全球昂贵的五种药它们凭什么卖这么贵！

2017-05-17 曹敏健康时报网

近日，全球最贵药物Glybera被其研发商荷兰生物技术公司uniQure叫停，并宣布将撤市。以Glybera为首的全球最贵药，费用每年至少几十万美元，相当于一辆超级跑车的价格。高昂的天价和暗淡的市场令这些药物陷入“有价无市”的尴尬境遇。Glybera世界首个基因药物药价：101万美元/年企业：荷兰的生物科技公司UniQureGlybera是针对治疗脂蛋白脂酶缺乏症的药物，每年的花费高达101万美元

近日，全球最贵药物Glybera被其研发商荷兰生物技术公司uniQure叫停，并宣布将撤市。以Glybera为首的全球最贵药，费用每年至少几十万美元，相当于一辆超级跑车的价格。高昂的天价和暗淡的市场令这些药物陷入“有价无市”的尴尬境遇。

Glybera

世界首个基因药物

药价：101万美元/年

企业：荷兰的生物科技公司UniQure

Glybera是针对治疗脂蛋白脂酶缺乏症的药物，每年的花费高达101万美元。它不仅是价格首贵，也是欧洲首个基因药物，曾在2004年获批欧盟的“孤儿药”。据《麻省理工学院技术评论》，2012年曾有一位病人接受过Glybera的治疗效果十分显着，但至今也仅有这一位。

此次的黯然退市，公司对此给出的理由是，该药的市场需求非常受限，未来几年有财力使用该药的病人也不会明显增加。

Spinraza

肌萎缩孤儿药

药价：75万美元/年

企业：美国的百健与 Ionis合作开发

逐渐恶化的肌无力是脊髓性肌萎缩(SMA)的显着症状，是一种罕见病。Spinraza是全球首个获批治疗SMA药物。

Spinraza的主要研发者之一、苏州大学神经科学研究所教授华益民称：“Spinraza纠正基因的缺陷，不论对于婴幼儿还是成人都有一定的效果。”

Spinraza是全球首个获批治疗脊髓性肌萎缩的药物。百健买断此药，去年12月获批，已于美国上市。第一年花费是高达75万美元，第二年降一半。

Lumizyme

治疗庞培病专项

药价：62.6万美元/年

企业：美国的百健与 Ionis合作开发

庞培病是由身体细胞中被称为糖原的一种糖积聚所引起的遗传疾病。Lumizyme(重组阿葡糖苷酶α)是2014年经美国FDA批准的用于治疗庞培病(Pompe disease，Ⅱ型糖原贮积症)，这种病的患者体内丢失或缺乏一种酶。

此类疾病主要的发病者是婴幼儿，发病率为四万分之一。

Lumizyme由医药巨头赛诺菲研发上市，每年该药的花费超过62.6万美元。

Ravicti和Carbaglu

尿素循环障碍药

药价：60万美元/年

企业：美国的Hyperion Therapeutics公司和意大利Recordati公司

Hyperion公司开发的Ravicti (苯丁酸甘油酯口服液)和Recordati公司开发的Carbaglu(carglumi酸)都是对于尿素循环障碍所开发的药物。这两个药不同的是，Carbaglu针对N-乙酰谷氨酸合成酶缺乏症，且是唯一一个。

在适用年龄上，Ravicti适用于2岁以上，Carbaglu则是各个年龄段。相同的一点就是，这两个药物的定价都较高，每年该药花费不低于60万美元。

专家观点：临床试验是高价主因

不难看出，这些全球最贵药都是用于治疗罕见病和危重疾病的“救命药”。药物方向定位罕见病，也就意味着药物的市场会小于一般的药物，如 Glybera，按发病率计算，整个欧洲的市场只有150到200人。一方面，市场的有限是影响因素，另一方面，极高的价格也是无法忽视的特点。作为药物最直接的研发者，华益民教授告诉记者，药价的确是非常的高。

以华益民教授研发的Spinraza为例，药企对于药品价格的决定力是高于研发方和政府的，而药企又会在临床试验的阶段投入大量的成本，这是导致药品价格高的重要原因；另一方面，药物能否被医保覆盖也是个影响因素，在美国，Spinraza的使用可以按照一定的比率部分报销，患者有更大的可能性使用药物。

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2017-05-20 虈亣靌

好东西大家一起学！

96 0
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2017-05-19 为啥子

市场定位，导致高价……

105 0
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2017-05-19 kane35

医药是某些集团的利润来源！不好！违背了医药的本质！

97 0
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2017-05-18 旺哥

天价啊！望药兴叹。

104 0
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2017-05-18 明天会更好！

太贵了，能有几个用的上啊！

81 0
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2017-05-18 Drhzm308

这种治疗少见疾病的药物，只能靠政府补贴了

63 0
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2017-05-18 七堇

好贵。

50 0
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2017-05-18 飛歌

学习了很有用

50 0
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2017-05-17 有备才能无患

确实贵，得了那些病只能等死

49 0
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time=2017-05-18, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=199561, encodeId=2ba919956183, content=太贵了，能有几个用的上啊！, beContent=null, objectType=article, channel=null, level=null, likeNumber=81, replyNumber=0, topicName=null, topicId=null, topicList=[], attachment=null, authenticateStatus=null, createdAvatar=http://cacheapi.medsci.cn/resource/upload/20160914/IMG57D82E721DB581755.jpg, createdBy=4fc81933929, createdName=明天会更好！, createdTime=Thu May 18 09:23:32 CST 2017, time=2017-05-18, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=199470, encodeId=c39b1994e0e1, content=这种治疗少见疾病的药物，只能靠政府补贴了, beContent=null, objectType=article, channel=null, level=null, likeNumber=63, replyNumber=0, topicName=null, topicId=null, topicList=[], attachment=null, authenticateStatus=null, createdAvatar=null, createdBy=ad4c106013, createdName=Drhzm308, createdTime=Thu May 18 06:59:02 CST 2017, time=2017-05-18, status=1, ipAttribution=), 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createdTime=Thu May 18 03:28:24 CST 2017, time=2017-05-18, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=199379, encodeId=39791993e94a, content=确实贵，得了那些病只能等死, beContent=null, objectType=article, channel=null, level=null, likeNumber=49, replyNumber=0, topicName=null, topicId=null, topicList=[], attachment=null, authenticateStatus=null, createdAvatar=https://wx.qlogo.cn/mmopen/NUyjXTCJjo7cJ6ejWUr2nIia8qOvAKNdLHr32Df6G8iaoaOx6pHaoiacwp26DaslxZHCGbibZhhCGven4rvmWP60GahtzgFjgCjR/0, createdBy=6b111703011, createdName=有备才能无患, createdTime=Wed May 17 23:36:49 CST 2017, time=2017-05-17, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=199372, encodeId=b8591993e290, content=宁肯人死也不造葯?唉, beContent=null, objectType=article, channel=null, level=null, likeNumber=54, replyNumber=0, topicName=null, topicId=null, topicList=[], attachment=null, authenticateStatus=null, 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2017-05-17 哈夫

宁肯人死也不造葯?唉

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