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 US healthcare system. Although opioid analgesics are commonly used to treat chronic noncancer pain, there are rising concerns regarding the appropriate use, misuse, and harms such as dependence and overdose. Overdoses involving opioids killed more than 47,000 people in the United States in 2017, and 36 percent of those deaths involved prescription opioids. 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.

One out of every four Americans experiences an anxiety disorder in his or her lifetime, making anxiety disorders the most prevalent group of psychiatric disorders in the United States.s. 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.

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.

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.

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.

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.

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.

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.