TUESDAY JUNE 26th, 2018
7:00 – 8:00am
Registration & Breakfast Buffet
8:00 – 8:05am
Welcome & Opening Remarks
Debbie Hart, MS, CAE, APR, President and CEO, BioNJ
8:05 – 8:55am
Opening Keynote: Precision Medicine – The Future of Cancer Care
Maurie Markman, MD – President of Medicine & Science, Cancer Treatment Centers of America
The following topics will be discussed:
Precision Medicine is not a new concept in the fight against cancer
Examples from the clinical research area of gynecologic malignancies emphasizing the critical need for a new research paradigm (including FDA review) that objectively considers clinically-relevant uncommon/rare “driver” molecular abnormalities
There have been several reported cancer trials/studies that have demonstrated the clinical relevance of precision cancer medicine- these trials/studies will be discussed
Possible approaches to objectively evaluate the utility of precision cancer medicine strategies, beyond the increasingly unrealistic traditional “gold standard” randomized trial.
8:55 – 9:05am
Precision Medicine – A Bridge to a World Without Diseases
Werner Verbiest, Global Head, Janssen Diagnostics, Johnson & Johnson
9:05 – 9:20am
Stopping the Wave Starts with you! Reflections on Lessons from Dementia Research
Luc Truyen M.D. Ph.D., Global Head Development and External Affairs, Neuroscience Therapeutic Area, Janssen Research and Development LLC
9:20 – 9:35am
Precision Medicine Today and Tomorrow: Science, Business and Policy. Patient’s Perspective
Andrea Stern Ferris, President and CEO, LUNGevity Foundation
9:35 – 10:05am
Fireside Chat: A World Without Disease
What can we learn from past approaches in drug design that will provide insights for developing the next generation of targeted therapeutics? Can Precision Medicine offer real hope in treating and managing Alzheimers and Lung Cancer? Werner Verbiest, Luc Truyen and Andrea Ferris discuss the social, economic and scientific challenges of tackling two complex and intractable diseases
Coffee & Networking
10:40 – 11:30am
Curing the Precision Deficit Disorder in Precision Medicine: A Unified,
Regulatory Network-Based Model for Cancer Drug Discovery
Moderator: Mariano Alvarez, Chief Scientific Officer, DarwinHealth, Inc
Panelists: Andrew Kung, MD, Ph.D., Chair, Department of Pediatrics – MSKCC, Kevin Kalinsky, MD, MS – Assistant Professor of Medicine, New York Presbyterian/Columbia University Medical Center.
Unfortunately, the gene-centric approach to precision-based cancer treatment which, for the most part, has relied on using a single drug to attack an individual mutation, has proved effective for only a relatively small percent of patients. This is because most patients lack actionable mutations and, even among those who do have such molecular alterations, most either fail to respond to the targeted therapy or rapidly develop drug resistance. Some call this the “precision deficit disorder” in precision medicine.
To find their way out of this impasse, many clinicians, scientists and researchers now believe that by complementing its initial focus on mutated genes, precision oncology must now also address the activity of otherwise normal proteins and molecular pathways that are ultimately responsible for cancer cells’ malignant behavior. The catch is that while genes can be readily sequenced to assess the presence of mutations, a complete tally of the wayward activity of potentially relevant proteins is much harder, thus thwarting previous attempts to identify the precise set of proteins that controls the cancer cell’s “engine room.”
In this panel we will discuss the progress that has been made using algorithms that can identify these critical proteins—dubbed “master regulators”—that hold the key to the malignant behavior of cancer cells. The algorithms use mRNA levels—easily measured with conventional methods—of the proteins’ numerous targets as an indirect but highly accurate indicator of protein activity. Importantly, unlike any other available approach, these algorithms uncover the hidden networks of proteins that work together to control abnormal cell activity in an individual patient’s tumor. These master regulators represent the ultimate on-off switches in the cancer cell’s regulatory ‘malware,’ providing a new class of targets for anticancer therapy, a potentially revolutionary advance in how we put more precision into precision medicine.
11:35 – 11:55am
Jeffrey R. Idle, PhD, Endowed Professor and Director, Arthur G. Zupko’s Systems Pharmacology and Pharmacogenomics, Arnold and Marie Schwartz College of Pharmacy and Health Sciences, Long Island University
12:00 – 1:00pm
1:00 – 1:50pm
Treatment, Patient, Time: Are We Getting it “Right”?
