425万美元一针，贵吗？
<p style="font-size: 16px; color: black; line-height: 40px; text-align: left; margin-bottom: 15px;"><span style="color: black;">源自</span>：药智网公众号</p>
<p style="font-size: 16px; color: black; line-height: 40px; text-align: left; margin-bottom: 15px;"><img src="//q9.itc.cn/images01/20240408/fbafed94f7484c16bb363e1466b04a97.jpeg" style="width: 50%; margin-bottom: 20px;"></p>
<p style="font-size: 16px; color: black; line-height: 40px; text-align: left; margin-bottom: 15px;"><span style="color: black;">照片</span><span style="color: black;">源自</span>：摄图网</p>
<p style="font-size: 16px; color: black; line-height: 40px; text-align: left; margin-bottom: 15px;">“五花马，千金裘，呼儿将出换美酒”。李白《将进酒》中的千古名句除了表达诗人豪迈之外，<strong style="color: blue;"><span style="color: black;">亦</span>透露了<span style="color: black;">针对</span>“钟鼓馔玉不足贵”的独特价值观。</strong></p>
<p style="font-size: 16px; color: black; line-height: 40px; text-align: left; margin-bottom: 15px;">这种非普适性价值观放在医药行业，<span style="color: black;">能够</span><span style="color: black;">指点</span>人们<span style="color: black;">针对</span>药价的态度，尤其是<span style="color: black;">药物</span>价格<span style="color: black;">持续</span>蹿升，药价纪录<span style="color: black;">持续</span>被刷新的当下。</p>
<p style="font-size: 16px; color: black; line-height: 40px; text-align: left; margin-bottom: 15px;">今年3月，Orchard Therapeutics为<span style="color: black;">她们</span>刚<span style="color: black;">得到</span>FDA监管<span style="color: black;">准许</span>的早发性异染性脑白质营养不良（MLD）一次性基因疗法Lenmeldy标出了425万美元的价格，一举将<span style="color: black;">全世界</span>最贵<span style="color: black;">药品</span>的价格<span style="color: black;">提高</span>了21%。之前的纪录由CSL Behring和uniQure的B型血友病基因疗法Hemgenix保持，一针350万美元。</p>
<p style="font-size: 16px; color: black; line-height: 40px; text-align: left; margin-bottom: 15px;"><strong style="color: blue;">就在人们对<span style="color: black;">瘦身</span><span style="color: black;">药品</span>的高价格<span style="color: black;">可否</span><span style="color: black;">恰当</span>争论不休之时，药价纪录的刷新却并<span style="color: black;">无</span><span style="color: black;">诱发</span>行业内的轩然大波。</strong>相反地，不少行业协会和非营利组织却认为这个“天价”基本<span style="color: black;">恰当</span>。就连向来“不食制药<span style="color: black;">公司</span>烟火”的临床与经济审查<span style="color: black;">科研</span>所(ICER)都罕见地对这个价格<span style="color: black;">暗示</span>出了“大体支持”的反常态度。要<span style="color: black;">晓得</span>ICER每年都会<span style="color: black;">发布</span>多篇<span style="color: black;">文案</span>，鞭笞制药业不<span style="color: black;">恰当</span>高药价的现象。例如ICER<span style="color: black;">近期</span>就对多发性硬化症<span style="color: black;">药品</span>定价远超<span style="color: black;">恰当</span>性阈值提出了“控诉”。但ICER认为，Lenmeldy的<span style="color: black;">恰当</span>价格在380万美元<span style="color: black;">上下</span>，但425万美元的标价不算太离谱。</p>
<p style="font-size: 16px; color: black; line-height: 40px; text-align: left; margin-bottom: 15px;"><strong style="color: blue;">人们对天价基因疗法的价格宽容，在<span style="color: black;">必定</span>程度上<span style="color: black;">表现</span>了<span style="color: black;">针对</span>彻底根治罕见疑难<span style="color: black;">疾患</span>的<span style="color: black;">剧烈</span><span style="color: black;">巴望</span>。</strong>尤其是在<span style="color: black;">非常多</span>凭借加速疗法上市但有效性存疑的<span style="color: black;">药品</span>的背景下，基因疗法的价值就<span style="color: black;">显出</span>尤其<span style="color: black;">明显</span>。ICER首席医疗官David Rind将Lenmeldy与Zolgensma进行了比较，Zolgensma是一种于2019年<span style="color: black;">准许</span>用于治疗脊髓性肌萎缩症的基因疗法，ICER估计Zolgensma每年<span style="color: black;">导致</span>的<span style="color: black;">病人</span>支出可能要高达210万美元。Rind<span style="color: black;">暗示</span>：“Lenmeldy赋予濒死<span style="color: black;">孩儿</span>生的<span style="color: black;">期盼</span>，它<span style="color: black;">持有</span>极高的价值。而<span style="color: black;">咱们</span>已为<span style="color: black;">哪些</span>几乎毫<span style="color: black;">没</span><span style="color: black;">功效</span>的<span style="color: black;">药品</span>付出了太多的金钱。”</p>
