Track 4 - Executive Summit:
Drug-Diagnostic Co-Development

Archived Content

Monday, May 21

1:00-2:00 Main Conference Registration

2:00-2:10 Welcoming Remarks from Conference Director

Julia Boguslavsky, Executive Director, Conferences, Cambridge Healthtech Institute

Biomarkers as Decision-Making Tools 

2:10-2:15 Chairperson's Opening Remarks

2:15-2:45 Turning an Active Compound into a Personalized Medicine: Do Biomarkers Help or Hinder?

Geert Kolvenbag, M.D., Ph.D., Global Product Vice President, AstraZeneca

The co-development of drug and biomarker has several inherent risks and challenges. In addition, the expectations and demands of the oncology community have increased over the last years. These challenges will be illustrated by a case study of a very active compound destined for a fast development program as a personalized medicine, but running into scientific, diagnostic and development challenges. This experience creates questions for the development of drugs and diagnostics today and in the future.

2:45-3:15 Evaluation of Biomarker Performance in the Real World: Comparative and Cost-Effectiveness Considerations

Felix W. Frueh, Ph.D., President, Medco Research Institute, Medco Health Solutions, Inc.

The difference between the potential to achieve an outcome and actually achieving it for a biomarker-guided intervention can be measured as the performance of a biomarker under controlled (optimized) conditions and its actual performance in the real-world.  Similar to post-approval safety and efficacy evaluations of drug therapies that differ from data found in pre-market assessments, the impact on clinical outcomes using biomarker-guided interventions are often not fully understood until these interventions are performed and analyzed in the heterogeneous environment of real-world clinical practice.  Following the two most critical factors, clinical outcomes and cost associated with reaching these outcomes, the Medco Research Institute has been conducting a series of real-world assessments of the performance of biomarkers to determine whether or not they should be integrated into everyday clinical practice.

3:15-3:45 Biomarkers: Finding the Balance between Driving Decision Making and Value or Distraction from Developing Drugs

Duncan McHale, Ph.D., Vice President, Global Exploratory Development, UCB Pharma

The past 5 years have seen an increasing focus on the identification and qualification of biomarkers. We now have tools which can generate proteomic or nucleotide data on hundreds of thousands of markers in single experiments and whole departments whose aim is to identify and qualify biomarkers. There are now 2 ICH guidances (E15 and E16) on genomic biomarkers and new regulatory processes developed specifically for qualifying biomarkers for use in regulatory decision making. This talk provides examples of where real value has been obtained using biomarkers as well as other areas where it has not. It will ask whether we have the balance right and how much should we invest in biomarkers and when.

3:45-4:15 Predicting Benefit to Therapy: Biomarkers and Molecular Profiles in Oncology Drug Development

Nicholas C. Dracopoli, Ph.D., Head, Oncology Biomarkers, Janssen R&D

The main goals of biomarker research in the pharmaceutical industry are to increase efficiency of the drug development process by improving understanding of the mechanism of action, deeper exploration of PK-PD interactions and predicting response to novel therapies in clinical development. However, predictive biomarkers are still only included in the labels of a minority of the oncology drugs approved in the U.S. since Herceptin in 1998. The development of novel biomarkers has enormous potential to impact drug development and improve patient outcomes. However, no complex molecular or protein profiles have been approved by the FDA to drive therapeutic use of any drug. This presentation will show how the small numbers of patients enrolled in clinical trials and the lack of long-term outcome data have limited the discovery of complex molecular profiles with strong predictive values (both negative and positive), and show how simple biomarkers measuring the status of the drug target or pathway (BRAF mutation, ALK-EML4 translocation, etc.) have been much more successful in the identification of highly predictive biomarkers which have been developed as companion diagnostics to drive therapeutic use of novel oncology drugs.

4:15-5:15 Welcome Reception in the Exhibit Hall with Poster Viewing

• • •

Tuesday, May 22

Sponsored by 

7:30-8:15 am Breakfast Presentation

Identification of Fluid Biomarkers of Treatment Response in Schizophrenia CSF and Plasma Samples

Eric Schaeffer, Ph.D., Director, Neuroscience Clinical Biomarkers, Bristol-Myers Squibb

Schizophrenia is a heterogeneous disease of complex and poorly understood etiology. Patients are often prescribed one of several approved medications based on their symptoms, but there is a high rate of switching among medications during a period of trial and error, while physicians seek to identify a drug(s) which will result in stabilization of symptoms. Given the variability in treatment response, there would be considerable value in the identification of a biomarker(s) which could provide an indication of whether a patient is responding to a particular medication early in the treatment regimen. This talk will discuss a biomarker identification and validation strategy focusing on highly multiplexed immunoassay panels offered by Myriad RBM.


