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2021-06-23

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FDA Approved Biogen Alzheimer’s Drug Despite Some Staff Concerns<blockquote>尽管有一些工作人员的担忧，FDA 还是批准了百健阿尔茨海默病药物</blockquote>

The U.S. Food and Drug Administrationapproved the first new Alzheimer’s drug in decadesover the obje

FDA Approved Biogen Alzheimer’s Drug Despite Some Staff Concerns<blockquote>尽管有一些工作人员的担忧，FDA 还是批准了百健阿尔茨海默病药物</blockquote>

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{"i18n":{"language":"zh_CN"},"data":{"magic":2,"id":123592828,"tweetId":"123592828","gmtCreate":1624428233064,"gmtModify":1634006240151,"author":{"id":3584774162933077,"idStr":"3584774162933077","authorId":3584774162933077,"authorIdStr":"3584774162933077","name":"Babaluke","avatar":"https://static.tigerbbs.com/cf1bdc263bcb63f2412eb36286eaedda","vip":1,"userType":1,"introduction":"","boolIsFan":false,"boolIsHead":false,"crmLevel":1,"crmLevelSwitch":0,"individualDisplayBadges":[],"wearingBadges":[],"fanSize":0,"starInvestorFlag":false},"themes":[],"images":[],"coverImages":[],"title":"","extraTitle":"","html":"<html><head></head><body>Good</body></html>","htmlText":"<html><head></head><body>Good</body></html>","text":"Good","highlighted":1,"essential":1,"paper":1,"likeSize":0,"commentSize":0,"repostSize":0,"favoriteSize":0,"link":"https://laohu8.com/post/123592828","repostId":1174011543,"repostType":4,"repost":{"id":"1174011543","kind":"news","pubTimestamp":1624425845,"share":"https://www.laohu8.com/m/news/1174011543?lang=zh_CN&edition=full","pubTime":"2021-06-23 13:24","market":"us","language":"en","title":"FDA Approved Biogen Alzheimer’s Drug Despite Some Staff Concerns<blockquote>尽管有一些工作人员的担忧，FDA 还是批准了百健阿尔茨海默病药物</blockquote>","url":"https://stock-news.laohu8.com/highlight/detail?id=1174011543","media":"WSJ","summary":"The U.S. Food and Drug Administrationapproved the first new Alzheimer’s drug in decadesover the obje","content":"The U.S. Food and Drug Administrationapproved the first new Alzheimer’s drug in decadesover the objection of agency statisticians who said there was insufficient evidence to support approval, according to newly released internal memos.<blockquote>美国根据新公布的内部备忘录，美国食品和药物管理局批准了几十年来首个阿尔茨海默氏症新药，但该机构的统计人员表示反对，称没有足够的证据支持批准。</blockquote> In the internal memos released Tuesday, FDA officials discussed whether to approve the drug fromBiogenInc.BIIB-1.71%over objections from the agency’s drug statistics office, which said that clinical trial data fell short of the proof typically required to put a new product on the market.<blockquote>在周二发布的内部备忘录中，FDA 官员讨论了是否批准 fromBiogenInc.BIIB-1.71% 的药物，因为该机构药物统计办公室表示，临床试验数据达不到将新产品推向市场通常所需的证据。</blockquote> Ultimately, top officials decided that the evidence indicating that the drug worked, while inconclusive, was strong enough to allow doctors and patients to decide for themselves whether to take it, according to the memos.<blockquote>根据备忘录，最终，高级官员决定，表明该药物有效的证据虽然不确定，但足以让医生和患者自行决定是否服用该药物。</blockquote> “FDA, then, is faced with a situation where the available evidence on the clinical endpoints is short of what we would require for standard approval of aducanumab,” wrote Peter Stein, director of the FDA’s office of new drugs, in a memo detailing his support for approval. “At the same time, patients with [Alzheimer’s] have a serious, progressive, ultimately fatal disease and are desperate for treatments.”<blockquote>“美国食品和药物管理局新药办公室主任彼得-斯坦（Peter Stein）在一份备忘录中详细说明了他对批准 aducanumab 的支持，他写道：”美国食品和药物管理局新药办公室主任彼得-斯坦（Peter Stein）在一份备忘录中写道。“与此同时，[阿尔茨海默氏症]患者患有严重的、进行性的、最终致命的疾病，他们迫切需要治疗”。</blockquote> The FDA made public its internal deliberations as it faces criticism from some doctors and scientists over the June 7 approval of the drug, called Aduhelm.<blockquote>FDA 公开了其内部审议，因为它面临一些医生和科学家对 6 月 7 日批准名为 Aduhelm 的药物的批评。