March 15, 2023 – The National Hemophilia Foundation (NHF) today announced that a series of manuscripts central to advancing patient-focused research for the inherited bleeding disorders (IBD) community have been published in the journal Expert Review of Hematology. These papers describe initial recommendations from six multi-disciplinary working groups established to evaluate and address the most urgent priorities for the community, and will form the basis for NHF’s community-driven National Research Blueprint (NRB).
“Despite incredible progress in the diagnosis and treatment of bleeding disorders in recent years, important unmet needs continue to burden this community, particularly for smaller underserved populations,” said Leonard Valentino, MD, NHF’s president and chief executive officer. “Through this collaborative process, we will direct research priorities and resources toward those areas that will make the most difference for individuals with these disorders and their families. This National Research Blueprint has the potential to redefine how IBDs are treated by the next generation.”
Since 2020, NHF in collaboration with the American Thrombosis and Hemostasis Network (ATHN) and other community organizations has championed this extensive community-driven effort to shape the future of research in IBDs, starting with a series of focus groups, listening sessions and surveys to collect input from patients and caregivers, researchers and allied healthcare professionals, chapter and member organizations, and industry partners. With this input, NHF convened a steering committee and working groups representing diverse disciplines of health professionals as well as patient representatives, referred to as lived experience experts (LEEs), to identify common themes and opportunities reflecting the community’s input. These groups convened during a State of the Science (SOS) Research Summit in September 2021 and then a series of workshops in 2022 to prioritize research opportunities based on feasibility, impact and risk.
Now, the newly published papers reflect the findings from each working group. All of this information will form the basis of the National Research Blueprint, which will aim to guide community collaboration and accelerate research progress.
“These manuscripts illustrate the incredibly methodical approach that has been undertaken to capture the most urgent needs of the IBD community and the most impactful opportunities to address them through collaborative research,” said Maria Santaella, PhD(c), MSN, RN-BC, CPHON, VP of Research Strategy at NHF. “We are so grateful to the diverse teams of trusted subject experts, LEEs and community members who have given so much time and energy to help build the manuscripts that will culminate in the NRB, a comprehensive research roadmap for the future.”
NHF encourages community dialogue on these papers, Building the foundation for a community-generated national research blueprint for inherited bleeding disorders, each highlighting the following:
- Executive Summary: The National Hemophilia Foundation State of the Science Research Summit Initiative
- Foreword: The National Hemophilia Foundation’s state of the science research summit: the foundation of a national research blueprint for inherited bleeding disorders
- Working Group 1: Building the foundation for a community-generated national research blueprint for inherited bleeding disorders: Research priorities to transform the care of people with hemophilia
- Building on the great progress in treating hemophilia, the focus now shifts toward advancing health equity and improving diagnostics and testing.
- Working Group 2: Building the foundation for a community-generated national research blueprint for inherited bleeding disorders: Research priorities for mucocutaneous bleeding disorders
- Focused research could improve diagnosis and management of these poorly understood disorders to reduce the substantial impact on quality of life.
- Working Group 3: Building the foundation for a community-generated national research blueprint for inherited bleeding disorders: Research priorities for ultra-rare inherited bleeding disorders
- New research opportunities can improve diagnosis, treatment and testing of new therapies for ultra-rare IBDs leveraging collaborative research networks and infrastructure.
- Working Group 4: Building the foundation for a community-generated national research blueprint for inherited bleeding disorders: Research to advance the health of women and girls and people with inherited bleeding disorders with the potential to menstruate
- Poor understanding of the line between normal and abnormal bleeding lead to disparities in proper diagnosis and care. Research can build foundational knowledge, standards for diagnosis and assessment, and patient-centered outcomes goals.
- Working Group 5: Building the foundation for a community-generated national research blueprint for inherited bleeding disorders: Research priorities in health services; diversity, equity, and inclusion; and implementation science
- Considerable gaps must be addressed to improve healthcare delivery and equitable access to ensure all people with IBDs can receive the care they need.
- Working Group 6: Building the foundation for a community-generated national research blueprint for inherited bleeding disorders: Facilitating research through infrastructure, workforce, resources and funding
- Accelerating IBD research requires focused investments in infrastructure, workforce development and funding to foster research collaborations and networks.
- Lived Experience Expert Editorial: Lived Experience Experts: A name created by us for us
- “Particularly in rare disorders, lived experience experts, or LEEs, have invaluable expertise to contribute at all stages of the research endeavor.” This commentary reflects on the impact of the patient perspective in defining research that will make a meaningful difference.
- International Perspective: Soliciting international perspectives on an American national research agenda for inherited bleeding disorders
- NHF seeks perspectives on how research priorities for IBDs identified by the U.S. community may apply to other countries. This summary calls on the entire international community, including care team members as well as LEEs, to participate in a survey that will further inform the global research dialogue.
The NRB process continues with the development of the research roadmap, expected in 2024. For more information on the NRB process, please visit https://www.hemophilia.org/research/national-research-blueprint. NHF encourages the entire community to consider participating in research. For more information on how to get involved, please email research@hemophilia.org.
