【直报网3月9日讯】（中国经济网）对我国数以万计的罕见病患者而言，“孤儿药”量少价高是他们无法回避的现实。记者近日调查发现，“孤儿药”尴尬处境背后，隐藏着一些有悖于市场导向的现象。

Direct reporting network March 9th news [] (Chinese Economic Net) on China's tens of thousands of rare disease patients, "orphan drugs" low prices that they cannot evasive reality. Reporter recently found, "behind the orphan drugs" embarrassing situation, hiding some contrary to market oriented phenomenon.

市场咨询公司EvaluateGroup发布的2014年“孤儿药”市场报告显示，2020年“孤儿药”有望占到处方药总销售额的19%，在不包括仿制药份额的前提下，年销售额将达到1760亿美元，年复合增长率接近11%，而常见病药物的年增长仅4%。

Market consulting company EvaluateGroup released 2014 "orphan drugs" market report shows, 2020 "orphan drugs" is expected to account for 19% of total sales of prescription drugs, in the premise of not including generics share, annual sales will reach $176000000000, the compound annual growth rate of close to 11%, and the drug of common diseases of annual growth of only 4%.

“孤儿药”已不再是一个缝隙市场，这也成为世界级制药企业的共识，目前辉瑞、罗氏等均已进入该研发领域。

"Orphan drugs" is no longer a niche market, it has also become a world-class pharmaceutical enterprise consensus, Pfizer, Roche is currently have to enter the field of research and development.

然而，对国内药企而言，研发“孤儿药”意味着面临研发、生产、销售风险。

However, the domestic pharmaceutical enterprises, R & D "orphan drugs" means facing R & D, production, sales risk.

跨国药企抢食市场“蛋糕”

Multinational pharmaceutical companies to snatch market "cake"

从美国上世纪80年代推出全世界首个“孤儿药”法案，到如今FDA公开的“孤儿药”目录收纳的数百种药品，不难看出欧美发达国家对这一特殊领域市场蓝海的支持力度。

Launched the first "orphan drug act in the world USA" from 80's of last century, the FDA has publicly "orphan drugs" directory containing hundreds of drugs, it is not difficult to see that the developed countries in Europe and America to support for the special field of market blueocean.

据了解，目前已有不少跨国药企巨头，如辉瑞、罗氏、赛诺菲、拜耳、葛兰素史克等涌向“孤儿药”市场，欲抢食这块万亿市场的大“蛋糕”。

It is understood, at present many multinational pharmaceutical companies such as Pfizer, Roche, giant, Senofi, Bayer, GlaxoSmithKline and other flocked "orphan drugs" market, to snatch the trillion market big "cake".

有观点认为，与常见病相比，罕见病病患数量虽然相对较少，但“孤儿药”价格高昂。2014年，每名罕见病患者在“孤儿药”上的平均花费约13.78万美元，是其他常见病用药平均费用的6倍。

There is the view that, compared with the common disease, rare disease although the number is relatively small, but "orphan drugs" high price. In 2014, per patient in the rare disease "orphan drugs" spend an average of about $137800, is 6 times the average cost of other common medicine.

一位不愿具名的某知名跨国药企人士向记者表示，以血友病为例，患者平均每年需注射100支含有凝血因子8的药物，而单价最低约为每支1000多元，即患者每年需最少支付10多万元的药费，“有时会出现供不应求，甚至断货的情况。”

A well-known multinational pharmaceutical companies who declined to be named, told reporters, in case of drug in patients with haemophilia, average annual injection of 100 containing factor of 8, while the lowest priced about each 1000 yuan a year, namely the patient required to pay about 100000 yuan fee, "sometimes appear in short supply, and even out of stock of the situation."

此外，“专利悬崖”也是跨国药企集体面临的困境，越来越多常见病药物同质化严重、竞争惨烈，“孤儿药”则成为其另辟利润来源的一个不错选择。

In addition, "facing the patent cliff" is also a multinational pharmaceutical companies collective dilemma, more and more medicine common disease, the homogenization of serious competition, "orphan drugs" has become another source of profit for its a good choice.

在政策扶持和市场空间的双轮驱动下，跨国药企的“孤儿药”之路显得较为平坦。FDA推行的“孤儿药”法案中提及，联邦政府对药企在“孤儿药”的研发经费退税最高可达50%，并且还有相关竞争性联邦经费支持。

Two wheel drive in the policy support and market space, multinational pharmaceutical companies "orphan drugs" road is relatively flat. FDA implementation of the "orphan drugs" mentioned in the act, the federal government to pharmaceutical companies in "orphan drugs" R & D spending tax rebate up to 50%, and support the relevant competitive federal funds.

