Coordinating Center for the NHLBI Catalyze Program

NHLBI 催化计划协调中心

• 批准号: 10209507
• 负责人: Diana M Severynse-Stevens
• 金额: $ 189.76万
• 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/10209507
• 关键词: Amendment; Area; Atmosphere; Cell Therapy; Cessation of life; Collaborations; Communication; Country; Custom; Development; Ensure; Fostering; Funding; Health; Heart Diseases; Hematological Disease; Infrastructure; Institutes; International; Investigational Drugs; Knowledge; Leadership; Licensing; Lung diseases; Mentors; Modeling; National Heart, Lung, and Blood Institute; Orphan Drugs; Pharmacologic Substance; Pharmacology and Toxicology; Process; Production; Regulatory Pathway; Research; Research Personnel; Resources; Role; Science; Services; Sleep Disorders; Support System; Technology; Training; Translational Research; United States National Institutes of Health; Work; clinical application; commercialization; design; expectation; experience; flexibility; gene therapy; innovation; insight; meetings; new technology; next generation; novel; novel therapeutics; product development; programs; skill acquisition; success; tool; translational scientist

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.

资助和支持产品从试验台研究过渡到临床的资金缺口 应用往往会导致潜在的重要发现的丢失。这种翻译鸿沟，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 美国国家卫生研究院(NIH)在过去20年的研究成果。国家心肺血液研究所(NHLBI) 近20年前就认识到了促进翻译研究的计划的必要性，从 细胞治疗生产援助计划，随后是基因治疗资源计划， 科学走向研究翻译和治疗(SMARTT)，以及美国国立卫生研究院 健康加速创新中心(NCAI)这些计划旨在提供多样化的 一系列服务，加快新产品的临床应用开发和 商业化。 CATALYE计划继续NHLBI对翻译科学的支持，通过资助、技术 支持和技能发展，以确保研究人员能够成功地推进新的疗法和 针对心、肺、血液和睡眠疾病和紊乱的平台技术。该程序将合并 翻译计划二十年来的最佳实践成为指导的典范 研究人员和先进的翻译研究。Catalyst将从以下方面提供技术指导 通过研究性新药(IND)研究发现，以及协助调查人员 开发里程碑，确定最佳监管途径，并为 他们的产品。CATALYE计划中的每个研究人员将由经验丰富的技术人员指导 团队期望他们不仅使用他们的知识来开发新技术或 治疗，但也是为了培养下一代翻译科学家。 作为催化协调中心(CC)，RTI International将借鉴20年来在以下方面的经验 翻译研究，提供卓越的运营和技术领导能力。通过我们的经验 作为Smartt的CC和NAI和REACH的独立评估者，我们拥有独特的洞察力 翻译计划中的最佳实践和挑战，并将应用这些知识来确保 Catalyst为调查人员提供了全面的开发支持系统和高效、 针对NHLBI的战略性且灵活的计划。我们认识到，催化的成功将取决于 调查人员在项目中的成功。作为Smartt的CC，我们能够与 全国各地的调查人员通过频繁的沟通和提供 合作的氛围。Smartt计划成功的关键是定制我们的团队 以及我们的方法，以满足每个调查员的需求。通过与每个人的全面讨论 调查人员及其团队，我们能够确定他们在产品方面的知识/经验水平 开发，项目中的差距/需求，以及与每个项目配合最好的沟通方式 调查员。这种方法对Smartt的成功体现在以下产品的数量上 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通过，11个IND前会议，2个IND 修正案，4个孤儿药物指定申请获得批准，4个药物合作伙伴关系或 许可协议。 作为催化的CC，我们将采取类似的方法来确保每个调查员都能收到 为他/她量身定制的支持和服务。我们将在为调查人员提供项目方面发挥我们的作用 具有敏感度、响应性和协作性的管理和技术指导。我们将作为 与NHLBI合作，使用我们已建立的基础设施、编程和 流程，我们将根据CATALYE计划的需要进行定制。因为程序灵活性是 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 报告每个项目的状态，并在我们预计会出现延误或技术挑战时进行通知 可能会阻碍产品开发。