突发公共卫生事件相关的药品审评审批政策思考

2019年的新型冠状病毒肺炎疫情对中国应对突发公共卫生事件的应急反应是一个严峻的考验。研究了美国食品药品监督管理局（FDA）医疗对策、紧急使用授权,以及药品审评审批加急程序的相关政策,为重大威胁相关的药品审评审批政策的制定提供参考,并为应对突发公共卫生事件的药品的战略性研制和储备提供思路。     

美国医药产品紧急使用授权风险管理研究

摘要：目的:研究美国医药产品紧急使用授权法规及指南,梳理对紧急使用授权产品监管措施,探讨建立适合我国国情的医药产品紧急使用授权制度。方法:查阅美国食品药品管理局官方网站及国内外文献数据库,分析美国医药产品紧急使用授权制度及实践经验。结果:美国医药产品紧急使用授权需要进行申请、审评、授权、定期收集安全性信息、持续风险效益评价等风险管理措施。结论:紧急使用授权制度对于加强公共卫生事件应对能力至关重要,我国应建立医药产品紧急使用授权制度,全面提升药械审评与评价能力及时发现授权产品风险,同时重视紧急使用授权产品的信息公开。     

建立我国突发事件应急状态下的医疗产品紧急使用授权机制探讨

目的： 通过对美国FDA的紧急使用授权（Emergency Use Authorization,EUA）制度进行研究,结合我国国情,探讨在我国建立医疗产品紧急使用授权机制。方法： 文献研究、专题访谈。结果与结论： 紧急使用授权制度有别于我国现有的特别审评程序,对于应对突发事件应急状态下所需药物的评估和使用授权,提高药物使用的安全性、时效性、可及性及合规性具有重要意义。本研究初步提出了在我国建立医疗产品紧急使用授权机制的考虑要点,包括建立紧急使用授权制度的必要性、制度的基本定位、上位法的支持、制度构建的关键要素、配套的技术指南及其他需要关注的问题。     

医药产品紧急使用授权

医药产品紧急使用授权(Emergency Use Authorization,EUA)指由美国食品药品管理局(FDA)在实际的或潜在的紧急状态下对未获批准的医药产品的使用及已获批准产品的未获批准用途的授权。本文结合实际操作层面,对EUA的法律基础、EUA在FDA使命范围内的作用、EUA的审评、发布和终止程序、应对2009年H1N1流感取得的成功经验以及EUA实施过程中遇到的挑战进行分析。同时与日本应对2009年H1N1大流行时对ArepanrixTMA/H1N1流感疫苗和Novartis H1N1流感疫苗的紧急授权进行比较分析。     

美国药品效期延长计划应用研究及启示

目的 探讨美国药品效期延长计划对我国应对紧急突发公共卫生事件过程中药品使用的启示.方法 通过文献研究法,对美国联邦政府与美国食品药品管理局合作建立的药品效期延长计划的基本情况及其与紧急使用授权的关系做简要介绍,重点从实践应用层面研究该计划的具体做法.结果与结论 美国药品效期延长计划的经验,为我国在应对紧急突发公共卫生事件时规范药品的使用提供了参考.     

美国药品不良反应的自愿报告系统

MedWatch是美国FDA的自愿报告系统，医疗专家和医学产品使用者使用此系统自愿提交不良事件报告，报告范围包括FDA已经批准的上市后的药品、生物制品、医疗器械和特殊营养产品。     

美国FDA医疗产品紧急使用授权管理简介及启示

介绍了美国医疗产品紧急使用授权的定义,法律依据、管理程序和判定标准,以及执行过程中具体操作条件和要求。以期对我国相关管理机构在公众健康安全紧急状态下,对医疗产品的紧急使用授权管理提供借鉴。     

新的经济环境下植物提取物行业动态

介绍了美国医疗产品紧急使用授权的定义,法律依据、管理程序和判定标准,以及执行过程中具体操作条件和要求。以期对我国相关管理机构在公众健康安全紧急状态下,对医疗产品的紧急使用授权管理提供借鉴。     

