2021 State of the Science Research Summit

项目总结/摘要 国家血友病基金会（NHF）拥有超过70年的经验， 多发性遗传性出血性疾病（IBD）随着IBD患者需求的发展， 诊断和治疗选择的进步，以及日益多样化的人口（例如种族/人种， 年龄、性别/性别），采用全面的方法来确定差距和问题，将确保患者获得 最佳护理例如，IBD生物医学研究的追求通常优先考虑一种出血性疾病， 另一个和Eclipse研究IBD对患者和护理人员的心理社会影响。虽然这 这种方法对一些人来说是一场医疗改革，它没有考虑到整个社会的需要， 尤其是在社区为基因治疗和第一代老年患者做准备的时候。之间 由于这些发展的不确定性，病人的声音可能会丢失，研究人员往往依赖自己的声音。 科学专业知识，而不是患者输入，以确定IBD社区结果，导致滥用时间 和资金。 为了应对IBD社区内的这些巨大变化，利益相关者必须联合起来， 患者因此，于二零二一年第二季度（帐篷。5月），NHF将举办国家科学研究 邀请IBD社区定义IBD研究和患者护理的下一个十年。客观 这个为期两天的峰会的目的是通过放大不同的患者来拆除IBD研究的传统方法， 的观点，使患者能够作为关键决策者，在创建一个统一的，国家研究 议程将由所有利益攸关方通过。首脑会议的具体目标如下： 1.介绍了传统IBD研究的概述，以及研究前的汇总结果， 会议利益攸关方倡议 2.利用分组工作组来确定、定义和优先考虑研究优先事项（4-6）， 会议与会者 3.确定和分配所有利益攸关方在新制定的议程中的作用 为了体现NHLBI的使命，本次峰会将改善这些人的健康和生活质量。 通过以患者人群的需求为中心， 考虑.除了以IBD患者为中心的议程外，峰会还将允许创建一个 其他罕见疾病组织可以用来开发自己的以患者为中心的 具体会议和议程。

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.