Rare Diseases Clinical Research Scholar’s Program

项目名称：罕见病临床研究学者项目： 项目摘要 罕见疾病（RD）是一个独特的医学领域，包括大量的疾病， 每一个病人的数量都很少。它们主要影响儿童，大多数具有遗传性 基础罕见病研究（RDR）中采用的方法需要模型和分析 这些工具不同于更常见的条件。该提案旨在开发一种混合的 使用罕见疾病特有的特定工具和方法学习课程。它将 继续与NIH资助的罕见疾病临床研究网络密切合作 （RDCRN），一个在大学和学术医疗中心的240个站点的合作。是 预计这一计划将继续吸引和留住新的研究人员在该领域的罕见的 通过网络发展、研究成功以及接触患者和家人来预防疾病 组罕见疾病学者计划是一个学年提供的课程， 它有6个组成部分：1）。最初为期两天的面对面RDR方法和政策研讨会 这将汇集学员，导师，病人倡导团体，联邦机构和 生物技术/制药2.）关于方法的一系列半月一次的网上互动研讨会 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.）第三章半月一次的“粉笔板”视频会议， 培训学员个人RDR项目，重点是实用设计和分析工具。4)一 提供课程内容的门户网站，学员之间以及 学员和教师/导师，以及其他互动功能。5)“顶点”研究项目 每个学员在半年度RDCRN会议上的演讲， 受训人员、患者权益团体、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量化对 在RDR之前和之后的课程涉及的主题。这些工具还将用于 监督课程和教学的质量。我们将跟踪该计划的结果， 跟踪学员的学术职业选择、在RDR领域的保留情况， 其他成功的衡量标准。20至25名学员将继续录取， 年该计划包括80小时的直接教学和至少40小时的准备工作 （家庭作业）以及起草和执行研究方案所需的工作，以及 把结果写出来。完成该计划的学员将获得RDR证书 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.