[survey] orphan drug supply security status: rare disease depressions to be filled

2014年,一场旨在帮助罕见病肌萎缩侧索硬化患者(也称“渐冻人”)的冰桶挑战赛活动在全球流行开来,让很多人了解到了“罕见病”这个概念。由于单种疾病患者人数较少,罕见病患者的用药也被称为“孤儿药”。如何让这样的极少数人也能享受到社会的福利和关怀?2016年12月21日,国务院常务会议确定了“十三五”期间深化医改重点任务,提出要健全药品供应保障体系,扶持低价药、“孤儿药”、儿童用药等生产。
来源| 经济日报
文 | 袁勇 续梦婷
目前,我国罕见病患者的药品供应保障存在哪些问题?造成这些问题的原因有哪些?有哪些可能的改善途径?带着这些问题,记者走访了患者、专家、药企以及有关部门等。
国际罕见病日期间,公益组织罕见病发展中心发起“罕见病家庭纪实摄影展”,介绍罕见病群体生活状态。刘云摄
张笑目前是某黏多糖贮积症患者组织的全职工作人员,平时经常在网上与病友及其家属分享医疗信息等。袁勇摄
1创新型“孤儿药”难寻“中国造”
近日,记者在北京见到了黏多糖贮积症I型患者张笑。黏多糖贮积症是一种罕见隐性遗传疾病,患者身体呈现全身关节僵硬、角膜浑浊、肝脾肿大、呼吸道阻塞等症状。病症给张笑带来了极大的痛苦和不便,“我甚至都无法自己洗头、穿袜子”。同时,张笑还面临一个无奈的现实:国内黏多糖患者长期处于无药可用的境况。目前,国内尚无针对此病种的有效药物,由美国拜玛琳公司生产的Aldurazyme是针对该病I型的特效药。该药于2003年上市,然而并未引入国内。
与张笑处境类似的患者还有不少。记者走访众多患者和专家后发现,目前国内很多罕见病患者完全依赖进口药物,如果药物未在国内上市,就会面临无药可用的处境,创新“孤儿药”难见“中国造”。
在山东省罕见疾病防治协会会长韩金祥看来,提升国内药企的“孤儿药”研发能力,生产出“中国造”的“孤儿药”,是解决国内罕见病患者用药困境的根本途径。但是相比发达国家,我国“孤儿药”研发能力严重滞后,创新型“孤儿药”产出几乎为空白。首都儿科研究所研究员宋昉对记者表示:“当前在全世界的创新药物中,‘孤儿药’已经占到了一半左右的比例,‘孤儿药’的研发能力已经成为医药领域研发创新能力的一个重要体现。在这方面,我国正处于一个十分尴尬的境地。”
产生这种差距的原因有哪些?非营利性组织罕见病发展中心主任黄如方认为,国内药企“孤儿药”研发乏力,很大程度上是因为激励机制欠缺。在欧美多个国家,都有不同类型的激励机制推动“孤儿药”研发。
相关研究文献显示,在1983年之前,美国的“孤儿药”研发同样举步维艰。转折点是美国1983年公布的《孤儿药法案》。2002年,美国《罕见病法案》出台,使罕见病研究有了明确的法律保障,罕见病研究基金也逐渐增加,使得美国“孤儿药”研制保障制度体系更加完善。当前,美国药企在“孤儿药”研发全过程享受临床研究奖助金经费支持和税收抵免政策,同时,药物上市后,可给予企业7年的市场独占期。自《孤儿药法案》颁布以来,美国“孤儿药”的注册申请数量大幅度提高。从1972年到1982年,只有10个“孤儿药”上市,从1983年到2015年,则有将近500个“孤儿药”上市,年销售额超过了400亿美元。同时,美国的“孤儿药”政策也引来世界多个国家和地区效仿。
对比之下,当前我国针对罕见病问题的专项立法还是空白,“孤儿药”相关的政策仅散见于药物安全监管制度等方面。多家药企对记者表示,国家层面的医药研发相关激励政策基本统一在创新药层面,药企要获得相关激励,“孤儿药”也只能与其他创新药物无差别地进入创新药相关激励政策申请通道。两类药物的上市成本几乎相同,激励机制也没有区别,但是市场需求却大相径庭,药企把研发精力集中于市场需求更大的常规药物也就不足为奇。
海外投资,沟通中国与当地文化的桥梁与方法:
全国人大代表孙兆奇自2006年起就坚持提交罕见病相关议案和建议。在他看来,要改变国内“孤儿药”研发乏力的状况,就必须通过专项立法予以政策倾斜,“加快罕见病专项立法,是解决当前罕见病患者困境和‘孤儿药’研发滞后问题的最迫切需要”。以利好政策推动市场力量来解决“孤儿药”的研发和产出问题,也是很多专家学者的共同观点。
