Our opening keynoters are Scott Berns, MD, MPH, and Leslie Gordon, MD, PhD, accomplished Boston-area pediatricians and researchers. They cofounded the Progeria Research Foundation (PRF) after their son, Sam, was diagnosed with that rare condition. Sam died in 2014, at age 17, although not before he became an online sensation with his TEDx talk, My Philosophy for a Happy Life, and the HBO documentary Life According to Sam. Thanks largely to PRF’s support, the gene that causes progeria was isolated and the first treatment developed. Berns, who is PRF’s Board chair, is also CEO of the National Institute for Children’s Health Quality. Gordon, the Foundation’s volunteer medical director, is professor of pediatrics research at Hasbro Children’s Hospital and the Alpert Medical School of Brown University in Providence, Rhode Island, as well as progeria clinical trial investigator at Boston Children’s Hospital and Harvard Medical School.

How can research help people make health care decisions that are individual to them? This session will review a few select PCORI-funded studies to help highlight PCORI’s effort to tailor healthcare choices to the needs of each person, based on their personal characteristics, biology, history, and preferences. This includes the adoption of the latest technological advances in screening for health conditions; making treatment decisions using tools that help predict outcome success; and using individualized plans to help improve care satisfaction.

Chronic pain exerts a significant burden on the general population, with approximately 50 million Americans suffering from chronic pain and costs of $635 billion dollars per year to the U.S. healthcare system. Although opioid analgesics are commonly used to treat chronic noncancer pain, there are rising concerns regarding appropriate use, misuse, and harms such as dependence and overdose. In 2017, prescription opioids were involved in approximately 36 percent of all deaths in the United States. Given the serious safety concerns and lack of long-term evidence of effectiveness, evidence-based alternatives are needed. This symposium will discuss three PCORI-funded studies that address nonpharmacological approaches for chronic pain, including methodological challenges and lessons learned for conducting comparative effectiveness research.

The Agency for Healthcare Research and Quality (AHRQ) is mandated to work with PCORI and others to disseminate patient-centered outcomes research (PCOR) findings into practice. AHRQ established a structured process to review and assess findings for strength of evidence, level of potential impact, and implementation feasibility to prioritize them for investment. In this session, AHRQ will highlight three of its dissemination and implementation initiatives: EvidenceNOW: Advancing Heart Health in Primary Care; TAKE Heart: AHRQ’s National Initiative to Increase Use of Cardiac Rehabilitation; and Screening and Management of Unhealthy Alcohol Use in Primary Care.

Anxiety disorders are the most prevalent group of psychiatric disorders in the United States, with a lifetime prevalence of one out of every four Americans. There is also a significant public burden, with annual costs of associated medical expenditures, lost productivity, and functional impairment estimated at $42.3–$46.6 billion annually. Anxiety disorders are among the earliest psychiatric conditions to develop, with a median age of onset of 11 years. This session will include presentations on PCORI-funded research studies comparing evidence-based interventions in the treatment of anxiety, followed by a discussion of how these patient-centered approaches have the potential to improve quality of life for those impacted by anxiety disorders.

Increasingly, patients are faced with health decisions that have many options, uncertain outcomes, and benefits and harms that are valued differently by each individual. There is no single right answer about how to proceed. Shared decision making recognizes the importance of having patients and providers work together to select tests and treatments, though more clarity is needed on what it means and how it is implemented in day‐to‐day care. This session will provide examples of shared decision making across the care continuum and across the lifespan. Panelists will highlight how shared decision making includes evaluation of options based on care goals, concerns, and personal context of individuals.

A hallmark of PCORI’s work has been the engagement of the public and patients in clinical comparative effectiveness research. This session will feature experienced patient partners who have participated in a range of research studies and engagement efforts. Panelists will share their experiences and provide practical advice for researchers and engagement coordinators on ways to operationalize engagement and build constructive relationships and teams. For patient partners, the panel will offer advice on applying personal experience to the design and implementation of a research study.

Studies have shown that it typically takes 17 years for the results of a randomized controlled trial to reach clinical practice, if it makes it there at all. The gap between what we know will improve health care and what is taken up in practice remains an important obstacle to improving the healthcare system and patients’ health. Implementation strategies to integrate evidence into practice settings where patients and clinicians can use it to actively inform decisions, as well as dissemination activities to enhance awareness of relevant information, are essential routes to improving health care and health outcomes. This session will highlight three PCORI-funded implementation projects that take the results of PCORI-funded comparative clinical effectiveness research studies and promote their uptake in real-world clinical practice settings.

To date, PCORI has demonstrated the feasibility of engaging patients and stakeholders in all phases of patient-centered outcomes research. Engagement in our funded studies can take many forms, however, and as such, PCORI’s practice of engagement has become a real-time, live experiment and laboratory for innovation that offers a rich set of experiences and data to generate practice-based evidence about engagement. This session will explore recent findings from an externally led investigation into how engagement has influenced the planning and conduct of PCORI-funded studies, what changes to the studies resulted from that influence, and how it occurred. This session will also highlight select projects and include stakeholder reactors to discuss the implications of the focused investigation for PCORI and the broader field.

This session will provide an overview of how patient-centered comparative clinical effectiveness research can inform decision making around prevention related to health conditions. Prevention activities broadly interpreted may aim to avoid the development of a disease or condition in healthy populations, or improve management after diagnosis and recovery, including the need for monitoring for disease recurrence. Attendees will hear from PCORI-funded research teams about their approach to prevention and from stakeholder representatives who will discuss the contributions PCORI findings can make in the field of public health.

