NBDF has convened a Steering Committee and working groups with healthcare providers, researchers, people affected by bleeding disorders and other subject matter experts to distill the important insights you’ve shared and define research priorities.
Diverse working groups have been convened based on the themes from the community’s input.
Each working group is tasked with addressing key research questions on these topics and defining opportunities, based on well-defined criteria, to foster actionable research to benefit the community.
- How can we use new technologies to discover therapies that make life with hemophilia easier?
- What is needed to introduce more targeted and accessible diagnostics and therapies for people with bleeding disorders?
- How can we better understand the biology of these rare diseases and what do we need to know to improve diagnosis and treatment?
- How can we improve care for women with bleeding disorders through new tools like non-invasive prenatal testing for therapies for reproductive system bleeding?
- Where can we make the greatest impact on equal access to care and more inclusive coverage? What digital tools could be implemented?
- How can we encourage more trainees to join our professional community?
2021 State of the Science Research Summit
The initial phase of the NRB culminated with the virtual State of the Science (SOS) Research Summit in 2021 where each of the working groups focused on different areas presented research questions and ranked them based on risk, feasibility, and impact. The SOS also provided the community a forum to comment and raise additional research questions, breakthrough opportunities and challenges. This process has formed the basis for the NRB now in development. NBDF expresses sincere gratitude to everyone who participated in the SOS activities and Summit.
Thank you to the State of the Science Steering Committee
State of the Science Manuscripts – Now Published!
The initial recommendations of each working group from the State of the Science (SOS) are now published in a Supplement of the journal Expert Review of Hematology.
These manuscripts define specific themes and research recommendations compiled by each working group that have the greatest potential to make a meaningful, lasting impact for people with inherited bleeding disorders. The guidance provided in these papers will form the basis of the National Research Blueprint.
The Foundation extends sincere thanks to everyone on the SOS Steering Committee, Working Group chairs and participants for their time and contributions to these important papers, each of which includes a simple language summary. These papers can be reviewed at the following links:
- 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
- Working Group 2: Building the foundation for a community-generated national research blueprint for inherited bleeding disorders: Research priorities for mucocutaneous bleeding disorders
- 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
- 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
- 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
- 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
- Lived Experience Expert Editorial: Lived Experience Experts: A name created by us for us
- International Perspective: Soliciting international perspectives on an American national research agenda for inherited bleeding disorders