目前,全球“孤儿药”市场正处于快速增长阶段,然而研发的滞后正让我国药企错失在巨大的“孤儿药”市场中分得更多利润的机会。医药市场研究机构Evaluate Pharma发布的2015年“孤儿药”市场报告显示,当前全球“孤儿药”市场有着近12%的年增长率,相比之下,2015年上半年,全球普通药品市场增长率仅为5.9%。报告预测,到2020年,全球“孤儿药”销量将达到1780亿美元,“孤儿药”在所有处方药销量总额的占比有望超过20%。全球各大制药巨头也瞄准这一市场,以并购等方式扩大自己在“孤儿药”市场中的占有率。与此同时,我国国内药企生产的“孤儿药”却几乎都是仿制药,利润率与创新药差距巨大,导致我国药企难以在“孤儿药”市场中分得高利润,其创新能力和市场价值也受到了很大制约。
2高价“孤儿药”能否入医保
对于药企来说,创新药的研发往往需要巨额投入。而“孤儿药”市场需求很小,药企要想通过销售利润覆盖研发和上市成本,并获得较好的回报,往往会利用专利保护将药物单价定得很高。
以黏多糖药物Aldurazyme为例,按照其上市价格,每个患者每年的药物花费至少要200万元,这让患者望药兴叹。张笑告诉记者,即使有途径从国外买到药,也根本买不起,“价格太贵了,所以我只能定期做一下检查,身体哪个器官出现问题了,做一下局部治疗。”张笑表示,目前她能够联系到的国内患者就有300多人,治疗状况基本和她类似。
拜玛琳中国区市场准入经理曹雯表示,如果药物不能进入医保目录,国内患者就很难用得上药物。“目前在国内推广‘孤儿药’的成本不小,包括临床试验和注册审批的投入、医保谈判、病人赠药等,同时Aldurazyme价格高昂,几乎不可能仅靠病人自费用药,必须在医保支付下才可能获得市场,国内目前尚不具备这样的条件。”曹雯说,Aldurazyme在多个国家和地区上市,都是通过纳入医保目录、减轻患者经济负担来获得市场的。曹雯还表示,针对Aldurazyme在国内获批上市,药企与多个地方政府有关部门进行过沟通,但是尚无进展,各地有关部门都表示药物要在获取注册资格之后才能考虑是否纳入医保目录。
在我国,由于罕见病特效药一般不属于基本药物,是否纳入医保的决定权一般在各地有关部门。但是对于地方来说,决定药物是否纳入医保必须在药物注册后再进行相关测算,因此,按照既有规定,各地有关部门的做法无可厚非。
推动更多“孤儿药”进入医保目录,压力有多大?人力资源和社会保障部发布的数据显示,我国2015年全年城镇基本医疗保险基金总收入11193亿元,支出9312亿元,年末城镇基本医疗统筹基金累计结存8114亿元,收入高于支出,结余数约为10个月平均支付水平。2009年人社部、财政部发布的《关于进一步加强基本医疗保险基金管理的指导意见》指出,统筹地区城镇职工基本医疗保险统筹基金累计结余原则上应控制在6—9个月平均支付水平。当前结余数略高于原则范围,显示出当前我国医保基金尚有较强支付能力。
但是,也有不少担忧的声音出现。韩金祥认为,由于中国罕见疾病种类和人群众多,进口“孤儿药”价格高昂,如果大规模纳入医保,很可能会逐渐影响到其他常见病、多发病患者的医疗支付,“事实上这种情况已在欧洲显现”。同时,有专家认为,医保基金较高的结余数掩盖了各统筹地区存在的差异,在部分地区,结余数甚至已经达到20个月以上的平均支付水平,但同时很多地区则已入不敷出,各地情况大有不同。
北京大学药学院药事管理和临床药学系主任史录文表示,由于全国各地存在医疗统筹水平和财政收入水平的差异,如果国家能够出台一个宏观政策,各地根据地区发病率和经济发展水平等自身情况建设救助体系,才是比较合适的救助体系建设方式。
事实上,国内经济发展水平较高的部分地区已经开始逐步探索将更多罕见病纳入医疗保障体系。2016年,浙江省将包括渐冻症、戈谢病、苯丙酮尿症在内的罕见病纳入医疗保障体系。渐冻症和苯丙酮尿症的治疗费用每年为十几万元,戈谢病的治疗药物Cerezyme在国内的购买费用则需每个患者每年至少200万元,且需终生服药,这些病种被纳入医保体系,大大减轻了患者的经济负担。据悉,被纳入保障范围的罕见病患者,将由基本医保、大病保险、医疗救助、财政专项资金等逐层分担化解其合规医疗费用。