</blockquote> The critics say the FDA ignored the scientific standards it typically holds drugmakers to and could prompt the federal government to spend billions of dollars on a drug that may not work.<blockquote>批评者称，FDA忽视了制药商通常遵守的科学标准，并可能促使联邦政府在一种可能无效的药物上花费数十亿美元。</blockquote> “While I fully support the Center’s recent decision to approve the therapy, I acknowledge that there are different opinions about this decision, including within FDA, and there likely will continue to be questions around our process for review and the data we considered to support the approval,” said FDA Acting Commissioner Janet Woodcock. “In the end, an experienced group of medical reviewers at FDA concluded that there was sufficient evidence to approve the Alzheimer’s therapy via the accelerated approval pathway.”<blockquote>“美国食品和药物管理局代理局长珍妮特-伍德科克（Janet Woodcock）说：”虽然我完全支持该中心最近批准该疗法的决定，但我承认，包括美国食品和药物管理局内部在内，对这一决定有不同的意见，围绕我们的审查程序和我们为支持批准而考虑的数据可能会继续存在疑问。“最终，美国食品和药物管理局一组经验丰富的医学审查员得出结论，有足够的证据通过加速审批途径批准阿尔茨海默氏症疗法。”</blockquote> Supporters say the FDA decision provides a lifeline to patients who have lacked any medicine promising to slow the progression of Alzheimer’s, and that the decision couldspur the development of more, better medicines.<blockquote>支持者说，美国食品和药物管理局的决定为那些缺乏任何有望减缓阿尔茨海默氏症进展的药物的患者提供了一条生命线，这一决定可能会刺激更多更好的药物的开发。</blockquote> The agency usually releases approval documents after a decision, though not typically as quickly as it did this time. The 83 pages of memos appear to be a portion of the FDA’s deliberations, largely reflecting the exchanges of top officials.<blockquote>该机构通常会在做出决定后发布批准文件，尽管通常不会像这次那么快。这 83 页的备忘录似乎是 FDA 审议的一部分，主要反映了高层官员的交流。</blockquote> Tristan Massie, a biostatistics reviewer for the FDA, “does not agree that the totality of the data provides sufficient evidence to support the efficacy of aducanumab in Alzheimer’s disease and does not recommend approval,” one of the internal documents shows.<blockquote>一份内部文件显示，美国食品和药物管理局（FDA）的生物统计学审查员特里斯坦-马西（Tristan Massie）“不同意所有数据提供了足够的证据来支持aducanumab治疗阿尔茨海默病的疗效，因此不建议批准”。</blockquote> Patrizia Cavazzoni, the FDA’s top-ranking drug-evaluation official, wrote in another memo that the agency’s drug biostatistics office “provided documentation for its recommendation that substantial evidence of effectiveness had not been provided in the application.”<blockquote>美国食品和药物管理局（FDA）最高级别的药物评估官员帕特里齐亚-卡瓦佐尼（Patrizia Cavazzoni）在另一份备忘录中写道，该机构的药物生物统计办公室 “为其建议提供了文件，即申请中没有提供实质性的有效性证据”。</blockquote> That office’s full dissenting analysis wasn’t included in the materials made available Tuesday, but portions of it were.<blockquote>该办公室的完整异议分析并未包含在周二提供的材料中，但部分内容包含在内。</blockquote> Despite the objections, Dr. Cavazzoni wrote that Biogen had met the requirements for an “accelerated approval,” a type of regulatory clearance that allows a drug to be marketed even when there remains uncertainty about its clinical benefit if it fills an unmet need for a serious disease and has effects against a biological sign of the disease.<blockquote>尽管有人反对，卡瓦佐尼博士还是写道，百健公司已经满足了 “加速批准 ”的要求，这是一种监管许可，允许一种药物在临床益处仍存在不确定性的情况下上市，只要该药物满足了严重疾病未得到满足的需求，并且对疾病的生物体征产生了影响。</blockquote> Dr. Cavazzoni wrote that Aduhelm met that standard because of its effect of reducing a sticky substance called amyloid which “is reasonably likely to predict clinical benefit.”<blockquote>Cavazzoni 博士写道，Aduhelm 之所以符合这一标准，是因为它能减少一种叫做淀粉样蛋白的粘性物质，而淀粉样蛋白 “很有可能预测临床益处”。</blockquote> Some doctors and scientists saytargeting the amyloid plaqueshasn’t been proven to slow Alzheimer’s. Some members of a panel of advisers that recommended the FDA reject the drug haveresigned from the committee, citing the agency’s approval.<blockquote>一些医生和科学家说，针对淀粉样斑块尚未被证明可以减缓阿尔茨海默氏症的进展。建议美国食品和药物管理局拒绝该药物的顾问小组的一些成员以该机构的批准为由辞去了委员会职务。</blockquote> Biogen listed the price of Aduhelm at $56,000 a year, and patients and Medicare could also face testing charges.<blockquote>百健将 Aduhelm 的价格列为每年 56,000 美元，患者和医疗保险也可能面临检测费用。