Abstract：
The National Hemophilia Foundation (NHF) conducted extensive, inclusive community consultations to guide prioritization of research in coming decades in alignment with its mission to find cures and address and prevent complications enabling people and families with blood disorders to thrive.
With the American Thrombosis and Hemostasis Network, NHF recruited multidisciplinary expert working groups (WG) to distill the community-identified priorities into concrete research questions and score their feasibility, impact, and risk. WG6 was charged with identifying the infrastructure, workforce development, and funding and resources to facilitate the prioritized research. Community input on conclusions was gathered at the NHF State of the Science Research Summit.
WG6 detailed a minimal research capacity infrastructure threshold, and opportunities to enable its attainment, for bleeding disorders centers to participate in prospective, multicenter national registries. They identified challenges and opportunities to recruit, retain, and train the diverse multidisciplinary care and research workforce required into the future. Innovative collaborative approaches to trial design, resource networking, and funding to surmount obstacles facing research in rare disorders were elucidated.
The innovations in infrastructure, workforce development, and resources and funding proposed herein may contribute to facilitating a National Research Blueprint for Inherited Bleeding Disorders.
Research is critical to advancing the diagnosis and care of people with inherited bleeding disorders (PWIBD). This research requires significant infrastructure, including people and resources. Hemophilia treatment centers (HTC) need many different skilled care professionals including doctors, nurses, and other providers; also statisticians, data managers, and other experts to process patients’ clinical information into research. Attracting diverse qualified professionals to the clinical and research work requires long-term planning, recruiting individuals in training programs and retaining them as they become experts. Research infrastructure includes physical servers running database software, networks that link them, and the environment in which these components function. US Centers for Disease Control and Prevention (CDC) and American Thrombosis and Hemostasis Network (ATHN) coordinate and fund data collection at HTCs on the health and well-being of thousands of PWIBD into a registry used in research studies.National Hemophilia Foundation (NHF) and ATHN asked our group of health care professionals, technology experts, and lived experience experts (LEE) to identify the infrastructure, workforce, and resources needed to do the research most important to PWIBD. We identified the types of CDC/ATHN studies all HTCs should be able to perform, and the physical and human infrastructure this requires. We prioritized finding the best clinical trial designs to study inherited bleeding disorders, identifying ways to share personnel and tools between HTCs, and innovating how research is governed and funded. Involving LEEs in designing, managing, and carrying out research will be key in conducting research to improve the lives of PWIBD.
With the American Thrombosis and Hemostasis Network, NHF recruited multidisciplinary expert working groups (WG) to distill the community-identified priorities into concrete research questions and score their feasibility, impact, and risk. WG6 was charged with identifying the infrastructure, workforce development, and funding and resources to facilitate the prioritized research. Community input on conclusions was gathered at the NHF State of the Science Research Summit.
WG6 detailed a minimal research capacity infrastructure threshold, and opportunities to enable its attainment, for bleeding disorders centers to participate in prospective, multicenter national registries. They identified challenges and opportunities to recruit, retain, and train the diverse multidisciplinary care and research workforce required into the future. Innovative collaborative approaches to trial design, resource networking, and funding to surmount obstacles facing research in rare disorders were elucidated.
The innovations in infrastructure, workforce development, and resources and funding proposed herein may contribute to facilitating a National Research Blueprint for Inherited Bleeding Disorders.
Research is critical to advancing the diagnosis and care of people with inherited bleeding disorders (PWIBD). This research requires significant infrastructure, including people and resources. Hemophilia treatment centers (HTC) need many different skilled care professionals including doctors, nurses, and other providers; also statisticians, data managers, and other experts to process patients’ clinical information into research. Attracting diverse qualified professionals to the clinical and research work requires long-term planning, recruiting individuals in training programs and retaining them as they become experts. Research infrastructure includes physical servers running database software, networks that link them, and the environment in which these components function. US Centers for Disease Control and Prevention (CDC) and American Thrombosis and Hemostasis Network (ATHN) coordinate and fund data collection at HTCs on the health and well-being of thousands of PWIBD into a registry used in research studies.National Hemophilia Foundation (NHF) and ATHN asked our group of health care professionals, technology experts, and lived experience experts (LEE) to identify the infrastructure, workforce, and resources needed to do the research most important to PWIBD. We identified the types of CDC/ATHN studies all HTCs should be able to perform, and the physical and human infrastructure this requires. We prioritized finding the best clinical trial designs to study inherited bleeding disorders, identifying ways to share personnel and tools between HTCs, and innovating how research is governed and funded. Involving LEEs in designing, managing, and carrying out research will be key in conducting research to improve the lives of PWIBD.
