罕见病政策国际经验及对我国的启示

本研究系统梳理了近年来有关罕见病政策和服务的文献及文件,归纳了美国、日本、欧盟等国家和地区罕见病方面的立法及相关法规、管理制度、激励措施、保障政策等方面的政策设计和实践经验。目前,发达国家和地区对罕见病制定了较为系统的政策和法规,罕见病药品的研发和医疗保障等在法律层面得到了不同程度的保障。我国尚未形成针对罕见病的系统性的政策体系,结合中国实际,我国应从逐步建立系统的罕见病政策法规、拓宽筹资渠道、鼓励孤儿药研发,扩大新生儿筛查疾病的范围等方面着手,推动我国罕见病政策发展。     

国内外罕见病用药保障模式的评述

目的：通过比较国际和国内关于罕见病药物保障模式的特征和侧重点,总结目前我国在这一领域实践的经验和不足,为保障模式的选择和相关政策的改进提供建议。方法：梳理美国、欧盟、新加坡、澳大利亚等国家和组织成员国针对罕见病出台的保障措施,以及国内各地区的探索经验,并分析不同保障模式之间的差异。结果：目前关于罕见病比较典型的保障模式有医疗保险报销、财政资金补助、专项基金补偿、医疗救助资金援助等,它们在筹资规模、保障水平、可持续性等方面有所不同。结论：我国需在考虑自身经济实力、罕见病流行特征等因素的基础上,采取多层次、多水平的综合保障模式,力求精准化资助罕见病患者的用药,共同分担其费用负担。     

浙江省、上海市及青岛市罕见病患者用药待遇水平比较分析

目的:为我国其他省市选择和设计罕见病药品保障模式提供参考。方法:梳理浙江省、上海市及青岛市3个典型地区的罕见病药品保障模式,以戈谢病为例,对比3个典型地区给予患者药品费用的报销比例,反映罕见病患者在3个典型地区的用药待遇水平差异,并分析产生待遇差异的原因。结果:浙江省实际报销比例最高、青岛市其次、上海市最低,原因是浙江省和青岛市筹资渠道的多元性与筹资来源的稳定性提高了其支付水平,药品谈判议价、用药风险管理等"节流"措施为其腾出了更多资金空间。结论:建议其他省市探索平建立渠道多元、来源稳健的罕见病药品筹资机制、通过谈判机制、用药管理等措施,将节约的资金用于提高罕见病患者待遇水平。     

哥伦比亚罕见病医疗保障制度研究及其启示*

罕见病保障是我国社会关注的热点话题。近年来,随着医疗保障体系的不断完善,罕见病保障能力得到提升, 医保目录已纳入多种罕见病用药,但在立法、制度建设等方面仍存在不足,患者治疗负担较重。哥伦比亚通过立法建立了独立于公共医保的“最高预算”基金,对罕见病患者进行保障。文章在构建罕见病政策分析框架的基础上,从“制度目标、制度设计、制度效能”3个层面剖析了哥伦比亚罕见病保障制度,基于保障经验,建议我国从优化登记系统、完善立法体系与探索专项基金保障3个方面完善罕见病保障体系。     

我国罕见病“友好型”普惠险评价指标体系构建及应用探索*

构建科学客观的罕见病“友好型”普惠险评价指标体系，为普惠险更好地参与罕见病多层次保障提供参考依据。文章以文献研究为基础，综合应用主成分分析法确定评价体系与指标权重，并对我国市面上保障范围不限制罕见病、基因型疾病或遗传性疾病的62款普惠险产品进行指标测算与应用。在保证基金稳定持续运营的基础上，为产品设置合理的既往症条款和责任免除条款；探索建立普惠险特药清单制度或地方性专项保障制度；在目录外住院自费保障的基础上，增加门诊的保障责任。     

