of MIT and Harvard, the Proteomics group has utilized high quality plasma samples from TB and control patients in conjunction with Myriad-RBM's Human Inflammation MAP to develop a specific and sensitive host response biomarker panel to diagnose adult persistent coughers with active TB disease. We will present results from this 5-year long prospective study and chart next steps to translate these promising results into a field deployable rapid TB diagnostic test. Such a test has the potential to be a game changer, saving many hundreds of thousands of lives every year.

4:15-4:45 Assay and Kit Lot Bridging Considerations for Single and Multiplex Biomarker Analysis in Support of Clinical Studies

Afshin Safavi, Ph.D., Founder and CSO, BioAgilytix Labs

Biomarker analysis has become a common practice by many pharmaceutical companies to help PK/PD modeling. The reliability of outcomes is heavily influenced by the quality of the reagents.  One of the challenges that bioanalytical labs face when running biomarker studies is the control of lot-to-lot variability of critical reagents and commercial immunoassay kits. Case studies will be presented to highlight the key bioanalytical considerations involved in running successful biomarker analyses in support of clinical studies.

4:45-5:00 Sponsored Presentation (Opportunity available) 

5:00-6:00 Welcome Reception in the Exhibit Hall with Poster Viewing

6:00-9:00 Dinner Courses*

  • Fit-for-Purpose Biomarker Assay Development and Validation
  • Non-Coding RNAs as Biomarkers and Diagnostics

(*Separate registration required)


Thursday, May 1  


7:30-8:15 am Breakfast Presentation (Sponsorship Opportunity Available) or Morning Coffee


Integrating Drug-Diagnostic Co-Development   


8:25-8:30 Chairperson’s Opening Remarks  

Kenneth Emancipator, M.D., Director, Companion Diagnostics, Merck Research Labs


8:30-8:55 Clinical Development of a Drug with a Companion Diagnostic: It’s Not Just about the Drug!

Kenneth Emancipator, M.D., Director, Companion Diagnostics, Merck Research Labs

When a companion diagnostic is co-developed with a therapeutic (drug or biologic), the clinic development program must support the registration requirements for both products. This presentation discusses the regulatory concepts, the key elements for planning successful clinical trials, various options for clinical trial design, and, most importantly, the common pitfalls encountered during the course of co-development programs.


8:55-9:20 Strategies for a Successful Integration of Diagnostics into Drug Development  

Felix W. Frueh, Ph.D., Executive Partner, Opus Three, LLC  

Drug-test co-development, or companion diagnostic development, requires a tight alignment of the diagnostic drug development processes. Inherently different timelines, milestones and expectations need to be coordinated and met. To the most part, a test is "expected to be ready" before used in a clinical setting — but what exactly does this mean? What are the clinically meaningful aspects and what are the regulatory requirements to integrate a test into the clinical decision making about the most appropriate use of a drug? Regardless of whether or not a diagnostic test is associated with the development of a drug, certain criteria must be met for the test to be used. The association of such test with the drug development can in fact pose several advantages for the test developer (at least during the development phase), but may also result in significant challenges, in particular in later stages once the test and the drug have been approved. The strategy for the successful development of a diagnostic therefore should not only include early considerations about the alignment of such test with drug development, but also how the test will be used in the market. The focus of this presentation is geared towards the earlier stages of drug-test co-development and how, within these early stages, questions about the market later on can be addressed simultaneously.

9:20-9:45 Regulatory Considerations for Companion Diagnostics

Eunice Lee, Ph.D., Regulatory Scientist, Office of In Vitro Diagnostics and Radiological Health, CDRH, FDA



9:45-10:45 Coffee Break in the Exhibit Hall with Poster Viewing


Regulatory and Reimbursement Strategies for Companion Diagnostics 

Chairperson's Opening Remarks

Tracy Bush, Ph.D., Director, Companion Diagnostics Regulatory Affairs, Roche Diagnostics

10:45-11:10 Policy Implications of Next-Generation Sequencing

Andrew Fish, Executive Director, AdvaMedDx

The Food and Drug Administration recently issued its first clearance of a next-generation sequencing platform to validate sequencing of any part of a patient's genome. In a concurrent statement, FDA noted that the platform would allow labs to develop tests for clinical use with greater confidence. This presentation will address ongoing questions about how FDA will regulate molecular and companion diagnostics, particularly with regard to next-generation and whole genome sequencing. The presentation also will address key issues related to diagnostics reimbursement challenges.

11:10-11:35 Global Regulation of Companion Diagnostic Products: Current Status and Future Trends

Tracy Bush, Ph.D., Director, Companion Diagnostics Regulatory Affairs, Roche Diagnostics

Globally, the regulatory frameworks for CDx and targeted drugs are still being established. This presentation will provide an introduction to current companion diagnostics regulatory definitions and basic registration pathway, both in the US and globally. Submission requirements and labeling expectations will be summarized and compared, and we will learn what changes to expect as the CDx regulatory paradigm continues to evolve.

11:35-12:00 pm Legal Developments Affecting Molecular Testing

Peter M. Kazon, General Counsel, American Clinical Laboratory Association

The regulation of molecular testing continues to be increasingly complex. The FDA has not explicitly regulated molecular testing performed as laboratory-developed tests, but it has taken a variety of actions that could have an impact in that area, including the issuance of its Guidance on Research Use Only/Investigational Use Only Products. Similarly, the Medicare Program has developed new processes for dealing with molecular testing, most importantly, the MolDx Program, which is overseen by Palmetto Government Services, one of its payment contractors. Finally, the payment for this testing is also in great flux, as CMS has just completed a year-long "gap filling" exercise that resulted in new pricing for most of this testing. This program will examine these various developments and attempt to predict what other initiatives lay ahead.

12:00-12:25 A Roadmap for Companion Diagnostics – Lessons Learned from Successful FDA Submissions

Steven Gutman, M.D., Strategic Advisor, Myraqa

Over the past ten years companion diagnostics have become viewed as a critical component in drug development. It is now common practice to use molecular targeting in early phases of drug study and selection and to consider clinical testing to reduce heterogeneity in treatment effect.  Although FDA guidance mandates review of companion diagnostics, to date few have received formal approval or clearance by FDA. Review information is publically available for these and provides a useful roadmap of secrets to success.

12:25-1:55 Enjoy Lunch on Your Own