<p style="font-size: 16px; color: black; line-height: 40px; text-align: left; margin-bottom: 15px;"><strong style="color: blue;">尽管ICER认为425万美元的标价还是有些过高，但相<span style="color: black;">针对</span>ICER审评的其它<span style="color: black;">药品</span>价格，Lenmeldy的溢价不算离谱。</strong>尤其是在美国<span style="color: black;">这般</span>一个药价<span style="color: black;">失去控制</span>的国家，基因疗法的高价格<span style="color: black;">最少</span>能换回<span style="color: black;">病人</span><span style="color: black;">必须</span>的治疗需求，满足<span style="color: black;">她们</span><span style="color: black;">针对</span>健康的<span style="color: black;">剧烈</span><span style="color: black;">巴望</span>，从这个<span style="color: black;">方向</span>来看，五花马和千金裘都是值得的付出。</p>
<p style="font-size: 16px; color: black; line-height: 40px; text-align: left; margin-bottom: 15px;">索性的是，<span style="color: black;">针对</span>大<span style="color: black;">都数</span><span style="color: black;">身患</span>早发性异染性脑白质营养不良的<span style="color: black;">病人</span><span style="color: black;">来讲</span>，<span style="color: black;">她们</span>很可能能够<span style="color: black;">得到</span>基因治疗<span style="color: black;">制品</span>，<span style="color: black;">由于</span>私人保险<span style="color: black;">机构</span><span style="color: black;">一般</span>会在与再保险<span style="color: black;">机构</span><span style="color: black;">签定</span>的合同中有所规定，<span style="color: black;">触及</span>到罕见病<span style="color: black;">病人</span>的治疗<span style="color: black;">一般</span>被视为不可预测的“天灾”（act of God），从而享有更大的<span style="color: black;">保证</span>。但州政府的健康保险计划Medicaid可能相对棘手<span style="color: black;">有些</span>，此前镰状细胞基因疗法<span style="color: black;">针对</span>健康保险的压力<span style="color: black;">已然</span>令Medicaid如芒在背。但<span style="color: black;">倘若</span>Medicaid和雇主保险<span style="color: black;">机构</span>能够甄别出<span style="color: black;">哪些</span>“皇帝新衣”<span style="color: black;">药品</span>并将它们剔除在计划之外，<span style="color: black;">她们</span>将会有<span style="color: black;">更加多</span>的空间去覆盖<span style="color: black;">哪些</span>昂贵但有效的疗法。</p>
<p style="font-size: 16px; color: black; line-height: 40px; text-align: left; margin-bottom: 15px;"><strong style="color: blue;">虽然仍然有人要诟病425万美元一针的<span style="color: black;">药品</span>，但<span style="color: black;">没</span>论<span style="color: black;">怎样</span>不可否认的是，它的<span style="color: black;">显现</span><span style="color: black;">最少</span>为人们<span style="color: black;">供给</span>了<span style="color: black;">选取</span></strong>，即使对<span style="color: black;">哪些</span><span style="color: black;">没</span>法支付的人<span style="color: black;">来讲</span>，有了<span style="color: black;">选取</span>可能会更加痛苦，但<span style="color: black;">期盼</span>是<span style="color: black;">没</span>价的。<span style="color: black;">此刻</span><span style="color: black;">能够</span>讨论的，是<span style="color: black;">怎样</span>在维持制药商<span style="color: black;">研发</span>意愿的<span style="color: black;">状况</span>下<span style="color: black;">掌控</span>成本。</p>
<p style="font-size: 16px; color: black; line-height: 40px; text-align: left; margin-bottom: 15px;">像Lenmeldy<span style="color: black;">这般</span>的<span style="color: black;">药品</span>，<span style="color: black;">必须</span>从<span style="color: black;">病人</span>身上取<span style="color: black;">流血</span>细胞，运送到某个<span style="color: black;">设备</span>，用病毒进行修饰，扩增，<span style="color: black;">而后</span>返回。制造每剂<span style="color: black;">药品</span>的成本可能高达50万美元。<span style="color: black;">按照</span>Kliegman<span style="color: black;">科研</span>所创建的<span style="color: black;">关联</span>模型，在最大程度降低成本的<span style="color: black;">状况</span>下，每年为200名<span style="color: black;">病人</span>进行基因治疗，<span style="color: black;">研发</span>商<span style="color: black;">必要</span>收费150万美元一剂<span style="color: black;">才可</span><span style="color: black;">得到</span><span style="color: black;">有些</span>微薄的利润。但<span style="color: black;">倘若</span><span style="color: black;">病人</span>人数<span style="color: black;">仅有</span>几十人时，<span style="color: black;">研发</span>商除了从单剂价格上做<span style="color: black;">文案</span>之外别<span style="color: black;">没</span>他法。这<span style="color: black;">便是</span><span style="color: black;">为何</span>Orchard Therapeutics放弃了四种针对超罕见病治疗<span style="color: black;">办法</span>的部分<span style="color: black;">原由</span>。尽管<span style="color: black;">科研</span>得到了强有力的数据，但<span style="color: black;">商场</span>模式上能否取得成功却面临着<span style="color: black;">极重</span>的<span style="color: black;">危害</span>。这些疗法去年被Kyowa Kirin收购，但只针对更<span style="color: black;">平常</span>的<span style="color: black;">疾患</span>，<span style="color: black;">例如</span>克罗恩病的遗传子集。</p>
