Five years ago, the National Bleeding Disorders Foundation (NBDF) set an ambitious goal: to transform how bleeding disorders research is conducted by ensuring patient voices and experiences are included in every phase, from study design and recruitment to outcomes and implementation. This initiative, now called the Bleeding Disorders Research Collaborative (BDRC), has published its first findings in the form of five white papers published in Expert Review of Hematology. Collectively, the papers establish a patient-centered research agenda for the future of bleeding disorders research.
The papers present a roadmap for bleeding disorders research, one in which people living with bleeding disorders, their caregivers and family members, play a meaningful role. These individuals, known as lived experience experts (LEEs), contribute firsthand knowledge that helps guide research priorities, study design, and the application of findings. LEEs have been essential partners in the development and execution of the BDRC and, the papers posit, should continue to play a pivotal role in shaping the future of patient-centered bleeding disorders research.
The publications are the product of seven multidisciplinary working groups, composed of LEEs, health care professionals, researchers, advocates, government partners and industry leaders, who collaborated to create a consensus on the areas where future bleeding disorders research should focus. The papers outline key priorities, including a community-informed research agenda, workforce development, infrastructure, community engagement strategies, and policies that support long-term success. They also emphasize the importance of including health equity, diversity, and inclusion across every aspect of the collaborative's work.
One of the most significant outcomes highlighted in the papers is the identification of 327 research priorities identified by the multidisciplinary groups. These priorities span a wide array of topics such as barriers to care, joint health, healthcare delivery, diagnostic tools, aging, mechanisms of bleeding, and inhibitor development. The breadth of these priorities reflects the value of bringing together patients, caregivers, researchers, clinicians, and other stakeholders to identify the questions that matter most to the bleeding disorders community. The findings also demonstrate the impact of LEE participation and the importance of achieving consensus across stakeholder groups.
Historically, patients have often participated in research primarily as study subjects. The BDRC model expands that role by engaging people with bleeding disorders and caregivers as partners in setting research priorities, informing study design, interpreting findings, and helping ensure research addresses real-world community needs.
Many aspects of the BDRC are being advanced within NBDF. A program run by NBDF’s LEE Research Advisory Council, the BDRC Research Ambassador program, prepares and empowers lived experience experts to participate effectively in research, collaborate with investigators, and even lead bleeding disorders research projects. Through training and education, the program helps ensure the next generation of patient-centered research partners will continue into the future.
Most importantly, these papers mark NBDF’s commitment to a patient-centered approach to research, one in which lived experience, scientific expertise, and health equity work together to accelerate discoveries and improve outcomes for people affected by bleeding disorders. By elevating the voices of lived experience experts and establishing a community-driven research agenda, NBDF's Bleeding Disorders Research Collaborative is helping shape a stronger, more inclusive future for bleeding disorders research.