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

8:30-9:00 Zelboraf: The Co-Development of Zelboraf and Its Companion Diagnostic

Walter H. Koch, Ph.D., Vice President and Head of Global Research, Roche Molecular Diagnostics

Cancers can be categorized based on their molecular etiology, including oncogenic driver mutations that are present. The development of targeted therapies alongside companion diagnostics that will identify patients most likely to receive benefit provides the opportunity to increase the success rate for oncology drugs and to decrease development time and associated costs. This presentation will detail the co-development of Zelboraf and the cobas 4800 BRAF V600 mutation test from the diagnostics perspective. This integrated process resulted in approval of the first personalized medicine for the treatment of metastatic melanoma within 6 years of the drug's discovery, a remarkably short time.

9:00-9:30 Crizotinib: The Xalkori, ALK CDx Partnership

Karen S. Long, Ph.D., Vice President, Medical, Regulatory and Clinical Affairs, Abbott Molecular

Pharmaceutical companies are increasingly engaging diagnostic companies to develop companion diagnostics to help select patients for their therapeutics. There are a number of factors that guide the pharmaceutical company's choice of diagnostic partner including IP considerations, diagnostic platform requirements and commercialization capabilities. However, in view of the relative size and importance of the U.S. market, the ability of the diagnostic company to navigate through the sometimes ambiguous regulatory process in the U.S. becomes a critical consideration, as this is vital to achieve product launch timing objectives and overall success of the therapeutic. There have been some recent examples of rapid co-development and approval of therapeutic/diagnostic product combinations in the U.S. including the recent Xalkori/ALK CDx approval. This session explores the factors that were most important in the successful co-development effort, approval and subsequent commercialization of the diagnostic.

9:30-10:30 Coffee Break in the Exhibit Hall with Poster Viewing


10:30-11:00 Personalized Medicine and Companion Diagnostics at Novartis

Michael C. Little, Ph.D., Global Head, Diagnostics Development, Novartis Molecular Diagnostics

Personalized medicine is here and now, after many years of promise and anticipation. Although early in the evolution of the impending revolution, the changes will bring positive impacts to healthcare. Nonetheless, the path to this future state is not a straightforward one, given the multiple elements such as development costs, clinical impacts, test performance, and regulatory constraints that must be aligned. Dr. Little will discuss how Novartis is approaching this space to ensure it provides the best solution for our patients, physicians, and payors.

11:00-11:30 Overcoming Challenges in Developing Companion Diagnostics

Ron Mazumder, Ph.D., M.B.A., Product Development Leader, Companion Diagnostics Center of Excellence, Johnson & Johnson

The development of companion diagnostics alongside targeted therapies presents a number of scientific, regulatory, organizational, and commercial challenges. This talk will highlight how the Companion Diagnostics Center of Excellence has addressed these issues in the context of Johnson & Johnson's drug development programs. Lessons learned from case studies will also be presented.

11:30-12:00 Drug and Diagnostic Co-Development: Under One Roof

Monica Reinholz, Ph.D., Senior Manager, Biomarker Strategy; Director, Clinical Studies, Translational Diagnostics, Ventana Medical Systems

12:00-12:30 Illuminating the Path for Co-Developing Drugs and Diagnostics: A Bayer Case Study

Carol Peña, Ph.D., Associate Director, Oncology Biomarkers, Bayer

This talk will present considerations impacting drug-diagnostic co-development, along with a Bayer case study. Dr. Peña will discuss the relevant regulatory guidance and precedents available. She will also address factors that need to be considered in designing a biomarker/CDx-based clinical development program, such as the prognostic value of the biomarker, and the challenge of defining biomarker "positive" vs. biomarker "negative." Lastly, Dr. Peña will present a Bayer case study in oncology, including pre-clinical/non-clinical data supporting a CDx-based project, and the design of trials supporting clinical validation of both drug and companion diagnostic.

Drug-Diagnostic Co-Development

12:30-2:00 Sponsored Presentations (Opportunities Available)

Contact Ilana Quigley at or 781-972-5457

Strategies for Companion Diagnostic Development 

2:00-2:30 Lessons Learned in the Development of Companion Diagnostics

Theo McCormick, Director, RxDx Services, Management Science Associates, Inc.

