2021 State of the Science Research Summit

项目摘要/摘要 国家血友病基金会(NHF)有70多年的经验，倡导为 患有多发性遗传性出血性疾病(IBD)。随着IBD患者的需求与两者的关系发生变化 诊断和治疗选择方面的进步，以及人口日益多样化(例如，族裔/种族， 年龄、性别/性别)，一种确定差距和关切的综合方法将确保患者获得 最佳护理。例如，对IBD生物医学研究的追求往往优先于一种出血性疾病。 另一项关于IBD对患者和照顾者的心理社会影响的研究。虽然这件事 这种方法使一些人的医疗保健发生了革命性的变化，它没有考虑到整个社区的需求， 尤其是在社区为基因治疗及其有史以来第一代老年患者做准备的时候。其中 这些发展的巨大，可能会失去耐心的声音，而研究人员往往依靠自己 确定IBD社区结果的科学专业知识而不是患者输入，导致滥用时间 和资金。 为了应对IBD社区内的这些巨大变化，利益相关者必须团结起来 病人。因此，在2021年第二季度(帐篷。5月)，NHF将持有科学研究状况 峰会，邀请IBD社区定义IBD研究和患者护理的下一个十年。目标是 这次为期两天的峰会的重点是通过放大不同的患者来打破IBD研究的传统方法 视角，使患者在创建统一的全国性研究时成为关键决策者 将由所有利益攸关方通过的议程。峰会的具体目标如下： 1.介绍了传统IBD研究的概况，以及Pre-Pre-Pre的汇总结果 会议利益攸关方倡议 2.利用分组工作组确定、定义和确定以下研究优先事项(4-6) 出席会议的人员 3.确定与新制定的议程相关的所有利益攸关方的角色，并为其分配角色 这次峰会反映了NHLBI的使命，将改善这些人的健康和生活质量 以患者群体需求为中心，以社区为中心诊断出IBD 对价。除了以患者为中心的IBD议程外，峰会还将允许建立一个 其他罕见疾病组织可以用来开发自己的以患者为中心和疾病的模板- 具体的会议和议程。

Project Summary/Abstract The National Hemophilia Foundation (NHF) has over 70 years of experience advocating for persons with multiple inherited bleeding disorders (IBD). As the needs of IBD patients evolve in relation to both advancements in diagnostics and treatment options, and an increasingly diverse population (e.g. ethnicity/race, age, gender/sex), a comprehensive approach to identifying gaps and concerns will ensure patient access to optimal care. For example, pursuits in IBD biomedical research often prioritize one bleeding disorder over another and eclipse studies into the psychosocial impacts of IBD on patients and caregivers. Though this method has revolutionized medical care for some, it fails to consider the needs of the entire community, especially as the community prepares for gene therapy and its first-ever generation of aging patients. Among the enormity of these developments, the patient voice can be lost, and researchers too often rely on their own scientific expertise rather than patient input to determine IBD community outcomes, resulting in misused time and funding. To respond to these monumental shifts within the IBD community, stakeholders must coalesce around patients. As such, in the second quarter of 2021 (tent. May), NHF will hold the State of the Science Research Summit, inviting the IBD community to define the next decade of IBD research and patient care. The objective of this two-day Summit is to dismantle traditional approaches to IBD research by amplifying a diverse patient perspective, enabling patients to serve as key decision makers in the creation of an aligned, national research agenda to be adopted by all stakeholders. Specific Summit aims are as follows: 1. Present an overview of traditional IBD research as well as the aggregated results of pre- Conference stakeholder initiatives 2. Utilize breakout working groups to identify, define, and prioritize research priorities (4-6) among conference attendees 3. Determine and assign roles to all stakeholders as they relate to the newly established agenda Mirroring the mission of the NHLBI, this Summit will improve upon the health and quality of life of those diagnosed with IBD by centering on the needs of the patient population as the community’s foremost consideration. In addition to the IBD patient-centric agenda, the Summit will allow for the creation of a template that other rare disease organizations may use to develop their own patient-centric and disease- specific conferences and agendas.