美国FDA对药品专家咨询委员会的管理及对我国的启示

目的加强我国药品专家审评委员会的管理并促进其有效发挥作用。方法通过介绍美国FDA对药品咨询委员会的管理,利用文献回顾和对比分析的方法探索我国药品专家审评委员会需要改善的方面。结果与结论合理设置专家成员、强化对专家委员利益冲突的管理、增加委员会工作程序的透明度并加强委员会网站的建设,将有利于发挥我国药品专家审评委员会在药品审评中的作用。     

突破性治疗药物程序在药品注册体系中的作用及展望

目的：进一步完善我国突破性治疗药物程序并发挥其在药品注册体系中的作用。方法：通过梳理美国食品药品管理局(FDA)突破性治疗认定(BTD)、欧洲药品管理局(EMA)优先药物计划 (PRIME)和我国国家药品监督管理局(NMPA)突破性治疗药物程序和案例,进一步探索优化我国突破性治疗药物程序在药品注册体系中的发展方向。结果与结论：FDA、EMA和NMPA的药品注册体系中均具有突破性药物治疗程序。FDA自2012年7月实行加快程序中的突破性治疗认定,通过大量案例积累了相对完善的管理经验,EMA的PRIME专人专管的政策支持以及明确的资格申请数据要求,均具有借鉴意义。我国突破性治疗药物法规于2020年落地实施后,至今也积累了一定案例。通过对FDA、EMA和 NMPA关于BTD的法规对比,为我国突破性治疗药物程序实施提供建议。     

美国食品药品管理局咨询委员会的作用及对我国的启示

通过分析美国食品药品管理局（FDA）咨询委员会及中国国家食品药品监督管理局药品专家审评委员会及各自咨询机制,探索我国药品专家审评委员会需要改善的方面,并为我国专家审评委员会制度的完善提出建议。认为FDA咨询委员会制度较成熟,在专家成员设置、遴选标准和会议的透明性等方面可以为我国药品专家审评委员会制度提供借鉴。     

欧盟药品加速审评政策及实证分析

目的:为我国新药审评审批制度的深入改革提供参考。方法:系统分析欧盟药品加速审评政策的法律基础,并与其他同类政策进行比较;对加速审评流程的关键环节,尤其是加速审评申请的递交前准备、许可前检查、申请的递交和评估等详细流程进行介绍。以Maviret为实例进行个案剖析,实证分析欧盟加速审评政策的应用效果。结果:加速审评是欧盟促进患者尽早获得新药的几项法律条款之一,与条件上市许可、同情用药、优先药物计划、孤儿药认定、医院豁免等政策一样作为在欧盟尽早获得新药的主要途径之一。当药物具备充分的治疗数据、具有重大公共健康利益和治疗创新性时,即有望获得加速审评。在欧盟法规(726/2004/EC)的基础上,欧洲药品管理局(EMA)不断完善加速审评政策体系,相继发布了相关指导原则(指南)与加速审评程序的执行时间表,为该政策的切实落地提供了详细、具体的指导。一旦药物进入加速审评程序,则其审评时间将由标准审评程序的210日缩短至150日。治疗丙型肝炎病毒感染的新药Maviret从2017年1月20日开始加速审评,直到2017年6月22日获得EMA人用药品委员会(CHMP)发布批准其上市许可的肯定意见,整个流程不到半年。结论:欧盟药品加速审评政策既有完善的立法支持,又有详细具体的实施细则和执行时间表,能够加快一些公众急需的、具有特殊医疗优势的药品的上市速度。     

健全与完善我国药品审评专家咨询制度之我见

药品审评所涉及的科学问题的判断,常常是超出药品监管部门公务员的自身知识水平,于是建立药品审评专家咨询制度便成为各国药品监管机构的通行经验。     

欧洲药品管理局：利益透明新政策

针对欧洲药品监管系统内部专家以及委员会成员,一项新政策得到了欧洲药品管理局（EMA）管理委员会的通过,并且征近日进行了公布。这一新政策与利益冲突和透明度问题相关,     

日本将加快药品审评

日本药品医疗器械管理局（PMDA）公布了一份最新五年计划,这个自下一财年（每年4月1日）启动的计划,将进一步缩短新药审评时间。到2011年,标准平均审评时间（包括回答问题或补充材料）缩短至12个月,对急需的优先产品缩短至9个月。目前计划正待日本卫生劳动福利部批准。     

简介美国药品专家咨询委员会及我国药品专家审评委员会

新药审评是一项系统工程,新药上市申请要经过多专业、多学科的综合审评和评价。各国在专职审评机构之外,都设立了外部专家审评机构,美国为药品咨询委员会,我国为药品审评委员会。     