但是在大部分地区,相比于患者需求,救助体系建设进展缓慢,用药难、用药贵的现象十分突出。在2016年两会上,孙兆奇建议在全国推广浙江省的罕见病医疗保障体制,以减轻患者负担,“很多患者及其家庭因病致贫、因病返贫,成为我国特别贫困人口中不可忽视的一部分”。
同时,有专家认为,如果高价“孤儿药”能够更进一步地纳入各地医保目录,减轻患者负担,有助于“孤儿药”获取更大的市场,对国内药企的研发也是一种巨大的激励。
3上市期待“绿色通道”

安全生产教育培训基本规定有哪些

按照规定,在创新药的专利保护期到期后,药厂可生产仿制药品,但需进行一致性评价等流程,合格后方可上市。仿制药的价格通常比创新药低很多,成为很多患者的替代选择。但是,记者调查发现,对于很多罕见病患者来说,使用仿制型“孤儿药”也面临重重困难。这是因为药物的上市成本与其他常规药物没有太大差别,且仿制药的低药价也导致药品利润较低,难以覆盖上市成本,抑制了企业推动药物上市的意愿。
陶子是一名罕见病先天性肾上腺皮质增生患儿的母亲,长期以来,她都被曲折的买药之路所困扰。氢化可的松是当前治疗先天性肾上腺皮质增生的必需药物,在国内,上海上药信谊药厂有限公司也生产可替代药物,但是只有20毫克剂量的产品。陶子介绍说:“这个病对于用药的剂量精准度要求很高,给小孩用药一般是5毫克剂量,20毫克的药品只能给成人用,如果用药不精准,会导致很大副作用。”而合适剂量的药物只能在国外购买。陶子表示,她曾和国外药企沟通,对方表示,因为药物利润太低,无法覆盖上市成本,因此不想进入中国市场。她只能从国外代购药物,这给陶子带来了很大的政策风险。
对此,记者联系了上药信谊药厂。上药信谊表示,如果公司获得5毫克或10毫克规格醋酸氢化可的松片的生产批文,且市场也有需求,公司也愿意适时组织生产,满足病人的临床需求。然而,要获得生产批文,药物的开发和审批周期较长,前期投入成本很大,如果市场很小,企业就不得不考虑经营负担的问题。
同样的两难还体现在“老药新用”上。涛子是一名罕见病结节性硬化症患者,主要服用的药物为仿制药西罗莫司。尽管疗效明显,但是西罗莫司的指定适应证并不包含结节性硬化症,在这种情况下,患者用药都需要通过医生申报的科研项目。此外,北京、深圳等地一些医院的专科门诊在通过伦理协会的试药备案后,也可以开药,但患者是以试药者的身份而非患者的身份用药,要跟医生签风险协议,这让患者的用药流程十分复杂繁琐。
“我们希望能够推进‘老药新用’,让更多患者能用上这个药,因为它价格便宜,患者可以负担得起。”涛子告诉记者,尽管国内有4家药企在生产西罗莫司,却只有华北制药表达了配合意愿,其余的药企态度消极,“所以华北制药也有很大顾虑,因为假如它花了很大成本推动药物增加适应证以后,其他药企也都受益,而其他药企已经进入很多医院,市场占有率更高”。

障碍的一面是激励机制欠缺,另一面则是相关成本高昂。华北制药相关人士焦金平表示,根据当前的政策,要新增适应证,一般需要3年左右的审批时间,“企业需要负担药物费用、临床治疗费用等,从资金和时间上来讲,都需要很大的成本,算一下收支账,其他企业也就不会有太多积极性”。
不少新功能有时会带来新的安全漏洞,所以安全需要有一个基本的思想,关闭不甚必要的功能和服务。
按照有关规定,新药进行临床试验往往需要获得足够多的病例支持。由于罕见病往往缺少足够的病例,在罕见病药物进入市场前,医药公司会提交“临床试验豁免”或“样本量减少”的申请,但是需要每次注册新药都进行单独提交。赛诺菲集团交流传媒部祝家莹表示,当前的审批方式会大大增加药企的时间成本,“希望相关部门出台明确的法规,以明确的原则规定罕见病药品可以获得免临床,或直接借鉴国外数据,并优先进行审评,而不需每次单独申请”。
事实上,为推动“孤儿药”上市速度,国家食品药品监督管理总局出台了一系列新药审批改革文件,提出针对罕见病患者等特殊群体用药给予加快审评、优先审评,但是实质效果并不明显。黄如方认为,首先是因为这些政策缺乏细则,相关部门如何执行并不明确。此外,我国罕见病用药和常规药物在同一审批渠道,缺少单独审批路径,审批效率也就无从提高。

