Stakeholders are increasingly placing a greater emphasis on the need to understand and define value in healthcare to drive improvements in decision-making. Evidence-based medicine holds the potential to enhance the quality of care and improve patients’ outcomes, but the diversity and complexity of the US healthcare system has created challenges in developing the targeted outputs that can inform clinical shared decision-making.

To address these challenges, PCORI was established under the Affordable Care Act in 2010 to coordinate and fund patient-centered, comparative effectiveness research (CER) in healthcare. Reauthorized by Congress in 2019 for another 10 years, PCORI now has a new mandate to consider the full range of patient outcomes. By design, PCORI does not conduct cost-effectiveness studies and instead is charged with funding and disseminating research that compares the benefits and risks of clinical interventions in real world settings. The institute awards 2 types of projects: broad funding awards, which are investigator-initiated research proposals on topics that respond to PCORI’s national priorities and research agenda, and targeted awards, which are applications on specific, high-impact topics that are generated through a multi-stakeholder process.

Since its inception, PCORI’s role has evolved in response to both internal and external factors, but its methods for both interventional and observational studies comparing 2 or more active interventions in realistic clinical settings have remained its primary focus. PCORI’s longstanding framework for funding research included assessment of care options, improving healthcare systems, dissemination of research outputs, tackling health disparities, and accelerating both patient-centered outcomes research and methodological research. Its other activities include building data infrastructure, improving research methods, and fostering broader stakeholder engagement in the research process by including patients, caregivers, and clinicians.

However, in response to changes in the US healthcare system and its recent reauthorization under new leadership, PCORI has selected a new set of proposed principles that will act as the foundation for its research agenda going forward. PCORI will accept stakeholder input beginning on June 28 for a period of 60 days. Their new proposed principles include:

1. Increasing evidence for existing interventions and emerging innovations
2. Enhancing infrastructure to accelerate PCOR through real-world data, patient and community engagement, and collaboration
3. Advancing the science of dissemination, implementation, and health communication between patients, clinicians, and stakeholders
4. Achieving health equity with an emphasis on research partnerships
5. Accelerating progress toward an integrated health learning system

The evolving drug pricing debate increasingly highlights concerns about gaps in medical evidence on the clinical effectiveness and relative value of innovation. A better understanding of PCORI’s evolution, research to date, and its new undertakings could position it for an increased role in clinical decision-making and policy. The institute’s investments in studying patient-reported outcomes, real-world resource utilization, and effectiveness that goes beyond clinical trial safety and efficacy data could be a way for stakeholders to inform decision-making and improve care value and affordability.

Consequently, there are 4 major reasons why PCORI’s role in healthcare decision-making could increase based on their proposed research agenda focused on patient-centered health:

1. PCORI makes significant investments in CER in the US, with an increasing focus on medical interventions, including drugs

Since its inception, the Institute has awarded over $2.7 billion to fund a range of patient-centered outcomes research and infrastructure-building activities. PCORI’s research portfolio is slated to further expand as the volume of completed research has increased rapidly in recent years, positioning PCORI to scale up its research dissemination activities and drive clinical implementation of its findings.

To understand PCORI’s continued evolution and investments in CER, Avalere analyzed PCORI’s more recent research funding allocations awarded between March 2019 and September 2020.

• The analysis found that, in just over a year, PCORI directed roughly $259 million for CER across 55 grants. This represents almost 80% of funded projects.
• Historically, a large share of the institute’s awards focused on health systems interventions that affect various aspects of care delivery but in recent years have increasingly shifted to medical interventions such as drugs, devices, and procedures. For the period between March 2019 and September 2020, Avalere found that allocations for research on medical interventions represented 51% of broad funding awards (also known as investigator-led applications) and 67% of targeted awards.
• Avalere also found that since 2019, CER on medical interventions have focused equally on both drug and non-drug studies. For comparison, a previous analysis showed that between 2016 and 2019 only 18% of medical intervention comparative effectiveness studies included drugs.

These findings suggest that PCORI is continuing to make robust investments in comparative effectiveness studies. As the institute’s creation of evidence on both new and existing healthcare interventions, use of real world evidence, and dissemination activities continues under its updated priorities, policymakers or other government agencies may look to PCORI as a reliable source of clinical evidence that values patient perspectives.

2. Since PCORI’s 2019 reauthorization, the organization is taking a patient-centered approach to considerations of the economic impact of treatments and services on patients

In 2019, Congress passed legislation to extend PCORI’s funding through 2029. This reauthorization outlines new priorities, including research on maternal morbidity and mortality, care for those with intellectual and developmental disabilities, and consideration of the economic impact of various treatments and services on patients.

Given standing concerns over care rationing, PCORI has been explicitly prohibited from supporting studies that conduct cost-effectiveness analysis. It also does not fund studies for which cost and economic impacts are the primary outcome. However, since the institute’s reauthorization, it has finalized their “Principles for the Consideration of the Full Range of Outcomes Data in PCORI-Funded Research” to guide future research on the full range of outcomes, including economic data and limited economic analyses under their new congressional mandate. This broader scope intends to take into account patient and family burdens (e.g., out-of-pocket costs, time costs) as part of secondary outcomes and to consider a range of factors, including treatment costs, insurance coverage, and benefit design. This patient-centered approach to economic considerations may increase the relevance for PCORI outputs in shared decision-making.

3. PCORI has been a pioneer in increasing the role of patients in research and thereby increasing stakeholder buy-in and support

PCORI is unique among US healthcare entities and large clinical research funders in the degree to which it requires patient engagement at all stages—from study design to output dissemination. As a result, the institute has the potential to play a meaningful role in capturing the full range of treatment benefits that are relevant to the patient and consumer perspective. Given that PCORI’s CER projects are informed by both scientific evidence and the lived experience of patients, the evidence generated from funded research could be very useful in real-world decision-making.

4. PCORI’s completed research and proposed national priorities position it to expand its work addressing health disparities and health equity

Given the growing focus on equity in the US healthcare system, PCORI’s funding of patient-centered outcomes research could be used to inform efforts to reduce disparities. Addressing disparities is part of both PCORI’s current and proposed National Priorities and Research Agenda and is in line with the vision of PCORI’s new executive director, Nakela Cook, who has stated publicly that addressing health equity and maternal health disparities are priorities for the organization. Part of their framework for funding research focuses on identifying disparities in prevention, diagnosis, and treatment effectiveness across populations such as those in rural areas and racial or ethnic minorities. PCORI has funded 105 CER studies related to improving health disparities as of March 2021, which aligns with Avalere’s findings that 17% of broad funding has been dedicated to addressing health disparities since the institute’s inception. Moving forward, the organization’s focus on engaging patients, families, and communities could produce outputs that inform meaningful changes based on their commitment to answering research questions that evaluate complex and interrelated factors that contribute to health disparities.

Download Avalere analysis of PCORI’s recent research funding allocations.

Methodology

Avalere analyzed PCORI’s publicly available grant funding data from pcori.org and classified projects based on definitions provided by “Research We Support” and PCORI’s annual reports. Avalere consulted PCORI’s website and annual reports on funding opportunities to understand how PCORI defined research categories. Funding projects not listed as research projects or methodological research were classified as general infrastructure, primarily composed of engagement award projects and PCORnet Clinical Data Research Networks. Non-drug interventions categorized as “other” included a range of other care approaches (e.g., non-medical interventions such as acupuncture) that did not fall within screening and diagnostic tests, devices and procedures, or behavioral health.

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