我国罕见病用药医疗保障政策及医保目录收载情况分析

目的:通过分析国际、我国国家及地方罕用药保障制度、政策以及我国医保目录罕用药收录品种,探索我国罕用药医疗保障可及性现状及发展建议。方法:计算机检索国家及地方官方政府网站和中文数据库等,获取罕见病及罕用药相关政策法规;系统提取《国家基本医疗保险、工伤保险和生育保险药品目录》中有关罕见病的药品信息。结果:我国尚无罕见病的官方定义和官方名录。近年来关于罕见病、罕用药研发审批政策措施正日渐丰满。在医疗保障方面,很多省、市地区已经针对地方罕见病的流行性特征出台并落实了罕见病的保障政策,但国家层面的整体政策方面仍是空白。2017年版国家基本医疗保险、工伤保险和生育保险药品目录其中罕用药共53种,与2009年版目录相比,新增7个品种,增幅为15.22%;共覆盖17种罕见病,涉及脑部或神经系统病变、先天性代谢异常、血液疾病、骨及软骨病变多个疾病领域。与往年相比,新版医保药品目录中罕用药的剂型也有所增多,其中甲类相对乙类的剂型种类更多。结论:我国罕见病保障政策集中于研发审批领域,医疗保障以地方政策为主,新版国家医保药品目录罕用药范围扩大,但配套政策有待完善。因此,我国罕用药保障问题需要进一步引起重视。     

常州市农村基本公共卫生服务研究

目的：了解常州市基本公共卫生服务现状,为完善农村公共卫生服务体系和保障机制提供科学依据。方法：通过座谈,利用相关统计报表和现场观察等方法,收集农村基本公共卫生服务组织体系、服务状况、经费投入等资料,并进行分析。结果：2008年全市农村基本公共卫生服务指标全部完成,服务工作基本规范,服务网络基本形成,财政保障基本到位。但农村基本公共卫生服务还存在不少问题,如农村人均筹资标准有待进一步提高,乡村医生的保障需要认真关注。结论：常州市农村基本公共卫生服务尚需健全组织体系和工作机制,提高筹资标准,明确工作内容,完善项目管理和建立科学考评机制。     

澳大利亚救生药物计划及对我国罕见病医疗救助制度的启示

目的:介绍澳大利亚救生药物计划,为完善我国罕见病医疗救助制度提供参考。方法:根据文献分析澳大利亚救生药物计划的主要内容和特点,并与国内罕见病医疗救助现状进行比较。结果:澳大利亚通过孤儿药资格认证和成本效益评估将符合条件的孤儿药纳入救生药物计划,并通过财政资助为罕见病患者提供免费的治疗药物。该计划规定了严格的孤儿药和患者纳入条件,建立了罕见病临床指南和专家咨询制度,并定期开展上市后再审查,提高了孤儿药的可及性。结论:我国应当借鉴澳大利亚经验,由国家主导罕见病和孤儿药的管理,在大病医疗保险的基础上探索符合我国国情的罕见病医疗救助制度。     

基于彩虹模型的医疗机构罕见病诊疗协作评价指标体系构建研究

目的 建立罕见病诊疗协作网内医疗机构视角下的罕见病诊疗协作评价指标体系,为促进罕见病诊疗协作工作的发展提供指导依据。方法 通过文献研究法选取初始评价指标,然后开展专家咨询完善评价指标,进而采用层次分析法计算各级指标的权重。结果 创建了涵盖制度协作、组织协作、专业协作、服务协作、功能协作和价值规范协作6个维度下的18个二级指标和61个三级指标的评价体系。专家权威系数为0.75。所有指标重要性和可操作性的综合评分均大于3.5分,变异系数均小于0.25。权重最高的一级指标是制度协作（0.242）,最低的是价值规范协作（0.063）。结论罕见病诊疗协作指标评价体系具有科学性和可靠性,可为协作网内罕见病诊疗协作机制的完善提供指引。     