阳泉商行综合业务系统通过信息安全等级保护三级测评

(文中“陶子”和“涛子”都为化名)
4工作开始上路
推动“孤儿药”审批效率提高,给予“孤儿药”审批绿色渠道至关重要。因此,如何认定罕见病和“孤儿药”,也是影响政策的重要因素。当前,我国对于罕见病尚未有明确的官方定义,世界各国则根据自身不同情况,有着不同的定义。史录文表示:“定义的不同,涉及实施的相关政策和措施的对象不同,因此如何定义罕见病,不但涉及发病率,也是一个经济性问题。”
在这个问题上,上海市先行一步。2016年2月份,上海市发布了《上海市主要罕见病名录(2016年版)》,56种疾病被纳入其中。国家有关部门也开始注意到这一问题,2015年12月,国家卫生和计划生育委员会组建了罕见病诊疗与保障专家委员会。国家卫计委对记者表示,“目前,卫计委依托专家委员会已经开展了以下工作:一是启动第一批罕见病目录的遴选,按照‘有明确诊断的技术方法、有药品治愈或明显控制缓解症状、社会反映比较突出’的原则,制订第一批罕见病病种遴选的标准和程序,并启动遴选工作;二是开展罕见病药品保障梳理和调查研究,专家委员会已经初步梳理我国罕见病药品保障情况,开始调查我国罕见病诊疗和用药现状,研究起草我国罕见病管理办法,为提高诊疗和保障水平提出意见建议。下一步专家委员会将依据职责,研究罕见病定义和病种范围”。
同时,有专家认为,加快“孤儿药”审评审批,需有非常详细的数据基础支撑,以确保药品的安全性和有效性,这恰恰也是我国“孤儿药”研发的短板,当前,我国流行病学数据失真严重、研究薄弱。因此有专家不断倡导并推广罕见病患者登记制度,以帮助建立罕见病数据库。“建立数据库可以补充流行病学数据的匮乏,完善‘孤儿药’的科学基础和核心知识,也可以为药物研发提供量化的数据支撑,使得药企在药物设计方面更加合理化。”黄如方说。
受此启发,国内众多的罕见病组织也开始收集各种罕见病相关数据,包括罕见病患者的分布和诊治信息,“孤儿药”研发、生产和销售信息,以及“孤儿药”的剂量、临床使用信息等,以期将来促进“孤儿药”临床试验的有效开展。而山东省罕见疾病防治协会等省级罕见病机构也正在组织进行全国范围内的罕见疾病摸底调查等基础研究,并得到了国家科技支撑计划的资金支持。
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In 2014, a rare disease to help patients with amyotrophic lateral sclerosis (also known as amyotrophic lateral sclerosis) of the ice bucket challenge activities worldwide popular, so many people understand the concept of rare disease. Because of the small number of patients with a single disease, rare disease patients are also known as orphan drugs. How can such a small number of people can enjoy the social welfare and care? In December 21, 2016, the State Council executive meeting to determine the 13th Five-Year during the key tasks of deepening medical reform, proposed to improve the system to ensure the supply of drugs, cheap drugs, support the orphan drug, children’s drug production.