FDA批准罗氏埃博拉病毒检测试剂用于应急使用

罗氏表示美国FDA批准其埃博拉病毒检测试剂用于应急使用,以应对这一全球性最严重疾病在西非的爆发。美国FDA批准罗氏LightM ix Ebola Zaire rRT-PCR Test用于有埃博拉病毒感染体征的患者,该瑞士制药商在一份声明中表示。罗氏表示,LightM ix试剂可以在3h内给出检测结果,快速地检测这种病毒,以便尽快治疗。根据该检测试剂紧急使用的资格,美国及其他国家的某些实验室已被授权在有限时间内使用这款检测试剂,用于检测在西非一直在传播的埃博拉病毒。     

美国FDA在应急医药产品应急准备中的职能分析

目的:对美国流行病和灾害性应急准备系列法案中美国食品药品监督管理局(FDA)职能定位进行分析,为我国政府部门的应急职能设计提供参考。方法:采取法案内容分析法,了解FDA在应急医药产品准备中的职能定位。结果:FDA在医药产品应急准备的根本职能是促进突发事件中应急医药产品的开发和使用的可获得性,包括发布医药产品的应急指导和安全警示信息,负责授权医药产品紧急使用权、延长医药产品保存期限及预置职能。结论:应重视国家级应急准备工作,完善应急准备的配套法律体系,准确定位"应急医药产品的应急预案",加强应急医药产品的应急信息化管理,建设应急医药产品的快速调剂和使用机制。     

公共卫生事件中医疗机构中药制剂应急管理探讨

目的 回顾与总结新型冠状病毒肺炎疫情下医疗机构中药制剂应急管理措施,为进一步改进和完善我国应对突发公共卫生事件中药品应急管理体系建设提供参考。方法 结合本次新型冠状病毒肺炎疫情中各省医疗机构中药制剂应急管理工作实践,梳理开展医疗机构中药制剂应急管理工作流程,探讨突发公共卫生事件中医疗机构中药制剂管理策略及措施。结果 目前,19个省份发布新型冠状病毒肺炎防控用医疗机构中药制剂应急管理文件,25个省份完成百余种医疗机构中药制剂备案审批并投入临床使用。结论 建立完善的药品应急管理体系,可以提高监管效率,协调指导相关工作有序进行,进一步提高突发公共卫生事件应对能力。     

The National Institutes of Health Chemical Countermeasures Research Program (NIH CCRP): A collaborative opportunity to develop effective and accessible chemical medical countermeasures for the American people

The United States Department of Health and Human Services (HHS) leads the nation in preventing, preparing for, and responding to the adverse health effects of public health emergencies and disasters. In addition to biological, radiological and nuclear agents, the risk of a high consequence public health emergency due to the intentional and/or accidental release of chemical agents is a major growing concern of the US government. As such, the federal government is fully committed to address public health security threats posed by chemicals. To enhance chemical emergency preparedness and response, HHS oversees the interdepartmental research, advanced development, regulatory review and approval, procurement, and stockpiling of medical countermeasures (MCMs). Within the National Institute of Health (NIH/HHS), the National Institute of Allergy and Infectious Diseases (NIAID) is responsible for the fundamental research and early development of MCMs to prevent deaths and/or treat injuries during and after emergencies due to large scale chemical exposure. This commentary provides an overview of the NIAID/NIH Chemical Countermeasures Research Program (CCRP) and resources to facilitate the research, discovery, and early development of chemical MCMs. Available product development resources include research funding opportunities, expert advice from the NIH, and preclinical and efficacy service support cores to reduce opportunity costs and entry barriers for prospective developers interested in entering or accelerating the development of chemical MCMs.     

