a Radiation and Nuclear Countermeasures Program (RNCP), Division of Allergy, Immunology and Transplantation (DAIT), National Institute of Allergy and Infectious Diseases (NIAID), National Institutes of Health (NIH), Rockville, Maryland.
b Office of Biodefense Research Resources and Translational Research (OBRRTR), Division of Microbiology and Infectious Diseases (DMID), NIAID, NIH, Rockville, Maryland.
Radiat Res. 2018 Dec;190(6):659-676. doi: 10.1667/RR15137.1. Epub 2018 Aug 30.
The risk of a radiological or nuclear public health emergency is a major growing concern of the U.S. government. To address a potential incident and ensure that the government is prepared to respond to any subsequent civilian or military casualties, the U.S. Department of Health and Human Services and the Department of Defense have been charged with the development of medical countermeasures (MCMs) to treat the acute and delayed injuries that can result from radiation exposure. Because of the limited budgets in research and development and the high costs associated with bring promising approaches from the bench through advanced product development activities, and ultimately, to regulatory approval, the U.S. government places a priority on repurposing products for which there already exists relevant safety and other important information concerning their use in humans. Generating human data can be a costly and time-consuming process; therefore, the U.S. government has interest in drugs for which such relevant information has been established (e.g., products for another indication), and in determining if they could be repurposed for use as MCMs to treat radiation injuries as well as chemical and biological insults. To explore these possibilities, the National Institute of Allergy and Infectious Diseases (NIAID) convened a workshop including U.S. government, industry and academic subject matter experts, to discuss the challenges and benefits of repurposing products for a radiation indication. Topics covered included a discussion of U.S. government efforts (e.g. funding, stockpiling and making products available for study), as well unique regulatory and other challenges faced when repurposing patent protected or generic drugs. Other discussions involved lessons learned from industry on repurposing pre-license, pipeline products within drug development portfolios. This report reviews the information presented, as well as an overview of discussions from the meeting.
美国政府越来越关注放射性或核公共卫生紧急情况的风险。为了应对潜在事件，并确保政府有能力应对随后的平民或军事伤亡，美国卫生与公众服务部和国防部负责开发医疗对策（MCM），以治疗因辐射暴露而导致的急性和迟发性损伤。由于研发预算有限，以及将有前途的方法从实验室推进到高级产品开发活动，并最终获得监管批准的成本高昂，美国政府优先考虑重新利用已经存在相关安全性和其他关于其在人类中使用的重要信息的产品。生成人体数据可能是一个昂贵且耗时的过程；因此，美国政府对已经建立了此类相关信息的药物（例如，用于其他适应症的产品）感兴趣，并确定它们是否可以重新用于治疗辐射损伤以及化学和生物损伤的 MCM。为了探索这些可能性，美国国家过敏和传染病研究所（NIAID）召集了一次研讨会，包括美国政府、工业界和学术界的专家，讨论为放射性适应症重新利用产品的挑战和益处。涵盖的主题包括讨论美国政府的努力（例如，资金、储备和提供产品供研究），以及重新利用受专利保护或仿制药时面临的独特监管和其他挑战。其他讨论涉及从行业中汲取的有关重新利用药物开发组合中许可前、管道产品的经验教训。本报告审查了所提出的信息，以及会议讨论的概述。
