2019 Archived Content

Cambridge Healthtech’s 10th Annual

Companion Diagnostics: Strategy and Partnerships

Winning Strategies for Drug-Diagnostics Co-Development

August 20-21, 2019

The co-development of drugs and diagnostics, i.e. companion diagnostics development, is delivering significant patient benefits, healthcare cost savings, and revenue opportunities. Historically, it carries out most technological advances both as IVD products and as laboratory developed tests with gene panels and whole exome sequencing playing a role of companion tests. The space of companion diagnostics is highly sensitive to the regulatory and reimbursement fluctuations, and it requires specific market access strategies. Cambridge Healthtech Institute’s Tenth Annual Companion Diagnostics: Strategy and Partnerships conference is designed to facilitate knowledge and opinion exchange between pharmaceutical and diagnostics executives, translational scientists, clinicians, business experts, regulators, international companion diagnostics leaders, and other parties involved in drug-diagnostics co-development and precision medicine. Case studies of successful collaboration between pharmaceutical and diagnostics companies’ researchers and executives will be presented.

Final Agenda

Recommended Short Course*

SC2: Discover How Machine Learning Can Complement Diagnoses through Medical Imagery

Helen Lam, Software Developer, Developer Advocate, IBM Watson

Nicholas Bourdakos, Software Developer, IBM Watson

In this hands-on session, learn how to train your own custom classifier using medical images to help augment your diagnoses. We will first cover the overview of available technologies for medical imagery in diagnosis, then will show examples by utilizing Watson Machine Learning and Tensorflow, an open source software library for high performance numerical computation to create a custom model.

Co-organized with

SC5: Tumor Mutation Burden: Unloading the Latest Challenges and Developments

Susan J. Hsiao, MD, PhD, Assistant Professor, Pathology and Cell Biology, Columbia University Medical Center

Larissa V. Furtado, MD, Medical Director, Molecular Oncology, ARUP Laboratories; Associate Professor of Pathology, University of Utah School of Medicine

Ahmet Zehir, PhD, Director, Clinical Bioinformatics, Memorial Sloan Kettering Cancer Center

In this short course, we will review the current state of the field for tumor mutational burden (TMB), a biomarker predictive of response to immunotherapy agents. This course will cover concepts including the clinical utility of measuring TMB, technical considerations and challenges in validating and measuring TMB in a clinical laboratory, issues surrounding clinical TMB interpretation, and future directions and applications of TMB testing.

*Separate registration required.

TUESDAY, AUGUST 20

7:30 am Registration and Morning Coffee

8:30 Chairperson’s Opening Remarks

Jonathan Beer, MBA, Director of Disruptive Technologies, Oncology Precision Medicine, Novartis

8:35 Companion Diagnostics in the Era of Consolidation and Globalization: Multiplexed Biomarkers Across Therapeutic Areas and Around the Globe

Omar Perez, PhD, RAC, Head of Precision Medicine and Diagnostics, R&D, GSK

Although biopharmaceutical pipeline products increasingly incorporate companion diagnostics into their clinical development efforts, commercial launch plans often do not adequately address the diagnostic component. Early planning and collaboration between biopharma and diagnostic companies can better support an optimal launch.

9:00 Companion and Complementary Diagnostics: Regulatory Challenges and Opportunities

Gallagher_Pamela Pamela Gallagher, PhD, Scientific Reviewer, FDA

Companion diagnostics are essential for the safe and effective use of therapeutic products and promise to deliver a clearer understanding of disease development at the individual patient level. The companion diagnostics industry is rapidly progressing along with rapid advances in technology and healthcare demands leading to unique regulatory complexities. The strategies for successful codevelopment (for companion and complementary diagnostic devices) will be reviewed and the FDA regulatory review process for some approved companion diagnostic devices will be discussed.

9:25 Liquid Biopsies Enabling Precision Medicine

Beer_Jonathan Jonathan Beer, MBA, Director, Disruptive Technologies, Oncology Precision Medicine, Novartis

Liquid biopsies are a minimally invasive source of biomarker data with clear benefits in monitoring response to therapy and early detection of disease progression. The US FDA has approved the detection of CTCs as a prognostic marker for several cancer types and has now approved two CDx assays to detect variants in ctDNA. Further technology advances are required in order to deliver on the promise of liquid biopsies utility in Precision Medicine.

9:50 NEW: How Clinical Practice is Shaping the Precision Medicine Ecosystem

Barrera_Lourdes Lourdes Barrera, PhD, Senior Director, Precision Medicine, Oncology Business Unit, Novartis

Pharma companies continue to build and invest in targeted therapy pipeline. New diagnostic platforms are being developed to identify the patient most likely to respond to a given treatment. There is an ever growing need to understand the precision medicine landscape thought the eyes of the practicing clinician. Geographical differences in the access to various testing modalities and reimbursement must be accounted for in clinical development programs and go-to-market strategies. This presentation will provide an overview of the considerations for the global development and life cycle management of patient diagnostic and monitoring tools.

