各国生物类似药立法发展现状和批准产品的研究进展

通过对美国、欧盟、日本、韩国及我国生物类似药的立法发展及法规进行梳理,了解我国生物类似药法规方面存在的主要差异。通过对美国、欧盟、日本、韩国及我国生物类似药的批准及在研情况进行梳理,摸清国内外生物类似药的上市及研发现状,为我国生物类似药法规体系研究奠定基础。主要综述了各国生物类似药立法发展现状和批准产品的研究进展。     

抗体类生物类似药命名管理及其影响分析

目的 规范我国抗体类生物类似药命名,完善其全生命周期管理体系。方法 深入研究世界卫生组织（World Health Organization,WHO）、欧盟、美国、日本及我国关于抗体类生物类似药的命名要求,分析不同命名方式对药品全生命周期管理的影响。结果 目前,国内外抗体类生物类似药的命名原则不尽相同,但总体遵循WHO的国际非专利药名系统不同的命名方式将对处方安全性、药物警戒、医保准入与支付等方面产生影响。结论 建议药品监管部门逐步与国际接轨,基于国际非专利药名系统并结合我国实际情况尽快出台生物类似药命名相关文件,以明确抗体类生物类似药命名。此外,建议加强药物警戒管理、制定科学审慎的生物类似药医保支付决策,从而构建生物类似药尤其是抗体类药物的全生命周期管理体系。     

典型国家创新药医保谈判准入评价标准研究及对我国的启示

本文旨在学习典型国家创新药医保谈判准入评价标准设计和框架,结合我国创新药医保谈判准入现状,为完善我国创新药医保谈判准入评价标准提供参考.查阅并总结新西兰、德国、法国创新药医保谈判准入相关的评价标准,分析其特点并与我国进行比较,探讨对我国的借鉴意义.典型国家在创新药医保谈判准入体系方面发展较为成熟,在评价内容的多样性、评...     

澳大利亚高值药物医保准入机制简介及对我国罕用药医保准入的启示

目的:介绍澳大利亚高值药物医保准入机制,为我国罕用药医保准入的完善提供建议。方法:运用文献研究和政策分析法,对澳大利亚高值药物医保准入的遴选原则、风险共担机制、限制使用原则进行概述分析,并对高值药物医保准入机制实施效果进行评价。结果与结论:澳大利亚药物福利计划对高值药物的纳入不仅考虑成本效益比,同时关注药物的临床疗效、不可替代性及药物的社会性原则,通过风险共担机制和限制使用原则在保障患者用药可及性的同时维持医保基金的可持续,在保障患者用药、加强基本医疗保障福利方面取得显著成效。建议完善我国罕用药医保遴选标准,引入风险共担机制及规范罕用药的使用和报销。     

刍议美国生物类似药行动计划

生物类似药是指在质量、安全性和有效性方面与已获准上市的参比制剂具有相似性的治疗性生物制品。结合美国、欧盟、日本和世界卫生组织对生物类似药研发及监管思路,重点介绍了2018年度美国食品药品监督管理局发布的《生物类似药行动计划——简介和概述：创新与竞争间的平衡》,以期对我国生物类似药监管政策的制定提供借鉴。     

各国生物类似药沟通交流机制及生物类似药可互换性对比研究及建议

药品注册中的沟通交流对于控制风险、提高研发注册效率具有重要作用。对美国、欧盟、日本、韩国等国家生物类似药沟通交流机制进行对比研究,结合我国沟通交流的现状,问卷调研和专家研讨,提出完善我国生物类似药沟通交流机制的建议。各国对生物类似药的"可互换性"有着不同的界定和管理。对美欧等国家生物类似药的"可互换性"进行研究,结合问卷调研和专家研讨,提出完善我国生物类似药可互换性的建议。     

澳大利亚高值药物管理准入政策及对我国的启示

为解决高值药物准入对医保可持续性的影响,各国积极探索管理准入在提高高值药物可及性、降低医保基金风险等方面的作用,并取得了一定成效.本研究系统梳理了澳大利亚高值药物管理准入的相关政策,包括药物遴选、协议制定、实施管理、数据收集以及药物再审查等,比较分析澳大利亚高值药物管理准入和我国谈判准入政策,为我国进一步完善高值药物医...     

国外创新药纳入医保目录机制研究以及对我国的启示

目的：从创新药纳入医保机制的典型国家案例中，汲取能够为我国医保目录管理工作提供思路的借鉴点，为加快创新药物医保准入，建立行之有效、符合国情的医保目录管理体系提出建议。方法：对德国、日本、美国和新西兰4个典型国家的创新药医保目录纳入机制进行纵向案例介绍，再从企业参与程度、价值评估机制、药品定价标准以及动态调整周期4个方面进行横向总结对比，分析其模式中创新及可借鉴之处。结果：4个典型国家在此方面已经具有相对成熟的流程与丰富的经验，并且已经形成一套相对完备且各具特色的体系，对我国有借鉴意义。结论：建议建立健全创新药准入机制，以保证企业充分参与创新药准入全程，建立公平权威的创新药评估机制，选取科学合理的创新药定价标准，实施动态长效的目录调整周期。     

