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INTRODUCTION AND OVERVIEW

In response to a growing national awareness that the development and use of new diagnostic, therapeutic, and preventive interventions was occurring at a pace that both quickened and outstripped the evidence necessary to make informed decisions about the comparative advantages of different interventions, 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 legislative 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 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. Recognizing early on that suitable methods for patient-centered CER warranted a deeper evidence base, as well as standards that ensure methodological rigor, PCORI has also made commensurate investments in projects designed to improve the science and methods of patient-centered clinical research. Another element of PCORI’s unique contribution to the research landscape since its inception was the establishment of PCORnet^®, the National Patient-Centered Clinical Research Network, in 2013. PCORnet^® is designed as a collaborative resource to accelerate research by leveraging real-world clinical and administrative data from health systems and payers, along with patient-generated data.

Based on important contributions completed and in progress—including research findings, a new paradigm for patient-engaged research, and a national research infrastructure, PCORI was reauthorized by Congress in 2019, adding two notable elements in the new statute. First, the 2019 amending legislation called for additional research priorities and prescribed that “such priorities should reflect a balance between long-term priorities and short-term priorities and be responsive to changes in medical evidence and in health care treatments.” In the process of engaging these additional responsibilities, PCORI’s leadership and Board of Governors has undertaken a strategic evolution from national priorities for research to national priorities for health that will guide its funding of research and other activities, emphasizing the desired impact on health and health care.

Second, the legislation expanded PCORI’s authority to study “the full range of clinical and patient-centered outcomes … [including] … the potential burdens and economic impacts.” The ability to study this broader constellation of direct and indirect economic impacts, ranging from out-of-pocket costs to productivity to health care utilization, positions PCORI to fund new research that can inform the value of health and health care from the perspective of patients and families.

The reauthorization provides a springboard for development of PCORI’s next phase, including development of its national priorities for health, its research agenda, and its strategic plan. As part of this development process and reflecting its deep commitment to broad stakeholder engagement, PCORI sought input via several forums, including its Advisory Committee meetings, internal discussions, and varied external stakeholder meetings. Insights from these forums have generated key considerations about how PCORI might maximize its impact and ability to address fundamental research topics in line with its mission and the most urgent questions in health and health care.

With this backdrop, PCORI enlisted the National Academy of Medicine (NAM) to organize and produce two virtual meetings, titled Priorities on the Health Horizon. The overarching objective of these meetings was to engage patients, clinicians, health system leaders, researchers, and other stakeholders from the broader health community to identify and discuss high-priority emerging issues in health, health care, and biomedical science and technology. This engagement leveraged the NAM’s deep experience in convening experts on matters of significant national importance, including its long-standing thought leadership role in the realization of a learning health system, its recent work conducting assessments of the impact of COVID-19 on nine health-related sectors, and cross-cutting analyses of the most compelling system-wide priorities. With assistance from PCORI, the NAM identified participants for each meeting, including patients, researchers, clinicians, policy makers, and purchasers. A multistakeholder

work group, comprising meeting participants, was established by NAM to co-develop this publication.

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 presentations on four broad topics: (1) technologies; (2) social and environmental factors; (3) optimizing value; and (4) infrastructure. Each topic was chosen intentionally for its ability to encompass several interrelated concepts (see Box 1). The same topics 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 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. The NAM summarized highlights of both meetings for presentation to PCORI’s Board of Governors and senior leadership, as background to their ongoing strategic planning discussions. Discussion highlights from each macro topic are synthesized here, including the broad stage-setting presentations that took place on March 15 and key points from the

breakout group discussions on March 16, along with deeper topical examinations from the April 27 meeting.

At the March meeting, each breakout group used the following questions to guide the conversation:

1. What are the additional concepts or topics to consider as part of this topic?
2. What are the potential disruptors, opportunities, and key trends on the horizon related to this topic?
3. What are the major obstacles or challenges to advancing progress related to this topic?
4. What else would it take for this topic to have a measurable and positive impact on health/health care in the next 5 years?

Similarly, at the April 27 meeting, the participants focused on two key questions that were designed to build on the discussions at the March meeting:

1. What will it take to create a patient-centered learning health system (with respect to infrastructure, technologies, incentives, and engagement)?
2. How can PCORI use its research strategies, unique role, and activities to improve patient experience, outcomes, and value in health and health care?

A synopsis of each broad topic is presented in the following chapters, followed by cross-cutting themes and insights for PCORI’s national priorities and the field at large.