</blockquote> About six million Americans suffer from Alzheimer’s, a progressive condition that robs people of their memories and cognitive abilities. Biogen has said that as many as two million of them may benefit from the medicine.<blockquote>约有 600 万美国人患有阿尔茨海默氏症，这是一种会剥夺人们记忆和认知能力的进行性疾病。百健表示，其中多达200万人可能会从这种药物中受益。</blockquote> The FDA approval sent Biogen’s stock soaring. The company has said it expects only minimal revenue from the drug this year, in part because of the logistical challenges in administering the medicine, but that sales would begin to increase next year.<blockquote>美国食品和药物管理局的批准使百健的股价飙升。该公司表示，预计今年该药物的收入将微乎其微，部分原因是管理该药物的后勤挑战，但销售额将在明年开始增长。</blockquote> Analysts estimate sales of $946.3 million in 2022, according to FactSet. Sales are seen growing to $5.2 billion in 2026.<blockquote>根据 FactSet 的数据，分析师预计 2022 年销售额为 9.463 亿美元。预计 2026 年销售额将增长至 52 亿美元。</blockquote> The memos show FDA officials attempting to balance the subpar study data for Aduhelm with the desire of Alzheimer’s patients and their families to try any new treatment that might help.<blockquote>备忘录显示，美国食品和药物管理局官员正试图在 Aduhelm 的研究数据与阿尔茨海默氏症患者及其家人尝试任何可能有帮助的新疗法的愿望之间取得平衡。</blockquote> “There remains residual uncertainty regarding clinical benefit,” Dr. Stein wrote. But if the FDA were to wait for the company to finish another clinical study, “any approval would occur after a multiple year delay” and patients “could suffer irreversible loss of brain neurons and cognitive function and memory,” he wrote.<blockquote>“斯坦因博士写道：”临床获益仍存在不确定性。但他写道，如果美国食品和药物管理局等待该公司完成另一项临床研究，“任何批准都将在多年的延迟之后进行”，患者 “可能会遭受不可逆转的大脑神经元、认知功能和记忆力丧失”。</blockquote> The FDA said it plans to release more documents regarding its decision making as early as next week.<blockquote>FDA表示，计划最早下周发布更多有关其决策的文件。</blockquote> The FDA’s approval was based primarily on two large Phase 3 clinical trials launched by Biogen to prove its drug helped slow the cognitive decline of people with early-stage Alzheimer’s.<blockquote>美国食品和药物管理局的批准主要基于百健公司启动的两项大型 3 期临床试验，以证明其药物有助于减缓早期阿尔茨海默氏症患者的认知能力下降。</blockquote> In March 2019, the companyhalted the studies earlyafter an independent monitoring board concluded the drug was unlikely to help patients.<blockquote>2019 年 3 月，在独立监测委员会得出结论该药物不太可能帮助患者后，该公司提前停止了研究。</blockquote> Seven months later, however,the company made a surprising reversaland said it was now planning to seek FDA approval after conducting additional analyses of trial data that came in after the studies were halted. The company said its analyses found the drug was successful in one study but failed in the second.<blockquote>然而，七个月后，该公司做出了令人惊讶的逆转，并表示在对研究停止后获得的试验数据进行额外分析后，现在正计划寻求 FDA 的批准。该公司表示，其分析发现该药物在一项研究中取得了成功，但在第二项研究中失败。</blockquote> The FDA memos provide some new details on Biogen’s about-face. In June 2019, the company met with FDA officials and both sides agreed the studies shouldn’t have been stopped, according to a “summary memorandum” document.<blockquote>美国食品和药物管理局的备忘录提供了一些关于百健转变的新细节。根据一份 “摘要备忘录 ”文件，2019 年 6 月，该公司会见了 FDA 官员，双方一致认为不应停止研究。</blockquote> At the meeting, FDA officials recommended that Biogen analyze the studies further to understand if it was still possible to draw conclusions from the data, despite the halt of the trials and conflicting results.<blockquote>在会议上，FDA 官员建议百健进一步分析这些研究，以了解尽管试验停止且结果相互矛盾，但是否仍然有可能从数据中得出结论。</blockquote> Several months later, in October 2019, the agency and company met again and agreed that the studies could still be used to evaluate the drug’s effectiveness, according to the memo.<blockquote>根据备忘录，几个月后，即 2019 年 10 月，该机构和公司再次会面，并一致认为这些研究仍可用于评估药物的有效性。</blockquote> The following day, on Oct. 22, Biogen announcedit would seek approval for the medicine, sending up its share price and adding billions of dollars to its market value.<blockquote>第二天，即 10 月 22 日，百健宣布将寻求该药物的批准，从而推高其股价，并为其市值增加数十亿美元。