Coordinating Center for the NHLBI Catalyze Program

NHLBI 催化计划协调中心

• 批准号: 10460683
• 负责人: Diana M Severynse-Stevens
• 金额: $ 191.88万
• 依托单位: RESEARCH TRIANGLE INSTITUTE
• 依托单位国家: 美国
• 财政年份: 2020
• 资助国家: 美国
• 起止时间: 2020-09-15 至 2025-08-31
• 项目状态: 未结题
• 来源: https://reporter.nih.gov/project-details/10460683
• 关键词: Coordinating; Center; NHLBI; Catalyze; Program

The gap in funding and support for the transition of products from bench research to clinical application often results in the loss of potentially important discoveries. This translational gap, also called the “Valley of Death,” has been an area of increased biomedical spending by the National Institutes of Health (NIH) during the past two decades. The National Heart, Lung, and Blood Institute (NHLBI) recognized the need for programs to foster translational research nearly 20 years ago, starting with the Production Assistance for Cellular Therapies program, followed by the Gene Therapy Resource Program, Science Moving Towards Research Translation and Therapy (SMARTT), and the National Institutes of Health Centers for Accelerated Innovations (NCAI) These programs were designed to provide a diverse array of services to accelerate the development of novel products into clinical application and commercialization. The Catalyze Program continues NHLBI’s support of translational science through funding, technical support, and skills development to ensure that investigators can successfully advance novel therapies and platform technologies for heart, lung, blood, and sleep diseases and disorders. This program will merge the best practices from two decades of translational programs to become a model for mentoring investigators and advancing translational research. Catalyze will provide technical guidance from discovery through Investigational New Drug (IND)–enabling studies, as well as assist investigators in the development of milestones, identify the optimal regulatory pathway, and build a commercial strategy for their product. Each investigator in the Catalyze Program will be mentored by an experienced technical team with the expectation that they will use their knowledge not only to develop a novel technology or therapeutic, but also to train the next generation of translational scientists. As the Catalyze Coordinating Center (CC), RTI International will draw on 20 years of experience in translational research to provide exceptional operational and technical leadership. Through our experience as the CC for SMARTT and the independent evaluator of NCAI and REACH, we have a unique insight into best practices and challenges in translational programs and will apply that knowledge to ensure that Catalyze provides a comprehensive development support system for investigators and an efficient, strategic, and flexible program for NHLBI. We recognize that the success of Catalyze will depend on the success of the investigators in the program. As the CC for SMARTT, we were able to work with investigators from across the country effectively by having frequent communication and providing an atmosphere of collaboration. The key to the success of the SMARTT program was to customize our team and our approach to meet the needs of each investigator. Through comprehensive discussions with each investigator and their team, we were able determine their level of knowledge/experience in product development, the gaps/needs in the program, and the communication style that worked best with each investigator. The success of this approach for SMARTT is evident in the number of products that advanced along the development path, including 25 manufacturing campaigns, 18 pharmacology/toxicology packages completed, 7 INDs cleared, 11 pre-IND meetings, 2 IND amendments, 4 Orphan Drug Designation applications approved, and 4 pharmaceutical partnerships or licensing deals. As the CC for Catalyze, we will take a similar approach to ensure that each investigator receives the support and services tailored to him/her. We will approach our role in providing investigators with project management and technical guidance with sensitivity, responsiveness, and collaboration. We will work as a partner with NHLBI to rapidly establish the CC using our established infrastructure, programming, and processes, which we will customize for the needs of the Catalyze Program. Because program flexibility is critical for NHLBI, we will be able to provide state-of-the-art tracking tools to ensure that NHLBI is apprised of the status of each project and informed when we anticipate delays or technical challenges that may impede product development.

产品从台架研究向临床过渡的资金和支持存在差距 应用往往导致潜在的重要发现的损失。这种翻译间隙，也称为 “死亡之谷”一直是美国国立卫生研究院增加生物医学支出的一个领域。 在过去的20年里，健康（NIH）。美国国家心肺血液研究所（NHLBI） 近20年前就认识到了培养转化研究项目的必要性， 生产援助细胞疗法计划，其次是基因治疗资源计划， 科学转向研究翻译和治疗（SMARTT），和美国国立卫生研究院 加速创新健康中心（NCAI）这些计划旨在提供多样化的 一系列服务，以加速新产品的临床应用开发， 商业化 该催化剂计划继续NHLBI的支持，通过资金，技术 支持和技能发展，以确保研究人员能够成功地推进新疗法， 心脏、肺、血液和睡眠疾病和障碍的平台技术。这个程序将合并 从二十年的翻译项目中获得的最佳实践，成为指导的典范 研究人员和推进转化研究。Catalyze将提供来自 通过研究性新药（IND）使能研究发现，并协助研究者 制定里程碑，确定最佳监管途径，并制定商业策略， 他们的产品。Catalyze计划中的每位研究人员都将由经验丰富的技术人员指导。 团队期望他们不仅能利用自己的知识开发新技术， 治疗，但也培养下一代的翻译科学家。 作为催化剂协调中心（CC），RTI国际将利用20年的经验， 转化研究，以提供卓越的运营和技术领导。通过我们的经验 作为SMARTT的CC以及NCAI和REACH的独立评估者，我们有独特的见解 了解翻译项目中的最佳实践和挑战，并将运用这些知识来确保 Catalyze为研究人员提供了一个全面的开发支持系统， 为NHLBI提供战略性和灵活的方案。我们认识到，Catalyze的成功将取决于 调查人员在该计划中的成功。作为SMARTT的CC，我们能够与 调查人员从全国各地有效地通过有频繁的沟通，并提供一个 合作的氛围。SMARTT计划成功的关键是定制我们的团队 以及我们满足每位调查员需求的方法。通过与每一个人进行全面讨论， 调查人员及其团队，我们能够确定他们在产品方面知识/经验水平 发展，计划中的差距/需求，以及最适合每个人的沟通风格 调查员SMARTT这种方法的成功体现在产品数量上， 沿着发展道路前进，包括25个制造活动，18个 完成药理学/毒理学包，7个IND获批，11个IND前会议，2个IND 修正案，4个孤儿药指定申请获得批准，4个制药合作伙伴关系或 许可协议。 作为Catalyze的CC，我们将采取类似的方法，以确保每位研究者都能收到 支持和服务，为他/她量身定制。我们将接近我们的角色，为调查人员提供项目 管理和技术指导，具有敏感性、响应性和协作性。我们将作为 与NHLBI合作，利用我们已建立的基础设施、规划和 我们将根据Catalyze计划的需要定制流程。因为程序的灵活性 对于NHLBI至关重要，我们将能够提供最先进的跟踪工具，以确保NHLBI 了解每个项目的状态，并在我们预计会出现延迟或技术挑战时通知我们， 可能会阻碍产品开发。