WEDNESDAY SEPTEMBER 23rd, 2020
Registration & Breakfast Buffet
Welcome and Opening Remarks
Precision Medicine Leaders’ Summit Chair: John Quackenbush, PhD, Henry Pickering Walcott Professor of Computational Biology and Bioinformatics, Chair, Department of Biostatistics, Harvard University T.H. Chan School of Public Health.
Nigel Russell, Founder & President, The Journal of Precision Medicine and the Precision Medicine Leaders’ Summits.
Keynote – Getting Up Close and Personal with Precision Medicine: Implications of Single Subject Studies (S3)
Yves Lussier, MD, FACMI, Associate Vice President for Health Sciences and Chief Knowledge Officer, Executive Director, Center for Biomedical Informatics and Biostatistics, University of Arizona Health Sciences.
This presentation will review recent innovative translational computational biology and bioinformatics analytics that substantially advanced our accuracy in predicting effect sizes and statistical significance in single-subject studies (S3). S3 can potentially increase clinical trials’ efficiency through reducing clinical trial cohort sizes by more than 50%. Indeed, S3 enable two paradigm-shifting analytics. First, each subject serves as their own case and control, generating significance metrics (pvalues) associated to the magnitude of their altered pathophysiological mechanisms (effect size). It follows that of these significantly altered biomechanisms are specific to that individual, however if we assume that the conditions (tissue, assay) between the two paired samples are relevant to therapeutic response ,then the union of statistically significant mechanisms, obtained in at least one subject regardless of the therapeutic response, provide substantially smaller and unbiased list of mechanisms likely relevant to the patient response than conventional inputs of all gene products in machine learning (ML) classifiers.
We will demonstrate the increase accuracy of these principles in simulations of biomarker discovery and in one published clinical trial validation where ML classifiers of therapeutic response can be derived from a small set of twenty subjects and led to identify children prone to asthma exacerbations in another independent validation.
Precision 20 Foundation Medicine Speaker & Topic
Precision 20 – Getting to Know the Person Behind the Patient
Dana Deighton, Cancer Patient, Survivor, and Advocate
Coffee & Meet the Expert
Leveraging Real-World Data in Regulatory Science
Moderator: Lori Anderson, Precison Medicine Informatics and Real-World Data Program Lead, XIFIN
Panelists: Trish Goede, PhD, Vice President of Clinical Informatics, XIFIN; Wendy Rubinstein, MD, PhD, FACP, FACMG, Director Personalized Medicine, FDA
The 21st Century Cures Act passed in 2016 mandates that the FDA increasing leverage real-world data (RWD) in its regulatory decision making. Real-world data comes from a number of sources including EHR’s, claims databases, and patient registries. However, there are limitations on the usefulness of RWD extracted from these sources. During this panel discussion, we will discuss the current uses of RWD in Regulatory Science and explore how uses could potentially be expanded by integrating patient data from multiple disparate data sources including clinical, financial, and biomarker data into a single enriched RWD source.
Is My Drug Right for a Synthetic Control Arm?
Moderator: C.K. Wang, MD, Senior Medical Director, COTA
Panelists: Daniel Lane, PhD, MBA, Vice President Research, COTA; Matthew Reynolds, PhD, Vice President, Real World Effectiveness, IQVIA
Randomized, placebo-controlled clinical trials have long been considered the gold standard in drug development, but they raise concerns about generalizability and are not feasible in all settings. Synthetic control arms offer an appealing alternative that relies on existing real-world data (RWD) collected from various sources. Using this data, synthetic control arms can replace or supplement a control group treated with a non-experimental approach, potentially accelerating the time it takes to get a drug to market and reducing the overall cost of drug development.
Advances in the curation of patient data, support from the FDA for the use of RWD in clinical trials, and growing concern over the rising costs of drugs have generated enthusiasm for the use synthetic control arms in clinical trials. However, there are risks inherent in omitting randomization.
Radiomics and Its Impact on Precision Medicine
John Quackenbush, PhD, Henry Pickering Walcott Professor of Computational Biology and Bioinformatics, Chair, Department of Biostatistics, Harvard University T.H. Chan School of Public Health
From Single-Gene Tests to Large Multi-Gene Panels: Should Genomic Testing in Cancer be “One Size Fits All”
Moderator: David Parker PhD, SVP Diagnostics Solutions, Precision For Medicine
Panelist: Sheryl Krevsky Elkin, PhD, Chief Scientific Officer, N-of-One, a Qiagen Company
Most personalized medicine therapies are targeted to the product of a single gene. When there were relatively few such drugs, single-gene tests generally sufficed to guide therapeutic decision making, and many approved CDx still fall in this category. Now, however, considerably more targeted therapies are available for use in various cancers, with additional ones in clinical testing. In response, NGS tests for cancer gene alterations are becoming available that encompass ever-larger panels of genes and have potential to guide patients toward both appropriate approved therapies and targeted drug candidates in development. While these multi-gene panel tests are acknowledged to have great clinical potential, many payer coverage policies reflect the perception that they don’t yet deliver utility commensurate with their cost. This panel will discuss issues of approved vs. investigational uses, clinical utility, cost-effectiveness, value, and others as it considers how each type of genomic testing assay best fits into the cancer care ecosystem.
