Rare Diseases Clinical Research Scholar’s Program

项目名称：罕见病临床研究学者项目： 项目总结 罕见疾病(RD)作为一个医学领域是独一无二的，包括大量与 每一个都有少量的病人。它们主要影响儿童，大多数人有一种基因 基础。罕见病研究(RDR)中使用的方法需要模型和分析 工具不同于更常见的情况。这项提议寻求开发一种混合的 使用针对罕见疾病的特定工具和方法的学习课程。会的 继续与美国国立卫生研究院资助的罕见疾病临床研究网络密切合作 (RDCRN)，由大学和学术医学中心的240个站点组成的协作体。它是 预计这一计划将继续吸引和留住稀有领域的新调查人员 通过网络开发、研究成功以及与患者和家人接触而患上疾病 组。罕见病学者项目是一学年开设的课程 它有6个组成部分：1)最初为期两天的RDR方法和政策面对面研讨会 这将把实习生、导师、患者权益倡导团体、联邦机构和 生物技术/制药。2.)关于方法学的一系列半月一次的互动式网上研讨会 包括研究设计、统计学、生物伦理学、治疗学、政策和方法 与该领域的其他关键贡献者互动，包括患者倡导团体、联邦 机构和生物技术/制药公司。3.)每半个月举行一次的视频会议，重点是 个别受训人员RDR项目，重点是实用的设计和分析工具。4)a 提供课程内容、学员之间以及学员之间互动机会的门户网站 实习生和教师/导师，以及其他互动功能。5)“顶峰”研究项目 每名受训人员在RDCRN半年一次的会议上的演讲，该会议将汇集 受训者、患者权益倡导团体、联邦机构和生物技术/制药公司。6.)利用 CTSA跟踪和评估系统以及前后测试，以量化对 课程前后RDR中涉及的主题。这些工具还将用于 监控程序和指令的质量。我们将通过以下方式跟踪该计划的结果 跟踪学员的学术职业选择，在RDR领域的留存，以及 其他衡量成功的标准。每名学员将继续招收25至25名学员 年。该项目包括80小时的直接教学和至少40小时的准备工作 (作业)以及起草和开展研究性学习方案所需的工作，以及 把结果写下来。完成该计划的学员将从 RDCRN.

Project Title: Rare Diseases Clinical Research Scholar's Program: PROJECT SUMMARY Rare diseases (RD) are unique as a medical field, comprising a large number of disorders with a small number of patients in each. They primarily affect children and most have a genetic basis. The methods employed in rare disease research (RDR) require models and analytical tools that are different from more common conditions. This proposal seeks to develop a blended learning curriculum employing specific tools and methods unique to rare diseases. It will continue to be work closely with the NIH funded Rare Diseases Clinical Research Network (RDCRN), a collaborative of 240 sites at universities and academic medical centers. It is expected that this program will continue to attract and retain new investigators in the field of rare disease through network development, research success, and exposure to patient and family groups. The Rare Disease Scholar's Program is a curriculum provided over an academic year that has 6 components: 1.) an initial two day face-to-face RDR methods and policy workshop that will bring together the trainees, mentors, patient advocacy groups, federal agencies and biotech/pharma. 2.) a series of semi-monthly interactive, web-based seminars on methodologies in RDR including study design, statistics, bioethics, therapeutics, policy and approaches to interacting with other key contributors to the field, including patient advocacy groups, federal agencies and biotech/pharma. 3.) a semi-monthly “chalk board” videoconference focusing on individual trainee RDR projects with an emphasis on practical design and analytical tools. 4) a web portal with course content, opportunities for interaction among trainees and between trainees and teachers/mentors, and other interactive features. 5) a “capstone” research project presentation by each of the trainees at the semi-annual RDCRN meeting that will bring together the trainees, patient advocacy groups, federal agencies and biotech/pharma. 6.) Utilizing the CTSA tracking and evaluation system and a pre- and posttest to quantify understanding of the topics involved in RDR prior to and following the curriculum. These tools will also be used to monitor the quality of the program and instruction. We will track the program's outcome by following the trainees in terms of academic career choice, retention in the field of RDR, and other measures of success. Twenty to twenty five trainees will continue to matriculate each year. The program involves 80 hours of direct teaching and at least 40 hours of prep work (homework) as well as the work required to draft and carry out a research study protocol, and write up the results. Trainees completing the program will receive a certificate in RDR from the RDCRN.