各国孤儿药政策对比分析

据统计,医学上明确诊断的罕见病有5 000~8 000 种。虽然因患病人数少而得名罕见病,但考虑到疾病种类之多,罕见病仍是各国不可忽视的公共卫生挑战。协助和激励医疗行业开发治疗罕见病的孤儿药,是各国政府一项重要的公共卫生政策。重点介绍了美国、日本和欧盟的孤儿药立法,激励政策以及对病患、医药行业的积极影响。     

罕见病基因治疗与孤儿药

孤儿药因面向的罕见病患者群小、市场需求低、研发成本高、缺乏政策支持等,其发展面临困境。随着精准医疗概念的提出,基 因治疗因能够从根本出发,给患者提供 “一劳永逸”的治疗,备受关注。基因治疗以单基因罕见病的治疗作为极佳切入点,为孤儿药的 研发带来了新的希望。概述基因治疗针对的疾病对象、实施策略和属性以及基因药物的结构及基因治疗的载体,以血友病的基因治疗为 例回顾罕见病基因治疗的发展,并分析罕见病基因治疗药物研发现状。     

美国生物制品中孤儿药的研发现状 及对中国孤儿药研发策略的思考

过去30 年, 美国在全球第一部《孤儿药法案》推动和相关政策法规引导下,生物科技飞速发展,尤其在孤儿药研发领域,成果卓著。 对这30 年来美国FDA 批准的生物制品中孤儿药产品作一解析,回顾美国对生物制品中孤儿药的研发策略,介绍孤儿药开发的新型商业 模式,同时反思中国孤儿药研发策略。     

“首届中国孤儿药研发论坛”专家观点

对2015 年4 月25 日召开的“首届中国孤儿药研发论坛”的专家报告内容进行归纳总结,旨在为从事罕见病诊断和孤儿药研发工 作的人士提供信息参考。报告内容涉及国内外孤儿药研发现状和前景、中国孤儿药政策和审评状况、罕见病诊断以及中国孤儿药研发的机 会等。     

“首届中国孤儿药研发论坛”专家观点（续）

对 2015 年 4 月 25 日召开的“首届中国孤儿药研发论坛”与会专家对话内容进行归纳总结,旨在为从事罕见病诊断和孤儿药研发工作的人士提供信息参考。来自产、学、研领域的专家从研发、临床、审评、投资、媒体等角度探讨了中国孤儿药研发策略。     

“孤儿药”激励机制亟待设立

在治疗罕见病的"孤儿药"的研发领域,一直活跃着许多新兴的中小型生物技术企业。而现在,随着越来越多的大型制药企业将研发重心转向孤儿药,这个领域呈现出群雄逐鹿的态势(见本期"产经"栏目)。对于我国的制药行业来说,这个领域也充满的着机会,因为在一定意义上来说,大家都处于同一起跑线上。但相比于发达国家日益健全的针对"孤儿药"研发的法规,我国在相关政策和立法上还处于起步阶段,如果不尽快推进相关法规和激励机制的设立,那必将制约中国     

欧盟罕用药政策的分析及其对我国的启示

目的 通过分析欧盟及其成员国的罕用药相关政策,为我国罕用药及罕见病相关制度政策的制定提供参考。方法 采用文献研究法对欧盟及其成员国法国、意大利、荷兰、英国的罕用药相关政策进行检索,并比较分析四国相关政策与制度的异同点。结果 除欧盟统一的罕用药政策外,法国、意大利、荷兰以及英国均制定了相应的罕用药制度,设立罕见病治疗指导中心,有针对罕用药研发与价格制定的政策与措施,对于罕见病的治疗有相应的医疗保障制度,相应的政策制度较为完善。 结论 我国可借鉴其经验尽快制定罕见病的相关定义,建立罕见病中心,出台相应的研发激励与价格制定政策,并完善罕用药的报销与罕见病的医疗保障。     

开发抗寄生原虫药的途径

开发抗寄生原虫药的途径许多热带寄生虫病的治疗迫切需要新的、有效和安全的药物。基础生物化学研究可以为这个问题提供帮助,方法是鉴定寄生虫独特而必需的代谢过程,成为以后合理的药物设计的基础。这项多学科研究可分为３个主要阶段：靶鉴定与验证、合理的抑制剂设计、...     

