2023 PCORI Annual Meeting

Patient engagement is more than a phrase; it is essential to effective treatment and care. In this one-on-one interview, hear the captivating stories of two patient advocates, Suzanne "Suz" Schrandt, JD, founder and CEO of ExPPect, and Catherine Ames, author of the Daily Trojan’s "Chronically Catherine" column and a recent graduate of University of Southern California, as they discuss how patient engagement has evolved, examine recent shifts within the patient engagement space, and share ways to actively support patient-centered care.

Twenty years have passed since the National Academy of Medicine released its report, Unequal Treatment: Confronting Racial and Ethnic Disparities in Health Care. While widespread recognition of disparities has increased, these disparities continue to persist. There are numerous sources of bias that affect whether people from traditionally underserved communities, including racial/ethnic populations, persons with disabilities, LGBTQIA individuals, and others, receive the appropriate treatment and care. Addressing discrimination at a health systems level is essential for achieving health equity and improving health outcomes for all.

This session explored:

• Evidence for health systems interventions designed to address structural discrimination.
• Development and use of such interventions within institutions, highlighted by health system representatives and patient advisors.
• Practical lessons learned from creating and implementing these interventions.
• Methodological challenges of conducting patient-centered comparative clinical effectiveness research (CER) related to systems interventions.

Comparing dissemination, implementation and health communication (DIHC) strategies for increasing the uptake and sustainment of evidence-based practices, or de-implementation of harmful or outdated practices, is integral in identifying the best ways to get evidence into the hands of patients, caregivers, providers, and relevant stakeholders to enable them to make the most informed decisions about healthcare. But once the best strategies are identified through comparative clinical effectiveness research (CER), how are they scaled up and out broadly to make the greatest impact and improve outcomes?

This session reviewed the aims of CER in DIHC and spotlight researchers who have received dissemination and implementation awards to promote awareness of their findings from PCORI-funded research and to facilitate the uptake of these findings into practice settings. A panel of researchers and patient partners presented an overview of their DIHC CER study, results and impact, then discussed best practices for facilitating uptake of results widely and broadly, beyond the health systems or centers that participated in the CER study.

In response to the COVID-19 pandemic, PCORI invested in an array of targeted studies, enhancements to existing studies, and other evidence products addressing telehealth modalities and the role they have in health care delivery—both in the immediate context of the COVID-19 pandemic and more broadly as the healthcare system adjusts to a "new normal" post-pandemic. This session highlighted several of these studies and products, with a focus on leveraging telehealth to facilitate equitable access to and delivery of quality care, as well as future directions for telehealth-related research post-pandemic.

PCORI is creating synergies among the data, technology, methods, and researchers, patients and other stakeholders need to transform health research. There is a critical need to conduct research at a large scale, faster and more efficiently—and to accelerate the participation of populations historically underserved or underrepresented in health research. PCORI has invested in PCORnet^®, a national research network, to meet these needs by generating real-world evidence through health data collected in everyday care encounters and settings. These data represent 30 million patients receiving care at more than 40 health systems, each with dozens of practice sites, reflecting the full diversity of the U.S. population. Session speakers highlighted the growth and contributions of PCORnet over the last decade and discussed how PCORnet is poised to continue to support national-scale research to speed evidence generation.

PCORI-funded studies require meaningful engagement throughout the research process and PCORI-funded engagement awards focus on building the capacity and convenings to foster an infrastructure that allows for that meaningful stakeholder engagement. This session offered illustrative experiences and learnings for how meaningful engagement can be done, as well as reviewed the library of free PCORI materials and resources to support these efforts.

Mental health issues are the most common complication during the perinatal period. At least one in five women will experience mental health issues during this time, with some populations being more at risk than others. Not only does this cause significant morbidity, but it is also one of the most frequent underlying causes of pregnancy-related death, accounting for more than 22 percent of maternal deaths according to the most recent Centers for Disease Control and Prevention data. There is wide consensus that repeated screening should take place both during pregnancy and in the first year postpartum. However, recent data from the Healthcare Effectiveness Data and Information Set showed 2021 screening rates during pregnancy were only 16 percent and nine percent for Medicaid patients and private insurance patients, respectively, and 17 percent and 11 percent in the postpartum period. In addition, it is estimated that fewer than 20 percent of those with mental health issues are treated for it, although numerous effective treatment methods are available. In this breakout session, attendees gained a deeper understanding of the scope of this issue, significant barriers for screening and treatment, possible solutions and options for comparative clinical effectiveness research.

