@article{5d9a347c03b54a59b1ebeea632f0ed90,
title = "Building the blueprint: Formulating a community-generated national plan for future research in inherited bleeding disorders",
abstract = "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.",
keywords = "advocacy, blueprint, community, health equity, inherited bleeding disorders, research",
author = "Valentino, {Leonard A.} and Witkop, {Michelle L.} and Santaella, {Maria E.} and Donna DiMichele and Michael Recht",
note = "Funding Information: Research reported in this publication was supported by the National Heart, Lung, and Blood Institute of the National Institutes of Health under Award Number R13HL158209. The content is solely the responsibility of the authors and does not necessarily represent the official views of the National Institutes of Health. Fiona Robinson, PhD provided professional medical writing support during manuscript development. The entire State of the Science Research Summit initiative and this manuscript were funded by National Hemophilia Foundation. Funding Information: 12–15 September 2021, NHF hosted the State of the Science (virtual) Research Summit (SOSRS), an opportunity for all community members to weigh in on the research questions and infrastructure initiatives prioritised by the WGs. The Summit, supported entirely by NHF, with no funding from industry or commercial partners, was open to all without charge. NHF, ATHN, and partner social media and email campaigns featured SME and HCP testimonials emphasising the opportunity to direct inherited BD research towards PWIBD priorities (Figure S1 ). The NHF SOSRS was well attended with 441 unique attendees from all stakeholder groups (Table S5 ). Funding Information: LAV, MES, and MLW state that they have no interests which might be perceived as posing a conflict or bias. The opinions expressed in this manuscript by LAV are his own and not necessarily reflective of those of the National Hemophilia Foundation or its Board of Directors. DDM acted as a paid consultant to the National Hemophilia Foundation in the design, organisation, and execution of the State of the Science Research Summit on Inherited Bleeding Disorders. MR's employers have received research support from Bayer, BioMarin, CSL Behring, Genentech, Grifols, Hema Biologics, LFB, Novo Nordisk, Octapharma, Pfizer, Sanofi, Spark, Takeda, and uniQure. MR has acted as a paid consultant to Catalyst Biosciences, CSL Behring, Genentech, Hema Biologics, Kedrion, Novo Nordisk, Octapharma, Pfizer, Sanofi, Takeda, and uniQure. He is on the Board of Directors of the Foundation for Women and Girls with Blood Disorders and Partners in Bleeding Disorders. Publisher Copyright: {\textcopyright} 2022 The Authors. Haemophilia published by John Wiley & Sons Ltd.",
year = "2022",
doi = "10.1111/hae.14588",
language = "English (US)",
journal = "Haemophilia",
issn = "1351-8216",
publisher = "Wiley-Blackwell",
}