Personalized Health Care & Prevention Stakeholder Engagement Workshop
February 25, 2015

Executive Summary

On February 25, 2015, the National Institutes of Health (NIH) Health Economics Common Fund Program convened a workshop to engage a range of stakeholders in discussions about how NIH-funded research can enhance the role of personalized medicine in improving the efficiency and effectiveness of health care. The goal of the workshop was to facilitate dialogue among researchers, stakeholders, and NIH staff to (1) inform stakeholders of ongoing NIH-funded research initiatives and (2) help researchers focus on questions of critical value to stakeholders.

Participants included invited panelists representing diverse stakeholder groups, investigators leading cooperative agreements funded under the auspices of the Common Fund program on Determinants and Consequences of Personalized Health Care and Prevention, and NIH staff. The invited panelists represented five broad stakeholder groups: (1) health care providers, (2) patient advocates, (3) guidelines organizations, (4) insurers, payers, and health technology assessment organizations, and (5) pharmaceutical and diagnostic developers, manufacturers, and regulators.

The workshop began with brief presentations summarizing the ongoing cooperative agreement research projects. Each project seeks to understand and identify strategies to maximize the potential value of genomic technology and other personalized medicine approaches. The projects utilize analyses of large data sets, multiple modeling and simulation techniques, cost-effectiveness and value-of-information analyses, and/or surveys to achieve their aims. The project investigators expressed a desire to produce tools and information that are useful to stakeholders. Panel presentations and discussions followed, during which each invited panelist shared his or her perspective on the research and clinical practice needs of the field. An overview of the recently launched Precision Medicine Initiative (PMI) was also included in the agenda.

Health Care Providers

The first panel highlighted the perspectives of health care providers. A major theme was the challenge posed by the rapid growth in genomic testing to clinical decision making at both the provider and system levels.

• Although genomic tests have great potential to inform diagnoses and treatment decisions, many tests currently lack evidence to clearly support clinical decisions.
• There are outstanding research needs as well as a need to educate both patients and providers about appropriate interpretation of genomic data and genetic risk factors of disease.
• The panelists also discussed a systems-level tension between the desire to keep up with the pace of technological development and the need to base decisions on sound evidence, which takes time to produce.
• New economic tools are required to support pragmatic decision making in an evidence-poor environment.

Patient Advocates

The second panel elicited insights from patient advocates on the importance of personalized medicine from the patient perspective.

• Patients have diverse preferences that influence their health care choices, including heterogeneous perceptions of risk and anxiety and different definitions of quality of life.
• Truly personalized medicine will consider these types of individual preferences.
• Patients have a hunger for information, and there is a need for greater education about risk and the distinction between clinically useful information and data that are not clinically useful or meaningful.
• Participants noted a potential tension between the preferences of individual patients and optimizing public health.

Guidelines

The third panel comprised representatives of organizations that issue formal clinical guidelines and recommendations.

• Clinical guidelines are beginning to incorporate aspects of personalized medicine, especially genomic tests and biomarkers, as the evidence base for clinical use grows.
• Participants agreed that information on cost-effectiveness could affect guidelines, provided the information is current, reliable, reproducible, and efficiently produced.

Payers

The fourth panel considered how payers view the challenges and opportunities posed by personalized medicine. Panelists included representatives of both government and private payers. There are substantial differences between public and private payment systems in the factors affecting their coverage decisions. Public payments are determined by statutes and regulations, whereas evidence of clinical utility is the primary criterion for coverage by private insurers.

• As new personalized medicine tests and clinical evidence emerge, payers and providers will need new tools to manage, interpret, and make decisions based on a plethora of new information.
• New incentives will also be needed to better align practice with emerging evidence.

Industry

The final panel presented perspectives from representatives of the pharmaceutical and diagnostic industries and of the Food and Drug Administration (FDA). The presentations identified two significant challenges facing the development of new personalized medicines: knowledge gaps in basic science and misaligned economic incentives for innovation. Regulatory approval of new treatments and diagnostics depends on the existence of sound evidence.

• Although the FDA has demonstrated an ability to quickly approve molecularly targeted therapies, targeted therapies are dependent on how well the science is understood—in particular the molecular and genetic causes and pathways of the disease.
• Panelists noted that the current pricing and reimbursement landscape poorly incentivizes industry to develop new personalized medicine technologies.
• Value-based pricing schemes and combined reimbursements for treatments and companion diagnostics would help promote innovation.

The workshop concluded with a summary discussion in which participants reflected on the themes raised throughout the meeting and suggested possible paths forward. In order to increase the relevance of research on the economics of personalized medicine for decision makers, participants suggested the following:

• Generate evidence on the use and impact of precision medicine technologies to inform economic assessments.
• Prioritize research investments to generate evidence.
• Present research results and cost-effectiveness measures in multiple formats.
• Develop transparent models that stakeholders could adjust to their needs.
• Encourage communication between the research and health care delivery communities.