RaRE

Rare Disease and Research Engagement: Developing a National Model for Cross-Cutting Stakeholder Engagement

Background:
While some rare disease organizations have succeeded in developing engaged communities, there is a lack of an organized engagement approach that allows collaboration on cross-cutting rare disease PCOR. RaRE will convene stakeholders across diverse rare disease communities to discuss and develop themes that are generalizable across rare diseases. The group will build a national network of engaged partners to improve readiness for future rare disease comparative effectiveness research and evidence dissemination, and will define a cross-cutting PCOR agenda for rare disease and mental health. PCORI’s proven engagement resources and multi-stakeholder engagement principles will be leveraged to ensure robust and inclusive engagement between these organizations. The project team includes diverse stakeholders who have demonstrated expertise in convening partners, developing rare disease communities, and leading PCORI-funded initiatives to ensure success of this project. The goal of this work is to develop a Rare Disease Partnership Model and a set of stakeholder-informed PCOR priorities at the intersection of rare disease and mental health/quality of life.

Key Objectives:

Build a national network of diverse rare disease stakeholders interested in PCOR for rare diseases
Develop a comprehensive and engagement model and governance structure that will enhance collaborations between disparate rare disease communities for PCOR partnership.
Identify cross-cutting patient-centered outcomes and research questions amenable to pragmatic comparative effectiveness research studies
Develop a roadmap to implement and sustain robust stakeholder engagement in cross-cutting rare disease PCOR.

Resources:

The Roadmap for Rare Disease Stakeholder Engagement was developed by stakeholders representing patient partners, advocates, health system leaders, payers, clinicians, researchers, public health professionals, and industry leaders with broad experience applying the engagement principals established by the Patient Centered Outcomes Research Institute (PCORI). The Roadmap is a model for building robust and sustainable stakeholder engagement to support rare disease patient-centered outcomes research (RD-PCOR) that transcends individual rare diseases and limited geographies. It is intended to guide researchers in building authentic, meaningful engagement with stakeholders who have not traditionally been included as equal partners in the research process.

The model is organized into two key phases, Pre-Engagement and Engagement. It is important to note that engagement can be initiated from any starting point, and movement within the roadmap can be bi-directional and non-linear based on the needs and experience of the research team.

An Implementation Guide (see link at bottom of the page) was designed to be used in parallel with the Roadmap and will assist users in identifying key activities under each goal and assessing their team’s readiness for effective and meaningful stakeholder engagement. The guide highlights critical questions that research teams may want to consider in order to achieve robust and sustainable stakeholder engagement. Teams may choose to work through the questions in this guide throughout the course of a project or initiative to document engagement efforts and provide opportunities for quality improvement.

It is important to note that the Roadmap and Implementation Guide are models. They were designed specifically for Rare Disease Stakeholder engagement; however the model can be applied to stakeholder engagement across most any research initiative.

Click below to download an editable copy of the Roadmap and Implementation Guide.

Rare Disease Stakeholder Engagement Roadmap
Roadmap Implementation Guide

This project was funded through a Patient-Centered Outcomes Research Institute Eugene Washington PCORI Engagement Award (EACC-18475).