C-Path Scientific Breakthrough Conference: Addressing unmet needs and challenges in underserved drug development areas through collaborative partnerships

C-Path 科学突破会议：通过合作伙伴关系解决服务不足的药物开发领域未满足的需求和挑战

• 批准号: 10827777
• 负责人: Kanwaljit Singh
• 金额: $ 15万
• 依托单位: CRITICAL PATH INSTITUTE
• 依托单位国家: 美国
• 财政年份: 2023
• 资助国家: 美国
• 起止时间: 2023-07-01 至 2024-06-30
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/10827777
• 关键词: Path; Scientific; Breakthrough; Conference; Addressing

ABSTRACT This three-day scientific conference; “C-Path Scientific Breakthrough Conference: Addressing unmet needs and challenges in underserved drug development areas through collaborative partnerships” will bring together representatives from an interdisciplinary team of industry and academic researchers, patient groups, and regulatory agencies – to discuss drug development challenges in neonatal medicine, alpha-1 antitrypsin deficiency (AATD), and lysosomal diseases (LD), and brainstorm how pre-competitive public private partnerships (PPPs) can help advance drug development in these underserved populations. Each year in the U.S., 10% of neonates are born preterm and there is an urgent unmet need to improve survival and outcome in this vulnerable population. In 2015 the FDA collaborated with C-Path to create International Neonatal Consortium (INC), a PPP of industry leaders, academic researchers, regulatory agencies, families, and nurses to advance medical product development tools for endorsement by FDA and other global regulatory agencies. AATD is a clinically under-recognized disease associated with an increased risk of chronic liver disease in adults and children and is the leading cause of chronic obstructive pulmonary disease (COPD) in adults, outside of smoking. Unmet needs in AATD pertain to both diagnosis, evaluation, and treatment of AATD – for both hepatic and pulmonary manifestations. To overcome the lack of tools that provide both comprehensive and objective assessment of outcomes in AATD, the Critical Path for AATD (CPA-1) has involved stakeholders from industry, academia, patient groups, and regulatory agencies, with the objective to achieve actionable solutions or tools for AATD drug development. Lysosomal diseases (LDs) are a group of inherited metabolic disorders caused by mutations in genes that code for enzymes involved in the breakdown of macromolecules in lysosomes. Challenges related to this group of rare diseases relate to early detection, lack of effective diagnostic tools and screening programs, and lack of effective treatments. Critical Path for Lysosomal Diseases (CPLD) is a PPP comprised of stakeholders from pharmaceutical companies, academic scientists with an interest in LD drug development, patient groups, and regulatory representatives. This conference will showcase the work done by INC, CPA-1, and CPLD and capitalize on strong existing pre-competitive relationships between the various stakeholders in the drug development communities to advance efforts to produce publicly available drug development tools aiming at improving the efficiency and safety of medical product development.

抽象的 这个为期三天的科学会议； “ C-Path的科学突破会议：未满足的解决 通过协作伙伴关系，服务不足的药物开发领域的需求和挑战”将会 汇聚一组工业跨学科团队和学术研究人员的代表 患者组和监管机构 - 讨论新生儿的药物开发挑战 药物，α-1抗胰蛋白酶缺乏症（AATD）和溶酶体疾病（LD）和集思广益 竞争前公共合作伙伴关系（PPP）可以帮助推进这些药物开发 服务不足的人群。在美国，每年，有10％的新生儿是早产的，有一个 紧急未满足的需求可以改善这一脆弱人群的生存和结果。 2015年FDA 与C-Path合作创建了国际新生儿财团（INC），该联盟（INC）是行业领导者的PPP， 学术研究人员，监管机构，家庭和护士推进医疗产品 FDA和其他全球监管机构认可的开发工具。 AATD是诊所 与成人慢性肝病的风险增加有关的公认疾病不足 儿童，是成人慢性阻塞性肺疾病（COPD）的主要原因 吸烟。 AATD中未满足的需求与AATD的诊断，评估和治疗有关 - 用于 肝和肺部表现。克服缺乏提供两者的工具 对AATD的结果的全面和客观评估，AATD的关键途径（CPA-1） 涉及来自行业，学术界，患者群体和监管机构的利益相关者与 目的是实现AATD药物开发的可行解决方案或工具。溶酶体疾病 （LDS）是一组由基因中的突变引起的一组代谢性疾病 参与溶酶体大分子分解的酶。与这个小组有关的挑战 罕见疾病与早期发现有关，缺乏有效的诊断工具和筛查程序， 缺乏有效的治疗方法。溶酶体疾病（CPLD）的关键路径是累积的PPP 制药公司的利益相关者，对LD药物感兴趣的学术科学家 发展，患者组和监管代表。这次会议将展示工作 由Inc，CPA-1和CPLD完成，并利用牢固的现有前竞争关系 在药物开发社区的各个利益相关者之间，以促进生产努力 公开可用的药物开发工具旨在提高医疗的效率和安全性 产品开发。