The source of | Economic Daily
This paper | Yuan Yongxu Meng Ting
At present, China’s rare disease patients with drug supply security problems? What are the causes of these problems? What are the possible ways to improve? With these questions, the reporter visited the patients, experts, pharmaceutical companies and relevant departments.
International rare disease day, the rare disease center of public interest organizations launched a rare disease family documentary photography exhibition, the introduction of rare disease groups living conditions. Liu Yun photo
A smile is now a full-time staff of a mucopolysaccharidosis patient tissue, usually in line with the patients and their families to share medical information etc.. Island.
1 of a new orphan drug to Chinese made
Recently, the reporter saw Zhang Xiao mucopolysaccharidosis type I patients in Beijing. Mucopolysaccharidosis is a rare genetic disease in patients with recessive body presentation body stiffness, corneal opacity, hepatosplenomegaly, airway obstruction and other symptoms. Illness has brought great pain and inconvenience to Zhang smile, I can not even wash their own hair, wearing socks. At the same time, Zhang also faces a helpless reality: the domestic long-term situation in patients with sticky polysaccharide drug free. At present, there is no effective drugs for this disease, by the United States Malin company Aldurazyme is a specific drug for the disease I. The drug was listed in 2003, but was not introduced into the country.
Zhang laugh situation similar to patients with many. The reporter visited many patients and experts found that many domestic rare disease is completely dependent on imports of drugs, if the drug is not in the domestic market, will face no drug use situation, innovative orphan drug invisible China made.
In the view of president of Shandong province Han Jinxiang rare disease prevention association, to enhance the capability of research and development of domestic pharmaceutical companies orphan drug, to produce the Chinese orphan drug, is the fundamental way to solve the domestic rare disease medication dilemma. However, compared with developed countries, China’s orphan drug R & D capability is lagging behind, innovative orphan drug output is almost blank. Capital Institute of Pediatrics Professor Song Fang told reporters: in the current new drug in the world, orphan drugs have accounted for about half the proportion of ‘orphan drugs’ research ability has become an important embodiment of research and innovation capacity in the field of medicine. In this respect, our country is in a very awkward position.
What are the reasons for this gap? A rare disease development center director of the nonprofit organization Huang Rufang believes that the domestic pharmaceutical companies orphan drug research and development is weak, largely because of the lack of incentive mechanism. In Europe and the United States, there are different types of incentives to promote orphan drug research and development.
Relevant research documents show that in 1983, the United States orphan drug development is equally difficult. Turning point is the United States in 1983 released the orphan drug act. 2002, the United States, the rare disease act was introduced, so that rare disease research has a clear legal protection, rare disease research fund has gradually increased, making the United States orphan drug development and protection system is more perfect. At present, the development of the pharmaceutical companies in the whole process of orphan drug enjoy clinical research grant funds support and tax credit policy, at the same time, the drug can be given after the listing, the enterprise 7 years of market exclusivity. Since the orphan drug act was enacted, the number of applications for orphan drugs in the United States has increased significantly. From 1972 to 1982, only 10 orphan drugs listed, from 1983 to 2015, there are nearly 500 orphan drugs on the market, with annual sales of more than $40 billion. At the same time, the United States orphan drug policy has attracted a number of countries and regions in the world to follow suit.
In contrast, the current our country for rare disease problems of special legislation is still blank, orphan drug policy related only in drug safety supervision system etc.. A number of pharmaceutical companies told reporters that the pharmaceutical R & D relevant incentive policies at the national level unified innovation in the aspects of medicine, pharmaceutical companies to obtain relevant incentives, orphan drug only with other innovative drugs blanketly into innovative drug related incentive policies for channel. The two drugs listed cost almost the same, the incentive mechanism is no difference, but the market demand continues to be quite different, pharmaceutical companies to conventional drug development focus on the greater market demand will be not at all surprising.