Navigating the scientific, medical, regulatory, contractual and political interactions and dynamics between healthcare professionals, the clinical laboratory, drug manufacturers, IVD diagnostic kit manufacturers and health plans are essential for optimal uptake of drug-diagnostic companion products. Those early decisions in the development cycle have complex downstream impact. This session will map out the trouble spots and potential effects of those decisions.

2:30-3:00 Strategies for Companion Diagnostic Development and Commercialization: Perspectives from a Global IVD Company

John F. Beeler, Ph.D., Director, Theranostics and Business Development, bioMerieux

The drug development process is witnessing a paradigm shift in which new therapies need to be tailored to well-defined patient subgroups via a companion diagnostic assay. With several theranostics partnerships in place at bioMerieux, the speaker will address the challenges of co-development programs and discuss strategies to achieve successful commercialization of these IVD products.

3:00-3:30 The Drug/Diagnostic Development Continuum:  Does the Ideal Co-Development Scenario Really Exist?

Rosanne Welcher, Ph.D., M.B.A., Vice President, pharmDx Research and Development, Dako North America

The ideal scenario for co-development of a diagnostic and targeted therapeutic is for both parties to engage at an early stage of drug development. The accepted framework for the development cycle is to align IUO assays to phases of the drug clinical program. Despite best efforts, there are numerous examples of diagnostic assays utilized in pivotal clinical trials that have not yet been fully validated or validated on the target indication.  What are the strategies employed to "cut in" or post-validate a diagnostic? This presentation will focus on various scenarios that deviate from the ideal, focusing on the risks and benefits to pharma and the diagnostic partner.

3:30-4:00 Combined CLIA and IVD Strategy Accelerates Timing and Mitigates Risk for Companion Diagnostics

Andrew Grupe, Ph.D., Senior Director, Pharmacogenomics, Celera/Quest Diagnostics

Personalized medicines are heralded as the future of drug development. Bringing a targeted medicine to market requires the concerted efforts of a drug developer and a diagnostics partner. There is no one-size-fits-all approach for these partnerships, and often a tailored relationship is forged between the two partners either before or during a pivotal Phase III trial. Late stage partnerships increase the drug candidate's risk profile when it is deemed to need a predictive biomarker for a pivotal Phase III trial. This presentation will provide specific examples that illustrate the benefits of working with a diagnostics organization with both experienced IVD manufacturing and an extensive CLIA laboratory infrastructure. The tangible benefits that mitigate the drug development risk and may be attractive to both partners if the relationship starts before Phase II will be described.

4:00-5:00 Refreshment Break in the Exhibit Hall with Poster Viewing


5:00-5:30 Zelboraf: Lessons Learned and Future Regulatory Implications for Drug/Diagnostic Contemporaneous Development

Linda Burdette, Ph.D., Director, Drug Regulatory Affairs, F. Hoffmann-La Roche

The recent approval of Roche/Genentech's BRAF-targeted therapy, Zelboraf™, with its companion diagnostic exemplifies the regulatory contemporaneous co-development process encouraged in FDA's 2011 In Vitro Companion Diagnostic Devices draft guidance. As a case study, "Zelboraf Lessons Learned" also highlights next steps for CDER/CDRH guidance development, including the regulatory pathway when the companion diagnostic is identified late in clinical development, when clinical utility data in attribute-negative population is needed, what level of evidence is required for approval of a new indication for an approved diagnostic, and conditions whereby a diagnostic may be granted approval for a class of drugs.

5:30-6:00 Bridging the Gap: Co-Development of Targeted Therapeutics and Companion Diagnostics in the U.S. and EU

Sabah Malek, Senior Regulatory Scientist, IVD/Medical Devices, Voisin Consulting Life Sciences

This presentation will focus on the major regulatory issues that concern diagnostic and pharmaceutical partners during co-development of a targeted therapeutic and companion diagnostic, including current development trends related to companion diagnostics, coordination of clinical trials, and the regulatory review of both products in the U.S. and EU. After months of discussions on personalized medicine and the use of companion diagnostics with targeted therapeutics, the much-anticipated draft FDA guidance document on in vitro companion diagnostics was finally released by the agency for comments. In contrast, the EU has yet to release any specific guidance on companion diagnostic development and/or finalize changes in the regulatory framework. We will discuss both the impact of the proposed draft guidance issued by the FDA and the expected major changes surrounding companion diagnostic regulation to occur in the EU.