Panelists: Andrea Sboner, Ph.D., Department of Pathology and Laboratory Medicine, Institute for Computational Biomedicine Director of Informatics and Computational Biology, Englander Institute for Precision Medicine, Jeffrey R. Idle, PhD Endowed Professor and Director, Arthur G. Zupko’s Systems Pharmacology and Pharmacogenomics, Arnold and Marie Schwartz College of Pharmacy and Health Sciences, Long Island University,Stephen A Williams MD PhD, Chief Medical Officer, Somalogic Inc.
The goal of precision medicine is to ensure that the right treatment is delivered to the right patient at the right time. Buried in that near-religious statement are assumptions about our ability to identify the “right” things that are proving to be, if not completely untrue, then at least less robust than hoped in the early heady days. The three “right” choices that have to be made in precision medicine are inextricably linked, but it might help to consider each in isolation.
Treatment: What do we mean by the “right” treatment, how do we select it, and how do we measure its efficacy?
Patient: Who is the “right” patient, how do we select him/her, how do we know we have chosen correctly?
Time: What is the “right” time, how do we select it, and how do we assess our choice?
Successful answers to all of these questions ultimately depend on making a sufficient number of the right measurements, identifying real signals over noise in those measurements, and interpreting those signals in a meaningful way. Early precision medicine focused primarily on genomics, which still drives most of the awareness and hope for the field (finding signal in genomic noise is difficult, but “if we only sequence enough people/genomes/exosomes/transcriptomes/epigenomes, the knowledge will fall into place”). More recently, advocates for measuring “everything” possible are questioning the genomics-alone paradigm (omics-mania), but are also be running into some of the same challenge of measuring enough of everything to get reliably meaningful insights to fall out of the data in a consistent way, without swamping out any potential “signal” with a lot of “noise.”
This panel will take a hard look at where we are in terms of being “right,” and advocate for varying approaches to get us closer.
1:55 – 2:15pm
An Overview of Rheumatologic Therapy, and the Need for Diagnostics to Direct Therapy.
David J. Ridley, MD Rheumatologist, Clinical Instructor, University of MN
2:20 – 3:10pm
No More Trial and Error – Developing the Right Trials for the Right Patients.
Moderator: Jonathan Wagg, MD, PhD, Head of Disease Modeling, Oncology, Roche
Panelists: Arturo Loaiza-Bonilla, MD MSEd Chief of Medical Oncology and Medical Director of Research at Cancer Treatment Centers of America – ERMC, and also Co-Founder and Chief Medical Officer at Massive Bio, Bob Powell, Pharm.D. Adjunct Professor, College of pharmacy, University of North Carolina Chapel Hill, Michele Becci, VP, Industry Strategy – Medidata Solutions
Industry has moved away from the classic three phased design to approvals based on data found as early as in Phase 1b open-label studies. There is increased use of adaptive designs and master protocols as well as discussion on the level of redundancy with control arms for studies of similar drugs in the same tumor type. Clinical teams must become more agile, adapting process more frequently with faster development paths; increasingly need to work across company boundaries; harness advanced analytics to drive effective and efficient clinical development through patient finding, improved site selection, in trial monitoring, other approaches Critical to success of oncology research is adopting a flexible, unified platform that supports the modern clinical research paradigms and streamlines study conduct across the entire process
Almost 80% of cancer patients are diagnosed in a community setting and require logistical support to reach the nearest trial center. Better patient engagement and more personalized study designs are becoming more and more desirable in clinical research. Virtual trial designs already implemented have been shown to dramatically reduce or eliminate the need for ongoing site visits, allowing for research to be done at a huge cost savings to the sponsor and in a much more inclusive and convenient way for patients. A future of hybrid trial models that would embrace the use of technology to allow patients to participate remotely with little disruption to their personal lives coupled with the reality that drug development will continue still require in-person tests and evaluation. The balance between virtual and in-person settings will vary according to the needs of the study We estimate that in the future about 25 percent of clinical trials in the U.S. could be done as [completely] virtual trials. By leveraging broad capabilities for both standard and virtual protocols, customers can use what Medidata calls the “Trial Dial” to choose the right balance of site-based visits and virtual visits best suited for achieving maximum study efficiency and data quality. This type of hybrid trial will play a central role in the future of clinical research.
