Background: Rare bone diseases account for 5 percent of all birth defects and virtually all rare bone diseases cause significant physical disabilities. Patients and caregivers are frequently isolated and uncertain about best care practices. Using virtual technology allows patients and clinicians to connect and to gain knowledge of their particular rare bone disorder.
Proposed Solution to the Problem: The project Leveraging Virtual Communication to Advance PCOR Adoption by the Rare Bone Disease Community will work to leverage the relationships among members of the Rare Bone Disease Alliance (RBDA) to host virtual meetings with leaders and stakeholders as well as clinicians/researchers who often care for a number of rare diseases. The RBDA is a network of patient groups and professionals dedicated to improving the lives of people with various rare bone diseases.
Objectives: The aim of the project is to share the successful PCOR experience of the Osteogenesis Imperfecta Foundation with the rare bone disease community. The project will pay particular attention to reaching stakeholders from underrepresented communities.
Activities: To achieve this goal, the project will convene a series of virtual meetings over one year that enhances collaboration among rare bone disease communities; increases underrepresented community participation in PCOR; engages with stakeholders who may be disadvantaged by the digital divide; initiates an engagement approach that fosters collaboration and partnerships; and develops a road map to implement and sustain robust stakeholder engagement built solely on virtual platforms. The project will convene a series of three large group virtual meetings that will introduce PCOR to the rare bone disease communities. In addition, the project will hold several focus group virtual meetings with patients and caregivers to ascertain the barriers to expert care from the patient perspective while identifying potential collaborative clinical research opportunities.
Projected Outcomes and Outputs: This project will strengthen the working relationships among members of the RBDA. While different, many of these rare bone diseases have similarities and there is much “shared knowledge” between the groups. A physician or researcher with expertise in one disorder often has expertise in several rare bone disorders. Similarly, patients with one disorder have many shared experiences with individuals affected by a different disorder. The convening award is a critical step toward developing the capacity to address shared concerns like access to adult care for people with rare bone diseases. The project will encourage clinicians and researchers who participate in this convening award to develop their own PCOR projects and lay the groundwork for future PCOR and CER projects.
Patient and Stakeholder Engagement Plan: The Leveraging Virtual Communication to Advance PCOR Adoption by the Rare Bone Disease Community Convening Award will engage the stakeholder community at every step. Patients, parents/caregivers, clinicians, and researchers will be invited to serve on the project advisory, leadership, and scientific committees that will help govern and guide the project. The advisory committee will be made up of representatives from each RBDA organization and, most importantly, committee members will report back to their communities both their experiences being part of this project as well as their commitment to supporting PCOR. The focus groups and convening meetings will be patient- and stakeholder-led from the planning stages through participation. A significant portion of the project team’s efforts will be devoted to engaging and identifying individuals with rare bone diseases who may not currently be reached by the team’s internet communication efforts. Using OIF and other RBDA members’ mailing lists, the team will conduct a traditional postal survey to establish contact with such patients, as this will provide a means for understanding internet access issues and the best ways for the team to engage.
Project Collaborators: The 13 RBDA patient organizations are invited to make up the project’s advisory committee along with other stakeholders, including rare bone expert clinicians. The Osteogenesis Imperfecta Foundation serves as the lead patient organization supported by OI expert and orthopedic surgeon Laura Tosi, MD, and Duke University’s Bryce Reeve, PhD, from its Populations Sciences Department, as subcontractors.