</blockquote> The company had been quietly re-examining the data for months, but didn’t have the confidence to move forward until the latest meeting with FDA officials, Biogen Chief Executive Michel Vounatsos said in an interview at the time.<blockquote>百健公司首席执行官米歇尔-沃纳索斯（Michel Vounatsos）当时在接受采访时说，几个月来，公司一直在悄悄地重新审查这些数据，但直到最近一次与美国食品及药物管理局官员会面，公司才有信心继续前进。</blockquote>","collect":0,"html":"<!DOCTYPE html>\n<html>\n<head>\n<meta http-equiv=\"Content-Type\" content=\"text/html; 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color:#2a4b87;}\n.meta .head { display: inline-block; overflow: hidden}\n.head .h-thumb { width: 30px; height: 30px; margin: 0; padding: 0; border-radius: 50%; float: left;}\n.head .h-content { margin: 0; padding: 0 0 0 9px; float: left;}\n.head .h-name {font-size: 13px; color: #eee; margin: 0;}\n.head .h-time {font-size: 12.5px; color: #7E829C; margin: 0;}\n.small {font-size: 12.5px; display: inline-block; transform: scale(0.9); -webkit-transform: scale(0.9); transform-origin: left; -webkit-transform-origin: left;}\n.smaller {font-size: 12.5px; display: inline-block; transform: scale(0.8); -webkit-transform: scale(0.8); transform-origin: left; -webkit-transform-origin: left;}\n.bt-text {font-size: 12px;margin: 1.5em 0 0 0}\n.bt-text p {margin: 0}\n</style>\n</head>\n<body>\n<div class=\"wrapper\">\n<header>\n<h2 class=\"title\">\nFDA Approved Biogen Alzheimer’s Drug Despite Some Staff Concerns<blockquote>尽管有一些工作人员的担忧，FDA 还是批准了百健阿尔茨海默病药物</blockquote>\n</h2>\n<h4 class=\"meta\">\n\nWSJ2021-06-23 13:24\n\n</h4>\n</header>\n<article>\nThe U.S. Food and Drug Administrationapproved the first new Alzheimer’s drug in decadesover the objection of agency statisticians who said there was insufficient evidence to support approval, according to newly released internal memos.<blockquote>美国根据新公布的内部备忘录，美国食品和药物管理局批准了几十年来首个阿尔茨海默氏症新药，但该机构的统计人员表示反对，称没有足够的证据支持批准。</blockquote> In the internal memos released Tuesday, FDA officials discussed whether to approve the drug fromBiogenInc.BIIB-1.71%over objections from the agency’s drug statistics office, which said that clinical trial data fell short of the proof typically required to put a new product on the market.<blockquote>在周二发布的内部备忘录中，FDA 官员讨论了是否批准 fromBiogenInc.BIIB-1.71% 的药物，因为该机构药物统计办公室表示，临床试验数据达不到将新产品推向市场通常所需的证据。</blockquote> Ultimately, top officials decided that the evidence indicating that the drug worked, while inconclusive, was strong enough to allow doctors and patients to decide for themselves whether to take it, according to the memos.<blockquote>根据备忘录，最终，高级官员决定，表明该药物有效的证据虽然不确定，但足以让医生和患者自行决定是否服用该药物。</blockquote> “FDA, then, is faced with a situation where the available evidence on the clinical endpoints is short of what we would require for standard approval of aducanumab,” wrote Peter Stein, director of the FDA’s office of new drugs, in a memo detailing his support for approval. “At the same time, patients with [Alzheimer’s] have a serious, progressive, ultimately fatal disease and are desperate for treatments.”<blockquote>“美国食品和药物管理局新药办公室主任彼得-斯坦（Peter Stein）在一份备忘录中详细说明了他对批准 aducanumab 的支持，他写道：”美国食品和药物管理局新药办公室主任彼得-斯坦（Peter Stein）在一份备忘录中写道。“与此同时，[阿尔茨海默氏症]患者患有严重的、进行性的、最终致命的疾病，他们迫切需要治疗”。</blockquote> The FDA made public its internal deliberations as it faces criticism from some doctors and scientists over the June 7 approval of the drug, called Aduhelm.<blockquote>FDA 公开了其内部审议，因为它面临一些医生和科学家对 6 月 7 日批准名为 Aduhelm 的药物的批评。</blockquote> The critics say the FDA ignored the scientific standards it typically holds drugmakers to and could prompt the federal government to spend billions of dollars on a drug that may not work.<blockquote>批评者称，FDA忽视了制药商通常遵守的科学标准，并可能促使联邦政府在一种可能无效的药物上花费数十亿美元。</blockquote> “While I fully support the Center’s recent decision to approve the therapy, I acknowledge that there are different opinions about this decision, including within FDA, and there likely will continue to be questions around our process for review and the data we considered to support the approval,” said FDA Acting Commissioner Janet Woodcock. “In the end, an experienced group of medical reviewers at FDA concluded that there was sufficient evidence to approve the Alzheimer’s therapy via the accelerated approval pathway.”