How Data and Technology Can Accelerate Precision Trials:
Nearly 90% of the data ever created originated in the past two years. Enormous volumes of data are generated by many data streams of different types, billions of nucleotides in the genome, hundreds laboratory test results, wearable sensor reading of dozens of kinds, medical imaging data containing many thousands or millions of pixels per image; These data present formidable challenges in how to structure, integrate and interpret this data for relevant insights to inform clinical trials. In order to achieve the tremendous gains of oncology research, data sharing and AI can fundamentally change the way trials are executed, bringing new comparators and approaches to the process. Extracting ‘fit-for-purpose’ value from the tsunami of data while maintaining quality of the data is required to rapidly bring these novel therapies to market.
3:10 – 3:40pm
Coffee & Networking
3:40 – 4:30pm
The Illusion of ‘Art’ in Medicine – Can Artificial Intelligence Bring Us Closer to Science?
Moderator: Spyro Mousses, PhD., CEO, Executive Scientific Advisor, Medical Intelligence and Innovation Institute Children’s Hospital of Orange County
Panelists: John Baldoni Ph.D., Senior Vice President, DPU Head, ln Silico Discovery, GSK Pharmaceuticals Morten Sogaard, Ph.D, Vice President & Global Head, Target Sciences & Technologies, Pfizer, Inc. Jim Fackler, M.D. Associate Professor, Anesthesiology and Critical Care Medicine, Johns Hopkins University School of Medicine
The tsunami of big data, advances in high performance computing, and innovations in machine learning have now converged, making it possible to transform how we discover, develop and deliver drugs. We will discuss how big pharmaceutical companies are using AI to enable and accelerate in silico discovery. We will then give examples of how AI is also being applied to bring precision to clinical development of experimental therapeutics. Finally, we will argue that in the near future, the same machine learning strategies that can improve clinical drug development can also be used in precision medicine – to find patterns in a single patient, and predict which treatment are safe and effective for that patient disease context. From acceleration to pharmaceutical sciences, to precision in drug development and care delivery, we will discuss how AI is rapidly evolving from niche tools for bioinformaticians to a ubiquitous and unifying platform that can add value across the entire spectrum.
4:35 – 5:25pm
Predictive Analytics in Precision Medicine – Harnessing the power of RWE, NGS and Liquid Biopsies to Inform and Help Refine Patient Selection Algorithms.
Moderator: Jessica Federer, Founder & CEO Innovayte
Panelists: Anne Marie Martin, PhD, SVP, Global Head of Precision Medicine, Novartis, Melanie Nallicheri, MBA, Chief Business Officer & Head Biopharma, Foundation Medicine, Ryan Fukushima, MBA, Chief Operating Officer, Tempus, Inc.
This panel will take the discussion beyond a companion diagnostic driven rhetoric and explore the data revolution in medicine which will ultimately provide the foundation for precision medicine to flourish. We will discuss how evolving technologies will support diagnostic development and data generation and we’ll highlight the need to work with a new breed of data companies that are complementing our understanding of diseases in the real world setting, as well as contributing to drug/diagnostic development. Finally, culminating in the incorporation of data analytics, leading in turn to predictive analytics. we shall focus on patient selection/monitoring and discuss how the intersection of robust biomarkers, technologies such as NGS and Liquid Biopsies coupled with new data insights will help us achieve that goal. The power of data analytics will create a new framework that will assist and help refine our selection algorithms
5:30 – 6:30pm
How to Maximize the Value of Your Genomics Portfolio? Discussion leader – Marc Grodman, MD, Co-Founder & CEO Genosity
Genomics has transformed medicine and patient care over the past decade, but much remains to be accomplished if the potential of these breakthroughs is to be fully leveraged. With decades of scientific, clinical and technological experience, we recognize that the greatest untapped opportunities in unlocking the value of genomics is to facilitate the intersection between genomics and therapeutics as well as the providing for the sustainability of clinical genomic programs in the marketplace. This will ultimately allow for the for greater access as well as the development of new and better drugs not only by discovering new targets and leads, but also by recruiting the right target patient population for clinical trials. Genomic testing needs to become more cost-effective and accessible, especially to large academic and hospital systems who will continue to see a larger portion of potentially consented patients
Challenges and solutions for optimal use of Omic data in clinical trials and precision medicine – Discussion Leaders – Ana Oromendia PhD, Genomics Business Analytics Manager at Medidata Solutions & Jason Mezey, PhD, Professor – Genetics, Cornell University and Weill Cornell
Omics data are becoming critical to driving scientific discoveries in oncology clinical research and treatment decisions in clinical practice. However, the systems and processes common in the life science community have usually failed to fully utilize the broad spectrum of data available in human clinical trials to yield new discoveries. Medidata’s new technology, Rave Omics with Biomarker Discovery, seamlessly integrates omic data with clinical data, performs data QC in real-time, and automates advanced machine learning and statistical analyses to uncover evidence of omic biomarkers.
