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NORD, University of Maryland on PCORI award to advance rare disease research

Danbury, Connecticut
Monday, July 20, 2015, 13:00 Hrs  [IST]

An award from the Patient-Centred Outcomes Research Institute (PCORI) will help the National Organisation 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-centred outcomes, and disease management, have received a PCORI Eugene Washington Engagement Award to support the development of a training programme on patient-centred outcomes research, also known as PCOR.

The goal of the training programme 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 & chief executive officer Peter L. Saltonstall.

“The goals of this programme are timely and critical to equip patient organisations with the knowledge and understanding of how PCOR works and how they can apply it in their advocacy, research, and patient service programmes. Patient involvement in rare disease research and development will be a critical part of medicine going forward.”

The training programme 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 October 21-22 in the Washington, D.C. area. The programme will be followed by an additional half-day session on October 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 organisations.

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 programme on PCOR specifically for NORD-member organisations 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 programme criteria. PCORI has awarded nearly $5.5 million to support 28 projects to date through this programme.

PCORI is an independent, non-profit organisation 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.


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