National People’s Congress Sun Zhaoqi since 2006 to adhere to the proposal and the proposal of a rare disease related. In his view, to change the domestic orphan drug research weakness of the situation, it must be through special legislation to tilt policy, to speed up the rare disease specific legislation, is to solve the most urgent need to lag the current plight of rare diseases and ‘orphan drugs’ research and development. Favorable policies to promote market forces to solve the orphan drug research and development and output problems, but also many experts and scholars of the common view.
At present, the global orphan drug market is in a rapid growth stage, however, the lag is R & d let our pharmaceutical companies miss in the huge orphan drug market gets more profit opportunities. The pharmaceutical market research firm Evaluate Pharma released the 2015 orphan drug market report shows that the current global orphan drug market with the annual growth rate of nearly 12%, compared to the first half of 2015, the global average market growth rate of only 5.9%. The report predicts that by 2020, the global orphan drug sales will reach $178 billion, orphan drugs in all prescription drug sales accounted for more than 20% of the total is expected to exceed. The world’s major pharmaceutical giants are also targeting this market, mergers and acquisitions and other ways to expand their share of the orphan drug market. At the same time, China’s domestic pharmaceutical companies to produce the orphan drug has almost all generics, huge profits and innovation gap, resulting in China’s pharmaceutical companies to share the high profits in the orphan drug market, innovation ability and market value are greatly restricted.
2 high priced orphan drugs can enter Medicare
For pharmaceutical companies, research and development of innovative drugs often require huge investment. The orphan drug market demand is very small, the pharmaceutical companies to want to cover costs of listing by R & D and sales profit, and get good returns, often use the patent protection of drugs price will be very high.
The mucopolysaccharide drug Aldurazyme as an example, according to the market price, the annual cost of each drug in patients with at least 2 million yuan, which makes the patient look medicine helplessly. Zhang Xiao told reporters, even if there is a way to buy drugs from abroad, or simply can not afford to buy, the price is too expensive, so I can only do regular check, the body which organ problems, do some local treatment. Zhang Xiao said that she was able to contact the domestic patients have more than and 300 people, the basic treatment and she is similar.
Malin, China’s market access manager, said Cao Wen, if the drug can not enter the health insurance directory, it is difficult for patients to use drugs. At present in the domestic promotion of ‘orphan drugs’ cost is not small, including clinical trials and the registration and approval of investment, health insurance negotiation, patient donated medicine etc. at the same time, the high price of Aldurazyme, almost impossible by patients at their own expense medication, must be obtained in the Medicare payment market, currently still does not have such conditions. Cao Wen said, Aldurazyme in a number of countries and regions, are included in the directory of health insurance, to reduce the economic burden of patients to obtain the market. Cao Wen also said that in view of the Aldurazyme in the domestic market is approved, the relevant departments of pharmaceutical companies and a number of local governments had to communicate, but there is no progress, all departments are said to be in after obtaining the qualification to consider whether the drug into the health insurance directory.
In our country, as a result of rare disease drugs generally do not belong to the basic drugs, whether or not the right to be included in the health care sector in the relevant departments. But for the place, the decision whether the drug into the medical insurance must be registered after the relevant calculation, therefore, in accordance with the existing provisions of the relevant departments of the practice of understandable.

郑州信息科技职业学院借力互联网开展学生成长教育

To promote more orphan drugs into the health insurance directory, how much pressure? Ministry of human resources and social security data show that the basic medical insurance fund in 2015 the annual total income of 11193 towns in China billion yuan, spending 931 billion 200 million yuan, the urban basic medical fund accumulated balance of 811 billion 400 million yuan, higher than the revenue expenditure, the balance of the number of about 10 months the average level of payment. In 2009 the Ministry of human resources and the Ministry of Finance issued on the further strengthening of the basic medical insurance fund management guidance and pointed out that the urban workers basic medical insurance pool fund accumulated balance principle should be controlled in 6 to 9 months on average pay level. The current balance is slightly higher than the scope of the principle, showing that China’s current medical insurance fund has a strong ability to pay.
