Project Summary  

For many of the over 6,800 identified rare diseases, treatments or cures do not exist. While research is ongoing, it often does not include input from rare-disease patients. It is important that researchers understand patient views on outcomes desired, current treatments, and the unique challenges faced by rare-disease suffers. Most rare-disease patient advocates are patients themselves or caregivers for those with rare diseases. With drive and passion, they can contribute to rare-disease patient-centered outcomes research (PCOR) if empowered with the “know-how.” 

The National Organization for Rare Disorders (NORD) recognized that to improve rare-disease patient engagement, training and support are needed. In partnership, NORD and the University of Maryland will provide training on PCOR for the NORD membership. The PCOR training needs expressed by NORD members will be the primary focus of the program, which will take place in fall 2015 as part of the NORD Annual Summit. The project will tap and adapt existing resources, and design, implement, and evaluate an educational program on PCOR specifically for staff and patient members (and/or family/caregiver members) of NORD-member organizations. It leverages tools and learnings from past PCORI-funded projects and the AHRQ-funded PATIENTS project. All training materials, as well as best practices identified, will be disseminated in partnership with NORD and its members.

The projected outputs from this project are the development and delivery of training on PCOR, effective collaboration skills with researchers, and basic research methods for NORD-member organizations; evaluation of training via participant-completed online assessments; dissemination tools developed and lessons learned through the evaluations; and development of a checklist for identifying successful PCOR partnership collaborations.

Project collaborators include NORD staff; NORD member-organization staff and volunteers; National Health Council; and training speakers.

Training Summary and Resources

In response to rare-disease patients’ desire to make research more meaningful to their community, we developed and conducted a two-day special training on PCOR for rare-disease patient advocates. Prior to hosting the training, a Patient Advisory Board composed of leading experts in PCOR and rare disease patient advocates was established to inform the direction and content of the training. The training program was offered at the October 2015 NORD Annual Summit over 2 days (October 22nd and 23rd).

The first day of the training was a half-day track, as part of NORD’s annual summit, focused on the key principles and methods of PCOR, stakeholder engagement, and effective collaboration with researchers. Over 100 participants attended this session.

Day One Materials

• Day One Agenda
• Session One – PCOR: What it Means to the Rare Disease Community
• Session Two – Reorienting the Research Environment
• Session Three – Partnerships and Funding for PCOR Projects
• Session Four – PCOR and Policy – Panel Discussion

The second day of the training was a full day session open only to rare disease patient advocates who were selected through an application process. The second day built on the previous day’s presentation, but included a more in depth treatment of the subject matter. Rare-disease patient advocates engaged in active learning exercises to apply the concepts and principles presented, as well as interactive discussions to brainstorm ways the information presented could be applied to their specific rare condition.

Day Two Materials

• Day Two Agenda
• Session One – The Natural History of Disease: The Trump Card?
• Session Two – The Different Levels of Patient Engagement
• Session Three – Putting your Best Foot Forward: Establishing Partnerships with PCOR Stakeholders
• Session Four – Optimizing Funding Applications

The training was supplemented with various materials to help participants better understand how to conduct and engage in PCOR including: the PCORI Patient and Family Engagement Rubric and the Ten-Steps of Continuous Patient Engagement Framework. In addition to this, new resources were developed including a Partnership Checklist.

Resources

• Acronym List
• Glossary of Terms
• Successful Partnership Checklist
• PCOR for Rare Disease Resource List

After the training, the patient advocates who participated in the training participated in Research Club teleconferences. The calls were designed to continue active learning on PCOR methods and discuss new opportunities to engage in PCOR. The topics originated from the patient advocates and included the PICOTS framework and patient-reported outcomes. One Research Club Teleconference focused on The PICOTS Framework: How to Write a Research Question.  Another Research Club Teleconference focused on Patient Reported Outcomes.

Research Club Teleconference Materials

• The PICOTS Framework: How to Write a Research Question
• Introduction to Patient Reported Outcomes (PROs)
• Introduction to Patient Reported Outcomes (PROs) Slide Deck

More on this Project

VIDEO: Patient-Centered Outcomes Research for Rare Disease Patients
Patients with rare diseases can become empowered through involvement in patient-centered research, says University of Maryland researcher Eleanor Perfetto, PhD.

VIDEO (below): Answering the Right Questions
PCOR focuses on answering the questions that matter most to patients and their caregivers.

Event Information

PCOR Training for NORD Members

October 19-23, 2015

Crystal Gateway Marriott
1700 Jefferson Davis Highway
Arlington, VA 22202

This is an in-person meeting and is open to NORD patient organization members only. See the conference website for details.

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