各国生物类似药适应症外推技术要求的比对研究及完善我国生物类似药适应症外推技术要求的建议

生物类似药的适应症外推,需要满足科学的前提条件。通过对美国、欧盟等国家以及WHO生物类似药适应症外推技术要求进行比对研究,提炼生物类似药适应症外推的前提条件,设计问卷、开展调研和研讨;结合对比研究、调研及专家研讨,提出完善我国生物类似药适应症外推技术要求考量的建议。     

多维度关注生物类似药的管理与临床应用

目的:为提高生物类似药的管理与临床应用水平提供参考。方法:检索国内外药品监管部门和世界卫生组织有关生物类似药审批和应用管理相关的政策法规,从药品全生命周期、通用名和处方、适应证外推、临床用药互换、药物警戒、医保支付体系、教育培训等多维度对生物类似药进行梳理。结果与结论:生物类似药是指在质量、安全性和有效性方面与已获准上市的参照药具有相似性的治疗性生物制品。在生物类似药的研发、生产、流通、使用、监管全生命周期系列环节中的管理在不同国家/地区/组织各有特点,其在研发阶段不需再独立验证安全性和有效性,只需用分析方法逐步从结构和功能上阐明其与参照药高度相似性即可;我国的生物类似药命名与原研药相同,采用通用名处方;美国FDA批准生物类似药的适应证外推需要基于其申请时的数据和信息、参照药的安全性和有效性信息及适应证相关科学要素的考量,需要经过评估,在监管下有条件地使用;美国FDA审批可替换生物类似物的标准严格,即实现互换的审批标准要高于生物相似的审批标准,但我国目前尚无这一概念;医药企业、监管机构、学术机构、医院药房之间需沟通交流,共同加强上市后风险控制和安全性监测;我国医疗保障部门需建立适宜的支付体系并通过支付制度鼓励生物类似药的使用;医务工作者要学习生物类似药的特点,才能在了解其技术审评的基础上,于实践中用好生物类似药。     

The regulatory framework of biosimilars in the European Union

In the European Union (EU), the regulatory policy for biosimilars has enabled different biosimilar products to be marketed through an abridged application, which allows the applicant to submit a reduced dossier. Nevertheless, some manufacturers of biological products that share some characteristics with copies have opted for a full application; therefore, the number and extent of clinical studies required in these cases is increased. Here, we focus on a comparison of recombinant human erythropoietin medicinal products. We analyse and discuss clinical studies submitted to the European Medicines Agency that relate to available biosimilars and biological medicinal products that are authorised with a full dossier. We also discuss the issues of interchangeability and substitution, given that the EU allows each Member State to set their own substitution policies.     

The regulatory landscape of biosimilars: Algeria's efforts and progress made from 2006 to 2021

Biologics are tremendously efficacious biological molecules that have enabled the treatment of many life-threatening diseases, which have previously been hard to treat. Biosimilars, also known as “follow-on biologics”, are highly similar versions of another already approved biologic, called the Reference Product. The European Union has been a pioneer in the regulation of biosimilars. WHO guideline on evaluation of biosimilars published in 2009 was an important landmark in biosimilar regulations worldwide, and several countries have adopted its principles in the development of their own regulatory pathway for the approval of biosimilars. Most countries in the Middle East North Africa (MENA) region still lack official and scientific guidelines for biosimilar approval pathways. This article explores the regulatory situation of biosimilar registration pathways in Algeria and describes the progress made and the regulatory landscape changes for biosimilars in Algeria during the past ten years. Our findings indicate that the development of biosimilar regulation in Algeria went through three major phases between 2006 and 2021, during which there has been much progress in drafting guidance documents for biosimilars. Since 2016, Algeria has used the EMA, FDA and WHO guidelines as the basis for approval of several biosimilars and no national guidelines or regulations have been adopted to date. Additionally, there has been no regulation on substitution/interchangeability. The Algerian regulatory authority has gained considerable experience with approval and use of increasingly complex biosimilars over the past 5 years and has the potential to create its own biosimilar-specific regulatory pathway in the near future.     

Biosimilars Are Here: A Hospital Pharmacist’s Guide to Educating Health Care Professionals on Biosimilars

Background:

Pharmacists are the recognized experts in pharmacotherapy. With the recent introduction of biosimilar agents into the US market, pharmacists are poised to play a pivotal role in evaluating their risks versus benefits within the framework of cost containment.

Purpose:

This article provides hospital pharmacists with the necessary information on the principles surrounding the development, approval process, and use of biosimilars.

Methods:

Information contained in this article enables hospital pharmacists to identify concerns relating to biosimilars, implement educational components, and successfully evaluate biosimilars for the addition to the formulary. Additionally, this article reviews the European experience with biosimilars, the US Food and Drug Administration approval process, and postauthorization safety and pharmacovigilance programs.

Conclusion:

It is important to educate health care providers about the differences between biosimilars and their reference biologics. The adoption of biosimilars is necessary to control long-term costs of biologics, increase patient access to care, and encourage innovation.     