<blockquote>“美国食品和药物管理局代理局长珍妮特-伍德科克（Janet Woodcock）说：”虽然我完全支持该中心最近批准该疗法的决定，但我承认，包括美国食品和药物管理局内部在内，对这一决定有不同的意见，围绕我们的审查程序和我们为支持批准而考虑的数据可能会继续存在疑问。“最终，美国食品和药物管理局一组经验丰富的医学审查员得出结论，有足够的证据通过加速审批途径批准阿尔茨海默氏症疗法。”</blockquote> Supporters say the FDA decision provides a lifeline to patients who have lacked any medicine promising to slow the progression of Alzheimer’s, and that the decision couldspur the development of more, better medicines.<blockquote>支持者说，美国食品和药物管理局的决定为那些缺乏任何有望减缓阿尔茨海默氏症进展的药物的患者提供了一条生命线，这一决定可能会刺激更多更好的药物的开发。</blockquote> The agency usually releases approval documents after a decision, though not typically as quickly as it did this time. The 83 pages of memos appear to be a portion of the FDA’s deliberations, largely reflecting the exchanges of top officials.<blockquote>该机构通常会在做出决定后发布批准文件，尽管通常不会像这次那么快。这 83 页的备忘录似乎是 FDA 审议的一部分，主要反映了高层官员的交流。</blockquote> Tristan Massie, a biostatistics reviewer for the FDA, “does not agree that the totality of the data provides sufficient evidence to support the efficacy of aducanumab in Alzheimer’s disease and does not recommend approval,” one of the internal documents shows.<blockquote>一份内部文件显示，美国食品和药物管理局（FDA）的生物统计学审查员特里斯坦-马西（Tristan Massie）“不同意所有数据提供了足够的证据来支持aducanumab治疗阿尔茨海默病的疗效，因此不建议批准”。</blockquote> Patrizia Cavazzoni, the FDA’s top-ranking drug-evaluation official, wrote in another memo that the agency’s drug biostatistics office “provided documentation for its recommendation that substantial evidence of effectiveness had not been provided in the application.”<blockquote>美国食品和药物管理局（FDA）最高级别的药物评估官员帕特里齐亚-卡瓦佐尼（Patrizia Cavazzoni）在另一份备忘录中写道，该机构的药物生物统计办公室 “为其建议提供了文件，即申请中没有提供实质性的有效性证据”。</blockquote> That office’s full dissenting analysis wasn’t included in the materials made available Tuesday, but portions of it were.<blockquote>该办公室的完整异议分析并未包含在周二提供的材料中，但部分内容包含在内。</blockquote> Despite the objections, Dr. Cavazzoni wrote that Biogen had met the requirements for an “accelerated approval,” a type of regulatory clearance that allows a drug to be marketed even when there remains uncertainty about its clinical benefit if it fills an unmet need for a serious disease and has effects against a biological sign of the disease.<blockquote>尽管有人反对，卡瓦佐尼博士还是写道，百健公司已经满足了 “加速批准 ”的要求，这是一种监管许可，允许一种药物在临床益处仍存在不确定性的情况下上市，只要该药物满足了严重疾病未得到满足的需求，并且对疾病的生物体征产生了影响。</blockquote> Dr. Cavazzoni wrote that Aduhelm met that standard because of its effect of reducing a sticky substance called amyloid which “is reasonably likely to predict clinical benefit.”<blockquote>Cavazzoni 博士写道，Aduhelm 之所以符合这一标准，是因为它能减少一种叫做淀粉样蛋白的粘性物质，而淀粉样蛋白 “很有可能预测临床益处”。</blockquote> Some doctors and scientists saytargeting the amyloid plaqueshasn’t been proven to slow Alzheimer’s. Some members of a panel of advisers that recommended the FDA reject the drug haveresigned from the committee, citing the agency’s approval.<blockquote>一些医生和科学家说，针对淀粉样斑块尚未被证明可以减缓阿尔茨海默氏症的进展。建议美国食品和药物管理局拒绝该药物的顾问小组的一些成员以该机构的批准为由辞去了委员会职务。</blockquote> Biogen listed the price of Aduhelm at $56,000 a year, and patients and Medicare could also face testing charges.<blockquote>百健将 Aduhelm 的价格列为每年 56,000 美元，患者和医疗保险也可能面临检测费用。</blockquote> About six million Americans suffer from Alzheimer’s, a progressive condition that robs people of their memories and cognitive abilities. Biogen has said that as many as two million of them may benefit from the medicine.<blockquote>约有 600 万美国人患有阿尔茨海默氏症，这是一种会剥夺人们记忆和认知能力的进行性疾病。百健表示，其中多达200万人可能会从这种药物中受益。</blockquote> The FDA approval sent Biogen’s stock soaring. The company has said it expects only minimal revenue from the drug this year, in part because of the logistical challenges in administering the medicine, but that sales would begin to increase next year.