<p style="font-size: 16px; color: black; line-height: 40px; text-align: left; margin-bottom: 15px;"><strong style="color: blue;">人间万事，毫发常重泰山轻。</strong>但<span style="color: black;">没</span>论价值观怎么扭曲，在健康面前一切都是其次。<span style="color: black;">倘若</span>真的能挽救人间疾苦，再贵的<span style="color: black;">药品</span>都是值得的。但人们经历了太多相反的例子。与其讨论针对罕见病的有效基因疗法的“天价”<span style="color: black;">可否</span><span style="color: black;">恰当</span>，不如探索一条可<span style="color: black;">连续</span>性的<span style="color: black;">研发</span>罕见病疗法的路线，并<span style="color: black;">奋斗</span>实现<span style="color: black;">研发</span>商、保险计划和<span style="color: black;">病人</span>的共赢局面。</p>
<p style="font-size: 16px; color: black; line-height: 40px; text-align: left; margin-bottom: 15px;"><strong style="color: blue;">管控高价<span style="color: black;">药品</span>，<span style="color: black;">已然</span><span style="color: black;">作为</span>了美国联邦政府和州政府的当务之急。</strong>《降低通货膨胀法案》<span style="color: black;">便是</span>从立法的<span style="color: black;">方向</span>对高药价的宣战，但遭到了制药<span style="color: black;">机构</span>和行业协会的<span style="color: black;">剧烈</span>抵制。</p>
<p style="font-size: 16px; color: black; line-height: 40px; text-align: left; margin-bottom: 15px;">IRA授予Medicare同选定的十款<span style="color: black;">药品</span>进行价格谈判，这十款<span style="color: black;">药品</span>都对Medicare和Mediaid造<span style="color: black;">成为了</span>巨大的财政压力（但并不都属于天价<span style="color: black;">药品</span>行列）。进入“价格谈判”的<span style="color: black;">药品</span>数量还有可能<span style="color: black;">逐步</span><span style="color: black;">增多</span>，这<span style="color: black;">暗示</span>了美国政府利用法律杠杆对<span style="color: black;">失去控制</span>的药价进行管控的决心。</p>
<p style="font-size: 16px; color: black; line-height: 40px; text-align: left; margin-bottom: 15px;">行业关注的<span style="color: black;">另一</span>切入点<span style="color: black;">便是</span>美国<span style="color: black;">药品</span>流通系统中独特的中间人（例如<span style="color: black;">药物</span>福利管理者PBM），以及非常不透明的折扣回扣制度。</p>
<p style="font-size: 16px; color: black; line-height: 40px; text-align: left; margin-bottom: 15px;"><strong style="color: blue;">要从管控中间人的<span style="color: black;">方向</span><span style="color: black;">掌控</span>美国的高药价，<span style="color: black;">能够</span>从下几个<span style="color: black;">方向</span><span style="color: black;">思虑</span>：</strong></p>
<p style="font-size: 16px; color: black; line-height: 40px; text-align: left; margin-bottom: 15px;"><strong style="color: blue;">监管和立法</strong>：政府<span style="color: black;">能够</span><span style="color: black;">经过</span>监管和立法手段来规范<span style="color: black;">药物</span>市场，例如实施价格<span style="color: black;">管理</span>、限制<span style="color: black;">药物</span>利润、强制<span style="color: black;">需求</span>透明度，以及加强对医药<span style="color: black;">机构</span>和<span style="color: black;">药物</span>分销商的监管。这<span style="color: black;">能够</span><span style="color: black;">经过</span>国家立法、执法<span style="color: black;">公司</span>以及<span style="color: black;">关联</span>政策<span style="color: black;">公司</span>的合作来实现。</p>
<p style="font-size: 16px; color: black; line-height: 40px; text-align: left; margin-bottom: 15px;"><strong style="color: blue;">促进竞争</strong>：鼓励<span style="color: black;">更加多</span>的<span style="color: black;">药物</span><span style="color: black;">机构</span>进入市场，<span style="color: black;">增多</span>竞争<span style="color: black;">能够</span>降低<span style="color: black;">药物</span>价格。政府<span style="color: black;">能够</span><span style="color: black;">经过</span>简化审批程序、减少专利<span style="color: black;">守护</span>的<span style="color: black;">乱用</span>以及<span style="color: black;">供给</span>激励<span style="color: black;">办法</span>来促进创新和竞争。</p>
