The inherited bleeding disorders community has seen significant advancements in care, thanks to new therapeutics and technologies, and improved diagnostics. Yet there are still important and persistent gaps in care today, particularly for those with rare disorders and underserved populations, such as women, girls, and people who have or had the potential to menstruate with bleeding disorders.

This is why the NBDF has led the development of a National Research Blueprint (NRB), an ambitious community-driven initiative to shape the future of research for bleeding disorders. The goal is to launch a research infrastructure that can markedly accelerate progress that matters to all persons affected by bleeding disorders and their families. In order to achieve this, the blueprint is intentionally designed to ensure the entire journey of transformational research is influenced by the experiences of people who live with bleeding disorders and their loved ones – recognized as lived experience experts (LEEs) – and is grounded in principles of health equity, diversity and inclusion (HEDI). 

An image that says: An ambitious initiative to build a transformational research enterprise through full community engagement

Our Vision: Thriving in the face of an inheritable bleeding disorder begins with community-inspired research.

Our Mission: The National Research Blueprint serves to advance an accessible standard of care and quality of life for all who are affected by inheritable bleeding disorders:

  • Through collaborative and meaningful scientific inquiry; 
  • Coordinated through an efficient research infrastructure; 
  • Undertaken by a diverse, capacitated workforce in partnership with an engaged community; 
  • Supported by facilitative research policy; 
  • Grounded in HEDI principles; and Importantly, fully informed by LEEs, who are key team members in research development and implementation.

“Including the patient voice in research is crucial for ensuring that studies address the real needs and concerns of those directly affected by bleeding disorders. Patients provide unique insights into the daily challenges and practicalities of living with a bleeding disorder, leading to more relevant and impactful research outcomes. Additionally, involving patients fosters a sense of ownership and empowerment, ultimately improving the trust and cooperation between researchers and the patient community.”

– Erin Cirelli, LEE

 

The process, which began in 2020, has evolved through three major phases:

An image regarding Years of Steady Progress in 3 phases: Assess the Research Stat of the Science, Develop the National Blueprint, and Implement
  • Phase 1: Listening and Learning. During Phase 1, NBDF convened members from across the bleeding disorders community to understand pressing needs, ask questions and propose research priorities. This process included physicians, researchers and allied health professionals, advocates, government partners, industry leaders, and most importantly, people affected by bleeding disorders – referred to as lived experience experts (or LEEs). 
  • Phase 2: Convening and Developing. During Phase 2, the NRB steering committee and multidisciplinary working groups defined the principles, scope, and priorities for a blueprint to address these questions. These groups identified fundamental resource and infrastructure needs to launch and scale an integrated research pathway with opportunities for active community participation. 
  • Phase 3: Implementing. Now, in Phase 3, we are activating the first iteration of what will be an evolving NRB. Together with LEEs and community leaders, NBDF is in the process of raising funds and planning for the critical first steps for the NRB as a catalyst to redefine priority bleeding disorders research. Read further for what lies ahead and how you can get involved.

"There are a lot of opportunities for research to understand more about the experience of living with a bleeding disorder, and to make lives better until we can find a cure."

– Listening Session Participant

 

Learn more about our journey since 2020, and access summit recordings, survey results, and working group publications.

 

What the NRB will mean for our community 

The National Research Blueprint for inherited bleeding disorders

 

“This initiative is an exciting opportunity to accelerate research progress for people with bleeding disorders by bringing together all the right people, including those who are directly affected, to cooperate on urgent areas of research.”

– NRB Steering Committee member Maria E. Santaella, VP Research, NBDF 

 

How can you participate?

  • Get involved as a lived experience expert or lend your professional expertise to the NRB effort! See here for more details and a form to opt-in
  • Join Community Voices in Research (CVR), an initiative aimed at understanding the personal experience of living with a bleeding disorder and providing critical data to help identify the community’s most pressing needs. Participants get access to a Personalized Dashboard connecting them to additional opportunities to participate in research, among other things. 
  • Participate in Virtual Advisory Panels and other research projects. For more information, contact NBDF’s Research Department
  • Learn more about the NRB during NBDF’s annual Bleeding Disorders Conference in Atlanta scheduled for September of 2024 – follow this link for details.
  • Bookmark or subscribe to NBDF’s YouTube page where you can access the recordings of multiple NRB building sessions and community conversations.
  • Spread the word! Talk with your friends and family about the NRB and encourage them to consider signing up to participate as LEEs in the process. 
  • Follow NBDF for progress updates. Follow NBDF on Facebook, Twitter, and LinkedIn, or Subscribe to receive NBDF email updates.