<blockquote>美国食品和药物管理局的批准使百健的股价飙升。该公司表示，预计今年该药物的收入将微乎其微，部分原因是管理该药物的后勤挑战，但销售额将在明年开始增长。</blockquote> Analysts estimate sales of $946.3 million in 2022, according to FactSet. Sales are seen growing to $5.2 billion in 2026.<blockquote>根据 FactSet 的数据，分析师预计 2022 年销售额为 9.463 亿美元。预计 2026 年销售额将增长至 52 亿美元。</blockquote> The memos show FDA officials attempting to balance the subpar study data for Aduhelm with the desire of Alzheimer’s patients and their families to try any new treatment that might help.<blockquote>备忘录显示，美国食品和药物管理局官员正试图在 Aduhelm 的研究数据与阿尔茨海默氏症患者及其家人尝试任何可能有帮助的新疗法的愿望之间取得平衡。</blockquote> “There remains residual uncertainty regarding clinical benefit,” Dr. Stein wrote. But if the FDA were to wait for the company to finish another clinical study, “any approval would occur after a multiple year delay” and patients “could suffer irreversible loss of brain neurons and cognitive function and memory,” he wrote.<blockquote>“斯坦因博士写道：”临床获益仍存在不确定性。但他写道，如果美国食品和药物管理局等待该公司完成另一项临床研究，“任何批准都将在多年的延迟之后进行”，患者 “可能会遭受不可逆转的大脑神经元、认知功能和记忆力丧失”。</blockquote> The FDA said it plans to release more documents regarding its decision making as early as next week.<blockquote>FDA表示，计划最早下周发布更多有关其决策的文件。</blockquote> The FDA’s approval was based primarily on two large Phase 3 clinical trials launched by Biogen to prove its drug helped slow the cognitive decline of people with early-stage Alzheimer’s.<blockquote>美国食品和药物管理局的批准主要基于百健公司启动的两项大型 3 期临床试验，以证明其药物有助于减缓早期阿尔茨海默氏症患者的认知能力下降。</blockquote> In March 2019, the companyhalted the studies earlyafter an independent monitoring board concluded the drug was unlikely to help patients.<blockquote>2019 年 3 月，在独立监测委员会得出结论该药物不太可能帮助患者后，该公司提前停止了研究。</blockquote> Seven months later, however,the company made a surprising reversaland said it was now planning to seek FDA approval after conducting additional analyses of trial data that came in after the studies were halted. The company said its analyses found the drug was successful in one study but failed in the second.<blockquote>然而，七个月后，该公司做出了令人惊讶的逆转，并表示在对研究停止后获得的试验数据进行额外分析后，现在正计划寻求 FDA 的批准。该公司表示，其分析发现该药物在一项研究中取得了成功，但在第二项研究中失败。</blockquote> The FDA memos provide some new details on Biogen’s about-face. In June 2019, the company met with FDA officials and both sides agreed the studies shouldn’t have been stopped, according to a “summary memorandum” document.<blockquote>美国食品和药物管理局的备忘录提供了一些关于百健转变的新细节。根据一份 “摘要备忘录 ”文件，2019 年 6 月，该公司会见了 FDA 官员，双方一致认为不应停止研究。</blockquote> At the meeting, FDA officials recommended that Biogen analyze the studies further to understand if it was still possible to draw conclusions from the data, despite the halt of the trials and conflicting results.<blockquote>在会议上，FDA 官员建议百健进一步分析这些研究，以了解尽管试验停止且结果相互矛盾，但是否仍然有可能从数据中得出结论。</blockquote> Several months later, in October 2019, the agency and company met again and agreed that the studies could still be used to evaluate the drug’s effectiveness, according to the memo.<blockquote>根据备忘录，几个月后，即 2019 年 10 月，该机构和公司再次会面，并一致认为这些研究仍可用于评估药物的有效性。</blockquote> The following day, on Oct. 22, Biogen announcedit would seek approval for the medicine, sending up its share price and adding billions of dollars to its market value.<blockquote>第二天，即 10 月 22 日，百健宣布将寻求该药物的批准，从而推高其股价，并为其市值增加数十亿美元。</blockquote> The company had been quietly re-examining the data for months, but didn’t have the confidence to move forward until the latest meeting with FDA officials, Biogen Chief Executive Michel Vounatsos said in an interview at the time.<blockquote>百健公司首席执行官米歇尔-沃纳索斯（Michel Vounatsos）当时在接受采访时说，几个月来，公司一直在悄悄地重新审查这些数据，但直到最近一次与美国食品及药物管理局官员会面，公司才有信心继续前进。