by Lindsay Stephens, Senior Operations Coordinator, Personalized Medicine Coalition

In his opening remarks during the 2019 Biotechnology Innovation Organization (BIO) International Convention’s “Personalized Medicine & Diagnostics Track,” which was co-hosted by BIO and the Personalized Medicine Coalition (PMC) from June 3 – 6, PMC Senior Vice President of Science Policy Dr. Daryl Pritchard contended that “the case for personalized medicine has been made.”

As evidence to support his claim, Dr. Pritchard noted that PMC classified 42 percent of the new drugs the U.S. Food and Drug Administration (FDA) approved in 2018 as personalized therapies.

The field’s champions, he suggested, are turning their attention to an even more important problem: how to integrate personalized medicine strategies into health care practice.

The track demonstrated that innovative business models and policy solutions, as well as the development of evidence showing the clinical utility and economic value of diagnostic testing and targeted therapies, will be instrumental to clinical adoption.

Three themes emerged.

1. Partnering and innovative collaborations across stakeholder groups are crucial for developing an ecosystem that brings diagnostic tests and personalized treatments to patients faster and at a reasonable cost.

Panelists across sessions agreed that tackling clinical adoption challenges will necessitate collaborating with other stakeholders. “Nobody can do this by themselves,” Art Swanson, Vice President, Genomics Product Management, Optum, argued during a session titled “Innovative Models for Real-World Evidence: Creating Value-Based Precision Health.”

Aurélie Deleforge, Associate Consultant, Bionest Partners, added during “Precision Medicine Beyond Oncology” that pharmaceutical companies and diagnostic companies, along with payers, providers, and patient groups, are necessary partners because access to diagnostic tests is a prerequisite to access to personalized treatments. She predicted that these types of partnerships will become more prominent as stakeholders continue conversations about how they can mutually benefit each other and how their partnerships can drive health care decisions that result in better patient outcomes.

Arguably the most needed partnership right now is with payers. During a session on the “Clinical and Economic Value of Personalized Cancer Treatment,” Ammar Qadan, Vice President, Global Head of Market Access, Illumina, acknowledged that without payer recognition of the value of next-generation sequencing (NGS), the technology will go nowhere. Qadan and Dr. Michael Sherman, Chief Medical Officer of Harvard Pilgrim Health Care, highlighted a partnership between Harvard Pilgrim and Illumina that is designed to facilitate improved access to genomic testing for previously non-covered individuals.

2. Health care decision-makers require evidence of the utility of personalized medicine. Large amounts of real-world and clinical data exist, but it’s still unclear how to unlock the power of this data to build the evidence that will result in more widespread patient access.

During “From Diagnostics to Treatment and Back Again in the Digital Era,” Kristen Pothier, Global Head of Life Sciences Strategy, Ernst & Young Parthenon, summed up issues that were raised throughout the track sessions by asking how we can “take the best data and put it to work.” With many datasets available, the opportunity is there, but as Elizabeth Donley, CEO, NeuroPointDX, mentioned in “Precision Medicine Beyond Oncology,” so much data makes it “difficult to draw meaningful conclusions” and “get your hands around it all.”

Another concern — How do we involve consumers in data collection and storage while convincing them that their data are in good hands? Art Swanson mentioned that some data are “ripe for misuse,” and as we begin looking for ways to convert this information into real-world evidence, it is important that the “consumer [has] confidence, trust and control over data.”

3. To remain innovative, policies will need to be flexible to keep up with new technologies and treatments.

With personalized medicine technology and treatments developing rapidly, it is important that regulatory and reimbursement policies keep up so as to ensure appropriate access and continued innovation. As Elizabeth Hillebrenner, Associate Director for Scientific and Regulatory Programs at FDA’s Center for Devices and Radiological Health, explained during “Precision Medicine and the FDA: Prospects for Regulation of Laboratory-Developed Tests (LDTs),” FDA is working with Congress to propose new regulatory oversight processes that proponents believe will “level the playing field” for LDTs and in vitro diagnostics (IVDs) to help assure safety and reliability while providing an environment that will allow for tests to rapidly come to market.

During “Coverage for Advanced Diagnostics and the Impact on Patient Access to Personalized Medicine,” panelists discussed how the Centers for Medicare and Medicaid Services (CMS) re-opened a National Coverage Decision (NCD) applicable to NGS-based tests amidst concerns over language that could be interpreted to mean non-coverage of germline testing. PMC Senior Vice President of Public Policy Cynthia A. Bens remarked that CMS’ decision to reconsider the policy demonstrates the importance of policy and science working together. Bens hopes all government agencies will continue to demonstrate such flexibility as health care evolves toward personalized medicine.

The Way Forward

These conversations underline that the field is filled with promise and dedicated leaders. To accelerate progress, personalized medicine’s advocates are shifting attention to implementation challenges, building evidence of value, innovative business models, and flexible policy solutions that can align health care toward a future of better patient outcomes and a more efficient health system.