摘要：
未经批准：国家血友病基金会（NHF）进行了广泛的，包容性社区协商，以指导未来几十年的研究优先次序，以与其寻找治疗方法、解决和预防并发症的使命相一致，使患有血液疾病的人和家庭能够茁壮成长。
未经批准：美国血栓和止血网络，NHF招募了多学科专家工作组（WG），将社区确定的优先事项提炼成具体的研究问题，并对其可行性进行评分，影响，和风险。WG6负责识别基础设施，劳动力发展，以及促进优先研究的资金和资源。在NHF科学研究峰会上收集了社区对结论的投入。
UNASSIGNED：WG6详细说明了最小研究能力基础设施阈值，以及实现这一目标的机会，出血性疾病中心参与前瞻性，多中心国家登记册。他们确定了招聘的挑战和机遇，保留,并培训未来所需的多元化多学科护理和研究人员。试验设计的创新协作方法，资源网络，并阐明了克服罕见疾病研究面临的障碍的资金。
未经评估：基础设施方面的创新，劳动力发展，本文提出的资源和资金可能有助于促进遗传性出血疾病的国家研究蓝图。
研究对于推进遗传性出血性疾病（PWIBD）患者的诊断和护理至关重要。这项研究需要大量的基础设施，包括人和资源。血友病治疗中心（HTC）需要许多不同的熟练护理专业人员，包括医生，护士,和其他提供者；还有统计学家，数据管理人员,和其他专家将患者的临床信息处理为研究。吸引各种合格的专业人员到临床和研究工作需要长期规划，在培训计划中招募个人，并在他们成为专家时留住他们。研究基础设施包括运行数据库软件的物理服务器，连接它们的网络，以及这些组件发挥作用的环境。美国疾病控制和预防中心（CDC）和美国血栓形成和止血网络（ATHN）协调并资助HTC收集有关成千上万PWIBD的健康和福祉的数据，并将其纳入研究中使用的注册表。国家血友病基金会（NHF）和ATHN要求我们的医疗保健专业人员小组，技术专家,和现场体验专家(LEE)来识别基础设施，劳动力，以及对PWIBD最重要的研究所需的资源。我们确定了所有HTCs应该能够进行的CDC/ATHN研究的类型，以及这需要的物理和人力基础设施。我们优先考虑寻找最好的临床试验设计来研究遗传性出血性疾病，确定在HTCs之间共享人员和工具的方法，创新研究的管理和资助方式。让LEE参与设计，管理,开展研究将是开展研究以改善PWIBD生活的关键。
未经批准：美国血栓和止血网络，NHF招募了多学科专家工作组（WG），将社区确定的优先事项提炼成具体的研究问题，并对其可行性进行评分，影响，和风险。WG6负责识别基础设施，劳动力发展，以及促进优先研究的资金和资源。在NHF科学研究峰会上收集了社区对结论的投入。
UNASSIGNED：WG6详细说明了最小研究能力基础设施阈值，以及实现这一目标的机会，出血性疾病中心参与前瞻性，多中心国家登记册。他们确定了招聘的挑战和机遇，保留,并培训未来所需的多元化多学科护理和研究人员。试验设计的创新协作方法，资源网络，并阐明了克服罕见疾病研究面临的障碍的资金。
未经评估：基础设施方面的创新，劳动力发展，本文提出的资源和资金可能有助于促进遗传性出血疾病的国家研究蓝图。
研究对于推进遗传性出血性疾病（PWIBD）患者的诊断和护理至关重要。这项研究需要大量的基础设施，包括人和资源。血友病治疗中心（HTC）需要许多不同的熟练护理专业人员，包括医生，护士,和其他提供者；还有统计学家，数据管理人员,和其他专家将患者的临床信息处理为研究。吸引各种合格的专业人员到临床和研究工作需要长期规划，在培训计划中招募个人，并在他们成为专家时留住他们。研究基础设施包括运行数据库软件的物理服务器，连接它们的网络，以及这些组件发挥作用的环境。美国疾病控制和预防中心（CDC）和美国血栓形成和止血网络（ATHN）协调并资助HTC收集有关成千上万PWIBD的健康和福祉的数据，并将其纳入研究中使用的注册表。国家血友病基金会（NHF）和ATHN要求我们的医疗保健专业人员小组，技术专家,和现场体验专家(LEE)来识别基础设施，劳动力，以及对PWIBD最重要的研究所需的资源。我们确定了所有HTCs应该能够进行的CDC/ATHN研究的类型，以及这需要的物理和人力基础设施。我们优先考虑寻找最好的临床试验设计来研究遗传性出血性疾病，确定在HTCs之间共享人员和工具的方法，创新研究的管理和资助方式。让LEE参与设计，管理,开展研究将是开展研究以改善PWIBD生活的关键。
