EXECUTIVE SUMMARY

In response to a growing national awareness that the development and use of new diagnostic, therapeutic, and preventive interventions had been occurring at a quickening pace—one far outstripping the evidence necessary to make informed decisions about their comparative advantage—the Patient-Centered Outcomes Research Institute (PCORI) was established in 2010 as part of the Patient Protection and Affordable Care Act legislation. Geared to helping patients, families, clinicians, and other health care stakeholders make better informed health care decisions and improve care and outcomes, PCORI’s initial mandate was to “identify national priorities for research, taking into account factors of disease…, gaps in evidence, practice variations and health disparities…, [and] the potential for new evidence” (PCORI, 2021). PCORI began funding comparative clinical effectiveness research (CER) in 2012 and, since then, has become a critical part of the U.S. research ecosystem, funding a substantial and growing portfolio of patient-centered outcomes research. With its portfolio, PCORI has crafted a new paradigm for engaging patients and stakeholders in the design, development, delivery, dissemination, and implementation of research findings on a wide array of topics. It has also focused on stimulating the development of new infrastructure imperative to facilitating the conduct of that research more quickly, in expanded settings and networks, and with a broader range of applicability.

In 2019, PCORI was reauthorized by Congress, adding two notable elements to the new statutory language. First, the 2019 amending legislation called for additional research priorities and prescribed that the research “reflect a balance between long-term priorities and short-term priorities, and [be] responsive to changes in medical evidence and in health care treatments.” Second, PCORI received expanded authority to study “the full range of clinical and patient-centered outcomes [including] … the potential burdens and economic impacts,” which positions it to fund new research that can inform the value of health and

health care from the perspective of patients and families (U.S. Congress, 2019). The reauthorization provides a springboard for development of PCORI’s next phase, currently being envisioned through a broad strategic planning initiative.

As part of this development process, and reflecting its deep commitment to broad stakeholder engagement, PCORI reached out to various stakeholder groups for input and enlisted the National Academy of Medicine (NAM) to organize and produce virtual meetings, which were titled Priorities on the Health Horizon. The overarching objective of the NAM meetings was to engage patients, clinicians, health system leaders, researchers, purchasers, and other stakeholders from the broader health community in identifying high-priority emerging issues on the health horizon. The key insights and themes from the meetings were summarized by the NAM organizers, with the guidance of a representative workgroup comprised of meeting participants.

Two particular recurring features of the conversations merit underscoring at the outset, one at the center of PCORI’s mission and vision, and a second that will directly affect its prospects for success. As to the first, the discussions reflected the broad concurrence that health care has become increasingly complex given tremendous advances in medical science, and until patients and families are at the center of all aspects of the health care delivery system and related research, the system will fall far short of the moral imperative to improve health for all communities. Their perspectives and guidance are critical to inform the reorientation of the business of health care; deploy effective, affordable, and efficient practices; steward the collection and use of data to improve care; and ensure equity for all. The second was the related and recurrent observation that the nation’s health system is built on a broken chassis of fee-for-service payment. As a result, progress in capturing the opportunities for research findings to catalyze improvements in the system’s effectiveness, efficiency, and equity faces a daunting barrier at the outset. The sense of the discussions was that the public’s interest will remain thwarted until a new chassis is built to replace what currently exists and that this is a potential area of opportunity for PCORI to consider research efforts focused on how emerging payment models may affect health care quality and health equity.

MEETING STRUCTURE

The first meeting of 40 invited participants on March 15–16, 2021 (https://nam.edu/event/priorities-on-the-health-horizon-informing-pcoris-strategic-plan-webinar), was designed to engage in “blue sky” thinking about emerging trends, priorities, and opportunities in health and health care. Day 1 was anchored by four macro topics: (1) emerging technologies; (2) social and environmental

factors; (3) optimizing value; and (4) infrastructure. (see Box 1). These broad topics were purposely selected to stimulate a wide-ranging discussion and were then discussed in smaller breakout groups on Day 2. NAM staff prepared topic briefs to provide context for each topic and identify potential research questions (see Appendix A). The second meeting of 25 invited participants on April 27, 2021 (https://nam.edu/event/priorities-on-the-health-horizon-informing-pcoris-strategic-plan-meeting-two) was designed to consider two topics that were of particular priority for deeper discussion based on the initial meeting: (1) development of a patient-centered learning health system, and (2) how PCORI could use its unique mission, capabilities, and core activities to improve patient experience, outcomes, and value in health and health care. Both meetings were chaired by Neil R. Powe, M.D., M.P.H., M.B.A., from the University of California, San Francisco, and Zuckerberg San Francisco General Hospital, and were open to the public.