Animal models for acute radiation syndrome drug discovery

Introduction: Although significant scientific advances have been made over the past six decades in developing safe, nontoxic and effective radiation/medical countermeasures (MCMs) for acute radiation syndrome (ARS), no drug has been approved by the US FDA. The availability of adequate animal models is a prime requisite under the criteria established by the FDA ‘animal rule’ for the development of novel MCMs for ARS and the discovery of biomarkers for radiation exposure.

Areas covered: This article reviews the developments of MCMs to combat ARS, with particular reference to the various animal models (rodents: mouse and rat; canine: beagle; minipigs and nonhuman primates [NHPs]) utilized for the in-depth evaluation. The objective, pathways and challenges of the FDA Animal Efficacy Rule are also discussed.

Expert opinion: There are a number of well-defined animal models, the mouse, canine and NHP, that are being used for the development of MCMs. Additional animal models, such as the minipig, are under development to further assist in the identification, efficacy testing and approval of MCMs under the FDA Animal Efficacy Rule.     

An Assessment of State Board of Pharmacy Legal Documents for Public Health Emergency Preparedness

Objective. To estimate pharmaceutical emergency preparedness of US states and commonwealth territories.Methods. A quantitative content analysis was performed to evaluate board of pharmacy legal documents (ie, statutes, rules, and regulations) for the presence of the 2006 Rules for Public Health Emergencies (RPHE) from the National Association of Boards of Pharmacy’s (NABP) Model Pharmacy Practice Act.Results. The median number of state-adopted RPHE was one, which was significantly less than the hypothesized value of four. Rule Two, which recommended policies and procedures for reporting disasters, was adopted significantly more than other RPHE. Ten states incorporated language specific to public health emergency refill dispensing, and among these, only six allowed 30-day refill quantities.Conclusion. Based on the 2006 NABP model rules, it does not appear that states are prepared to expedite an effective pharmaceutical response during a public health emergency. Boards of pharmacy should consider adding the eight RPHE to their state pharmacy practice acts.     

College/School of Pharmacy Affiliation and Community Pharmacies' Involvement in Public Health Activities

Objectives

To examine the relationship between pharmacy college/school affiliation and community pharmacies'' involvement in immunization and emergency preparedness activities.

Methods

Telephone interviews were completed with 1,704 community pharmacies randomly sampled from 17 states to determine the pharmacies'' involvement in immunization promotion, vaccine distribution, in-house immunization delivery, and health emergency preparedness and response, affiliation with college/school of pharmacy, and selected pharmacy and public health-related characteristics.

Results

Pharmacy college/school-affiliated community pharmacies were more likely than non-affiliated pharmacies to participate in immunization and emergency preparedness when controlling for pharmacy characteristics. College/school affiliation generally became nonsignificant, however, when public health-related characteristics were included in the analysis.

Conclusions

Affiliation with a college/school of pharmacy was related to community pharmacies'' involvement in immunization and emergency preparedness.     

美国应急管理的法制建设及FDA相关部门设置对我国的启示

目的:为我国应对突发公共事件等紧急状况提供参考。方法:通过查阅美国食品与药品管理局(FDA)应急管理指南,采用文献研究法和比较分析法进行研究。结果:美国建立了完善的应急法律法规体系,FDA建立了以应急运行中心为支点、各办公室和各中心提供支持的系统的应急响应组织结构,可以处理不同类型的紧急事件。结论:我国应进一步完善应急法律法规体系,建议在国家食品药品监督管理局(SFDA)中建立一个应急运行中心,并为之配备一套支持机构,当务之急是制定切实可行的应急预案。     

公共卫生事件中FDA应对短缺药品指导原则介绍

为应对新型冠状病毒肺炎（COVID-19）突发公共卫生事件,2020年3月,FDA发布“FD&C法案506C规定的永久停产和生产中断报告指导原则”。该指导原则为申请人和生产企业及时和详尽地报告药品和生物制品生产变化提出建议,以便FDA采取及时有效的措施应对可能的短缺药品,保障美国药品供应链稳定。该指导原则对完善我国药品生产中断报告制度具有参考价值。