10:15 Coffee Break in the Exhibit Hall with Poster Viewing

10:55 Chairperson’s Remarks

John Lubniewski, President, CEO, HTG Molecular Diagnostics

11:00 Going Global – Setting Up a European Subsidiary

Lubniewski_John John Lubniewski, President, CEO, HTG Molecular Diagnostics

The speaker will provide a high level and lighthearted walk through the thinking behind (and the process and actual work of) setting up your first European entity. In HTG’s case, we evaluated setting up in the UK, the Netherlands, Germany or France. We chose France. Buckle up.

11:30 From a Streamlined Companion Diagnostic to a Next Generation Sequencing Analysis in Acute Myeloid Leukemia: Opportunities, Challenges, and Global Considerations

Berisha_Flora Flora Berisha, Director, Companion Diagnostics, Translational Sciences, Global Oncology R&D, Daiichi-Sankyo, Inc.

Internal tandem duplications in fms-like tyrosine kinase 3 (FLT3-ITD) are common in Acute Myeloid Leukemia (AML) and confer a poor prognosis. The LeukoStrat^® CDx FLT3 Mutation Assay is a PCR-based in vitro diagnostic test designed to detect FLT3 internal tandem duplications (ITD) mutations and tyrosine kinase domain (TKD) FLT3 mutations. Although PCR-based assays are predominantly utilized for molecular detection of mutations/translocations in AML, the field is moving toward a more rapid, robust and sensitive method. Given the importance of Minimal Residual Disease (MRD) in hematologic malignancies, particularly in AML, a standardized and highly sensitive NGS assay is needed for the detection of in FLT3-ITD mutations to enable and guide clinical and therapy decisions during the patient journey.

12:00 pm Presentation to be Announced

12:30 Luncheon Presentation: Challenges and Strategies for Successful Global Companion Diagnostic Market Access

Hull_Stephen Stephen Hull, President, Founder, Hull Associates LLC

Companion diagnostics (CDx) have emerged as a distinct group of IVDs shaping the personalized health care spectrum. Global markets, each with distinct market access processes, evidence requirements and evaluation measures create challenges to market access, and are a key barrier to identifying patients, and subsequently accessing precision medicines for those who may benefit. The challenges of successful CDx programs will be reviewed, emphasizing strategies and activities that will facilitate optimal reimbursement and access.

1:00 Cookie & Refreshment Break in the Exhibit Hall with Poster Viewing

1:30 Chairperson’s Remarks

John Sninsky, PhD, Consultant, Translational Sciences

1:35 Evolving Trends in Respiratory Infections Diagnostics and the Impact in Drug Development

Villacian_Jorge Jorge Villacian, MD, Medical, Digital and Technical Solutions Leader, Janssen Pharmaceutica

Accurate and reliable diagnostics are important in assessing the etiology of acuter respiratory infections. This has great impact in drug development for viral respiratory diseases. New trends in diagnostic development include home testing. The interaction of these elements will be discussed in the presentation.

1:55 ctDNA Utility and Challenges

Barrett_Carl J. Carl Barrett, MD, Vice President, Oncology Translational Sciences, AstraZeneca

Circulating tumor DNA (ctDNA) is becoming increasingly used in clinical practice and clinical/translational research. Examples of this utility will be given for patient selection, monitoring disease response and elucidating mechanisms of resistance to targeted therapies. Despite the common use in many commercial and academic labs , issues remain with sensitivity and specificity of some assays and this will be discussed in this and other talks from our laboratories.

2:15 Design and Implementation of Rare and Orphan Disease Testing Programs

Mathews_Charles Charles Mathews, Principal, ClearView Healthcare Partners

Overview of the challenges and dynamics associated with identifying patients with rare or orphan disease post FDA approval of therapy. Review of examples of different testing programs and therapy/laboratory partnerships. Discussion of test program best practices. Thoughts on future of testing and implications for therapy development

2:45 Sponsored Presentation (Opportunity Available)

3:15 PANEL DISCUSSION: Precision Medicine Beyond Oncology

Moderator: John Sninsky, PhD, Consultant, Translational Sciences

Panelists: Speakers of the Session

Flora Berisha, Director, Companion Diagnostics, Translational Sciences, Global Oncology R&D, Daiichi-Sankyo, Inc.

Scott Patterson, PhD, Vice President, Biomarker Sciences, Gilead Sciences, Inc.

It’s been 20 years! Why so little success for a great idea (right drug, right patient, right time)? What are we doing wrong? Are health care providers outside of Oncology actually ready to implement Personalized Medicine? Where is the value for Co-Dx outside of oncology? Prediction of R? Prediction of NR? Prognosis? How good would a Co-Dx need to be to change care paradigms from requiring patients to fail “cheaper” drugs first? Is there a robust value proposition?