About 21 million people in the United States exhibit long COVID symptoms resulting in reduced quality of life, diminished productivity, and further exacerbating health disparities. This session highlighted ways stakeholder engagement is driving patient-centered long COVID care, including innovative ways patients have been engaged in research, emerging therapies and symptom management strategies, as well as the impact of novel health system models and care delivery methods to address long COVID. In addition to discussions that drew insights from two current ongoing/recently completed PCORI-funded projects identifying promising areas of future research, panel discussions included both patient and provider perspectives on the management of long COVID symptoms.

This session was an opportunity for prospective applicants and those with existing PCORI awards to submit questions to PCORI program staff and funded investigators about PCORI-funded research and the application process. This session included PCORI-funded investigators discussing their experience in applying for funding and a live, moderated question-and-answer session where audience members can ask questions or submit questions to the moderator.

PCORI is committed to long-term funding of patient-centered research in intellectual and developmental disabilities (IDD). Individuals with IDD are disproportionately impacted by high disease burden and tremendous barriers to care, especially as it relates to mental health and wellness. This session highlighted the spectrum of PCORI’s funded research—from engagement to comparative clinical effectiveness research to dissemination and implementation—aimed at improving mental health in individuals with IDD and the vital role of patient partners in this work. The session included presentations from PCORI-funded investigators and patient partners, followed by a moderated discussion and audience Q&A.

Rare disease is a long-standing focus for PCORI. Recently, PCORI acknowledged rare disease as one of twelve Topic Themes of focus for future investment. The absence of approved treatments for many rare diseases makes comparative clinical effectiveness research of head-to-head treatment options a challenge. This session highlighted PCORI-funded rare disease comparative clinical effectiveness research studies (with one example leveraging PCORnet^®, The National Patient-Centered Clinical Research Network) and engagement awards, and concluded with an equity-focused roundtable discussion of future directions for rare disease research and engagement.

Take a deep dive into the past, present and future of healthcare research with two prominent figures in the field. Join Nakela L. Cook, M.D., MPH, PCORI Executive Director, and Vivian W. Pinn, M.D., Founding Director (Retired) of the Office of Research on Women’s Health at the National Institutes of Health for a one-on-one discussion about the history of publicly funded healthcare research, PCORI’s current and future role within the field, and their shared experiences as physicians and research leaders.

Achieving health equity is one of PCORI’s National Priorities for Health. A key component of health equity research is social determinants of health (SDOH) data, which capture context and concepts including social, economic, physical infrastructure, and education. These data can be both self-reported by patients (e.g., employment, housing, food availability), and population-level measures (e.g., community safety, environmental conditions). While it is widely recognized by healthcare systems, community organizations, researchers, and other stakeholders that these data are essential for understanding and addressing healthcare inequities, the routine collection, standardization, and use of these data is not yet realized. This session highlighted PCORI-funded work that supports comparative clinical effectiveness research and patient-centered outcomes research, methods, and research infrastructure related to SDOH data needed to advance health equity.

Heart disease and stroke cause one in three deaths among women in the United States each year. Life events such as pregnancy and menopause, as well as psychosocial factors and fragmented care, may increase women’s risk for cardiovascular disease. However, women may be unaware of their risk and are less likely than men to undergo non-invasive diagnostic testing at the initial point of care. This results in delayed diagnosis, delayed therapeutic interventions and poorer results and outcomes. Further, despite the significant disease burden and gaps in care, women are still underrepresented in clinical research focused on heart disease and stroke.

In this session panelists:

• Described recent evidence on how pregnancy, the menopause transition, and psychosocial factors, including stress and interpersonal violence, impact cardiovascular risk in women.
• Learned how PCORI-funded researchers are testing innovative patient and community-centered care models to improve heart health for birthing people.
• Discussed gaps in care and opportunities for future comparative effectiveness research to improve women’s cardiovascular health across the life course.

This session provided an overview of PCORI’s investment and stakeholder engagement in post-traumatic stress disorder (PTSD), an important topic affecting a large number of people living in the U.S. Three projects were highlighted.

