随着国际医药市场不断变革，针对罕见病的“孤儿药”市场发展近年来呈现迅猛势头，跨国巨头纷纷抢占先机，其背后的政策引导经验值得借鉴。当前生物技术发展为全球“孤儿药”产业带来新机遇，而我国产业发展要迎头赶上还需弥补多重缺失。

Along with the international medicine market change constantly,In view of the rare disease"Orphan drug"Market development present rapid momentum in recent years,Taking transnational giants have initiative,Behind the policy guidance of experience is worth using for reference.The current biological technology development for the world"Orphan drug"Industry brought new opportunities,To catch up and the development of China's industry still need to make up for lack of multiple.

　　全球“孤儿药”产业发展迅猛跨国公司借机“分羹” Around the world"Orphan drug"Industry developing rapidly multinational companies are dealing"Points rewards"

　　世界卫生组织(WH O )将罕见病定义为患病人数占总人口0.65%至1%的疾病，常见的有白血病、地中海贫血、血友病、苯丙酮尿症、白化病、法布瑞病、成骨不全症、戈谢病等。“孤儿药”指用于预防、治疗、诊断罕见病的药品，具有研发成本高、药品定价高等特点。如罗氏治疗非霍奇金淋巴瘤的“孤儿药”美罗华年销售额为64亿美元，在全球所有药品市场排名中位列第八。

The world health organization(WH O )Define a rare disease presenting material 0.65% to 1% of the total population,Common with leukemia/The Mediterranean anemia/hemophilia/Phenyl acetone urine disease/albino/Method of bree osbourne disease/According to the disease/Gaucher disease, etc."Orphan drug"Used to prevent/The treatment/Diagnosis of a rare disease,With research and development cost is high/Drug pricing etc.Such as roche in the treatment of non-hodgkin's lymphoma"Orphan drug"Mabthera in annual sales of $6.4 billion,For the top spot in the global pharmaceutical market all ranked eighth.

　　汤森路透报告指出，2001年至2010年间“孤儿药”市场的增长(年复合增长率为25.8%)相对非“孤儿药”市场(年复合增长率为20.1%)呈现显著优势。2011年全球“孤儿药”市场销售额为500亿美元，占全球医疗市场约6%。

Townsend Reuters report,Between 2001 and 2010"Orphan drug"The growth of the market(Compound annual growth rate of 25.8%)Relative to the non -"Orphan drug"The market(Compound annual growth rate of 20.1%)Has significant advantages.In 2011 the global"Orphan drug"Market sales of $50 billion,Accounts for about 6% of global medical market.

　　鉴于全球“孤儿药”市场的广阔前景，跨国制药企业近年来纷纷抢占“孤儿药”市场先机。2009年辉瑞公司与以色列生物技术公司Protalix达成协议开发用来治疗戈谢病的药物，开始涉足“孤儿药”市场。戈谢病为遗传性罕见病，每年治疗费用约为20万美元至25万美元，患者需终身服药。同年，罗氏公司以468亿美元全资收购生物技术公司基因泰克，充分利用其在生物制药领域里的专业知识进军“孤儿药”领域。

In view of the world"Orphan drug"The broad prospects of the market,Multinational pharmaceutical companies in recent years in succession"Orphan drug"Market opportunities.2009, Pfizer Protalix biotechnology companies to reach an agreement with Israel development of drugs for treatment for gaucher disease,Began to set foot in"Orphan drug"The market.Gaucher disease is rare hereditary disease,Treatment costs about $200000 to $200000 a year,Patients need lifelong treatment.In the same year,Roche in biotech companies wholly owned $46.8 billion offer genentech,Make full use of their professional knowledge in the field of biopharmaceutical"Orphan drug"In the field of.

　　2011年作为全球第三大制药公司的赛诺菲以逾201亿美元收购罕见药巨头美国健赞，借此进入“孤儿药”市场。此外，诺华公司也十分关注“孤儿药”市场，旗下重磅产品“孤儿药”格列卫目标人群是慢性髓性白血病患者，年销售额已达50亿美元。

In 2011 as the world's third largest pharmaceutical company sanofi to more than $20.1 billion acquisition of rare drug giant health in the United States,Like to take this opportunity to enter the"Orphan drug"The market.In addition,Novartis is also very attention"Orphan drug"The market,Its blockbuster products"Orphan drug"Gleevec target groups is chronic myelogenous leukemia patients,Has amounted to $5 billion in annual sales.

　　刚刚参加完第十六届北京国际生物医药产业发展论坛的一些业内人士认为，国际医药市场原来所谓的重磅炸弹商业模式———靠一种药能卖几十亿甚至上百亿美元的时代已经一去不复返了，个性化用药将成为全球药业的大趋势，即根据每个病人的具体状况，针对性用药。

Just in the 16 th Beijing international biological medicine industry development BBS some of the personage inside course of study thinks,International medicine market was the so-called "heavy bomb" business model -- by a kind of medicine can sell billions and billions of dollars in time has gone,Personalized medicine will become the trend of the global pharmaceutical industry,That is according to the specific situation of each patient,Targeted drug.