Blockchain: Current and Future Viable Applications in Addressing the Data Challenges of Precision Medicine – Discussion Leader – Maria Palombini – Director, Emerging Communities & Opportunities Development, Global Business Strategy & Intelligence, IEEE Standards Association
When we think about precision medicine, we have two equal partners: the science and the data. Currently they are many challenges with collecting, managing, utilizing, leveraging and any other action around an individual’s health data – this is where Blockchain potentially can solve 50% of the problem. This “workgroup” like discussion will discuss some of the current health use cases for blockchain and they related to precision medicine while introducing specific use cases in this area and finally assessing whether blockchain is “worth” further investigation to address those use cases. Key questions to be addressed include:
– handling and sharing of people and population data
– overcoming the barriers with the current state of the EHR as it relates to collection, storing and utilizing genomic and/or precision medicine data
– the ethical considerations of precision medicine (privacy, security and rights of patients data)
– a trusted framework for ML and AI applications in precision medicine to distribute and secure data
Change is Coming! Case Studies and Learnings from Recent Payer Engagements in Precision Medicine. Discussion Leader: Lena Chaihorsky, V.P. Payer Innovation, ALVA10
Public and Private Payers have been wrestling with the concepts and value propositions of Precision Medicine for some years now. Participants will be briefed on a few notable and recent payer pilots in Precision Medicine, and discuss the changes in managed care that these programs represent. Alternatively, participants will examine where Precision Medicine stakeholders need to continue to work with payers – what are the persistent blind spots as the relationship between these two industries evolves?
The Use of Precision Oncology Decision Support Systems in the clinical practice. Discussion Leader: Stephane Richard, CEO CureMatch, Inc.
85% of cancer patients are treated in the community. Most of these oncologists are outside of the best academic centers where the best research is taking place. This creates a gap in knowledge and quality of care for both the physician and the patient. How can you identify the best possible treatment on your own when there is such a huge amount of data and knowledge to process?
Precision Medicine powered by Artificial Intelligence can bridge that gap, giving the community oncologist access to the best research, the latest clinical results, the best combination therapies, all optimized for their patient based on their molecular profile.
Deriving Transformation Value from EMR and Ancillary System to Enable Precision Care” Discussion Leader: Rahul Patel, MS, EVP & GM – Healthcare & Life Sciences, Persistent Systems, Inc.
The implementation of the EMR and other investments in data assets over the last few years has made available enormous volumes of clinical and social data. At the same time, evolving techniques in genomics and other sciences are adding to the types of data that are useful for patient care. However health systems have to overcome some key challenges. Just like data silos, there are also knowledge generation silos that have evolved over the years. Bringing together the data as well as the knowhow and techniques for making sense of this data requires a cohesive approach as part of a top down strategy for transformation. What is required is a technology architecture that utilizes the diverse data types to construct a ‘digital twin’ of the patient and use this construct to precisely map their needs to the right therapeutic choices, experience types and resources?
Diagnostic Methods and Precision Medicine Inventions – Still Patentable, Discussion Leader Beverly Lubit, Partner, McCarter & English
The challenge of subject matter eligibility in the U.S.