药品审评认证机构信息化的几点建议

药品审评认证机构是近年来为适应食品药品监督管理系统机构体制改革而设立的。主要是受省级食品药品监督管理局委托,开展药品、医疗器械、保健品和化妆品技术审评和认证工作,并为上级单位提供技术支持。目前,许多省(市)相继成立了药品审评认证机构,对保证食品药品监管系统行政许可工作的顺利进行发挥着越来越重要的作用。随着机构职能的进一步落实和技术审评工作的逐步展开,     

我国药品附条件批准程序实施情况及相关思考

目的：对我国药品附条件批准上市相关政策和实施情况进行深入分析和探讨,参考美国与欧盟药品附条件上市政策,对我国附条件批准上市的实施和推进提出建议。方法：通过梳理药品注册管理办法发布后国家药品监督管理局(NMPA)药品附条件批准上市申请审评审批法规政策实施情况,重点围绕当前法规中的准入条件、准入程序、上市后监管要求、撤销情形以及撤销程序进行综述,针对实施过程中发现的问题,借鉴美国食品药品管理局(FDA)药品加速审批(Accelerate Approval)与欧洲药品管理局(EMA)药品附条件批准(Conditional Marketing Authorisation)经验以及对各国附条件政策进行比较分析,探讨我国药品附条件批准上市政策的发展方向。结果与结论：为了加快具有突出价值的临床急需药品上市,缩短新技术临床应用时间,美国与欧盟均设立了相对完备的附条件上市法规政策及程序。我国的附条件批准制度虽然建立时间较短,但有欧美的经验作为参考,结合我国的临床实践和监管需要, 相关法规也在趋于完善。未来,监管部门更多需要考虑的是对程序和技术要求的细化、制度之间的衔接 (如疫苗的紧急使用授权与附条件批准制度),以及加强上市后监管等方面。     

美国和欧盟儿科用药审评机构职责与作用探析

目的：为提高我国儿科用药的审评质量和效率提供借鉴。方法：收集与整理美国、欧盟儿科用药审评、管理机构的职责与作用,并加以研究分析。结果与结论：近两年,我国成立了与两个儿科用药相关的专家委员会,分别隶属于药品审评中心及国家卫生和计划生育委员会,这两个专家委员会成员仍然都是儿科临床专家,相对于美国FDA和欧盟EMA,缺少了专门的药学、药理毒理、药物警戒、统计等方面的专家。目前,国家药品审评中心并没有专门的儿科用药审评团队。建议我国借鉴美国与欧盟的经验,在药品审评中心内部成立专门的儿科用药审评部门。为各审评机构在技术审评过程中遇到的与儿科用药相关问题提供日常内部咨询;为审评中心制定与儿科用药相关的技术规范、指导原则等做全面的统筹规划;为企业进行儿科用药研发提供科学建议。     

美、欧、日三国药物审批趋势解读（2004—2013年）

2014年5月汤森路透集团的科学创新监管中心（CIRS）发表了一份＂R＆DBriefing54＂的报告。基于该报告对新活性物质的统计数据,分析美、欧、日三个药物审批机构,即美国食品与药品管理局、欧洲药物管理局和日本药品与医疗器械管理局的新药审批趋势。     

Clinical pharmacology as a cornerstone of orphan drug development

A recent US Food and Drug Administration (FDA) advisory committee meeting highlighted the potential of clinical pharmacology to overcome challenges in orphan drug development.     

药品技术审评时限纳入法规的合理性讨论

目的 探讨将药品技术审评时限写入法规中的合理性问题.方法 通过分析人用药品注册技术要求国际协调会（ICH）成员国美国、日本及欧盟在法律中对药品技术审评时限的规定,并对美国、日本药品实际审评时限作简要统计.结合我国药品审评现状,进一步分析我国将药品技术审评时限写入法规中是否合理.结果与结论 药品技术审评是一项复杂的技术工作,其考虑因素的复杂性远非法定时限的简单切割.所以,将药品技术审评时限写入《药品注册管理办法》中有欠妥之处.     