三省（市）医疗保险特殊用药谈判制度比较

目的:了解山东省青岛市、江苏省和浙江省医疗保险特殊用药谈判制度的基本情况。方法:查阅山东省青岛市、江苏省和浙江省与医疗保险特殊用药谈判制度相关的政策法规,从谈判机制、保障对象、支付范围、筹资机制及补偿水平五个方面进行比较。结果:青岛市筹资来源于财政拨款,谈判涉及药品和医用材料,覆盖重大疾病、罕见病及慢性病;江苏省无进行额外筹资,有针对性地救助乳腺癌和慢性髓性白血病患者,并将仿制药纳入了谈判范围;浙江省建立了财政补贴与个人缴费的分担机制,没有限定医疗保险特殊用药的临床指征,实行按费用分段补偿。结论:为进一步完善制度,可鼓励医疗保险特殊用药赠送谈判;增加罕见病用药,逐步纳入医用材料;建立医疗保险特殊用药保障目录的动态调整机制。     

医疗服务供方支付制度改革的挑战与建议

简要回顾了我国医疗服务供方支付方式改革进展,分析了目前医疗服务支付体系面临的主要挑战,包括：医疗服务各方职责关系不明确、缺乏系统的质量标准和质量改进机制以及缺乏对供方的有效激励与引导等。指出单一的支付方式改革无法解决系统问题,必须通过开展系统、综合性改革才能在支付改革领域取得突破;建议推动经办机构成为战略服务购买者,通过需方筹资改善服务绩效,完善支付制度作为医疗保险战略采购政策工具的功能。     

德国社会保障体系及医疗运营对我国医改的借鉴

目的随着医药卫生体制改革的不断深入,建立适合中国国情的医疗服务体系,逐步缓解群众看病难看病贵问题日益成为需要解决首要矛盾。在完善医疗保障和服务方面,德国在社会保障体系的架构、医疗服务的运行、管理和补偿等环节实现了良性的运转。其完善的社会保障体系、政府主导的医疗补偿机制、高效的区域卫生服务网,以及规范的医疗服务质量管理,和DRG-s结算方式等方面的经验和实践,对完善我国医改的相关政策、措施具有一定的借鉴和指导意义。     

Social and occupational health protection for self-employed farmers in the European Union

Aim The focus of this article deals with the diversity of national social security provisions specific to self-employed farmers in the European Union (EU) and attempts an initial research and categorisation of social protection provisions applying to the European population of self-employed farmers. Methods An extensive internet search was performed to identify national social security provisions for self-employed farmers. A crude categorisation of social security provisions for self-employed farmers is attempted aiming to identify EU Member States (MS) that apply: (1) a general social protection scheme for all the population, (2) a social protection scheme specific for the totality of the self-employed population, and (3) a social protection system specific for self-employed farmers. National provisions for the coverage of self-employed farmers for occupational injuries and diseases are also categorised. Moreover a care study from Finland is presented describing the innovatory Finnish system of substitute farmers’ services that allows self-employed farmers to be substituted in their farms in case of sickness, injury or holidays. Conclusion Persons occupied in agriculture in the EU MS, while facing many of the same occupational risks, are covered to varying degrees by national social security schemes. An initial conclusion that was derived is that countries that apply special social security systems for self-employed farmers are more likely to include in the provisions of their systems the coverage for occupational injuries and diseases as well. The increased diversity of national provions regarding the protection of self-employed farmers should be further researched at the European level and efforts for the coordination of relevant national policies towards a more comprehensive coverage of farmers should be discussed.     

老年慢性心功能不全患者的延续性护理进展

目前人类社会逐渐步入老龄化,老年性疾病威胁着人们的健康,心脑血管疾病患病率逐年升高。慢性心功能不全是心脏病的终末期表现,反复出现的心功能衰竭,增加了患者住院的频率和死亡风险,如何解决这一问题显得尤为重要。以传统生活方式干预为主的护理措施,已经不能满足人们对于护理服务的需求,只有更加多元化医疗服务才能满足社会需要。延续性护理的出现对护理工作提出了新的概念,护理工作不止局限在医疗机构之内,而是延伸到医院以外。延续护理的出现,满足了各种人群的不同需求,适应了社会发展。我国延续性护理服务体系尚不完善,本文就延续性护理在慢性心功能不全的应用情况进行综述,为慢性心功能不全患者延续性护理工作的开展提供借鉴和参考。     

Tackling rare diseases at European level: why do we need a harmonized framework?