Coffee & Networking
How a Collaborative Ecosystem can Revolutionize the Next Generation of Precision Medicine
Kevin Hrusovsky, MBA, Chairman & CEO, Quanterix
As we find ourselves on the precipice of a renewed movement in precision medicine and preventative care, a hesitation on the part of drug companies to enact companion diagnostics marks the last mile of moving this into full-blown revolution. Breaking through that hesitation will require an ecosystem of collaboration to embolden the stratification biotechnology that enables this enhanced personalized performance of drugs and payers. This talk will focus on how to build this collaborative ecosystem where the influence of payers and advocacy groups can pervade traditional profit models. It will also explore how increased collaboration across stakeholders will further accelerate the transformation of precision health.
mCODE: Powering Learning Health Systems with a FHIR-enabled Data Model
Brian Anderson, MD, Chief Digital Health Physician, MITRE Corporation
Panelists: Monica Bertagnolli, MD, Chief, Division of Surgical Oncology, Brigham & Women’s Hospital at Brigham and Women’s / Dana-Farber Cancer Center, Professor of Surgery, Harvard Medical School; Greg Simon, JD, President, Biden Cancer Initiative, Director, Biden Foundation; Carole Tremonti, RN, MBA, Dana-Farber Cancer Institute
Please join us for a panel discussion on the future development and implementation of the Minimum Common Oncology Data Elements (mCODE). We will discuss real world data use cases of mCODE in application development for patient care, clinical trial research and patient data ownership.
Fireside Chat – The Role of CROs and Innovation to Support Clinical Trials
Moderator: Madhushree Ghosh, Ph.D, Senior Director, Global Strategic Partnerships, Life Sciences Solutions, Thermo Fisher Scientific
Panelists: Kellie Yarnell, Vice President, Global Genomic Operations, Q² Solutions Patrick Hurban, PhD., Senior Director and Global Head, Translational Genomics, Q² Solutions
When it comes to supporting clinical trials, having a global footprint is simply not enough for contract research organizations (CROs) to maintain a competitive edge. Access to the latest technology to help meet the stringent development demands from pharmaceutical clients has become equally important in the age of multi-omic driven medicine. Recently, Q² Solutions announced a $73-million expansion of its center of excellence genomic testing center in Durham, North Carolina. The new facility will house a translational science and innovation laboratory where state-of-the-art technology for biomarker discovery will be tested with the intent to deploy it across its laboratories in China, Scotland and Singapore. This fireside chat will discuss both the business and scientific strategy of staying on the leading edge to support clinical trials.
Networking Reception – enjoy drinks and hors d’oeuvres with fellow attendees, speakers and sponsors
THURSDAY SEPTEMBER 24th, 2020
Day One Recap
Precision Medicine Leaders’ Summit Chair: John Quackenbush, PhD, Henry Pickering Walcott Professor of Computational Biology and Bioinformatics, Chair, Department of Biostatistics, Harvard University T.H. Chan School of Public Health
8:30 – 9:20am
How to Overcome the Challenges to Build a Precision Medicine Program
Moderator: Clynt Taylor, CEO Intervention Insights
Panelist: Arturo Loaiza-Bonilla, MD, MSEd, FACP, Vice Chair Medical Oncology, CTCA Philadelphia; Shridar Ganesan, MD, PhD, Associate Director for Translational Science, Section Chief, Molecular Oncology, Omar Boraie Chair in Genomic Science, Professor of Medicine, Rutgers Cancer Institute; Huntington Willard, PhD, Chief Scientific Officer & SVP Medical Affairs, Genome Medical, Inc.
With rapid advancements and growing complexity in the field of precision medicine, more than ever cancer centers need better ways to deliver, manage and measure quality care.
In this session, we’ll discuss the challenges physicians, lab and practice directors face in this new age of precision oncology and offer solutions for standardizing processes, improving patient access, increasing clinical trial accrual and creating a true value-based care environment.
Keynote – Understanding True Impact of PGx
Kristine Ashcraft, MBA, Founder and CEO, Youscript
Systems implementing precision medicine programs have warned that EHR-integrated clinical decision support tools (CDST) must be a priority. See the added value pharmacogenomics and YouScript CDST have provided in impacting clinical decisions and reducing ED visits, hospitalizations, readmissions, and costs. In addition to peer-reviewed studies, we will cover recent real-world validations. These include a Highmark BCBS VITAL innovation program at an Allegheny Health Network clinic and a large-scale longitudinal claims analysis completed by a major U.S. payer.