药品注册专员岗位胜任力评价指标体系的构建

目的:构建科学客观的药品注册专员岗位胜任力评价指标体系。方法:通过查找药品注册胜任力模型文献、注册相关法规性文件,结合Spencer胜任力辞典和Hay胜任力辞典,初步拟定药品注册专员岗位胜任力评价指标体系。通过两轮德尔菲专家咨询法对药品注册相关领域专家进行函询,筛选与修正评价指标,并运用优序图法对指标进行权重计算;基于问卷调查法,以232名药品注册工作的相关人员为研究对象,再运用探索性因子分析、验证性因子分析和信度分析对构建的评价指标体系进行检验。结果:构建的药品注册专员岗位胜任力评价体系涵盖4个核心维度(注册专业能力、关系管理能力、专业发展能力、个人综合素质)、11个子维度、41个测量条目,并确定了各维度指标权重。该评价体系建立过程中专家积极性高、协调性好,探索性因子分析和验证性因子分析证明了该指标体系的科学合理性。结论:所建药品注册专员岗位胜任力评价指标体系具有全面性、综合性、科学性,可为药品注册专员的评价和管理提供依据。     

Risk communication efforts for drug safety in the US and EU, and its present situation in Japan

In pharmacovigilance, it has been recognized that it is essential to share the information regarding the risk of drugs among stakeholders and to have risk communication (RC) which enables consumers to make their own judgments regarding the risk. In particular, RC between the governmental agencies and consumers is given a high priority. Hence, its provisions and initiatives should be considered thoroughly. I present a brief overview of current proactive approaches for RC to protect the patient's rights, to increase openness and transparency and to share information in the US and EU. Following the Food and Drug Administration Amendment Act (FDAAA), Institute of Medicine of the National Academies (IOM) recommended that FDA makes efforts for RC. The RC Advisory Committee was established and consumers are involved as its members. FDA published "Guidance Drug Safety Information" in 2007 and "Strategic Plan for RC" in 2009. Thus, its framework has been developed. European Medicines Agency (EMA) issued "Work programme 2010" and "EMA Communication on safety related issues", which indicated its policies and measures. EMA is promoting development of the framework for Patient Involvement (e.g., members of scientific committees). Regarding the direct patient reporting of adverse drug reactions, FDA began that in 2003 and EMA recommends to encourage it for EU Member States, and it has already started in some countries. RC has important roles to take safety measures for drugs and to protect patient's rights, therefore such an approach for it be implemented is expected in Japan.     

Patient representatives' contributions to the benefit‐risk assessment tasks of the European Medicines Agency scientific committees

In the European Medicines Agency (EMA), the involvement of patients has been increasingly recognized as valuable and necessary. Specifically in scientific committees, patients through patient representatives are actively involved in deliberations and decision making processes. These scientific committees are meant to ensure that licensed medicines have a positive benefit–risk ratio in favour of the patients and users. To investigate what the contributions are of patient representatives in benefit–risk assessment, we interviewed 15 scientific committee members, 10 of whom are/were EU-state regulatory representatives and five are/were patient representatives. We asked the participants questions related to the benefit–risk assessment tasks of their committees, the connection between patient representatives and the patient perspective, and the contribution of patient representatives in the various benefit–risk assessments tasks. We found that the contribution of patient representatives benefit–risk assessment may be a variable of the benefits and the risks involved in the drug such that the necessity of their contribution is strongly felt when both benefits and risks are high, when benefits are almost equal or are equal to risks and when both benefits and risks are low. In terms of the various benefit–risk tasks, patient representatives contribute to benefit–risk analysis by providing criteria that help define the benefit–risk picture. In benefit–risk evaluation, patient representatives aid in providing a specific basis for the values and weights given to specific benefits and risks and in decision making, they provide what may be a crucial patient perspective in terms of the acceptability of risks. Hence, patient representatives provide a specific expertise in these scientific committees.     

Drug evaluation and registration in Hungary

In Hungary, the actual drug evaluation and registration system reflects international standards and national traditions. The compulsory drug registration system that was established in 1933 was among the first in Europe. Laboratory control (since 1927), clinical trials (since 1951) and human clinical pharmaceutical experiments (since 1967) are prerequisites for new-drug approval. Applications should be sent to the National Institute of Pharmacy, which has the overall responsibility for the registration of pharmaceutical products. Applications are assessed on the basis of the drug's quality, safety, and efficacy. The procedure follows several steps: evaluation of chemical and pharmaceutical data by the staff of the National Institute of Pharmacy; evaluation of toxicologic and pharmacologic documentation with the help of the Committee on Drug Administration; after consultation with the Committee on Medical Research Ethics (mandatory in cases of original new drugs), authorized clinical pharmacologic investigations are conducted in the units of the Clinical Pharmacological Network, which are supervised by the National Center for Clinical Pharmacology; clinical trials; application for registration (scientific evaluation); and finally, application to the Ministry of Health for a marketing authorization. The process may be facilitated appreciably for preparations already registered in another country. Moreover, Hungary is an active member in the World Health Organization (WHO), Pharmaceutical Inspection Convention of the European Free Trade Association (EFTA PIC), the Council of Mutual Economic Assistance (COMECON), and other international pharmaceutical and clinical pharmaceutical collaborations.