仿制药一致性评价品种的美国简略新药申请申报策略

文章从产品的开发以及申报资料的撰写等方面探究仿制药一致性评价品种的美国简略新药申请申报策略,为中国仿制药国际化战 略提供参考。     

药品专利监测与非专利药开发策略

随着国际化战略、市场竞争和运作的日益加强，知识产权保护的意识越来越强。如何有效地应用专利战略，合理地规避专利风险已经成为医药研发中的一个主要问题。在“未来十年内，将有大量‘重磅炸弹’级药品专利到期”的一片声音中，我们不禁提出问题，     

在美国进行植物药开发的路径、要求及策略探讨

近年来,越来越多药企开始在美国进行植物药的临床试验申请和新药申报,美国 FDA 日益重视植物药的开发,于 2016 年 12 月 发布并实施了修订版的《行业指南:植物药》,为植物药后期开发提供更多专业建议。结合 FDA 已经批准的 2 个植物药案例,详细介绍 植物药 CMC(化学、生产、控制)、临床前研究和过往人用历史方面的基本要求,浅谈 FDA 的申报要求、审评思想和申报策略,以期 为中国中药企业的国际化开发和注册提供参考。     

溶酶体贮积症和其他罕见疾病适应证治疗药物的相关交易情况

介绍汤森路透Cortellis 竞争情报分析数据库报道的市场上高额的溶酶体贮积症和其他罕见疾病治疗药物的合作协议以及该领域某些重要的有前景的候选治疗药物。     

Stream Restoration Practices in the Southeastern United States

We collected information on 860 stream restoration projects in four states in the southeastern United States—Georgia, Kentucky, North Carolina, and South Carolina—to gain a better understanding of the practice of stream restoration in this area of high aquatic biodiversity and rapid metropolitan expansion. This was completed as a part of the National River Restoration Science Synthesis, with the larger goal of understanding the state of the science of stream restoration. Stream restoration project density, goals, and monitoring rates varied by state, although southeastern monitoring rates were higher than in other parts of the country. North Carolina had the most projects in the Southeast, of which 36% were monitored. In‐depth phone interviews with project managers from a random subsample of projects provided insights into the process of stream restoration. Land availability was the most common basis for site prioritization, and 49% of projects involved mitigation. Although 51% of projects were associated with a watershed assessment, only 30% of projects were done as part of a larger plan for the watershed. Projects were monitored using physical (77% of monitored projects), chemical (36%), and biological (86%) variables, although many projects were planned and ultimately evaluated based on public opinion. Our results suggest that stream restoration in the southeastern United States is at an exciting point where better incorporation of a watershed perspective into planning and establishment and evaluation of stated, measurable success criteria for every project could lead to more effective projects.     

高通量测序技术在罕见病分子诊疗中的 应用及临床实例分析

罕见病病种繁多,且表型复杂多样,不仅仅体现在疾病间的不同,同一种疾病的不同患者在表型上也可能大相径庭。这种普遍存 在的遗传异质性和临床异质性,使罕见病的诊疗极具挑战。近年来,在后人类基因组计划时代,各种测序技术快速发展,使得大规模测 序如疾病目标基因集测序、全外显子组测序、全基因组测序等成为了现实。高通量测序技术可实现对多个靶基因进行高通量平行测序, 有效节约了成本与时间,越来越广泛地应用到临床疾病分子诊疗领域。分析传统测序技术与高通量测序技术的优缺点,介绍罕见病诊疗 中常用的高通量测序策略,并结合临床实例,综述高通量测序技术在罕见病诊疗中的应用。     

全球抗HIV 药物研发进展报告

近年来,艾滋病在世界范围内的迅速传播和蔓延,已严重威胁着人类的健康,且引发了一系列社会问题。报告以Thomson Reuters Cortellis 数据库为数据源,从总体研发态势、研发阶段、作用靶点、研究地区、研发机构、销售情况以及交易合作等角度对全球 抗HIV 药物的研发情况进行多角度、多层次的分析,旨在为我国抗HIV 药物的研发提供参考。     

The Psychologizing of Chinese Healing Practices in the United States

This paper explores ways in which Chinese healing practices have undergone acculturation in the United States since the early 1970s. Reacting to what is perceived as biomedicine's focus on the physiological, those who describe themselves as favoring a holistic orientation often use the language of 'energy blockage' to explain illness, whether thought of as 'physical,' 'emotional,' or 'spiritual'. Acupuncture in particular has been appropriated as one modality with which to 'unblock' such conditions, leading to its being used by some practitioners in conjunction with more psychotherapeutic approaches which include valuing the verbalizing of feelings. Some non-Chinese practitioners in the United States, returning to older Chinese texts to develop 'an American acupuncture,' are reinserting diagnoses eliminated from Traditional Chinese Medicine (TCM) by the People's Republic of China as 'superstition'. The assumption has been that many such diagnostic categories refer to psychological or spiritual conditions, and therefore may be useful in those American contexts which favor this orientation. Among these categories are those drawn from traditions of demonology in Chinese medicine. What was once a religious category in China turns psychological in the American setting. At the same time, many who use these terms have, since the late 1960s, increasingly conflated the psychological and the religious, the latter being reframed as 'spiritual'. Thus, this indigenization of Chinese practices is a complex synthesis which can be described as simultaneously medical, psychotherapeutic, and religious.