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.

摘要 这次为期三天的科学会议;“C路径科学突破会议：解决未满足的 通过合作伙伴关系解决得不到充分服务的药物开发领域的需求和挑战”， 汇集了来自行业和学术研究人员的跨学科团队的代表， 患者群体和监管机构-讨论新生儿药物开发挑战 药物，α-1抗胰蛋白酶缺乏症（AATD）和溶酶体疾病（LD），并集思广益如何 竞争前的公私伙伴关系（PPP）可以帮助推动这些国家的药物开发， 服务不足的人群。每年在美国，10%的新生儿早产， 改善这一弱势群体的生存和结果的迫切需求尚未得到满足。2015年，FDA 与C-Path合作创建了国际新生儿联盟（INC），这是一个由行业领导者组成的PPP， 学术研究人员，监管机构，家庭和护士，以推进医疗产品 FDA和其他全球监管机构认可的开发工具。AATD是一种临床 与成人慢性肝病风险增加相关的认识不足的疾病， 慢性阻塞性肺疾病（COPD）的病因有哪些？ 吸烟。AATD中未满足的需求涉及AATD的诊断、评价和治疗-对于 肝脏和肺部表现。为了克服缺乏工具， 全面客观地评估《消除对妇女一切形式歧视公约》的成果，《消除对妇女一切形式歧视公约》的关键路径（CPA-1） 涉及来自工业界、学术界、患者团体和监管机构的利益相关者， 目标是为AATD药物开发提供可行的解决方案或工具。溶酶体疾病 (LDs)是一组遗传性代谢紊乱，由编码以下疾病的基因突变引起： 参与溶酶体中大分子分解的酶。与这一群体有关的挑战 罕见疾病的发病率与早期发现、缺乏有效的诊断工具和筛查方案有关， 缺乏有效的治疗。溶酶体疾病的关键路径（CPLD）是PPP，包括 来自制药公司的利益相关者，对LD药物感兴趣的学术科学家 开发、患者群体和监管代表。本次会议将展示工作 由INC、CPA-1和CPLD完成，并利用现有的强大竞争前关系 在药物开发界的各种利益攸关方之间， 公开可用的药物开发工具，旨在提高医疗效率和安全性 产品开发