System Wide Operations for Rare Disorders (SWORD)

项目摘要/摘要 标题：针对罕见疾病的全系统手术(剑) 了解和开发治疗人类罕见疾病的新疗法有许多 挑战。它们是如此罕见的事实意味着可以参与的患者很少 疾病临床研究网络(RDCRN)和其他研究由于 每项研究的地点数量有限，以及患者必须走多远才能到达 这些网站中的一个。新冠肺炎大流行带来的一件好事是 接受临床护理和研究都可以通过以下方式远程进行 远程医疗和将记录和文档邮寄到远程站点。虽然许多组件 研究研究或临床试验可以远程进行，有些方面需要实际人员- 面对面的接触。这些组件之一是抽血、处理和运送到核心 研究实验室遵循研究方案。生物标记物研究抽血的主要障碍 包括IRB监督和向符合以下条件的机构偿还费用的漫长过程 在离他们居住地更近的地方为少数患者设立一个网站是非常不切实际的。 作为我们BVMC6211项目解决这一问题的手段，我们在这里提出了开发 之剑：所有CTSA程序的网络，能够提取和处理血液 样本充当罕见疾病研究和试验的患者门户。剑代表着 《罕见疾病的全系统手术》。 这项建议将利用我们的BVMC 6211研究，并为传播我们与 “韦伯基金会”(SWF)传播利剑，用鲜血应对挑战 根据美国国立卫生研究院资助的多个地点的研究方案，提取、处理并运往核心实验室 学习。剑术的总体目标是开发和传播一种有效的方法来增强 参加需要为患者进行静脉采血、标本处理和运送的研究 参加远离招生地点的RDRCN学习。以剑取胜 抽血可能会迅速扩展到其他服务，包括放射科和获得其他 生物标本。

PROJECT SUMMARY/ABSTRACT Title: System Wide Operations for Rare Disorders (SWORD) Understanding and developing new treatments for human rare disorders presents many challenges. The fact that they are so rare means that patients who could participate in Rare Diseases Clinical Research Network (RDCRN) and other studies do not participate due to a limited number of sites for each study and the distance the patient would have to travel to reach one of these sites. One good thing that has come from the COVID-19 pandemic is the general acceptance that both clinical care and research studies can be performed remotely through telehealth and mailing records and documents to a distant site. While many components of a research study or clinical trial can be conducted remotely, some aspects require actual person- to-person contact. One of these components is blood draws, process, and shipment to core study laboratory following study protocols. Major barriers to blood draws for biomarker studies include the lengthy process of both IRB oversight and reimbursements to institutions that make it highly impractical to set up a site for a handful of patients closer to where they reside. As a means to solve this problem for our BVMC6211 project, here we propose the development of ‘SWORD: A network of all CTSA programs capable of drawing and processing blood samples to act as a patient portal for rare disorder studies and trials. SWORD stands for “System Wide Operations for Rare Disorders”. This proposal will leverage our BVMC 6211 study and for dissemination our partnership with the “Sturge Weber Foundation” (SWF) to disseminate SWORD and address challenges with blood draws, processing, and shipping to core lab as per study protocol for NIH funded multi-sites studies. The overall goal of SWORD is to develop and disseminate an efficient way to enhance enrollment in studies requiring phlebotomy and specimen processing and shipping for patients participating in RDRCN studies who live far from enrollment sites. Success with SWORD for blood draws could quickly expand to other services including radiology and for obtaining other biological specimens.