</blockquote>\n<div class=\"bt-text\">\n\n\n 来源：<a href=\"https://www.wsj.com/articles/fda-approved-biogen-alzheimers-drug-despite-some-staff-concerns-11624393800?mod=hp_lead_pos2\">WSJ</a>\n为提升您的阅读体验，我们对本页面进行了排版优化\n\n\n</div>\n</article>\n</div>\n</body>\n</html>\n","type":0,"thumbnail":"","relate_stocks":{"BIIB":"渤健公司"},"source_url":"https://www.wsj.com/articles/fda-approved-biogen-alzheimers-drug-despite-some-staff-concerns-11624393800?mod=hp_lead_pos2","is_english":true,"share_image_url":"https://static.laohu8.com/e9f99090a1c2ed51c021029395664489","article_id":"1174011543","content_text":"The U.S. Food and Drug Administrationapproved the first new Alzheimer’s drug in decadesover the objection of agency statisticians who said there was insufficient evidence to support approval, according to newly released internal memos.\nIn the internal memos released Tuesday, FDA officials discussed whether to approve the drug fromBiogenInc.BIIB-1.71%over objections from the agency’s drug statistics office, which said that clinical trial data fell short of the proof typically required to put a new product on the market.\nUltimately, top officials decided that the evidence indicating that the drug worked, while inconclusive, was strong enough to allow doctors and patients to decide for themselves whether to take it, according to the memos.\n“FDA, then, is faced with a situation where the available evidence on the clinical endpoints is short of what we would require for standard approval of aducanumab,” wrote Peter Stein, director of the FDA’s office of new drugs, in a memo detailing his support for approval. “At the same time, patients with [Alzheimer’s] have a serious, progressive, ultimately fatal disease and are desperate for treatments.”\nThe FDA made public its internal deliberations as it faces criticism from some doctors and scientists over the June 7 approval of the drug, called Aduhelm.\nThe critics say the FDA ignored the scientific standards it typically holds drugmakers to and could prompt the federal government to spend billions of dollars on a drug that may not work.\n“While I fully support the Center’s recent decision to approve the therapy, I acknowledge that there are different opinions about this decision, including within FDA, and there likely will continue to be questions around our process for review and the data we considered to support the approval,” said FDA Acting Commissioner Janet Woodcock. “In the end, an experienced group of medical reviewers at FDA concluded that there was sufficient evidence to approve the Alzheimer’s therapy via the accelerated approval pathway.”\nSupporters say the FDA decision provides a lifeline to patients who have lacked any medicine promising to slow the progression of Alzheimer’s, and that the decision couldspur the development of more, better medicines.\nThe agency usually releases approval documents after a decision, though not typically as quickly as it did this time. The 83 pages of memos appear to be a portion of the FDA’s deliberations, largely reflecting the exchanges of top officials.\nTristan Massie, a biostatistics reviewer for the FDA, “does not agree that the totality of the data provides sufficient evidence to support the efficacy of aducanumab in Alzheimer’s disease and does not recommend approval,” one of the internal documents shows.\nPatrizia Cavazzoni, the FDA’s top-ranking drug-evaluation official, wrote in another memo that the agency’s drug biostatistics office “provided documentation for its recommendation that substantial evidence of effectiveness had not been provided in the application.”\nThat office’s full dissenting analysis wasn’t included in the materials made available Tuesday, but portions of it were.\nDespite the objections, Dr. Cavazzoni wrote that Biogen had met the requirements for an “accelerated approval,” a type of regulatory clearance that allows a drug to be marketed even when there remains uncertainty about its clinical benefit if it fills an unmet need for a serious disease and has effects against a biological sign of the disease.