MEETING INSIGHTS

The critical discussion highlights from the two meetings identified four cross-cutting themes that are especially important to informing and shaping PCORI’s opportunities and priorities over the next decade (see Box 2). Foremost is the imperative for strategies that will advance health equity and dismantle the structural racism that contributes so greatly to health inequities. Because of the magnitude of the related disparities, the structural factors that affect health status demand dedicated, well-designed research activities. Untangling how clinical factors and social determinants of health work alone or in combination to reduce

or exacerbate inequities was viewed by meeting participants as an essential area for PCORI to prioritize. An important consideration that resonated throughout the April meeting was the need to differentiate and examine both health equity and health care equity, given that they are multifaceted and intersecting but with distinct knowledge gaps and research questions.

A second theme was the apparent value disconnect—that is, the gap between perceptions of value in the economic sense and values in the moral, cultural, or personal sense. The disconnect between the current structure, financing, and organization of health care, and what patients, families, and communities need and value is increasingly acknowledged as a key driver of this disconnect. Timely access to responsive, affordable, high-quality, person-centered health care is essential, yet the experience for many is a health system that is fragmented, uncoordinated, expensive, inequitable, and of uneven quality. Meeting participants concurred with the fact that health care in the United States has been constructed on a “broken chassis” of fee-for-service payment, which is unable to deliver the results needed by the nation on most important performance dimensions. The fundamental need to build a new chassis is in the public’s interest, rather than trying to fix what currently exists.

A third theme that cut across the discussions related to the need for an agile learning health system—one in which the alignment of evidence, informatics, incentives, and culture naturally improves and accelerates advances in health system effectiveness, efficiency, equity, and continuous learning. Given immense data capabilities, proliferation of potential sources of evidence, and rapid advances in technologies, there is a compelling need to be more nimble and coordinated in the

ability to learn from every patient and family and embed evidence into health care at every opportunity. Thus, an important element of the learning health system relevant to PCORI’s core capabilities is the relationship between the data infrastructure supporting health and health care, and that supporting health research—inclusive of real-world data, common data models, and standards. The experience of the National Patient-Centered Clinical Research Network (PCORnet^®) with consolidating data models, ensuring data completeness, and applying insights from coronavirus disease 2019 (COVID-19) research can enable a more coherent, aligned approach to moving data from electronic health records (EHRs) to research and then back into practice.

A final cross-cutting theme related to the need for a better understanding of the heterogeneous impacts of emerging technologies on patients, families, and communities. Technologies encompass cutting-edge medical and diagnostic therapies and digital innovations supporting health and care delivery, data use, and connectivity. Better understanding of their myriad effects and interplay will help create a more complete view of a person’s entire experience and predispositions related to individual circumstances (from genetics to social needs). This could inform population health as well as reduce disparities. An observation that permeated both meetings was that the full potential of precision medicine, informed by predictive analytics, can be realized only if equity is its cornerstone.

Given the scope of the issues in the four domains considered during the Priorities on the Health Horizon meetings—emerging technologies, social and environmental factors, optimizing value, and infrastructure—a formidable set of pressing health and health care research needs were identified and discussed. In addition, certain fundamental strategic priorities emerged as basic and critical to progress in the field: (1) the need to reorient research perspectives and activities to patient and family priorities and values, and in particular those conditions that drive inequities; (2) the need to foster strategic learning partnerships across groups, organizations, communities, and sectors; and (3) the need to build the continuous learning infrastructure to produce new insights at the pace and scale necessary for health and health care improvement.

These three strategic priorities for the field align well with PCORI’s adopted National Priorities for Health (see Box 3), which were released in June 2021 with an invitation for broad public comment. The adopted National Priorities orient PCORI’s enhanced strategic emphasis on learning what works best for improving people’s health and the health of the nation; stewarding the development of the infrastructure capacity to broaden and accelerate that learning process; quickening the pace at which lessons learned are disseminated and put into practice; reversing the persistent health inequities in the nation; and, through these efforts,

accelerating health system transformation from one that is often too fragmented and inefficient to one that is integrated and continuously learning.

In advancing these priorities, PCORI is signaling a bold commitment to deepening the systematic approach to ensuring that better, more reliable information is available to guide personal, community, clinical, organizational, and policy decisions to improve health and health care. PCORI further recognizes the importance of taking on a more facilitative role that draws broader public and private stakeholders to help mobilize synergy into building the aggregate strategy and infrastructure. Certainly, no single organization can meet the ever-increasing need to improve decisions central to health and health care delivery and outcomes. Given the size and complexity of these challenges, the imperative is for system participants to work together as seamlessly as possible to build the aggregate capacity to continuously improve learning and sharing throughout the system. PCORI’s commitment to the themes of marshaling, connecting, integrating, and accelerating is a strong and promising step toward evidence-driven, equitable health in the decade ahead.