WHO原料药预认证项目

目的 帮助国内原料药厂家了解并通过WHO预认证项目。方法 全面论述WHO原料药预认证项目的背景、申请流程、文件要求和意义。结果与结论 通过加深对WHO原料药预认证项目的认识,可以提高生产厂家参与WHO原料药预认证的积极性,增强通过预认证的信心。     

美国与欧盟孤儿药研发上市管理制度及对我国的启示

目的通过对美国与欧盟对孤儿药研发上市相关管理政策的分析,为我国孤儿药研发上市管理提供借鉴。方法分析美国和欧盟药政管理部门公开的法规文献和数据库检索数据,总结其对孤儿药的激励政策,分析获得孤儿药资格认定和批准上市的药物特点。结果美国和欧盟对孤儿药研发均颁布实施了诸如市场独占期、政府资助、审评专家对研究方案的指导等相应的激励政策。研发机构应采取及早申请孤儿药认定、多途径发现孤儿药及孤儿药的再开发等研发策略。结论美国和欧盟对孤儿药的研发与上市激励政策,刺激了制药企业对罕见病治疗药物的研发热情,有效缓解了罕见病无药可治的现状,对我国制定相关孤儿药政策提供了有益的借鉴。国内创新型制药企业应尽早布局孤儿药的研发,应重点关注国内已经被大众接受的罕见病和治疗,以及超说明书使用的问题;重点关注生物仿制药如单克隆抗体的研发动态;重点关注国际孤儿药专业公司的研发动态,使孤儿药在国内相关法律法规建立健全后,立即有所响应,尽早抢占孤儿药研发的领先地位和市场地位。     

How should we regulate risk in biomedical research? An ethical analysis of recent policy proposals and initiatives

The existing regulatory framework for research is increasingly attacked for its “one-size-fits-all” approach. Many stakeholders contend that existing regulations formulate the same regulatory requirements for research involving very different levels of risk, and thereby unnecessarily stifle medical progress. To address this criticism, regulators are currently developing more risk-adapted approaches to regulating research. A key feature of these approaches is that they aim to calibrate subject protections, including ethical review and safety monitoring, to the risks that studies pose to participants.     

中成药集中带量采购分组规则对比研究

目的：系统梳理全国及各省、市(区)中成药集中带量采购(以下简称集采)有关政策文件,对比分析各地集采分组规则和方法,为完善我国中成药集采分组规则提供借鉴。方法：通过文献分析法和比较分析法,对纳入研究的22个政策文本进行深入分析,总结全国及省际联盟中成药集采的进展情况。结果：全国和省际联盟中成药集采按照功能主治、给药途径、市场情况划分采购组,分组规则与药品剂型及质量的关联度不高;根据市场竞争格局划分竞价组别,忽视了部分中成药的创新价值,亦未考虑企业及其产品特有的品牌价值和中医药文化传播能力,分组规则的完备性有待提升。结论：中成药剂型繁多,质量控制指标复杂,现有分组规则沿用化学药集采分组思路,缺乏以中成药价值为导向的顶层设计。建议采取多元分组方式,将现有入围规则中的技术评价“前置”,即在制定分组规则时考虑药品剂型、质量、创新价值、弘扬中医药文化等因素,并对分组规则做进一步完善与细化。     

Emerging roles of basophils in allergic inflammation

Basophils have long been neglected in immunological studies because they were regarded as only minor relatives of mast cells. However, recent advances in analytical tools for basophils have clarified the non-redundant roles of basophils in allergic inflammation. Basophils play crucial roles in both IgE-dependent and -independent allergic inflammation, through their migration to the site of inflammation and secretion of various mediators, including cytokines, chemokines, and proteases. Basophils are known to produce large amounts of IL-4 in response to various stimuli. Basophil-derived IL-4 has recently been shown to play versatile roles in allergic inflammation by acting on various cell types, including macrophages, innate lymphoid cells, fibroblasts, and endothelial cells. Basophil-derived serine proteases are also crucial for the aggravation of allergic inflammation. Moreover, recent reports suggest the roles of basophils in modulating adaptive immune responses, particularly in the induction of Th2 differentiation and enhancement of humoral memory responses. In this review, we will discuss recent advances in understanding the roles of basophils in allergic inflammation.     

欧洲药品管理局(EMA)对大黄药用的评估报告及有关启示

2020年5月欧洲药品管理局(EMA)发布了"掌叶大黄和药用大黄及其根的评估报告",根据大量文献对大黄的活性成分、大黄及其制剂有效性和安全性以及临床应用做了全面和深入细致的讨论.该报告特别指出大黄制剂仅限于偶发便秘短期应用,有遗传毒性风险和致癌性尚未完全排除,限制每日用量和给药持续时间;该内容与中医药理论对大黄的传统评...     

黄芩-金银花药对治疗新型冠状病毒肺炎潜在作用机制的网络药理学研究

目的:基于网络药理学探讨黄芩-金银花药对与新型冠状病毒肺炎(COVID-19)关联关系及潜在作用机制。方法:利用TCMSP和STRING数据库平台过滤黄芩-金银花的有效化合物及对应作用靶点;运用Cytoscape 3.7.0统计软件建立并分析黄芩-金银花有效化合物-作用靶点网络;利用STRING数据库平台和Cytoscape 3.7.0统计软件建立并分析黄芩-金银花有效化合物作用靶点PPI网络;利用Bioconductor对作用靶点进行GO功能和KEGG通路富集分析。结果:黄芩-金银花59个有效化合物通过171条通路作用于202个靶点发挥其中医临床功效及潜在的COVID-19治疗作用。结论:黄芩-金银花所含的活性化合物可能通过抗病毒、抗炎等和肺、肝及心血管保护等作用,对COVID-19引发的肺损伤、肝损伤、心血管疾病和炎性反应具有潜在的预防与治疗作用,研究结果可为中医药治疗新冠肺炎提供一定的理论基础与科学依据。     

The effect of enzyme replacement therapy on clinical outcomes in female patients with Fabry disease – A systematic literature review by a European panel of experts

BackgroundHeterozygous females with Fabry disease have a wide range of clinical phenotypes depending on the nature of their mutation and their X-chromosome inactivation pattern; it is therefore important to examine outcomes of enzyme replacement therapy (ERT) in the female patient population specifically. This paper presents the findings of a systematic literature review of treatment outcomes with ERT in adult female patients.MethodsA comprehensive systematic literature review was conducted through January 2017 to retrieve published papers with original data on ERT in the treatment of Fabry disease. The review included all original articles that presented ERT outcomes data on patients with Fabry disease, irrespective of the study type.ResultsClinical evidence for the efficacy of ERT in female patients was available from 67 publications including six clinical trial publications, and indicates significant reductions in plasma and urine globotriaosylceramide (GL-3) accumulation (in female patients with elevated pre-treatment levels) and improvements in cardiac parameters and quality of life (QoL). To date, data are insufficient to conclude on the effects of ERT on the nervous system, gastrointestinal manifestations, and pain in female patients with Fabry disease.ConclusionsThis review of available literature data demonstrates that ERT in adult female patients with Fabry disease has a beneficial effect on GL-3 levels and cardiac outcomes. The current evidence also suggests that ERT may improve QoL in this patient population, though further studies are needed to examine these results.