Computer-implemented inventions: overcoming “mental steps”
Why simply identifying biomarkers is not enough
How predictable are biological effects
6:45 – 8:45pm
WEDNESDAY JUNE 27th, 2018
Precision Analytics: Enabling Precision Medicine in the Clinic
Andrew L. Pecora, MD, FACP CPE
President Physician Enterprise and Chief Innovations Officer Hackensack Meridian Health
Identifiable and actionable molecular mutations have transformed clinical care particularly in oncology. Malignancies that historically rapidly led to meaningful morbidity and ultimately death have been transformed with Precision Medicines into chronic diseases and possibly cures. Optimizing the use of Precision Medicine remains a challenge in the clinic. Use of Precision analytic tools at the point of care can be enabling.
Haematological Malignancies – Progress on Patient Selection and Response Prediction
Moderator: Jim Cassidy, Corporate Vice President, Celgene Corporation
Panelists: Anjan Thakurta, PhD. Executive Director, Translational Development, Celgene, Samir Parekh, MD, Associate Professor, Icahn School of Medicine at Mount Sinai, Anita Gandhi, PhD, Senior Director, Translational Development, Celgene
8:40 – 8:50am
Samir Parekh – Network Modeling and Personalized Therapy for Myeloma
Multiple Myeloma (MM) is a fatal cancer of plasma cells affecting 30,000 new individuals each year. The MM Program at Mount Sinai is one of the largest in the nation, with 3500 active patients and 650 new referrals each year for complex clinical management. Over the past 4 years, we have developed a unique platform for integrative analysis of multi-omics data to identify treatment options in real time for relapsed and refractory patients who have failed all FDA-approved MM drugs. We have conducted a pilot trial with relapsed patients with excellent results from RNA and DNA based drug repositioning. In addition to the clinical results, the sequencing data from these patients has yielded insights into pathogenic mechanisms and immunologic applications (vaccine development). The software and infrastructure developed by our computational team and engineers at Mount Sinai, could be useful for other teams of oncologists and researchers.
Our long-term plan is to integrate our platform into the routine clinical management of our patients, with additional refinement in terms of computational prioritization of drug recommendations and concurrent validation using in vitro (chip based) and in-vivo (PDX) models. Additionally, we are integrating single-cell analysis for clone-specific drug prediction and microenvironment assessment to direct precision immunological approaches complementing our drug matching algorithms. We intend to produce a CLIA-certified assay to sequence most our MM patients by 2020 and plan to extend our platform to help patients with other hematological malignancies and solid tumors.
8:50 – 9:00am
Anjan Thakurta – Myeloma genome project and its application to future studies in Myeloma
MGP is a collaborative large scale data sharing and analysis project. It was initiated and sponsored by Celgene. The plan was to use publically available data sets which incorporate multiple types of molecular data associated with clinical outcomes information. The ultimate objective being to define biologically distinct sub sets of patients in both newly diagnosed and relapse / resistant setting to enable more efficient clinical trial designs. Anjan will outline some of that data and discuss its relevance to future clinical studies
9:00 – 9:10am
Anita Ghandi – Patient selection approaches for immunomodulatory agents in Diffuse large b cell lymphoma
Celgene has a long track record of discovering and developing drugs in the – imid class from the thalidomide backbone. These agents have pleomorphic mechanism of actions which we now understand to be the consequence of degradation of specific transcription factors substrates. One aspect of these agents MOA is to modulate the immune response to disease. DLBCL remains a disease with significant unmet needs which we hope to address – at least in part by selecting the right patients for an immune modulatory approach
9:10 – 9:40am
All participants reconvene for panel discussion on topics presented above
Translating Digital Biomarkers into Precision and Predictive Medicine Solutions
Vaibhav A. Narayan, PhD, MBA, Vice President of Research IT, Janssen Pharmaceuticals LLC & Head, Integrated Solutions, Janssen Neuroscience.
With rising health-care costs, health care stake-holders (payers, physicians, patients) are shifting the onus from a ‘pay for intervention’ to a ‘pay for performance’ model. This change in focus towards overall outcomes will drive a paradigm shift towards ‘stratified medicine’. i.e. matching therapeutic (drug and non-drug) intervention to optimal data-derived disease subtypes and disease interception, i.e. move from a ‘diagnose and treat’ to a ‘predict and pre-empt’ approach. It is now possible to develop predictive models of disease state changes and progression that include streaming data from on-body/off body sensors found in wearable devices and smartphones. Integration of these new data-types with extant clinical history data and available biomarker data (genetic, imaging, molecular profiling) presents a new opportunity to develop real time predictive models and disease classification algorithms and change how diseases are detected, monitored and treated. This talk will explore some of the data, analytics and translational challenges in taking such digital solutions to patients.