Impact of Dx in Market Now
Moderator: Hannah Mamuszka, Founder & CEO, Alva10
Panelists: Mark Hiatt, MD, MBA, MS, Vice President, Medical Affairs, Guardant Health, former Executive Medical Director, Regence BCBS; Debbie Ledet, Vice President, Market Access, Biotheranostics, Inc; Debbie Windsor, Head of Market Access, ExosomeDx, a Bio-Techne Brand
Diagnostic developers have many challenges once they reach the clinical market- educating physicians on use, confirming clinical utility, demonstrating competitive advantage; but the largest market challenge is gaining broad reimbursement. During this panel, we will discuss what the challenges to broad coverage are for established diagnostic companies and how they are developing strategies to overcome them.
Coffee, Networking & Meet the Expert
Precision 20 – Collaborating with Payers Beyond Coverage
Heather A. Brown, PhD, Senior Vice President, Market Access and Reimbursement, HeartFlow
Reimbursement strategies for new products often focus on obtaining positive coverage policies. While this is essential to success, there may be opportunities to leverage a product’s value proposition beyond coverage into specific care pathways. This talk will focus on how to engage various aspects of the payer system to promote innovative care pathways and influence decision making in the provider network.
Precision 20 –
Gabriel A. Bien-Willner, MD, PhD, FACP, Medical Director, MolDX, Chief Medical Officer, Palmetto GBA
Emerging Diagnostics and the Impact Patients, Providers, Payers Will See in 2020 and Beyond
Moderator: Hannah Mamuszka, Founder and CEO, Alva10
Panelist: Slava Akmaev, MS, PhD, Scipher Medicine; Tony Grover, MBA, Chief Commercial Officer, Prescient Medicine
2020 is shaping up to be an exciting year for new diagnostic technology, particularly for precision medicine outside of oncology. We will discuss these novel diagnostic tools entering the market to address clinical challenges in autoimmune diseases and opioid use; how to gain consensus on clinical utility from physicians and payers; what the thresholds are to garner reimbursement coverage
12:55 – 1:45pm
Diagnostics Up Next Where is Diagnostics Technology Going?
Moderator: Hannah Mamuszka, Founder & CEO, Alva10
Panelist: Amrie Grammer, PhD, MS, Chief Operating Officer and Chief Science Officer, AMPEL BioSolutions, LLC; Christiana Iyasere, MD, MBA, Vice Director, Massachusetts General Hospital Department of Medicine Innovation Program & Co-Founder, Dyrnamix, Inc; Satabhisa Mukhopadhyay, PhD, Founder and Chief Scientist, 4DPath, Inc
Advances in diagnostic technology are making what used to seem inconceivable now possible. Using genomics to guide the use of a device in heart failure patients? Watch for Dyrnamix. Analyzing biomarkers for oncology with only a digitized H&E image? Check out 4D Path.
What’s coming next?
Unleashing the Power of Precision Medicine by Bridging the Divide Between Payers and the Diagnostics Industry
Matt Tucker, MBA, Head of Vital Program, Highmark Health & Chief Marketing Officer, NightWare; Lena Chaihorsky, Vice President, Alva10
Diagnostics of the Future: the Tests Payers Need from Our Industry
Moderator: Lena Chaihorsky, Vice President, Alva10
Panelists: James Almas, MD, Vice President and National Medical Director, Clinical Effectiveness, LabCorp, and former Medical Director, MolDX; Arthur Hermann, MS, Principal Strategic Technologist, Kaiser Permanente; Sumanth Reddy, EIR – National Accounts, Anthem; Ken Schaecher, MD, Associate Chief Medical Officer of University of Utah Health Plans
Diagnostics have historically developed their clinical utility value propositions and evidence in a vacuum, prior to seeking payer coverage and prior to seeking any real payer input. Using the Alva10 model of payer-partnered diagnostic development, this panel will discuss the areas where payers want and need to see the diagnostic industry developing solutions. What are payers’ areas of high costs and poor outcomes? What diagnostic value propositions will enable them to better manage tomorrow’s members? What kind of evidence is required to prove these new value propositions? These are the topics where payers and diagnostic companies must agree in order to provide valuable solutions, and have them be utilized for patient benefit.
Fireside Chat – Factors Influencing the Acceptance of a Novel Diagnostic Technology in the Coverage Policy of a Health Care Plan: Case Study
Mark Hiatt, MD, MBA, MS, Vice President, Medical Affairs, Guardant Health and Eugean Jiwanmall, MPH, MBA, Senior Research Analyst for Medical Policy & Technology Evaluation, Independence Blue Cross
This study of one plan’s evaluation and ultimate acceptance of liquid biopsy, in the context of ascertaining the genomic drivers of lung cancer, will illustrate the factors influencing such coverage decisions. Factors discussed include the description of the technology, published studies in support of the technology, and the opinion of other plans and their intermediaries.
Closing Remarks – Adjourn
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