The Bleeding Disorders Research Collaborative

Abstract

The Bleeding Disorders Research Collaborative (BDRC) aims to advance an accessible standard of care and quality of life for all people living with inheritable bleeding disorders. This goal will be achieved through collaborative and meaningful scientific inquiry, coordinated by an efficient research infrastructure, and undertaken by a diverse, capacitated workforce in partnership with an engaged community. The BDRC is supported by facilitative research policy and grounded in the principles of health equity, diversity, inclusion, accessibility, and belonging, striving for dignity, safety, well-being, and opportunities, leading to health justice. Importantly, the initiative is fully informed by lived experience experts, people affected by inheritable bleeding disorders, who are key members in the research development, implementation, and dissemination team.

Graphical abstract

Figure 1.

Core areas targeted by the BDRC to create a transformational, community-engaged research enterprise. These core areas include re-envisioning the approach to research, creating a novel process to prioritizing and conducting research, and focusing on outcomes that are meaningful to people who live with BDs. This included prioritizing meaningful research topics, streamlining and democratizing research processes, and ensuring outcomes are relevant, representative, and broadly shared.

Figure 2.

Evolution of the BDRC initiative. The 4 stages of the BDRC initiative are shown schematically: initial visioning and community engagement, identification of research priorities, infrastructure and governance development, and future goals for cross-condition leadership in research. The model anticipates long-term impact through broader adoption of LEE leadership in rare disease research. The first phase of the project, implementation, began with “blue sky thinking,” bringing together healthcare professionals, community leaders, industry and federal partners, and, most importantly, LEEs. In the second phase, a steering committee worked with interdisciplinary WGs to define the principles, scope, and research priorities addressing the community’s most pressing needs. As part of the process, they identified resource and infrastructure requirements to launch and scale an integrated research pathway with opportunities for community participation at every step. A blueprint emerged, designed around the principle that LEEs serve as essential partners in research closely integrating health justice including equity, diversity, inclusion, acceptance and belonging.

Figure 3.

Visualization of the BDRC priority-setting model. Rooted in the foundational themes of people centricity, HEDI, and collaboration, this tree structure maps the development from spanning research topics and scientific areas of interest established through the SOS to a rich canopy of community-driven research priorities established through the NRB phase. Main branches reflect broad spanning topics, whereas smaller branches denote scientific areas of interest. The leaves symbolize specific research priorities developed and refined through NRB WGs. A leaf may be taken from the tree to create a specific research project.

Figure 4.

The evolution of the WGs from the SOS to the NRB. This figure illustrates the transition of key research priorities identified during the SOS process into the strategic pillars of the NRB. The left side of the figure outlines the 6 WGs established during the SOS. The right side depicts the 7 WGs of the NRB that generated the overarching research themes, which together demonstrate how the initial research priorities have informed and broadened into a comprehensive national agenda for BD research.

Figure 5.

Research governance and study activation workflow. (A) The dynamic organizational framework of the BDRC with a hierarchical governing structure that includes key stakeholders provides oversight of research activities. (B) Project map outlining the stages from research proposal solicitation to active studies, highlighting critical review and approval points, including LEE/HEDI considerations. IRB, institutional review board.

Figure 6.

The evolution of LEE involvement in the BDRC and the RA program participation continuum. This figure illustrates the progression of individuals from being human subjects in research to engagement as LEEs, for their expertise in living with a BD, and subsequently participating as RAs. The top portion depicts this evolution, highlighting the transition and central role of LEEs. The bottom portion presents the RA program participation continuum, showcasing the increasing levels of engagement and leadership roles that trained LEEs can assume, ranging from engaged LEE and informed participant to research consultant, research collaborator, and ultimately research leader. Broad viewpoints and perspective will be welcomed among the LEEs that participate in the BDRC. Those selected for the RA Program will need to be highly motivated and have ample time to commit to the project.