COFFEE & NETWORKING
Pre-competitive Partnerships: Improving Patient Outcomes Through Precision Medicine
Moderator: Christina Bender, PhD, MBA, Director, Global Diagnostic Pipeline Strategy – Novartis
Panelists: J.D. Alvarez, M.D., Ph.D.,Vice President, Oncology Diagnostics, for the Janssen Pharmaceutical Companies of Johnson & Johnson, Sarah Hersey, MS, MBA, RAC, Head of Precision Medicine and Companion Diagnostics, Celgene CorporationSuso Platero, PhD, Head Biomarker Solution Center, Global Leader Precision Medicine, Covance, Inc., Wayne Caldwell, Director, Diaceutics, Inc.
• Collaborate on diagnostics, compete on the drugs
• Accelerate and advance precision medicine
• Reduce complexity for physicians
• Winning for patients
Is Data Mining of Real World Evidence from Large Populations the Fastest Route to Routine Precision Medicine?
Moderator: Michael Kolodziej, MD – VP and Chief Innovation Officer, ADVI Health
Panelists: Mark G Kris, MD, FACP, FACCP, Oncologist, MSKCC, Nicholas Robert, MD, Medical Director, Health Economic Outcomes Research/Health Informatics, McKesson Speciality Health/US Oncology, Robert S. Miller, MD, FACP, FASCO Vice President and Medical Director, CancerLinQ, by ASCO, Nate Nussbaum, MD, Medical Director, Flatiron Health
Despite the profound progress in the technical aspects of tumor genotyping, the clinical utility of NGS panels for somatic mutation analysis of solid tumors has continued to be an area of considerable controversy. With the recent finalization of the CMS National Coverage Determination for NGS in solid tumors, there is a real possibility that many more patients will be tested identifying potential targets. But because of coverage policy governing use of therapeutic agents directed against these targets, we will hit a new impasse in moving precision medicine forward. Certainly clinical trials like ASCO’s Tapur and NCI Match offer solutions, but the eligible patient base is huge compared to clinical trial opportunities. One potential solution is the use of real world evidence to rapidly expand the knowledge base and identify the clinical utility of novel marker/therapeutic agent combinations. But real world evidence has its own challenges. Our panelists are leaders in the real world evidence space. They will describe the current state of affairs in real world evidence; identify challenges in data collection, curation, and application; and offer a vision of how the promise of real world evidence can be realized.
The Power of Prediction – New Models for Neurodegenerative Disease
Moderator: Todd Sherer, Ph.D. CEO – The Michael J. Fox Foundation for Parkinson’s Research
Panelists: Darryle D. Schoepp, Ph.D., Vice President, Neuroscience Discovery Research, Merck, Melissa Nirenberg, MD, PhD – CMO – The New York Stem Cell Foundation
2:40 – 3:30pm
Getting to Yes- How to Drive Precision Medicine into the Market Through Investment
Moderator: Hannah Mamuszka, Founder and CEO of Alva10,
Panelists: Glenn Miller, PhD, Innovation’s Market Sector Leader, Partners Healthcare, Blake Long, MD, MBA, President, Long Health Care Consulting, Carol Berry, MBA, Senior Vice-President and Chief Business Officer, Caprion Biosciences Inc. Jeff Waldron, MBA, Managing Director, Sherborn Consulting Group
The success of Precision Medicine is reliant on proportional use of all the tools in healthcare- not only precision therapeutics, but precision diagnostics and tools for patient stratification for risk, response, adverse events, monitoring. In recent years, while investment on the therapeutic side has been strong, investment in biomarkers, diagnostics, and platforms has waned, as investors and companies struggle to find a path to return on their investments. Can we reframe the conversation and bring in new stakeholders to pull more technology into the market?
