Faculty at the University of Maryland School of Pharmacy have received a $250,000 Patient-Centered Outcomes Research Institute (PCORI) Eugene Washington Engagement Award to support the development of a training program on patient-centered outcomes research (PCOR). The goal of the training program, which will be delivered at the annual Rare Diseases and Orphan Products Breakthrough Summit hosted by the National Organization for Rare Disorders (NORD) in October 2015, is to give NORD members, largely patients and caregivers, the knowledge and tools they need to become more engaged in PCOR.
Eleanor Perfetto, PhD, a professor in the Department of Pharmaceutical Health Services Research (PHSR) at the School of Pharmacy, will lead the engagement project. “Patients and patient groups want to become more engaged in PCOR, but when they try to take an active role or are first approached by a researcher, they may feel uncomfortable, unprepared, or intimidated,” says Perfetto. “Some don’t know what is expected of them or how they can most effectively contribute. Some may also be unfamiliar with the technical lingo used by medical researchers. Our goal with this PCORI Engagement Award is to break down those barriers by training NORD members, many of them rare-disease patients themselves, to feel confident, prepared, and ready to engage in PCOR. The intent of this training will be to give the patients and patient groups the knowledge and tools they need to be active patient partners in PCOR, not merely filling a traditional, passive patient or study subject role.”
According to NORD, the leading independent nonprofit dedicated to helping people with rare diseases, nearly 7,000 rare diseases have been identified that affect nearly 30 million Americans, but treatment or cures do not exist for the majority of them. This collaborative project will design, implement, and evaluate an educational program on PCOR specifically for NORD member organizations to empower them to participate in research that could ultimately impact the treatment of their disease.
With the award, Perfetto and her collaborators will develop PCOR training materials or adapt existing training materials to provide instruction on PCOR, research methods, and patient engagement in the research process. The end result will be hands-on, active learning exercises for approximately 100 participants at NORD’s annual summit in Washington, D.C., the largest multi-stakeholder event dedicated to rare disease treatment, innovation, and access, in October 2015, followed by an additional half-day session for invited participants selected through an application process. Following the training sessions, the engagement award team will conduct evaluations of the sessions, tweak the training, and disseminate what was learned to additional NORD members and researchers from other organizations.
“We are excited about this collaboration with the University of Maryland and the opportunity to provide PCORI training to the organizational members of NORD,” says Peter L. Saltonstall, president and CEO of NORD. “The goals of this program are not only timely but critical to equip patient organizations with the knowledge and understanding of how PCOR works and how they can apply it in their advocacy, research, and patient service programs. Patient involvement in R&D will be a critical part of medicine going forward."
The project is part of a portfolio of projects approved for PCORI funding to help develop a skilled community of patients and other stakeholders from across the entire health care enterprise and to involve them meaningfully in every aspect of PCORI’s work.
“This project was selected for Engagement Award funding not only for its commitment to engaging patients and other stakeholders, but also for its potential to increase the usefulness and trustworthiness of the information we produce and facilitate its dissemination and uptake,” said Jean Slutsky, PA, MSPH, PCORI’s chief engagement and dissemination officer. “We look forward to following the project’s progress and working with the School of Pharmacy and NORD to share the results.”
The School of Pharmacy project and the other projects approved for funding by the PCORI Engagement Award Program were selected through a highly competitive review process in which applications were assessed for their ability to meet PCORI’s engagement goals and objectives, as well as program criteria. PCORI has awarded nearly $5.5 million to support 28 projects to date through this program. For more information about PCORI’s funding to support engagement efforts, visit http://www.pcori.org/content/eugene-washington-pcori-engagement-awards/.
“Currently, patient involvement in rare disease research is focused on raising public awareness, aiding in clinical trial recruitment, and developing informed consent documents,” says Perfetto. “These efforts represent only a fraction of areas where patients can guide and improve rare disease research. Patient input can guide research topics and all aspects of study design from selecting desired outcomes to selecting the ‘real-world’ comparator. Our hope is that by training rare disease patients on how to be more engaged and sharing that information with researchers themselves, we can improve treatment options for those who suffer from rare diseases.”
To apply for the training program, visit: http://rarediseases.org/summit-event-details/ and download the scholarship application.
PCORI is an independent, nonprofit organization authorized by Congress in 2010 to fund comparative effectiveness research that will provide patients, their caregivers, and clinicians with the evidence needed to make better-informed health and health care decisions. PCORI is committed to seeking input from a broad range of stakeholders to guide its work.
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About the University of Maryland School of Pharmacy
Established in 1841, the University of Maryland School of Pharmacy is a thriving center for professional and graduate education, pharmaceutical care, research, and community service. We create the future of pharmacy by pioneering new roles for pharmacists in advanced clinical practice and conducting cutting-edge research in drug discovery and development, comparative effectiveness and patient-centered outcomes, and disease management. Our faculty inspire excellence in our more than 800 students, residents, and postdoctoral fellows through a contemporary curriculum, innovative educational experiences, and strategic professional relationships. We are critical thinkers, lifelong learners, and leaders who are sought for our expertise.
About the National Organization for Rare Disorders (NORD)
The National Organization for Rare Disorders (NORD), established in 1983, is the leading independent nonprofit organization representing all patients and families affected by rare diseases in the U.S. NORD is committed to the identification, treatment, and cure of all 7,000 rare diseases, 95 percent of which have no treatment, and affect 30 million Americans, or 1 in every 10 people. NORD provides programs of advocacy, education, research and patient/family services to improve the lives of all people living with rare diseases. NORD represents more than 230 disease-specific member organizations and partners with many other organizations in specific causes of importance to the rare disease patient community. Join NORD at www.rarediseases.org/ and on Twitter at @RareDiseases.