\nDr. Cavazzoni wrote that Aduhelm met that standard because of its effect of reducing a sticky substance called amyloid which “is reasonably likely to predict clinical benefit.”\nSome doctors and scientists saytargeting the amyloid plaqueshasn’t been proven to slow Alzheimer’s. Some members of a panel of advisers that recommended the FDA reject the drug haveresigned from the committee, citing the agency’s approval.\nBiogen listed the price of Aduhelm at $56,000 a year, and patients and Medicare could also face testing charges.\nAbout six million Americans suffer from Alzheimer’s, a progressive condition that robs people of their memories and cognitive abilities. Biogen has said that as many as two million of them may benefit from the medicine.\nThe FDA approval sent Biogen’s stock soaring. The company has said it expects only minimal revenue from the drug this year, in part because of the logistical challenges in administering the medicine, but that sales would begin to increase next year.\nAnalysts estimate sales of $946.3 million in 2022, according to FactSet. Sales are seen growing to $5.2 billion in 2026.\nThe memos show FDA officials attempting to balance the subpar study data for Aduhelm with the desire of Alzheimer’s patients and their families to try any new treatment that might help.\n“There remains residual uncertainty regarding clinical benefit,” Dr. Stein wrote. But if the FDA were to wait for the company to finish another clinical study, “any approval would occur after a multiple year delay” and patients “could suffer irreversible loss of brain neurons and cognitive function and memory,” he wrote.\nThe FDA said it plans to release more documents regarding its decision making as early as next week.\nThe FDA’s approval was based primarily on two large Phase 3 clinical trials launched by Biogen to prove its drug helped slow the cognitive decline of people with early-stage Alzheimer’s.\nIn March 2019, the companyhalted the studies earlyafter an independent monitoring board concluded the drug was unlikely to help patients.\nSeven months later, however,the company made a surprising reversaland said it was now planning to seek FDA approval after conducting additional analyses of trial data that came in after the studies were halted. The company said its analyses found the drug was successful in one study but failed in the second.\nThe FDA memos provide some new details on Biogen’s about-face. In June 2019, the company met with FDA officials and both sides agreed the studies shouldn’t have been stopped, according to a “summary memorandum” document.\nAt the meeting, FDA officials recommended that Biogen analyze the studies further to understand if it was still possible to draw conclusions from the data, despite the halt of the trials and conflicting results.\nSeveral months later, in October 2019, the agency and company met again and agreed that the studies could still be used to evaluate the drug’s effectiveness, according to the memo.\nThe following day, on Oct. 22, Biogen announcedit would seek approval for the medicine, sending up its share price and adding billions of dollars to its market value.\nThe company had been quietly re-examining the data for months, but didn’t have the confidence to move forward until the latest meeting with FDA officials, Biogen Chief Executive Michel Vounatsos said in an interview at the time.","news_type":1,"symbols_score_info":{"BIIB":0.9}},"isVote":1,"tweetType":1,"viewCount":3632,"commentLimit":10,"likeStatus":false,"favoriteStatus":false,"reportStatus":false,"symbols":[],"verified":2,"subType":0,"readableState":1,"langContent":"EN","currentLanguage":"EN","warmUpFlag":false,"orderFlag":false,"shareable":true,"causeOfNotShareable":"","featuresForAnalytics":[],"commentAndTweetFlag":false,"andRepostAutoSelectedFlag":false,"upFlag":false,"length":4,"xxTargetLangEnum":"ORIG"},"commentList":[],"hasMoreComment":false,"orderType":2}