<p style="font-size: 16px; color: black; line-height: 40px; text-align: left; margin-bottom: 15px;"><strong style="color: blue;">减少中间人的利润</strong>：中间人<span style="color: black;">包含</span>制药<span style="color: black;">机构</span>、批发商、零售商以及医疗保险<span style="color: black;">机构</span>等。政府<span style="color: black;">能够</span><span style="color: black;">经过</span>监管<span style="color: black;">办法</span>来限制这些中间人的利润，例如限制<span style="color: black;">药物</span>价格上涨、<span style="color: black;">调节</span><span style="color: black;">医疗保险</span>支付政策、削减中间人的<span style="color: black;">补助</span>和津贴等。</p>
<p style="font-size: 16px; color: black; line-height: 40px; text-align: left; margin-bottom: 15px;"><strong style="color: blue;">改革医疗保险</strong>：改革医疗保险制度，<span style="color: black;">增多</span>对<span style="color: black;">药物</span>价格的谈判能力和影响力。这可能<span style="color: black;">包含</span><span style="color: black;">增多</span>公共医疗保险计划的覆盖范围、<span style="color: black;">加强</span>对<span style="color: black;">药物</span>价格的谈判能力、降低<span style="color: black;">病人</span>的<span style="color: black;">药物</span><span style="color: black;">花费</span>等。</p>
<p style="font-size: 16px; color: black; line-height: 40px; text-align: left; margin-bottom: 15px;"><strong style="color: blue;">鼓励<span style="color: black;">运用</span><span style="color: black;">广泛</span>的<span style="color: black;">药品</span></strong>：政府<span style="color: black;">能够</span>鼓励医疗保健<span style="color: black;">供给</span>者和<span style="color: black;">病人</span><span style="color: black;">运用</span>更为<span style="color: black;">广泛</span>的<span style="color: black;">药品</span>或替代品，这些<span style="color: black;">药品</span>可能价格更低、<span style="color: black;">或</span>在效果上更为接近，并且不会<span style="color: black;">由于</span>专利<span style="color: black;">守护</span>而<span style="color: black;">引起</span>价格居高不下。</p>
<p style="font-size: 16px; color: black; line-height: 40px; text-align: left; margin-bottom: 15px;"><strong style="color: blue;"><span style="color: black;">加强</span>透明度</strong>：政府<span style="color: black;">能够</span><span style="color: black;">需求</span>制药<span style="color: black;">机构</span>和医疗保险<span style="color: black;">机构</span><span style="color: black;">供给</span><span style="color: black;">更加多</span>的透明度，<span style="color: black;">包含</span><span style="color: black;">药物</span>定价和成本的公开信息，以便监管<span style="color: black;">公司</span>和公众更好地监督和<span style="color: black;">掌控</span><span style="color: black;">药物</span>价格。</p>
<p style="font-size: 16px; color: black; line-height: 40px; text-align: left; margin-bottom: 15px;">以上这些<span style="color: black;">办法</span>可能<span style="color: black;">必须</span>政府、医疗保健行业和公众<span style="color: black;">一起</span><span style="color: black;">奋斗</span>，<span style="color: black;">才可</span>有效地管控美国的高药价。</p>
<p style="font-size: 16px; color: black; line-height: 40px; text-align: left; margin-bottom: 15px;">Ref.</p>
<p style="font-size: 16px; color: black; line-height: 40px; text-align: left; margin-bottom: 15px;">1.Mast,J.Why the world’s most expensive drug might not be all that overpriced.STAT.28.03.2024.</p>
<p style="font-size: 16px; color: black; line-height: 40px; text-align: left; margin-bottom: 15px;">2.ICER Publishes Final Evidence Report on Gene Therapy for Metachromatic Leukodystrophy.ICER.30.10.2023.</p>
<p style="font-size: 16px; color: black; line-height: 40px; text-align: left; margin-bottom: 15px;">3.Mast,J.et al.New gene therapy,to be priced at$4.25 million,has already transformed children’s lives.20.03.2024.</p>
<p style="font-size: 16px; color: black; line-height: 40px; text-align: left; margin-bottom: 15px;">4.ICER Comments on the FDA Approval of Zolgensma for the Treatment of Spinal Muscular Atrophy.ICER.24.05.2017.</p>
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