Each aim to:

• Build capacity for research to generate new PTSD-related evidence.
• Disseminate targeted PTSD Evidence Updates to those who can use the findings.
• Implement PCORI-funded PTSD findings in large, diverse healthcare systems

Discussions highlighted how each project leverages their patient and stakeholder partners in unique ways to target distinct audiences. Together, these projects exemplify how PCORI can help facilitate the path from research and evidence generation to dissemination and sustainable uptake.

The plenary featured two Dissemination and Implementation Program awardees, each of whom has developed thoughtful strategies for promoting the uptake of evidence from their PCORI-funded research in practice. Their strategies take findings out of the research context, address barriers to uptake, and are designed to establish scalable models for widespread future implementation.

This session:

• Demonstrated PCORI’s commitment to, and achievements in, getting research results into practice to inform patients’ and others’ healthcare decisions.
• Demonstrated the use of implementation science principles to inform putting PCORI-funded research findings into practice settings.
• Raised awareness and understanding among PCORI-funded researchers about opportunities to extend their work, from testing the comparative effectiveness of interventions in the research setting to adapting approaches and demonstrating implementation in real-world settings.

Ableism is rooted in the assumption that people living without a disability are the ideal. Improving our knowledge of the term, disability can reduce stereotypes and stigma and improve disability inclusion in comparative clinical effectiveness research. In this session, we drew on the insights from three PCORI awardees to discuss how these considerations helped inform research design and disability inclusion in clinical trials. One project featured a Truth and Reconciliation resource on building trust between researchers and people with disabilities. Another project described efforts to create and disseminate an accessible research ethics training program that can be adopted by teams in collaboration with people with intellectual disabilities. Finally, the Disability Equity Collaborative described their efforts to provide evidence-based knowledge and practical solutions to address complex problems in disability access in healthcare.

This session presented accomplishments and lessons learned from the AHRQ-PCORI Institutional Mentored Career Development Program, which funded 11 institutions to support the training of embedded researchers to conduct patient-centered outcomes research and comparative clinical effectiveness research (CER) within learning health systems (LHS).

A recent article describes approaches developed by the 11 funded institutions to promote partnerships between scholars and health system leaders and to provide mentored. The AHRQ-PCORI Learning Health System Embedded Scientist Training and Research (LHS E-STaR) Centers program is the next phase of this work, and integrates lessons learned from the K12 award.

The session also explored the implementation of AHRQ-developed core competencies for training LHS scientists, and the approaches taken to develop LHS embedded research infrastructure, implement training and mentorship activities, strengthen partnerships among health system and community stakeholders, and the impacts on care delivery.

This session highlighted PCORI’s Foundational Expectations for Partnerships, which updates the 2014 PCORI Engagement Rubric and expands guidance for meaningful engagement to advance patient-centered outcomes research and health equity. Specifically, this interactive session provided:

• Brief introduction into why an update was needed, why now, and how the new Foundational Expectations were developed.
• Deep dive into the six new Foundational Expectations and the implications for principal investigators, study teams and patient partners.
• Opportunity to provide input on dissemination strategies for the new PCORI Foundational Expectations.

The number of people living in the United States over the age of 65 is rapidly growing and is projected to reach around 95 million by 2060. The U.S. healthcare system faces many barriers to addressing the complex care needs of older adults in the country. This session highlighted PCORI’s range of investments over the years to promote the health of older adults in the U.S including the funding of comparative clinical effectiveness research and research related projects to engage patients and the broader health and healthcare community, synthesize findings from existing research, and support the dissemination and implementation of effective interventions. PCORI-funded awardees discussed their projects and engaged the audience in an interactive discussion to identify promising directions for future work.

This half-day workshop sought to equip potential PCORI applicants with strategies for success in applying for PCORI funding and conducting PCORI-funded awards. Attendees could engage in both didactic and interactive sessions, with opportunities to meet with PCORI staff on individual and group interests, as well as hear from successfully funded PCORI awardees.

Key topics that were covered during breakout sessions, below, included understanding PCORI Funding Announcements (PFAs), developing a strong engagement plan, and developing effective Letters of Intent. The primary focus of this workshop was on applications for funding for comparative clinical effectiveness research (CER), although some content also provided insights on special considerations for other types of PCORI funding opportunities. View Workshop Agenda