成本、技术成“拦路虎”

Cost, technology into "a lion in the way"

记者在调查中发现，“孤儿药”市场未来具备高成长性在药企眼中已是共识，其中也包括国内药企。

Journalists in the survey found, "orphan drugs" market in the future have high growth potential in the pharmaceutical eyes is a consensus, including domestic pharmaceutical enterprises.

“这几年，随着社会对罕见病的认识不断提高，市场空间确实大了不少，我们不能否认这个事实。”万全万特制药公司研发部人士表示，但这对大多数药企来说，吸引力还远远不够。

"In recent years, with the social awareness of rare diseases continue to improve, the market space is a lot, we can not deny the fact." People in the R & D department Wanquan wante pharmaceutical company said, but that for most enterprises, the attraction is not enough.

市场增长强劲却难以调动企业的积极性，这似乎像一个伪命题。

Strong growth is difficult to mobilize the enthusiasm of enterprises, this seems like a pseudo proposition.

“国内药企研发立项大多会尽量避开‘孤儿药’。”罕见病发展中心主任黄如方表示，除了政策上的空白外，技术壁垒和成本考量往往会令药企失去研发勇气。

"Domestic pharmaceutical enterprises R & D project most of them will try to avoid the 'orphan'." Rare disease development center director Huang Rufang said, in addition to the blank on policy, technical barriers and cost tends to lose courage pharmaceutical R & D.

罕见病发展中心提供的资料显示，根据国际先例，开发和上市一个“孤儿药”的成功率在1/5000左右，需要投入的资金和时间成本都较高。

Provide a rare disease development center data show that, according to the international precedent, developed and launched a "orphan drugs" success rate about 1/5000, it needs capital and time cost is higher.

“对新药研发的不重视不仅存在于‘孤儿药’领域，这是我国生物制药领域的普遍现象。”海南一家制药企业人士表示，绝大多数跨国药企研发投入比例为年销售收入的15%～20%，我国则大多还停留在5%以下的水平。

"For the new drug research and development do not pay attention to not only exists in the 'orphan drug' field, which is a common phenomenon in our country in the field of biological pharmacy." A Hainan pharmaceutical companies said, the vast majority of multinational pharmaceutical companies R & D investment proportion for the annual sales income of 15% ~ 20%, our country most remained below 5% levels.

药企不愿加大研发投入，一个重要原因是，其研发水平尚无法与跨国药企匹敌。

Pharmaceutical companies are reluctant to increase investment in research and development, and an important reason is that the level of R & D, still cannot compete with multinational pharmaceutical companies.

上述跨国药企业务负责人向记者介绍，她所在公司生产的治疗血友病的药物主要成分凝血因子8是通过生物提取，纯度较高，且需要250多天的生产周期。“国产的凝血因子8一般是从人体血液中提取，可能含有病毒因子，纯度有限。”

The multinational pharmaceutical companies business person in charge told the reporter, the main component of coagulation factors in her company production of the drug treatment of hemophilia 8 is through the biological extraction, high purity, and the need for more than 250 days of production cycle. "Blood coagulation factor of domestic 8 is usually extracted from human blood, may contain virus factor, purity co.."

对企业而言，利润永远是其决策的关键考虑。“和常见药相比，‘孤儿药’基数小，药企还要担负新设备购入、人员成本、批量生产后的保质期限红线以及与跨国药企直接竞争的风险，药企很难得到实惠。”上述海南制药企业人士表示。

Speaking of the enterprise, profit always is the key decision making. "Compared with the common medicine, 'orphan' small base, pharmaceutical companies need to undertake the quality guarantee period of the red line new equipment purchasing, personnel cost, mass production and risk in direct competition with multinational pharmaceutical companies, pharmaceutical companies it is difficult to get the benefits." The Hainan pharmaceutical companies said.

诸多业内认为，基于我国生物医药研发的现状，至少在10～20年内，我国的“孤儿药”研发还是会以仿制欧美已过了专利保护期的“孤儿药”为主。▲

Many industry believes that the current situation, bio medicine R & D in China based on, at least 10 to 20 years, China's "orphan drugs" R & D will still in imitation of Europe and the United States have passed the patent protection period "orphan drugs" mainly. *

（原标题：跨国药企抢食“孤儿药” 成本技术成 “拦路虎”）

(original title: multinational pharmaceutical companies snatch "orphan drugs" cost technology into "stumbling block")

责任编辑：小申

Editor: small Shen