　　美国强生集团创新中心资深总监夏明德认为，孤儿药其实就是个性化用药趋势的一个具体表现形式。孤儿药单个品种市场规模虽然不大，但由于是针对特殊人群，竞争程度低，市场进入门槛相对较高，盈利能力更强，当前全世界都在注重个性化用药的趋势下，孤儿药的研发尤其受到关注。

The Johnson & Johnson group innovation center senior director XiaMingDe think,Orphan drug is personalized medicine a concrete manifestation of the trend.Although orphan drug varieties of single market scale is not big,But because is for special crowd,Low degree of competition,Market entry threshold is relative taller,Profitability is stronger,The current under the pay attention to the trend of personalized medicine all over the world,Orphan drug research and development of especially attention.

　　罕见病致医疗保障压力增大“孤儿药”产业亟待政策支持 Rare disease medical security pressure increasing"Orphan drug"Industrial policy support urgently

　　目前罕见病在我国尚无官方定义，疾病种类及患病人群尚无确切统计数据。按W T O罕见病定义粗略估计，我国罕见病患者至少在1000万以上，常见罕见病包括白血病、血友病、白化病、法布瑞病、戈谢病、成骨不全症、苯丙酮尿症等。

At present rare disease in our country there is no official definition,Disease types and sick people there is no exact statistics.According to W T O rare disease definition to a rough estimate,China's rare disease at least 10 million above,Common rare disease including leukemia/hemophilia/albino/Method of bree osbourne disease/Gaucher disease/According to the disease/Phenyl acetone urine disease, etc.

　　北京生物技术和新医药产业促进中心的相关报告指出，我国至今还没有一种自主研制生产的“孤儿药”上市，仍主要依赖进口，且引进困难，这使得许多罕见病患者得不到有效的诊断和治疗，加剧医疗保障压力，如“瓷娃娃”常被误诊为缺钙、小儿麻痹；国内罕见病患者使用“格列卫”必须通过中华慈善总会接受国际捐赠。

Beijing biotechnology and new medicine industry promotion center of the related report,Our country still does not have a kind of independent development and production"Orphan drug"listed,Is still mainly rely on import,And the difficulty in,This makes many rare disease can not get effective diagnosis and treatment,Rising health care,Such as"Porcelain doll"Is often misdiagnosed as calcium/polio;The domestic rare disease"gleevec"Must be accepted by China charity federation international donor.

　　另外，国内针对孤儿药发展的相关政策扶持滞后，仅于2009年发布了《关于印发新药注册特殊审批管理规定的通知》，对“孤儿药”新药注册申请可实行特殊审批。

In addition,Domestic for orphan drug related policy support for the development of the lag,Just released in 2009[About print and distribute new drug registration special stipulations on the management of the examination and approval notice],to"Orphan drug"New drug application for registration can be a special examination and approval.

　　相比而言，发达国家高度重视“孤儿药”产业发展。美国、欧盟、日本、新加坡等超过30个国家和地区先后颁布了“孤儿药”法案，通常给予权益拥有者市场独占权保护、大量研发资金资助、临床研究和新药报批相对时间较短等特殊国家政策优待。如美国在1983年《罕见病药法案》实施前仅有不足10个“孤儿药”上市，到2008年FD A批准上市的“孤儿药”达到325种；欧盟在2000年的“孤儿药”法规实施前，仅有8种“孤儿药”审核通过，到2009年已有47种“孤儿药”审核通过。

compared,Developed countries attach great importance to"Orphan drug"Industry development.The United States/The European Union/Japan/Singapore successively promulgated more than 30 countries and regions"Orphan drug"bill,Usually give the owner of the rights and market monopoly right protection/A large number of research and development funding/Clinical research and drug approval relative short time special national policies such as preferential treatment.Such as the United States in 1983[Rare disease drugs act]Only less than 10 before implementation"Orphan drug"listed,To the approval of FD A listed in 2008"Orphan drug"Up to 325 kinds of;The European Union in 2000"Orphan drug"Before the implementation of laws and regulations,Only eight"Orphan drug"Through audit,In 2009 for 47"Orphan drug"Through audit.

　　美国强生集团创新中心资深总监夏明德指出，美欧一系列扶持政策的出台，为这些国家产业发展奠定了良好基础，不仅鼓励社会资金进入“孤儿药”的研发环节，而且通过市场手段保障研发投入者有一定时间回收成本，同时价格也给予一定优惠，以此来引导“孤儿药”产业进入良性发展壮大轨道，控制社会医疗保障成本，值得借鉴。

Johnson & Johnson group senior director XiaMingDe pointed out that innovation center in the United States,The us and Europe a series of supporting policies,Laid a good foundation for the national industrial development,Not only encourage social capital to enter"Orphan drug"The development of links,And through market means security research TouRuZhe have certain time cost recovery,At the same time also offer certain preferential price,In order to guide the"Orphan drug"Industry into the benign development track,Control the cost of social medical insurance,Is worth using for reference.