NETWORKING & EXHIBITS
Legal Challenges in the Field of Precision Medicine
Moderator: Nephi Walton, MD, MS, Assistant Professor of Genomic Medicine, Geisinger Health
Panelists: Beverly Lubit, JD, MBA, PhD, Partner, McCarter English, Arthur E. Peabody Jr. JD, Principal at Arthur E. Peabody Jr. PLLC, Jennifer K Wagner, JD, PhD, Associate Director, Bioethics Research Assistant Professor Center for Translational Bioethics and Health Care Policy, Geisinger Research, Steven Tjoe, JD, MS, Associate, Goodwin Procter LLP
As the floodgates open and healthcare systems are inundated with new information in the form of genomics, other omics, and data from wearable devices many possibilities arise as to how to utilize this data to improve patient care using AI and other technologies. At the same time the regulatory environment and policies around this new information are still in their infancy with several cases now in litigation. In this session we discuss the legal challenges that surround precision medicine, discussing current litigation, and concerns to watch for as this new data becomes an integral part of our healthcare system.
Using Rare Benign Tumors to Cure Malignant Cancer
Moderator: Annette Bakker, PhD, President and Chief Scientific Officer, the Children’s Tumor Foundation
Panelists: Stefan Scherer, MD, PhD, Vice President, Head Early Development, Strategy and Innovation, Novartis, Antonio Iavarone, MD, Professor of Neurology and Pathology, Institute for Cancer Genetics, Columbia University
Not so long ago, benign tumors were barely spoken of. Most cancer organizations didn’t consider benign tumors because they were (mistakenly) treated as a ‘non-problem’. With the focus on complex, metastatic malignant cancers, those with benign tumors received scant attention, but yet faced serious issues nonetheless. Today, the world is changing and there is new hope for this underserved patient population. At the recent ASCO meeting in Chicago, the NCI reported the outcomes of the SPRINT trial. SPRINT is the registration trial of the MEK inhibitor selumetinib for inoperable plexiform neurofibromas. The results are stunning: 70% of the patients respond with tumors shrinking anywhere from 20-50%.
What now? This panel will address how benign tumors and the study of how ‘normal’ biology can contribute to the development of better malignant cancer treatments. Topics like tumor kinetics; tumor evolution; regulatory networks and AI will be included.
Free Evening – Enjoy the sights and sounds of Jersey City or visit the vibrant nightlife of the City that never sleeps.
THURSDAY JUNE 28th, 2018
Keynote: Building a Robust Biobanking Infrastructure for Precision Medicine
Dina Avery, DHSc, MA, MAEd Assistant Professor & Regulatory Affairs Specialist University of Alabama at Birmingham
Biobanking is key for the advancement of Precision Medicine. All biobanks should have an infrastructure in place that promotes compliance and quality. This presentation will provide key facts related to:
• Establishing the tissue banking team
• Collaboration needed among the tissue bank, patient and medical professionals
• Best practice to collect clinical & pathological data
• How to increase the specimen collection rate
• Building collaborative research relationships outside and within the institution
Can Digital Medicine Help Facilitate a Move to Precision Health?
Moderator: Ashish Atreja, MD, MPH, Asst. Professor & Chief Innovation Officer, Icahn School of Medicine at Mount Sinai
Panelists: Gregory J. Downing, D.O., Ph.D. Founder of Innovation Horizons, LLC, Yauheni Solad, MD, Medical Director, Digital Health, Yale New Haven Health, Rahul Patel, MS, EVP & GM – Healthcare & Life Sciences, Persistent Systems, Inc.
• Going beyond genes to look at digital biomarkers for precision population health
• Remote Monitoring and its impact on the delivery of precision medicine/health
• Real time data from a patient, including environmental and social determinants of Health
• Digital Engagement: Assessing the behavior of patients in terms of their interaction with healthcare providers, and the subsequent intervention by those providers.
COFFEE & NETWORKING
The Third Wave: How Precision Medicine will Accelerate the Move to Value and Bring Joy Back to the Practice of Medicine
Grace Terrell, MD, CEO – Envision Genomics
Three waves of change have been transforming the health care industry over the past ten years. The first wave has been a focus on patient-centered models of care. The second has been focused on consumerism. The third wave, just at the beginning of its crest, promises to redirect health care back to its core mission: providing highly effective and personalized solutions to individual patients. Precision medicine’s 21st-century technology redirects care back to what makes an individual unique, rather than focusing on 20th-century statistical modeling of averages and variances. The application of these technologies at the population level will lower costs and improve quality to a degree heretofore impossible to design. Understanding how to redesign care, payment models, and delivery systems with 21-st century precision technologies as a core organizing component is the essential work we need to focus upon throughout the health care industry.