3:40 Refreshment Break in the Exhibit Hall with Poster Viewing

4:25 Chairperson’s Remarks

Carolyn Hiller, Program Director, Clinical Diagnostics Initiative, Medical Device Innovation Consortium (MDIC)

4:30 Utility of a Novel Precision Medicine Maturity Model

Gustavsen_Gary Gary Gustavsen, Partner, Precision Medicine, Health Advances

The Personalized Medicine Coalition, alongside several industry leaders, recently commissioned a study to assess the adoption of personalized medicine across health systems in the US. As part of this broad survey, the team developed a novel maturity model to objectively and iteratively detail the extent to which personalized medicine has been adopted. Health Advances will share this quantitative data output and suggest how it may be useful to shape future CDx tactics by pharma.

5:00 NEW: The SPOT/Dx Quality Assurance Pilot: Reference Samples for NGS Assays

Barbara Zehnbauer, PhD, FACMG, Adjunct Professor, Pathology, Emory University School of Medicine

5:20 PANEL DISCUSSION: Somatic Reference Samples for NGS Dx

Hiller_Carolyn Moderator: Carolyn Hiller, Program Director, Clinical Diagnostics Initiative, Medical Device Innovation Consortium (MDIC)

Panelists:

Barbara Zehnbauer, PhD, FACMG, Adjunct Professor, Pathology, Emory University School of Medicine

Timothy K. McDaniel, PhD, Senior Vice President of Emerging Opportunities, TGen

J.D. Alvarez, MD, PhD, Vice President, Oncology Diagnostics, Janssen Pharmaceutical Companies

Zivana Tezak, PhD, Associate Director for Science and Technology, Personalized Medicine Staff, Office of in vitro Diagnostic Device (IVD) Evaluation and Safety (OIR), Center for Devices and Radiological Health, FDA

Clinical oncology is being transformed by the use of next-generation sequencing (NGS) based diagnostics. NGS lays out a promise for personalized medicine in other therapeutic areas. However, lack of agreed upon, well-characterized and community-validated reference samples and data benchmarks creates a potential challenge for the efficient development of these critical tests and for understanding their results. The ultimate goal of this effort is to develop properly consented, widely shareable reference samples that can be made available to the public and scalably produced in order to enable efficient development and improve the accuracy, reliability and transparency of NGS-based oncology tests.

6:00 Wine & Cheese Pairing Welcome Reception in the Exhibit Hall with Poster Viewing

7:00 Close of Day

WEDNESDAY, AUGUST 21

7:15 am Registration

Independence B-E and Foyer
7:30 Problem Solving Breakout Discussions with Continental Breakfast

Disruptive Technologies for Companion Diagnostics: Challenges and Solutions

Moderator: Jonathan Beer, MBA, Director, Disruptive Technologies, Oncology Precision Medicine, Novartis

Why is TMB not yet a validated IO CDx biomarker?
Which technology class is most likely to become an approved CDx?
What factors are preventing approval of more liquid biopsy CDx assays?

Companion and Complementary Diagnostics in Immuno-Oncology: The FDA Perspective

Moderators: Pamela Gallagher, PhD, Scientific Reviewer, FDA

Anand Pathak, MD, PhD, MPH, Medical Officer, FDA

8:25 Chairperson’s Remarks

Daryl Pritchard, PhD, Senior Vice President, Science Policy, Personalized Medicine Coalition

8:30 Influence of Genomic Tests on Decision Pathways for Reimbursement Practices for Oncological Therapeutics

Jiwanmall_Eugean Eugean Jiwanmall, MPH, MBA, Senior Research Analyst, Technology Evaluation & Medical Policy, Claim Payment Policy Department, Facilitated Health Networks, Independence Blue Cross

Achievement of optimal clinical care must be driven by multiple objective factors. Understanding and establishing the confidence in these contributing factors are essential due to their impacts on any proposed net improvement in relevant health outcomes. Development, advancement, integration, and routine clinical usage of options in oncological therapeutics are closely tied to availability, reliability, and acceptance of certain aspects of genomics. We will explore the impact of different facets of genetic testing on multiple phases of oncological therapeutics, and payment considerations based on these relationships.

8:45 Humana’s Perspective on Personalized Medicine

Bordenave_Kristine Kristine Bordenave, MD, FACP, Corporate Medical Director, NMCARE, Healthcare Services, Humana

9:00 Global Commercial and Partnership Considerations for Companion Diagnostics

Ferrara_Joseph Joseph Ferrara, President & CEO, Boston Healthcare Inc.

Key commercialization considerations for drug and test innovators, including balancing test access and quality, and embedding CDx global commercial considerations in pharma and diagnostic company partnerships will be highlighted.

