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!

    0

  2. 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    2017-05-19 为啥子

    市场定位,导致高价……

    0

  3. 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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=22, 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=28, replyNumber=0, topicName=null, topicId=null, topicList=[], attachment=null, authenticateStatus=null, 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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!

    0

  4. 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    2017-05-18 旺哥

    天价啊!望药兴叹。

    0

  5. 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    2017-05-18 明天会更好!

    太贵了,能有几个用的上啊!

    0

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createdAvatar=https://cdnapi.center.medsci.cn/medsci/head/2017/08/03/49a8b8e59b1110232178c436948997ae.jpg, createdBy=90151681386, createdName=哈夫, createdTime=Wed May 17 23:09:18 CST 2017, time=2017-05-17, status=1, ipAttribution=)]
    2017-05-18 Drhzm308

    这种治疗少见疾病的药物,只能靠政府补贴了

    0

  7. 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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=54, 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=32, 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=22, 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=28, replyNumber=0, topicName=null, topicId=null, topicList=[], attachment=null, authenticateStatus=null, createdAvatar=https://cdnapi.center.medsci.cn/medsci/head/2017/08/03/49a8b8e59b1110232178c436948997ae.jpg, createdBy=90151681386, createdName=哈夫, createdTime=Wed May 17 23:09:18 CST 2017, time=2017-05-17, status=1, ipAttribution=)]
    2017-05-18 七堇

    好贵。

    0

  8. 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    2017-05-18 飛歌

    学习了很有用

    0

  9. 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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=22, 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=28, replyNumber=0, topicName=null, topicId=null, topicList=[], attachment=null, authenticateStatus=null, 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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

    0

  10. 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createdTime=Fri May 19 15:47:16 CST 2017, time=2017-05-19, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=199783, encodeId=eb8f199e83c9, content=医药是某些集团的利润来源!不好!违背了医药的本质!, beContent=null, objectType=article, channel=null, level=null, likeNumber=64, replyNumber=0, topicName=null, topicId=null, topicList=[], attachment=null, authenticateStatus=null, createdAvatar=https://wx.qlogo.cn/mmopen/ajNVdqHZLLCxzIqGgHGpnv0v7a7MZQHVVjxvBfHWenpTPmFhDxa6vn3qno88vft2DRqYcyUBQFCRouANp2WgxQ/0, createdBy=b35a44222, createdName=kane35, createdTime=Fri May 19 05:12:05 CST 2017, time=2017-05-19, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=199726, encodeId=0683199e2655, content=天价啊!望药兴叹。, beContent=null, objectType=article, channel=null, level=null, likeNumber=65, replyNumber=0, topicName=null, topicId=null, topicList=[], attachment=null, authenticateStatus=null, createdAvatar=, createdBy=1ee71931136, createdName=旺哥, createdTime=Thu May 18 23:17:48 CST 2017, 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=54, 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=32, 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=22, 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=28, replyNumber=0, topicName=null, topicId=null, topicList=[], attachment=null, authenticateStatus=null, createdAvatar=https://cdnapi.center.medsci.cn/medsci/head/2017/08/03/49a8b8e59b1110232178c436948997ae.jpg, createdBy=90151681386, createdName=哈夫, createdTime=Wed May 17 23:09:18 CST 2017, time=2017-05-17, status=1, ipAttribution=)]
    2017-05-17 哈夫

    宁肯人死也不造葯?唉

    0