Since 1999 the European Commission has gradually developed a proactive approach towards rare diseases (RD). Despite the progress made over the last years, a comprehensive and evidence based approach is still missing in many EU Member States (MS), leading to an incomplete and often inadequate framework to address rare diseases. Healthcare systems in EU MS differ to great extent among countries in respect to their organization and funding. In general, they are not ready to face the specific problems and needs of people with rare diseases for possible prevention, timely diagnosis, adequate treatment and rehabilitation. Access to new advanced treatment and approved orphan drugs by EMEA is also a big challenge for many MS. A public health approach is needed to properly tackle rare diseases. It is a while that the idea of a comprehensive approach addressing the different challenges of rare diseases is under discussion. In our opinion, the first step to build a comprehensive approach is to properly plan the activities to undertake accordingly to needs, gaps and resources available in a Country. It is therefore important to develop a strategic plan. Adopting a strategic planning approach to rare diseases implies taking advantage of ongoing actions and building on it to adjust, re-orient or expand the response. So far only France has developed a national strategic plan for rare diseases, Bulgaria is in the process of approving its national plan for RD and Spain is in the process of developing it. In this context, considering the importance of developing national plans for RD, it would be very useful to develop recommendations for RD national plan development in order to provide an instrument to support Countries in designing their national plans. The three MS initiatives presented in this paper confirmed the availability of great experiences and expertises among many EU MS and supported the idea that all these different experiences available at the EU level should form the basis for developing recommendations on how to develop strategic plans for RD. The recommendations will provide an instrument to support Countries in designing national plans contributing to the development of a harmonised and evidence based approach for addressing RD in EU MS. The elaboration of a European Commission Communication on rare diseases will ensure that common policy guidelines are shared everywhere in Europe. The availability of recommendations for developing national plans on rare diseases will link MS efforts with a common strategy at European level.     

我国慢性病管理现状、问题及发展建议

慢性非传染性疾病对我国居民健康造成较大威胁,慢性病管理工作是我国卫生工作的重点。本文通过文献和相关政策研究,从宏观—政策,中观—组织机构间的协调,微观—患者和人群三个层面对我国慢性病管理工作进程进行梳理归纳,并立足我国慢性病管理实际提出我国慢性病管理仍存在的一些问题。建议未来的慢性病管理工作应注重提炼慢性病管理理论模型;结合医改分级诊疗工作的推行,构建整合式慢性病管理服务网络;加快提升慢性病管理"终端"能力建设。     

“Kindhearted People,Please Save My Family”: Narrative Strategies for New Media Medical Crowdfunding

ABSTRACT

With the development of new financing methods in the networked society, and due to the underdeveloped social security system in China, more and more patients and their families have to choose crowdfunding as an important way to raise treatment funds. Using thematic narrative analysis, this paper studied 100 texts of medical crowdfunding on Easy Fundraising from February 27, 2018 to May 1. It is found that the requests used a series of strategies including: constructing the identity of the patient in order to build a disadvantaged image worthy of help; using tragic narration based on the traditional Chinese cultural elements such as “family concept” and “filial piety” and contrast of the patients’ experience before and after the illness to mobilize the sympathy of potential donors; and downplaying the needs itself in order to maintain patients’ self-esteem.     

Medical social work practice in child protection in China: A multiple case study in Shanghai hospitals

With the rapid development of the child welfare system in China over recent years, medical social work has been increasingly involved in providing child protection services in several hospitals in Shanghai. Focusing on five cases in this paper, the exploratory study aims to present a critical overview of current practices and effects of medical social work for child protection, based on a critical analysis of the multidimensional role of social work practitioners engaged in the provision of child protection services as well as potential challenges. Implications and suggestions for future improvements of China’s child protection system are also discussed.