NORD and University of Maryland Partner on PCORI Award to Advance Rare Disease Research

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Training Program for Patients and Caregivers Will Be Offered at NORD’s Annual Rare Diseases and Orphan Products Breakthrough Summit in October

Pcori logoAn award from the Patient-Centered Outcomes Research Institute (PCORI) will help the National Organization for Rare Disorders (NORD) and the University of Maryland School of Pharmacy train patients and caregivers to become more involved in research in ways that may ultimately impact the treatment of their disease.

NORD, the nation’s leading independent nonprofit for helping rare disease patients and families, and the University of Maryland School of Pharmacy, known for shaping the future of pharmacy by pioneering new roles for pharmacists in clinical practice, research, comparative effectiveness, patient-centered outcomes, and disease management, have received a PCORI Eugene Washington Engagement Award to support the development of a training program on patient-centered outcomes research, also known as PCOR.

The goal of the training program is to give NORD members, largely rare disease patients and caregivers, the knowledge and tools they need to become more engaged in PCOR to advance research and understanding of rare diseases.

“We are excited about this collaboration with the University of Maryland and the opportunity to provide PCOR training to NORD’s members,” says NORD President & CEO Peter L. Saltonstall. “The goals of this program are timely and 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 rare disease research and development will be a critical part of medicine going forward.”

The training program will be offered at NORD’s annual Rare Diseases and Orphan Products Breakthrough Summit, the largest multi-stakeholder event dedicated to rare disease treatment, innovation, and access, on Oct. 21-22 in the Washington, D.C. area.  The program will be followed by an additional half-day session on Oct. 23 for participants selected through an application process.

Eleanor Perfetto, Ph.D., a professor 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.”

Following the training, 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.

According to NORD, there are 7,000 rare diseases that affect nearly 30 million Americans, yet the majority of rare diseases have no treatment and there are zero cures.  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.

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 in meaningful ways 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, 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.”

This Engagement Award was 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/.

PCORI is an independent, non-profit 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 healthcare decisions. PCORI is committed to seeking input from a broad range of stakeholders to guide its work.

To apply for the training program, visit: https://rarediseases.org/summit-event-details/ and download the scholarship application.


About 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.

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.