9:30 PANEL DISCUSSION: Funding Common Ground with Payers and Policy Makers to Advance Personalized Medicine in Oncology and Beyond

While emerging personalized medicine technologies and platforms have the potential to make drug development more economical and make health systems more efficient by targeting treatments to only those who will benefit, they are also challenging reimbursement systems accustomed to one-size-fits-all medicine. Indeed, personalized medicine has led payers to think differently about coverage and reimbursement of high-value diagnostics and novel cell-based and genetic therapies that have the potential to cure diseases with one or only a few treatments. The approval of tissue-agnostic drugs such as Keytruda and Vitrakvi, for example, will force payers to figure out policies for pan-cancer indications and associated genetic testing, while the approval of cellular-based CAR-T therapies and gene therapies such as Luxturna, Kymriah, and Yescarta are challenging the traditional way that high-cost therapeutics can be reimbursed.

Moderator:

Pritchard_Daryl Daryl Pritchard, PhD, Senior Vice President, Science Policy, Personalized Medicine Coalition

Panelists:

Bordenave_Kristine Kristine Bordenave, MD, FACP, Corporate Medical Director, NMCARE, Healthcare Services, Humana

Sen_Shuvayu Shuvayu S. Sen, PhD, Executive Director, CORE Oncology, Merck & Co., Inc.

Jiwanmall_Eugean Eugean Jiwanmall, MPH, MBA, Senior Research Analyst, Technology Evaluation & Medical Policy, Claim Payment Policy Department, Facilitated Health Networks, Independence Blue Cross

Stergiopoulos_Stella Stella Stergiopoulos, Associate Director, Health Economic Outcomes Research and Payer Policy, Foundation Medicine, Inc.

Szarama_Katherine Katherine B. Szarama, PhD, Presidential Management Fellow, CMS

Dumanois_Robert Robert Dumanois, Manager, Reimbursement Strategy, Thermo Fisher Scientific

Topics to be Discussed:

What are the key reimbursement challenges for advanced diagnostic tests?
What strategies are emerging for the coverage and reimbursement of cell-based and genetic therapies?
What evidence is necessary to demonstrate the value of personalized medicine technologies to payers and providers?

How can practice-based evidence be developed in an environment of limited reimbursement and reduced access?

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

Constitution A&B

11:30 Plenary Keynote Session

11:30 Chairperson’s Remarks

Charles Mathews, Principal, ClearView Healthcare Partners

11:40 NEW: Plenary Keynote Presentation: FDA Updates: Now and Looking to the Future

Katherine Donigan, PhD, Acting Director of Personalized Medicine, Office of In Vitro Diagnostics and Radiological Health, Center for Devices and Radiological Health, U.S. Food and Drug Administration

Introduction and background of the new Office Director of OIR and updates on precision medicine and other initiatives at the FDA.

12:10-1:05 pm Plenary Keynote Discussion: Proposals and Solutions for Diagnostic Reform Including Oversight of Laboratory Developed Tests (LDTs)

Co-Organized with

Moderator:
Cynthia A. Bens, Senior Vice President, Public Policy, Personalized Medicine Coalition

How are stakeholders influencing congressional activity on the Verifying Accurate Leading-edge IVCT Development (VALID) Act?
How will the VALID Act change the current oversight landscape for diagnostics, including LDTs?
How are policymakers addressing the role of CMS and CLIA in the VALID Act?
How will increased regulatory and oversight activities at the FDA affect the diagnostics industry?
What impact will changes in diagnostics regulation and oversight have on patient care?

Panelists:

Julie Khani, MPA, President, American Clinical Laboratory Association (ACLA)

Donald E. Horton, Jr., Senior Vice President, Global Government Relations & Public Policy, Laboratory Corporation of America Holdings

Susan Van Meter, Executive Director, AdvaMedDx

Tara Burke, PhD, Senior Director, Public Policy & Advocacy, Association for Molecular Pathology (AMP)

Lasiter_Laura Laura Lasiter, PhD, Science Policy Analyst, Friends of Cancer Research

1:05 Luncheon Presentation: CDx Development in the 21st Century: Leveraging the Clinical Genomics Community for Precision Medicine

Adrian_Willig Adrian Willig, PhD, Associate Head of Lab, SOPHiA GENETICS
With several CE-IVD solutions developed to support clinicians in Europe, SOPHiA GENETICS is well-positioned to support pharmaceutical and biotech companies develop the next generation of targeted therapies where a CDx application is required. SOPHiA GENETICS brings expertise in solution design and validation and can deploy CDx solutions throughout its community of over 970 healthcare institutions in 81 countries. Together, we will give many patients around the globe access to new targeted therapies.

1:35 End of Companion Diagnostics: Strategy & Partnerships