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).

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.