Rare Disease Research Training Program

罕见病研究培训计划

• 批准号: 9145741
• 负责人: Debra S Regier
• 金额: $ 9.52万
• 依托单位: CHILDREN'S RESEARCH INSTITUTE
• 依托单位国家: 美国
• 财政年份: 2015
• 资助国家: 美国
• 起止时间: 2015-09-18 至 2020-06-30
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/9145741
• 关键词: Academic Medical Centers; Address; Affect; Award; Biochemical; Bioethics; Biotechnology; Career Choice; Chalk; Child; Childhood; Clinical; Clinical Research; Collaborations; Complement; Course Content; Development; Disease; Doctor of Philosophy; Educational Curriculum; Educational process of instructing; Educational workshop; Evaluation; Event; Exposure to; Faculty; Family; Funding; Genetic; Goals; Grant; Hour; Individual; Internist; Knowledge; Learning; Measures; Medical; Mentored Clinical Scientist Development Program; Mentors; Methodology; Methods; Modeling; Monitor; Network-based; Neurologist; Nurse Practitioners; Nutritionist; Online Systems; Outcome; Participant; Patients; Pharmacologic Substance; Policies; Pre-Post Tests; Programmed Instruction; Protocols documentation; Publications; Rare Diseases; Research; Research Design; Research Methodology; Research Personnel; Research Project Grants; Research Training; Resources; Scientist; Series; Site; System; Testing; Therapeutic; Time; Training; Training Programs; Translational Research; United States National Institutes of Health; Universities; Videoconferences; Videoconferencing; Work; Writing; analytical tool; base; career development; certificate program; design; experience; interest; investigator training; medical specialist; meetings; novel therapeutics; patient advocacy group; pediatrician; peer; programs; public health relevance; research and development; research study; statistics; success; symposium; teacher mentor; tool; web portal

 DESCRIPTION (provided by applicant): 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 is sponsored by the NIH funded Rare Diseases Clinical Research Network (RDCRN), a collaborative of 240 sites at universities and academic medical centers. It is hoped that this program will attract and retain new investigators in the field of rare disease through network development, research success, and exposure to patient and family groups. The RDCRN Certificate Program in RDR 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 post- test 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 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.

 描述(申请人提供)：罕见疾病(RD)是一个独特的医学领域，由大量的疾病组成，每个疾病的患者人数都很少。它们主要影响儿童，大多数都有遗传基础。罕见病研究(RDR)中使用的方法需要与更常见的情况不同的模型和分析工具。这项提议寻求开发一种混合学习课程，使用针对罕见疾病的独特工具和方法。它是由美国国立卫生研究院资助的罕见疾病临床研究网络(RDCRN)赞助的，该网络由大学和学术医学中心的240个站点组成。希望通过网络开发、研究成功以及接触患者和家庭群体，该计划将吸引和留住罕见疾病领域的新研究人员。RDR中的RDCRN证书课程是一学年提供的课程，包括6个组成部分：1.)最初为期两天的面对面的RDR方法和政策研讨会，将把学员、导师、患者权益倡导团体、联邦机构和生物技术/制药公司聚集在一起。2.)每半个月举办一次互动式网络研讨会，内容包括研究设计、统计学、生物伦理学、治疗学、政策以及与该领域其他主要贡献者互动的方法，其中包括患者权益倡导团体、联邦机构和生物技术/制药公司。3.)每半个月举行一次“粉笔板”视频会议，重点是个别受训人员的RDR项目，重点是实用的设计和分析工具。4)提供课程内容、学员之间以及学员与教师/导师之间的互动机会以及其他互动功能的门户网站。5)每个受训者在每半年一次的RDCRN会议上发表的“顶峰”研究项目报告，该会议将把受训者、患者权益倡导团体、联邦机构和生物技术/制药公司聚集在一起。6.)利用CTSA跟踪和评估系统以及前后测试来量化课程前后对RDR中涉及的主题的理解。这些工具还将用于监控程序和教学的质量。我们将通过跟踪受训者在学术职业选择、在RDR领域的留存和其他成功衡量标准方面来跟踪项目的结果。每年将有25到25名实习生入学。该计划包括80个小时的直接教学和至少40个小时的准备工作(家庭作业)，以及起草和实施研究性学习方案并撰写结果所需的工作。完成课程的学员将获得RDCRN颁发的RDR证书。