However, there are many voices of concern. Han Jinxiang believes that due to Chinese rare disease type and the number of people who, high import orphan drug price, if the mass into health care, is likely to gradually affect other common diseases, the patient’s medical payment, the fact that this situation has emerged in europe. At the same time, some experts believe that the balance of higher medical insurance fund to cover up the difference exists in the whole area, in some areas, the balance has even reached the average level of payment for more than 20 months, but at the same time, many regions have to make ends meet, the situation is quite different from.
Shi Luwen, director of pharmacy administration and clinical pharmacy department of Peking University School of Medicine said that due to differences across the country are medical overall level and financial income level, if the country can introduce a macro policy, according to local area incidence and level of economic development and its situation of construction assistance system, is the more appropriate assistance system.
In fact, some areas of the country’s high level of economic development has begun to gradually explore more rare diseases into the health care system. In 2016, a rare disease in Zhejiang province will include Gaucher disease, amyotrophic lateral sclerosis, phenylketonuria, into the medical security system. Amyotrophic lateral sclerosis and phenylketonuria treatment costs each year for more than ten million, Cerezyme treatment of Gaucher disease in the domestic purchasing costs need each with at least 2 million yuan a year, and need lifelong medication, these diseases were included in the health care system, greatly reducing the financial burden of patients. It is reported that the rare disease patients are included in the scope of protection, will be the basic health insurance, serious illness insurance, medical assistance, financial special funds, such as the level of sharing to resolve its compliance medical expenses.
But in most areas, compared to the needs of patients, the rescue system is slow, difficult to use, expensive medication is very prominent. In 2016 NPC and CPPCC, Sun Zhaoqi suggested that the protection of rare disease medical system in the country to promote the Zhejiang Province, in order to reduce the burden of patients, many patients and their family poverty caused by illness, become an indispensable part of our country especially poor population.
At the same time, some experts believe that if the price of orphan drug can be further incorporated into the local health insurance directory, reduce the burden of patients, can obtain the bigger market for orphan drug research and development of domestic pharmaceutical companies, is also a huge incentive.
3 listing looks green channel
In accordance with the provisions of the patent protection period after the expiration of the new drug, pharmaceutical production of generic drugs, but the need for consistency evaluation process, qualified rear can be listed. Generic drugs are often much cheaper than innovative drugs and become an alternative for many patients. However, the reporter found that, for many rare disease patients, the use of imitation orphan drug is also facing difficulties. This is because the cost of listed drugs did not differ with other conventional drugs and generic drugs also lower price lead to lower profits, to cover costs of listing, the enterprise will promote the inhibition of drug.
Momoco is a rare disease in children with congenital adrenal hyperplasia of the mother, for a long time, she has been plagued by twists and turns to buy medicine. Hydrocortisone is essential drugs, the treatment of congenital adrenal hyperplasia in China, Shanghai Xinyi Medicine Pharmaceutical Company Limited also the production of alternative medicine, but only the 20 mg dose of the product. Momoco said: this disease for the drug dose accuracy requirements are high, give children medication is generally 5 mg dose, 20 mg of drugs only to adults, if the medication is not accurate, will cause a lot of side effects. The appropriate dose of drugs can only be purchased abroad. Momoco said that she had to communicate, and other foreign pharmaceutical companies said, because the drug profits is too low, can not cover the costs of listing, so do not want to enter the market Chinese. She can only buy drugs from abroad, which brings a lot of policy risk to Momoco.
In this regard, the reporter contacted the drug Xinyi pharmaceutical factory. The drug Xinyi said that if the permit production company acquired 5 mg or 10 mg of specifications for Hydrocortisone Acetate Tablets, and the market demand, the company will timely organize production, meet the clinical needs of patients. However, to obtain production approval, drug development and approval cycle is longer, the cost is large, if the market is very small, the enterprise must consider the management burden problem.