Improving the power to establish clinical similarity in a Phase 3 efficacy trial by incorporating prior evidence of analytical and pharmacokinetic similarity

ABSTRACT

To improve patients’ access to safe and effective biological medicines, abbreviated licensure pathways for biosimilar and interchangeable biological products have been established in the US, Europe, and other countries around the world. The US Food and Drug Administration and European Medicines Agency have published various guidance documents on the development and approval of biosimilars, which recommend a “totality-of-the-evidence” approach with a stepwise process to demonstrate biosimilarity. The approach relies on comprehensive comparability studies ranging from analytical and nonclinical studies to clinical pharmacokinetic/pharmacodynamic (PK/PD) and efficacy studies. A clinical efficacy study may be necessary to address residual uncertainty about the biosimilarity of the proposed product to the reference product and support a demonstration that there are no clinically meaningful differences. In this article, we propose a statistical strategy that takes into account the similarity evidence from analytical assessments and PK studies in the design and analysis of the clinical efficacy study in order to address residual uncertainty and enhance statistical power and precision. We assume that if the proposed biosimilar product and the reference product are shown to be highly similar with respect to the analytical and PK parameters, then they should also be similar with respect to the efficacy parameters. We show that the proposed methods provide correct control of the type I error and improve the power and precision of the efficacy study upon the standard analysis that disregards the prior evidence. We confirm and illustrate the theoretical results through simulation studies based on the biosimilars development experience of many different products.     

药品集中带量采购政策与我国医保支付制度的协同作用探讨

目的:探讨我国药品集中带量采购政策与医保支付制度的协同作用及尚存不足,为进一步提升其协同水平提出可行性建议.方法:采用文献分析法和政策评估法,分别对目前我国药品集中带量采购政策与医保支付制度的直接协同与间接协同作用进行阐述,分析其存在问题,并提出可行性建议.结果与结论:药品集中带量采购政策对我国医保支付制度在技术层面的...     

A prospective view on European pharmaceutical research and development. Policy options to reduce fragmentation and increase competitiveness

This article analyses 3 areas of policy that could reduce the fragmentation and improve the competitiveness of the European pharmaceutical sector. It argues that a potential solution to the issue of fragmentation of pharmaceutical research, development and innovation may be the development of policies at the European level, in those areas that European institutions have a competence. These areas may not necessarily rely exclusively on solving the issue of pricing and reimbursing pharmaceuticals as European Union (EU) Member States invoke the subsidiarity principle to claim policy exclusivity in this area. By contrast, policy areas where European institutions have a competence may include: i) a more intensified collaboration in science and technology policy (supporting the science base, identifying education needs for the future, collaborating in the development of new technologies and fostering university-industry collaboration); ii) support of research and development (R&D) by means of directly channelling funds into basic pharmaceutical research, avoiding duplication of the research effort, developing a set of research priorities, tackling the issue of technology transfer, promoting university-industry and cross-border collaborations or providing incentives that would induce private R&D activities in areas with large socioeconomic impact; and iii) an improvement in the environment for the financing of innovation in the EU, by means of selective use of tax policy at the national level (and where applicable, at the EU level), institutional reform in order to widen the pool of available funds for private investment, and the introduction of schemes that would encourage individuals and institutions to hold equity in innovative companies. The article identifies specific research, regulatory, medical and financing needs that require policy intervention, evaluates the possible dynamic implications of such interventions and highlights the benefits that may accrue from their implementation.     

Effective pharmaceutical regulation needs alignment with doctors

Concerns emanating from the medical community about the safety and efficacy of biosimilars indicate an increasing distrust of the outcome of the drug regulatory process. To illustrate this, we analysed the creation of the European biosimilar regulatory framework, specifically focussing on the guidelines outlining approval criteria for biosimilar erythropoietins, which have been recently adopted. We observed an absence of the organised medical community in the public process of creating and updating the guidelines. In this article we argue that, to ensure that innovative medicines continue to find their way to the patients who might benefit from them, a closer collaboration between the organised medical community and regulators is needed.     

药品集中招标采购机制招标采购主体设置规律述评——基于欧盟国家不同医疗保障制度实践经验的总结

目的:为完善我国药品招标采购体系,为解决药价虚高和药价畸低带来的质量问题提供参考。方法:将部分欧盟国家药品招标采购机制,依照社会医疗保险和国家卫生服务两种医疗保障模式分类,分别进行经验总结。以医药费用支付方式为纽带,建立相应主体之间的财务联系,以此分析各国在相应医药费用支付方式组合下的药品招标采购系统,讨论相应机制下医药费用超支承担者或分担者与药品招标采购主体之间的关系。结果与结论:医疗保障模式及其相关设计与药品集中招标采购机制设计关系紧密,决定着招标采购系统的具体设计。各国药品集中招标采购的主体都为医药费用超支责任的承担者。这遵循了博弈论分析招标采购机制相关文献中所提的"利益直接相关者作为招标主体参与招标博弈才能使其本身利益得到最大化满足"的基本原则。