Pembrolizumab

编者按： 2024年12月18日，美国默沙东以20亿美元的高价，购买了中国生物制药公司Hansoh的在研GLP-1受体激动剂HS-10535。而就在1年前，英国葛兰素史克就与Hansoh达成过协议，推进ADC产品HS-20089的开发和商业化。 实际上，最近一年，多个中国生物制药公司达成对外转让/开发许可协议， 德国 BioNTech 获得Duality生物两种 ADC 管线资产 DB-1303 和 DB-1311 的开发许可。 英国阿斯利康 与 Eccogene 签订了独家协议，开发并商业化小分子GLP-1受体激动剂ECC5004。 美国百时美施贵宝以84亿美元获得了中国 Biokin 药业针对 HER3/EGFR 的双特异性抗体药物偶联物的开发和商业化权利。 除此之外，中国生物制药公司在美国开设研发中心，在美国做3期临床试验以期直接在美国获批上市的举措也日益兴盛。 经过近20年的发展，中国的生物制药公司的研发终于呈现蒸蒸日上之势；但研发成功并不意味着商业会成功。其出路，到底在哪。。。 下面内容为王宇歌博士的分享，可以给予我们更多思考。 今天和几个朋友聊了一下美国医保的话题；及由此涉及的、近期在国内讨论较多药物定价的问题。 在医药行业的一般都知道一个常识，就是全球大药厂都是靠在美国挣钱获得盈利的，得美国者得天下。 药品不同于一般商品，大部分国家对药物定价都有严格管控；美国则相对没有那么严格的定价管控，美国的医药消费能力也是世界其他国家无法企及的，所以全球一半的医药利润都产生于美国。因为美国盈利高，所以药企也会在美国投入更多的研发，即使欧洲的五大药企，其研发业也主要布局在美国。 美国有全球最好的医药人才库，而药物销售产生的高利润会产生高工资的工作，从而进一步吸引医药研发人员到美国工作。这一方面导致了极高的药物定价，但另一方面也保证了很多最新研发出的“救命”药能够让美国患者最早使用，比如2013年上市的人类第一个病毒病治愈药物 - 丙肝病毒（HCV）治愈药物NS5B抑制剂Sovaldi以及2014年上市的开创免疫治疗先河、可以治愈部分恶性肿瘤的PD-1抑制剂Keytruda。 这两个发现分别获得了2020和2018年诺贝尔生理学及医学奖；而美国是最早可以使用这些诺奖发现转化成最先进药物的国家。许多国内的朋友这几天一直在讨论中国相关药物定价问题。 就像我讲的，医药不是一般商品。中国虽然有最大的患者群体，按人口算，数量约为美国4倍，然而，中国的医药市场仅为美国的1/4（这其中还包括很多传统医药）。虽然很多全球主要制药厂家一直十分重视中国医药市场，然而中国一个困局是这些药厂销售原研药按照定价销售，太过昂贵，没有销路。 作为我微博一个重要内容，我帮助中国患者群体追踪了十几年很多进口药物定价；在新冠疫情后，越来越多普通患者向我询问药物定价，尤其肿瘤靶向治疗药物。如果按照原研药品的定价，很少有中国患者能够承受得起，比如Keytruda虽然显著缓解中国常见的非小细胞肺癌（NSCLC）患者病情，但是刚上市时400mg一次注射需要23138美元，一年则需要196000美元，相当于人民币143万元。如果站在国内普通患者角度考虑，我十分同意药物进入医保，以让更多普通患者能够承受。我这些年见到了很多患者必须用高度靶向性的EGFR突变抑制剂，他们热切期待这些药物进入医保，以能够治疗自己或家人的肺癌，因此，他们对这些药物能否进入医保十分焦虑。但是当这些药物进入医保后，医保会把原研药药价压得非常低，最终让很多产品线以显著低于成本的价格进入医保。 这虽然对患者有很大裨益，但是这些公司难以在国内盈利，而考虑到他们高成本的运营，那么很多公司则会选择淡出中国市场，包括减少产品线、裁员、关闭部门及国内区域总部。 药厂必须盈利才能生存，无法保证盈利而需要全球总部倒贴，那么这些公司迟早退出该市场。药厂除了病人，还要服务自己的stakeholders（员工、股东、董事）等等。这也是最近几年十分频发的现象。 而有意思的是，很多国内创新药公司的新药，也是优先选择在美国上市，而显然这是因为在美国能迅速盈利，以赚回研发投入成本。 识别微信二维码，可添加药时空小编 请注明：姓名+研究方向！

TUESDAY, Dec. 24, 2024 -- When supplies of certain generic, platinum-based cancer chemotherapies dwindled in 2023, oncologists feared it might lead to under-treatment and many more cancer deaths. Fortunately, that did not turn out to be the case, a new study published recently in the Journal of the National Cancer Institute shows. “When we looked at the data on prescribing practices over the shortage period, compared to the previous year, we found that although reporting of the shortages was widespread, it didn't affect as many patients as we had feared," said lead study author Dr. Jacob Reibel . He's a third-year fellow in hematology-oncology at Penn Medicine’s Abramson Cancer Center in Philadelphia. As the research team explained, in the late winter and spring of 2023 the U.S. Food and Drug Administration first warned of shortages of two platinum-bearing chemotherapy drugs, cisplatin and carboplatin . Both have been around for decades and are widely used to treat cancers of the lung , head and neck, breast, bladder, ovaries, uterus and testicles. “At the time, national surveys showed that most cancer centers in the U.S. were reporting shortages of these platinum chemotherapies, but it wasn’t clear how the shortages were actually affecting patients,” Reibel said in a Penn news release. The shortages peaked in June of 2023 and slowly eased thereafter. How badly did these temporary shortages affect cancer patients? To find out, Reibel's team tracked data on nearly 12,000 cancer patients treated in 2023 at centers across the United States. All had advanced, solid-tissue cancers for which platinum-based chemotherapies are often recommended. The study found that the shortages led to only a 2.7% dip in use of the two drugs between February 2023 and January 2024. That means that about 137 fewer patients in the study group got platinum chemotherapy than would have been expected had no shortage happened, Reibel and team estimated. Extrapolated to the nationwide population, they calculated that about 1,000 patients with advanced solid tumors were probably affected overall. At the peak of the shortage, about 15% of patients probably had trouble accessing a platinum chemotherapy drug, the study found. But there was good news, too: Despite the shortages, the study team could find no difference in death rates for patients with solid tumor cancers in 2023 versus the year before. Why might that be so? Probably because oncologists now have more drug treatment options; medications such as immune checkpoint inhibitors, targeted therapies or other forms of chemotherapy, the researchers said. Of course, these newer drugs can be much more expensive that generic cisplatin and carboplatin. “We always want to prioritize the best treatments that we have for patients, and platinum chemotherapies just happen to also be very cost-effective because they’re generic and have been around for decades,” senior study author Dr. Ronac Mamtani noted. Cost can be a factor in prescribing any medication, of course. “While the alternative options may be effective, we want to be able to provide the ‘standard-of-care’ medications to any patient in need," said Mamtani, who is chief of genitourinary cancers at the cancer center. "Even one hundred patients who can’t get the preferred chemotherapy for their cancer type due to supply chain issues is far too many.” The researchers noted that shortages for cisplatin were over by June of 2024, although a shortage of carboplatin remains. However, prescribing levels for both drugs have by now returned to normal. Whatever your topic of interest, subscribe to our newsletters to get the best of Drugs.com in your inbox.