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Introduction: Decades of inherited bleeding disorders (BD) research transformed severe haemophilia from a childhood killer to a disorder managed across a full lifespan for many in economically developed countries. Health equity, a life unimpaired by disease complications, however, remains unimaginable for most people with an inherited BD (PWIBD). Aim: The National Hemophilia Foundation (NHF) and American Thrombosis and Hemostasis Network (ATHN) undertook the development of a community‐driven United States (US) National Blueprint for Inherited Bleeding Disorders Research to transform the experience of all PWIBD and those who care for them. Methods: Extensive community consultations were conducted to identify the issues most important to PWIBD and those who love and care for them. Expert multidisciplinary teams distilled these key areas of need into prioritised research questions, and identified the resources and infrastructure required to pursue them. A summit was held to gather feedback and inform the detailed blueprint. Results: Community‐prioritised research areas fell into three broad categories: issues common across inherited BDs, those specific to individual disorders, and issues of infrastructure and capacity. NHF State of the Science Research Summit discussions of the research questions derived from the community priorities by six working groups provided important input for the drafting of the research blueprint for the coming decades. Conclusion: The inherited BD community came together to develop the US National Blueprint for Inherited Bleeding Disorders Research dedicated to transforming the lives of all PWIBD including innovating solutions for the rarest disorders and under‐represented populations. [ABSTRACT FROM AUTHOR]

The ways in which the social drivers of health, also known as the social determinants of health (SDOH), affect health outcomes for people with inherited bleeding disorders (PwIBDs) is unclear. This systematic review of the published literature examines the impact of SDOH on health outcomes in PwIBDs. Articles that included the following parameters in PubMed informed this study: published in English between 2011–2021; available in free full text; study population diagnosed with an inherited bleeding disorder; and study measured at least one of the clinical/non-clinical outcome measures: bleeding frequency, chronic pain, mortality, quality of life (QOL), and/or cost. The main findings from the 13 included articles emphasized the unmet need for reducing the economic burden with sustainable population health strategies and treatment options for PwIBDs. Rural location was also a significant contributor to both delayed diagnosis and decreased access to care. Furthermore, the need for a multidisciplinary comprehensive care team to address physical, psychosocial, and emotional needs of PwIBDs was raised as a priority target in the desire for equitable and optimal health. This systematic literature review suggests that the SDOH are associated with inferior health outcomes and may influence the clinical progression of inherited bleeding disorders. [ABSTRACT FROM AUTHOR]

Introduction: The inherited bleeding disorders (IBD) community has witnessed significant therapeutic advances recently, yet important gaps persist, particularly for those with rare disorders and historically underserved populations. Aims: ‐To create a national research blueprint agenda, led by the National Hemophilia Foundation (NHF), enhancing patient‐centric principles, accelerate research progress and address important gaps in care.‐To review critical gaps that remain to be addressed in women with IBDs, who face specific bleeding challenges. Methods: The NHF research blueprint research agenda was defined by input from across the community, including caregivers and patients who are considered subject matter experts of their IBD, research leaders, allied health professionals and specialists, and representatives of the biopharmaceutical industry. In addition, two medical experts in the field of IBDs performed a comprehensive review to address the knowledge gaps in women with IBDs. Results: Two foundational principles of the NHF blueprint are: (1) it must deliver on key issues that significantly impact the lives of those affected by IBDs, and (2) the priorities defined are relevant and actionable aimed to achieve health equity among all those affected by IBDs. A multidisciplinary approach is necessary for an optimal management of puberty, transition to adulthood and pregnancy. Even if strict guidelines are followed, recent studies show that women with IBDs are still facing a high burden. Conclusion: NHF blueprint will be issued in 2022. A specific research agenda is needed in women with IBDs to further improve their management and quality of life. [ABSTRACT FROM AUTHOR]