　　生物技术带来发展新机遇国内产业提升需补三大缺失 Biotechnology brings new opportunities domestic industry development promotion to fill three big loss

　　业内人士指出，近年来，借力生物技术发展，国外“孤儿药”的研发效率已经大幅提高。由于80%的罕见病都是遗传病，随着疾病相关生物学研究的深入，很多特异性作用靶点都有可能成为研发“孤儿药”的突破口，生物技术的大量介入，为“孤儿药”研发和市场快速扩张带来机遇，美国生物技术公司健赞因此被誉为“孤儿药之王”。

The personage inside course of study points out that the,In recent years,Borrows the biological technology development,In foreign countries"Orphan drug"The r&d efficiency has been greatly increased.Because 80% of the rare disease is a genetic disease,With the deepening of the research on biology associated with disease,Many specific targets are likely to become the research and development"Orphan drug"The breakthrough,Biological technology of intervention,In order to"Orphan drug"Research and development and market expansion brings opportunities,American biotechnology company health blessing so is known as"The king of the orphan drug".

　　神威药业集团有限公司研究院院长刘军博等业内专家认为，由于“孤儿药”针对的病比较罕见，而我国还没有建立针对孤儿药发展的专门政策体系，因此目前在国内开发大规模孤儿药研发还比较困难，综合来看，产业发展需弥补三大缺失。

Power research institute LiuJunBo pharmaceutical group limited company, etc. Industry experts say,Due to the"Orphan drug"In view of the disease is rare,And our country has not set up according to the special policy of development of orphan drug system,Therefore at present large-scale orphan drug research and development in domestic development are more difficult,Comprehensive view,Industrial development need to compensate for three missing.

　　一是建立“孤儿药”研发公共平台，完善基础数据。相关部门应引导产业尽快明确罕见病和“孤儿药”定义，构建“孤儿药”研发基础平台，借助医改和发展移动医疗的契机，建立统一的罕见病患者电子病例档案，统计国内罕见病患者类别和人数，以便于“孤儿药”研发企业针对性选择项目和为开展临床试验招募患者。

One is to establish"Orphan drug"Research and development of the platform,Perfect basic data.Related department should guide industry rare disease and clearly as soon as possible"Orphan drug"definition,To construct"Orphan drug"Research and development platform,With the reform and development of mobile medical,Establish unity of rare cases of patients with electronic files,Statistics of domestic rare disease categories and number,In order to"Orphan drug"Research and development enterprise specific choice of projects and to recruit patients in clinical trials.

　　二是健全市场机制，调动市场主体积极性凝聚产业发展动力。一些业内人士指出，目前整个国内的药物注册体系、包括医疗体系，都不利于孤儿药的发展，企业即便投入研发了，得到的利润也比较小，市场动力不足。政府应尽快制定我国的《罕见疾病防治法》，制订罕见病管理和罕见病药物管理制度；效仿国外法规授权“孤儿药”拥有者附加的市场垄断权、新药定价、税收优惠政策等，打通市场机制。

Second, improve the market mechanism,Arouse the enthusiasm of market main body condensed industrial development power.Some of the personage inside course of study points out,At present the domestic drug registration system/Including medical system,Go against the development of orphan drug,Enterprise if investment in research and development,The profit is small,Lack of market power.The government should develop as soon as possible in our country[Rare disease prevention and control],Develop rare disease management and rare disease drug management system;Follow the laws and regulations authorized abroad"Orphan drug"Owners added monopolies in the market/New drug pricing/Preferential tax policies, etc,Through the market mechanism.

　　三是大力促进国内企业与国际企业合作创新。创新和合作是全球药业的趋势，没有创新就没有新药，就没有仿制药，目前许多企业出于短期视角考虑，主要做仿制药，发展处于被动。

Three is to promote domestic and international enterprise cooperative innovation.Innovation and cooperation is the trend of the global pharmaceutical industry,Without innovation, there is no new drugs,There is no generic drugs,At present many enterprises for short-term perspective,Mainly do generics,Development in a passive.

　　2012年10月，跨国制药企业赛诺菲与中国医药工业研究总院签署合作备忘录，共同进行我国首个罕见病领域疾病诊断及治疗的联合研究。政府应支持产业内类似的国际合作创新，同时借助政产学研联合支持一批“孤儿药”研发项目，鼓励制药企业投入“孤儿药”研发。(记者　张舵　王晓洁）

In October 2012.,Multinational pharmaceutical companies sanofi pharmaceutical industry research institute signed memorandum of understanding with China,Common in our country the first rare disease diagnosis and treatment in the field of joint research.The government should support similar international cooperation within the industry innovation,At the same time by political union support a batch"Orphan drug"Research and development project,To encourage the pharmaceutical enterprise"Orphan drug"Research and development.(Reporter zhang rudder Braun c a.manufacturing process controls)