Meaningful Research Needs Collaboration with Patients to Enable Transformative Clinical Outcomes
Moderator: Peter Kapitein – Patient Advocate, Inspire2Live
Panelists: Laura Thrall, Healthcare Consultant, Shridar Ganesan, MD, PhD. Chief Molecular Oncology, Rutgers Cancer Institute of New Jersey, Ilona Schelle – Patient Advocate, Inspire2Live, Owen Roberts,CEO Nobelex Biotech Inc & M4K Pharma
Healthcare and connected research has forgotten their main purpose; that their aim is to benefit the patient. This is crucial but the patient has not been invited to participate. We can clearly see that the patient has to take what they are given; rather like a dog that eats what it is offered. The one who is consuming is not paying for what’s on the menu and therefore does not determine what’s on it either. And we all know ‘If you’re not at the table you’re on the menu’.
Do patients really want to participate in research? Of course, patients are not researchers but we want to be at the table with the researchers and clinicians and then have a good and solid discussion about what research should be performed. There is no question about participating because of course we want to participate in our own research. This is about us and we should be involved.
This panel is about cooperation between the stakeholders in healthcare and their contribution to research that benefits the patient. Healthcare should have the patient in the centre and allow them to be a real consumer who determines what they need. Today, there are good examples of research that is performed in this way, so let’s follow these examples in order to achieve a better quality of life for patients. There is a great need for this to change because we have invested hundreds of billions of dollars in researching healthcare over the decades and continue to spend it in the same way
Is Precision Medicine Exacerbating Disparities in the Health Care System?
Moderator: Ysabel Duron, CEO – Latino Cancer Institute
Panelists: Jennifer Manly, Ph.D. Associate Professor of Neuropsychology in Neurology at the Taub Institute for Research in Alzheimer’s Disease and the Aging Brain, Columbia University, Nishadi Rajapakse, Ph.D. Director of the Transdisciplinary Collaborative Centers for Health Disparities Research Focused on Precision Medicine, NIH, Michael Yudell, Ph.D., MPH, Associate Professor & Chair – Community Health and Prevention – Drexel University, Jay Kaufman, Ph.D., Professor, Dpt. of Epidemiology, Biostatistics and Occupational Health, McGill University
• Historical background of how new technologies have affected disparities. Evidence showing that new technologies tend to exacerbate disparities when they diffuse unequally because advantaged populations get access first (e.g. HAART).
• Epidemiological/Public Health perspective on how disparities have shrunk over the last 50 years due mostly to mass public health campaigns that were not targeted (diet, blood pressure,cholesterol, healthy pregnancy, etc).
• Aging and dementia and the search for therapies, including the new move to allow novel “precision” therapies to be tested against surrogate outcomes to lower the bar for approval.
• Programmatic approach at NIMHD, how to encourage precision medicine research that is relevant for disparities and how it will act to close those gaps.
Molecular Imaging Solutions for Precision Medicine
Susanta K. Sarkar PhD, President, CadenzaMed, Adjunct Associate Professor Department of Radiology, University of Pennsylvania
Development of targeted therapeutics and immunotherapy has made a significant impact on the treatment of cancer patients. However, despite the remarkable clinical efficacy of these agents, the benefits from these approved drugs are still limited to subset of patients. This is mainly because of the lack of optimal ways to better identify responsive patients and improve outcome. Similarly, many targeted drugs that are being developed do not reach the market because of the lack of clinical benefit, likely due the testing of the drug in wrong patient population. This adds significant patient burden and major costs to the healthcare system. Recent advances in molecular imaging offers solutions to these challenges. This talk will outline how development of molecular imaging based approaches can help patient stratification in real time and potentially aid the physicians manage treatment regimens to benefit patients.
A Brave New Future – Gene Editing and Regulatory Oversight
Moderator: James N. Czaban, Partner & Chairman of the FDA and Medical Products Regulatory Practice Group at DLA Piper LLP
Panelists: Eric B. Kmiec, Ph.D, Founder and Director of the Gene Editing Institute at the Helen F. Graham Cancer Center & Research Institute at Christiana Care Health System