by David L. Davenport, Manager of Public Policy, Secretary to the Board, Personalized Medicine Coalition

Citing decreasing genetic sequencing costs, the development of new targeted therapies, and the ability to bring together genetic and clinical information with new technologies, Harvard Medical School’s Paul C. Cabot Professor of Genetics Raju Kucherlapati, Ph.D., who also serves as Scientific Director, Partners HealthCare Personalized Medicine, opened the Personalized Medicine & Diagnostics Track at the 2018 BIO International Convention by asserting that “the field of personalized medicine [is positioned to] have an even greater impact on patients and the health of the human population [in the future].”

The Personalized Medicine & Diagnostics Track, held in Boston from June 5 – 7, was co-organized by the Personalized Medicine Coalition (PMC) and sponsored by Thermo Fisher Scientific. The track brought together thought leaders to discuss the latest innovations and policies in personalized medicine. From those conversations several themes emerged:

1. Innovative, multi-stakeholder collaborations promise to bring personalized medicines to patients faster.

With thoughtful approaches to multi-stakeholder collaborations, patients stand to benefit. In Partnerships Power Precision Health: Collaborative Models for Advancing Personalized Medicine, moderated by the University of North Carolina School of Medicine’s Terry Magnuson, Ph.D., and featuring representatives from the BlueCross BlueShield Association, the National Institutes of Health (NIH)’s All of Us Research Program, and Color Genomics, panelists concluded that collaboration between and within multiple sectors of the health care system will be crucial to advance personalized medicine. Several collaborative models were discussed during the conversation, including: providers partnering with other providers to share data; multiple community stakeholder groups partnering with the NIH’s All of Us Research Program to collect a million genomes; and joint research endeavors involving industry, payers, providers, and patient groups.

2. Advances in data science will help integrate genomic, clinical, and economic information that is necessary to deliver new personalized medicines to patients.

During Personalized Medicine in Action: Examples of Clinical Implementation, moderated by Daryl Pritchard, Ph.D., Senior Vice President, Science Policy, PMC, and featuring representatives from Agendia, the Swedish Cancer Institute, and Stanford Health Care, panelists discussed constraints to delivering holistic, value-based personalized medicine and concluded that reimbursement paradigms must evolve to facilitate this approach. Building and combining genomic, clinical, and economic data sets would help provide evidence not just for clinical utility but for cost effectiveness, the latter of which is crucial to coverage and reimbursement. First and foremost, however, data must be “accurate” and “complete” to be useful.

Echoing Dr. Kucherlapati’s opening comments, panelists during the session Is Biotechnology Drowning in Health-Related Data? agreed that analyzing many kinds of data will be the next step in advancing personalized medicine. They pointed to artificial intelligence and machine learning as new tools that could help identify promising personalized medicine strategies. The panel, moderated by Damian Garde, National Biotech Reporter, STAT News, and featuring representatives from Novartis Institutes for Biomedical Research, GNS Healthcare, and Sema4, discussed the need to develop new personalized medicines for unmet medical needs and acknowledged the benefit of existing diagnostic strategies that help determine who will not benefit from a treatment.

Moderated by Alan Sachs, M.D., Ph.D., Chief Scientific Officer, Thermo Fisher Scientific, and featuring representatives from Metabolon, King Faisal Specialist Hospital and Research Center in Saudi Arabia, and the University of Pittsburgh, a discussion of large-scale public and private research programs aimed at screening healthy populations for actionable genetic risk factors reiterated that while some data already show the value of personalized medicine, the generation of additional evidence, along with provider education, is needed to facilitate clinical implementation. Panelists coalesced on implementation strategies that keep a broad vision for personalized medicine in mind but focus first on screening for genetic risk factors where evidence of value is strongest.

In a session titled Bridging the Gap: Adding Dynamics to the Diagnostic/Therapeutic Interface, moderated by Alice Jacobs, M.D., Advisor, Third Rock Ventures; Entrepreneur-in-Residence, Caltech, and featuring representatives from Cedars-Sinai Medical Center, Ceres Nanosciences, and Genome Profiling LLC, panelists discussed the potential for ongoing monitoring of biomarkers and patient data through wearables and direct-to-consumer genomics to develop dynamic wellness and treatment plans, and suggested that this may be the future of personalized medicine.

The track’s emphasis on data echoed thought leaders’ conclusions about the field during other Convention sessions outside of the track. In a fireside chat between U.S. Food and Drug Administration (FDA) Commissioner Scott Gottlieb, M.D., and BIO President and CEO James Greenwood, for example, Commissioner Gottlieb commented on the growing importance of managing data. In particular, he pointed to the increasing potential of real-world evidence (RWE) in product approvals or authorizations as the tools for establishing statistical significance of large RWE data-sets are improved and as investments are made in large data-set models.

3. Reforms to regulatory and coverage decision-making processes are needed to keep pace with the rapid development of new personalized medicine products, services, and technologies.

During The Next Generation of Personalized Medicine: A New Regulatory Paradigm for Next-Generation Sequencing Panels, moderated by Cynthia A. Bens, Senior Vice President, Public Policy, PMC, representatives from the American Cancer Society Cancer Action Network (ACS-CAN), FDA’s Center for Devices and Radiological Health, the U.S. Centers for Medicare & Medicaid Services (CMS)’ Coverage and Analysis Group, and Thermo Fisher Scientific discussed the parallel review process for medical devices and diagnostics at CMS and FDA, and how the agencies are working together to improve transparency and to help companies that approach FDA for clearance avoid common pitfalls in bringing to market new technologies facilitating personalized medicine. According to Tamara Syrek Jensen, J.D., Acting Director, Coverage and Analysis Group, CMS, “parallel review is important for how it is changing the communications between CMS and developers going into regulatory review [from retrospective] to prospective communication.” ACS-CAN Policy Principal Mark Fleury, Ph.D., said parallel review is the first step in “harmonizing” an oversight system that “doesn’t quash innovation.”

In Precision Diagnostics: Trends in Evidence Development for Medicare and Commercial Payers, moderated by Brian P. Carey, J.D, Partner, Foley Hoag LLP, with representatives from ADVI, CareFirst BlueCross BlueShield, and Foundation Medicine, panelists addressed the emerging need for more evidence of personalized medicine’s value. Parallel review, coverage with evidence development, and joint studies between commercial payers and diagnostic companies will be key in developing that evidence. Referencing Foundation Medicine’s recent experience with parallel review, the company’s Senior Director for Payer Policy and Health Outcomes Ingrid Marino encouraged other developers considering parallel review to prepare for contrasting points of view and bring solutions to their discussions with the government agencies.

During a panel on business models providing clinical and consumer genomic services, moderated by Vivek Mittal, Ph.D., Partner, Health Advances LLC, with representatives from Helix, MindStrong Health, and Canaan Partners, speakers agreed that companies must secure and protect the privacy of patient data, as well as help establish the clinical utility of reported variants in order to drive progress in consumer-directed personalized medicine. Panelists commended FDA for being progressive and trying to keep pace with innovation, particularly as it relates to digital health.

A New Age of Personalized Medicine

As PMC President Edward Abrahams, Ph.D., contended in his opening remarks, “one-size-fits-all medicine has taken us about as far as it can.” Recent innovations in multi-stakeholder collaborations, data science, and regulation discussed at this year’s BIO Convention will continue to help usher in a new age of personalized medicine that brings greater health system efficiency and, most importantly, better outcomes for patients.

PMC would like to thank all of the speakers for their contributions to the track, many of whom are PMC members (in bold above). The complete track agenda can be downloaded here.