Series available in English and Spanish - Created in partnership with the FDA and Critical Path InstituteWASHINGTON, Oct. 16, 2023 /PRNewswire/ -- Today, the National Organization for Rare Disorders (NORD®) launched a new education series in English and Spanish titled, 'Rare Disease Drug Development: What Patients and Advocates Need to Know,' designed to help patients and patient advocacy groups understand the drug development process..

英文和西班牙文系列-与FDA和Critical Path InstituteWASHINGTON合作创建，2023年10月16日/PRNewswire/-今天，国家罕见疾病组织（NORD®）推出了一个新的英文和西班牙文教育系列题为“罕见病药物开发：患者和倡导者需要知道的，“旨在帮助患者和患者代表团体了解药物开发过程。。

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'The goal of this educational series is to engage the rare disease community on their essential role in the drug development process and ultimately help produce more and better treatments for rare disease,' said Rebecca Aune, Director of Education Programs at the National Organization for Rare Disorders.

国家罕见病组织教育项目主任丽贝卡·阿恩（Rebecca Aune）说：“这个教育系列的目标是让罕见病社区参与其在药物开发过程中的重要作用，并最终帮助他们为罕见病提供更多更好的治疗方法。疾病。

'It was created to address key educational needs and gaps with input from patient organizations, patient advocates, and FDA reviewers, all of whom have a variety of experience with the drug development process for rare diseases. Moreover, this series is presented in the two most spoken languages in the United States.'.

“它的创建是为了解决关键的教育需求和患者组织，患者支持者和FDA审查人员的意见差距，他们都有罕见疾病药物开发过程的各种经验。此外，本系列还以美国两种口语表达。

New education series in English and Spanish: 'Rare Disease Drug Development: What Patients and Advocates Need to Know.

英语和西班牙语的新教育系列：“罕见疾病药物开发：患者和倡导者需要知道的内容。

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Each module is available in a mix of formats to accommodate multiple learning styles, including animated videos, expert interviews, infographics, fact sheets, checklists, and interactive quizzes. The first modules, on 'Drug Development Process', 'Stakeholder Roles in Drug Development' and 'Natural History Studies,' are now available for free at learn.rarediseases.org..

每个模块都有多种格式可供选择，以适应多种学习风格，包括动画视频，专家访谈，信息图表，情况说明书，清单和交互式测验。关于“药物开发过程”，“利益相关者在药物开发中的作用”和“自然历史研究”的第一个模块现在可以在learn.rarediseases.org免费获得。。

Future modules, to be rolled out in 2024, will cover 'Patient Experience Data,' 'Designing Trials for Small Populations,' 'Clinical Trial Endpoints and Clinical Outcome Assessments,' and more. Each module is designed to be valuable on its own, and together they provide a comprehensive understanding of the drug development process, the role of the patient, and the evidence needed to demonstrate that a new treatment for a rare disease works.The curriculum is a collaboration between NORD, the U.S.

将于2024年推出的未来模块将涵盖“患者体验数据”，“小群体设计试验”，“临床试验终点和临床结果评估”等。每个模块本身都是有价值的，它们共同提供对药物开发过程，患者角色以及证明罕见疾病的新疗法有效所需证据的全面了解。课程是美国诺德之间的合作。

Food and Drug Administration (FDA), and the Critical Path Institute (C-Path) funded through the Rare Disease Cures Accelerator—Data and Analytics Platform (RDCA-DAP). It was announced today by NORD President and CEO Peter L. Saltonstall at the organization's 2023 Rare Diseases and Orphan Products Breakthrough Summit® to an audience of patient advocates, researchers, drug developers, and regulators, including FDA Commissioner Robert M.

食品药品监督管理局（FDA）和由Rare Disease Cures Accelerator数据和分析平台（RDCA-DAP）资助的Critical Path Institute（C-Path）。这项研究今天由北欧总裁兼首席执行官Peter L.Saltonstall在该组织的2023年罕见疾病和孤儿产品突破峰会上宣布，由患者支持者，研究人员，药物开发者和监管机构（包括FDA专员Robert M。

Califf, M.D., who will address Summit attendees tomorrow morning.'C-Path is proud to support this education series and it's great to see the roll out begin,' said Collin Hovinga, Pharm.D., M.S., FCCP, Vice President of Rare and Orphan Disease Programs at C-Path. 'We're committed to listening to people with lived experience as their insight, experiences, and perspectives play a vital role in drug development for rare diseases.

Califf，M.D.，明天早上将向峰会与会者发表演讲。”C-Path很自豪地支持这个教育系列，看到推出的开始非常重要，“C-Path罕见和孤儿疾病项目副总裁，医学博士，FCCP的Collin Hovinga说。”我们致力于倾听具有丰富经验的人们，因为他们的洞察力，经验和观点在罕见疾病的药物开发中发挥着至关重要的作用。

C-Path's rare and orphan programs, including RDCA-DAP, rely on patient data for progress and their contributions are not just data points to us; they are the heartbeat of the mission to create efficient treatments for all.'There are more than 7,000 different rare diseases, of which 95% lack any FDA-approved treatment.  Rare diseases impact small patient populations of fewer than 200,000 people in the United States—sometimes far.

C-Path的罕见和孤儿计划，包括RDCA-DAP，依靠患者数据取得进展，他们的贡献不仅仅是数据点给我们；它们是为所有人创造有效治疗的使命的心跳有7000多种不同的罕见疾病，其中95%缺乏FDA批准的治疗方法。罕见疾病有时会影响美国不到20万人的小患者人群。