The patient data routinely collected and curated as part of electronic health records, data registries, and research networks can be leveraged to estimate the benefits and risks of healthcare interventions, especially in circumstances limiting the use of randomized controlled trials. However, limitations in the design and analysis of observational studies can produce biased results that could have serious consequences for patients (e.g., overestimating benefits of treatments and underestimating risks). Advances in the design and analysis of observational studies have the potential to support more robust causal inferences and generate the high-quality evidence needed to support informed healthcare decisions. This session will highlight PCORI-funded methods projects that have developed and examined approaches to addressing challenges in the design and analysis of observational studies using routinely collected patient data.

More than 60 million Americans find coverage through Medicaid. This session will explore how comparative clinical effectiveness research can serve this population and those who care for them. It features two PCORI-funded projects focused on Medicaid and two reactors/discussants presenting their perspectives. Attendees will learn current clinical care concerns in Medicaid and where decisional dilemmas remain.

Veterans, members of the armed services, and their families are a population of interest for PCORI. By serving a complementary role to that of the Department of Veterans Affairs, PCORI-funded projects help to ensure that new clinical research meets the needs of those who have served. In this session, attendees will hear about PCORI-funded projects that focused on engaging veterans and their families in research. The session also will highlight the results of completed projects that examined the comparative effectiveness of clinical care provided to veterans.

Telehealth is a dynamic, disruptive technology with the potential to reduce fragmentation of care, provide better patient access, and improve outcomes for a wide range of conditions. This session highlights two PCORI-funded projects, one illustrating telehealth’s potential to improve hearing loss screening in isolated areas of Alaska, and the other illustrating its challenges when applied to a home-based exercise program for patients with peripheral artery disease. A question underscored in this session is how the research enterprise can keep up to meet the evidence needs of decision makers in a rapidly evolving field. Commentary on both projects is provided by a payer representative and an expert on rural healthcare policy.

Our closing keynoters are Giselle Corbie-Smith, MD, MSc, Kenan Distinguished Professor of Social Medicine and Medicine and director of the Center for Health Equity Research at the University of North Carolina-Chapel Hill, and Al Richmond, MSW, executive director of Community-Campus Partnerships for Health, which promotes health equity and social justice initiatives between communities and academic institutions. Corbie-Smith is nationally recognized for her scholarly work on the practical and ethical issues regarding engagement of disparity populations in research. Richmond, a PCORI Ambassador, has led two Eugene Washington PCORI Engagement Awards that have explored the drivers, barriers, and challenges to patients being engaged in research. The closing session will highlight the 20-year partnership that Richmond and Corbie-Smith have sustained that has included local, regional, and national initiatives. Through its shared commitment to equity, this partnership has served as the quintessential example of authentic partnerships.

It often takes years for new evidence from clinical research to influence the delivery of health care. Many times, these findings never reach patients and families who could benefit from the information. The missing link is implementation: the process of getting research results into practice by adapting evidence and facilitating behavior change and decision making across individuals, communities, and healthcare systems. This session will highlight three PCORI-funded implementation projects that incorporate active strategies designed to put the evidence developed by PCORI-funded comparative clinical effectiveness research studies into practical use to improve health care and patient outcomes.

During this session, panelists will share their perspectives on how employers consider patient-centered outcomes research in the design and purchase of employee health benefits. Topics include how patient-centered evidence can be used to impact healthcare costs and quality; the need for linking broader outcomes, such as work absence and performance, to patient-centered evidence and the role of research in meeting this need; and identifying the issues and needs that employers see as relevant to maximizing the use of patient-centered evidence.

Populations most at risk for disparities are often underserved and unable to receive equitable care, resulting in worse health and healthcare outcomes. As such, improving healthcare equity is critical to addressing overall healthcare quality and improving patient outcomes. This session will highlight PCORI-funded projects focused on diverse populations, including racial/ethnic minorities, LGBTQ members, and individuals with disabilities. Presenters will discuss successes and challenges in engaging their targeted communities, key study results, and lessons learned from conducting patient-centered outcomes research. This session will highlight how addressing the social determinants of health can help improve healthcare equity and conclude with discussions on how to use research results to change policy and practice.

PCORnet®, the National Patient-Centered Clinical Research Network, was established with funding from PCORI in 2014 to enable clinical research that is faster, less costly, and—most importantly—patient-centered. PCORI’s vision for PCORnet is to be a sustainable national resource that enables patient-centered research that answers questions most important to patients, caregivers, clinicians, and the broader healthcare community. The goal of this session is to highlight PCORI-funded research projects that are utilizing the PCORnet infrastructure. Presentations will include discussion from researchers, patients, health plans, and health system leaders.

PCORI has funded several studies that aim to reduce the burden of chronic conditions for patients, their family members, and the healthcare system. During this session, the audience will hear from PCORI-funded investigators and their stakeholder partners about their research, which focuses on a variety of chronic conditions. After presentations from our investigators and their stakeholders, a discussant will facilitate audience and presenter discussion.

The public’s investment in PCORI has helped transform the culture of research, produced impactful evidence that is improving patient care, and built an infrastructure to help leverage the power of real-world data. This forward-looking session will discuss how we can build on PCORI’s initial success and identify new opportunities to advance patient-centered outcomes research.