• • •

Wednesday, May 23

7:30-8:15 am Breakfast Presentation (Opportunity Available)

Contact Ilana Quigley at or 781-972-5457


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

8:30-9:00 Partnering Strategies for Companion Diagnostic Development and Commercialization

Cynthia Gawron-Burke, Ph.D., Director, Scientific Liaison, External Scientific Affairs—Oncology Licensing, Merck Research Laboratories, Merck, Sharpe, & Dohme Corp.

Successful partnerships between biopharmaceutical and diagnostic companies are a critical component of realizing the future promise of personalized medicine. Partnering considerations such as collaboration models, challenges in negotiating collaboration agreements, and best practices to ensure successful partnerships will be discussed.

9:00-9:30 Pharma's View on Strategic Partnering for Companion Diagnostics

Cecilia Schott, Ph.D., M.B.A., Business Development Director, Personalized Healthcare, AstraZeneca

9:30-10:00 Challenges of Introducing Pharmacodiagnostics to Drug Development Teams

George A. Green, IV, Ph.D., Director, Pharmacodiagnostics, Bristol-Myers Squibb

Therapeutic development strategies that include companion diagnostic products have become common in today's pharmaceutical programs.  Incorporating diagnostics into the strategy for delivering a drug program has influence on the development, clinical, regulatory, and commercial strategies, and has overall implications for the program plan and schedule.  In addition, many companion diagnostic programs are partnered with external diagnostic development companies, further complicating the strategy.  Many drug development organizations do not have extensive experience with the diagnostic product development process, regulatory guidelines, and business models.  In this complex and highly matrixed environment, co-development of the companion diagnostic and therapeutic strategies depends on proper education, decision-making, and alliance management to ensure timely and effective delivery of the programs.

10:00-11:00 Coffee Break in the Exhibit Hall with Poster Viewing

11:00-12:00 Panel Discussion

12:00-1:30 Lunch on Your Own

Pharmacogenomic Biomarkers to Predict Benefit from Therapy 

1:30-2:00 Discovery of Pharmacogenomic Biomarkers in Cardiovascular and CNS Disorders through Expression Genomics

Wolfgang Sadee, Dr.rer.nat., Professor and Chair, Pharmacology; Director, Program in Pharmacogenomics, The Ohio State University

Genetic biomarkers have potential clinical utility for predicting disease risk and outcomes, including response to therapy. Pharmacogenomic biomarkers are rapidly gaining acceptance in drug discovery, development and therapy; however, a substantial portion of the responsible genetic factors is still uncertain ("missing heritability").  Available evidence suggests that genetic variants affecting expression regulation and RNA processing/translation may account for a large portion of genetically-determined phenotypic variability. We have developed comprehensive assays to detect regulatory variants, recently including the use of second-generation sequencing. Results have revealed the existence of frequent genetic variants with strong influence on clinical phenotypes, including drug response, even in important pharmaco-genes that had been under intense study for some time (e.g., CYP3A4, DRD2, DAT, HTR2A, CETP, ACE, NAT1). Several of these variants have promise as biomarkers for drug therapy, possibly as companion diagnostics in specific cases.

2:00-2:30 Improving Efficacy and Reliability for Personalized Medicine: The Role of Companion Diagnostics in Drug Development

Amelia Warner, Ph.D., Director, Clinical Pharmacogenomics, Merck

Identification of subpopulations of patients who either have subclinical response or toxicity to a drug in clinical development is a challenge.  Historically, identification of patients who have less than optimal response to drugs has occurred late in large global clinical trial review.  However, the pharmaceutical industry is working to optimize preclinical models, known genetic variation across populations, and earlier inclusion of surrogate biomarkers in early development programs to allow for early identification of populations that will benefit from alternate dosing or alternate therapies.  Adapting development strategies to include companion diagnostics remains a challenge, but pharmaceutical companies are developing clear working models to enable development of personalized therapies.

2:30-3:00 Applying Pharmacogenomics Effectively in Psychiatry and How to Overcome the Hurdles

Nadine Cohen, Ph.D., Senior Director, Head of Pharmacogenomics and Biomarker Execution Leader, Neuroscience Biomarkers, Janssen Pharmaceuticals Companies of Johnson & Johnson

An overview of pharmacogenomics applications at Janssen Neurosciences in Psychiatry will be discussed. Learn about using genomics to redefine mental disorders, managing complexity in psychiatric diseases with heterogeneity in disease biology and treatment response, and identifying genetic markers of clinical utility associated with treatment response for psychiatric drugs.

3:00 Close of Conference



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