The same dilemma is also reflected in the new use of old drugs. Tao Zi is a rare disease in patients with tuberous sclerosis, taking drugs mainly for generic pharmaceutical sirolimus. Despite the obvious curative effect, but does not contain the specified sirolimus indications of tuberous sclerosis, in this case, patients are required by the doctor application item. In addition, some hospitals in Beijing, Shenzhen and other places in the clinic by medicine ethics Association for the record, can also prescribe medication, but the patient is in the reagent status rather than the identity of patients with medication, to sign an agreement with the risk of doctors, which makes the patient’s medication process is very complicated.
We want to be able to promote the use of new drugs’, so that more patients can use this medicine, because it is cheap, patients can afford. Tao Zi told reporters that although there are 4 domestic pharmaceutical companies in the production of sirolimus, but only the North China Pharmaceutical expressed willingness to cooperate, the rest of the negative attitude of pharmaceutical companies, so the North China Pharmaceutical is also a great concern, because if they spent a lot of cost push drugs to increase the indications, other companies also benefit, while other companies have entered the many hospitals, higher market share.
One obstacle is the lack of incentives, the other side is related to high cost. North China Pharmaceutical official Jiao Jinping said, according to the current policy, to new indications, it normally takes about 3 years of processing time, companies need the burden of drug costs, treatment costs, and funds from the time of speaking, it takes a lot of cost calculate accounts, other companies will not have too many enthusiasm.
In accordance with the relevant provisions of the new drug clinical trials often need to get enough cases to support. Due to a rare disease often lack sufficient cases, in rare disease drugs before entering the market, the pharmaceutical companies will submit the clinical trial exemption or reduced sample size applications, but need each drug separately submit registration. Sanofi communication departments may Jiaying said that the current approval will greatly increase the industry cost of time, I hope the relevant departments issued clear regulations, with clear principles of rare disease drug can get free clinic, or directly from abroad data, and priority review, and do not need to apply separately for each.
In fact, to promote the orphan drug market, the State Food and Drug Administration issued a series of new drug approval documents of the reform, proposed a rare disease and other special groups to give up drug review, priority review, but the effect is not obvious. Huang believes that first of all, because of the lack of rules of these policies, the implementation of the relevant departments is not clear. In addition, China’s rare disease drugs and conventional drugs in the same approval channels, the lack of a separate examination and approval path, there is no way to improve the efficiency of examination and approval.
In the text, Momoco and Tao Zi are aliases
4 work started on the road
On this issue, the first step in Shanghai. 2016 in February, Shanghai issued a list of major rare diseases in Shanghai (version), the 56 diseases were included. National authorities have begun to pay attention to this problem, in December 2015, the national health and Family Planning Commission set up a rare disease diagnosis and protection of the expert committee. The State Planning Commission, told reporters, at present, Wei Planning Commission relies on the Committee of experts has carried out the following work: one is the selection of the first batch of rare disease directory to start, according to technical methods, a diagnosis of drug cure or significantly relieve symptoms and control society reflects the more prominent principle, make the first selection of rare disease standards and procedures, and start the selection work; two is the rare disease drug security review and investigation, the Committee of experts has been combing the rare disease drug security situation in China, began investigating the status quo of China’s rare disease diagnosis and treatment, research and drafting of regulations of our country in order to improve the diagnosis and treatment of rare diseases, and the level of protection is put forward opinions and suggestions. Next expert committee will be based on the responsibility to study the definition of rare diseases and disease.
At the same time, some experts believe that to speed up the orphan drug review and approval, have very detailed data base support, to ensure the safety and effectiveness of drugs, this is China’s orphan drug research and development of the short board, at present, China’s serious distortion of epidemiological data and weak research. Therefore, experts continue to advocate and promote the registration system for rare disease patients to help establish a rare disease database. The database can complement the lack of epidemiological data, improve the scientific basis and core knowledge of orphan drugs, can also provide quantitative data to support drug development, the pharmaceutical companies in drug design is more reasonable. Huang Huang said.
Inspired by this, many domestic rare disease organizations began to collect all kinds of rare disease related data, including the distribution and treatment information of rare diseases, orphan drug research and development, production and sales information, and orphan drug dosage, clinical use of information, in order to effectively promote the future orphan drug clinical trials . Shandong Province rare disease prevention association provincial rare disease organizations are organized based on a nationwide investigation of rare diseases, and obtained the national science and technology support program funding.
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