Senators urge action in Novartis gene therapy data manipulation scandal


2019-08-12 12:25:00 medcitynews


Five Democratic senators – two of them presidential candidates – are calling on the head of the Food and Drug Administration to take action in the wake of revelations last week that one of the world’s largest drugmakers had submitted manipulated data to the agency in its approval application for a gene therapy. In the letter, sent Friday to acting FDA Commissioner Ned Sharpless, the senators wrote that the scandal that emerged last week when the agency revealed allegations that certain animal testing data for Swiss drugmaker Novartis’ gene therapy Zolgensma were manipulated, but that the company did not inform the agency until after it was approved, highlights what they called the drug industry’s “privilege and greed.” The letter was signed by Sens. Dick Durbin, Illinois, Tammy Baldwin, Wisconsin, Bernie Sanders, Vermont, Elizabeth Warren, Massachusetts, and Richard Blumenthal, Connecticut. “It is unconscionable that a drug company would provide manipulated data to federal regulators in order to rush its product to market, reap federal perks, and charge the highest amount in American history for its medication,” they wrote. “Such greed cannot be condoned by the FDA.” Zolgensma – originally developed by AveXis, which Novartis acquired for $8.7 billion last year – received FDA approval in May for spinal muscular atrophy, and was given a $2.1 million list price. A Novartis spokesperson pointed to a statement the company released last week, standing by the therapy. However, the larger issue is not Zolgensma’s benefit-risk profile. Indeed, FDA officials have recommended it remain on the market. Rather, it’s that the company allegedly knew of the data manipulation issue in March, but did not tell the agency until late June, more than a month after Zolgensma’s approval. The company said that upon learning of the problem in the middle of March, it launched a two-part investigation. During a conference call with analysts last week, when asked when the news landed on his desk, CEO Vas Narasimhan did not answer directly, though the spokesperson wrote in an email that he found out in May. But in the letter Friday, the senators noted that federally funded research by the National Institutes of Health had contributed to Zolgensma’s development, and that the company received taxpayer-funded benefits designed to expedite the approval process like Fast Track, Breakthrough and Priority Review designations. In response, they urged the agency to use its authority to hold AveXis accountable, including through criminal, civil and regulatory actions. The FDA had stated last week the civil and possible criminal penalties were possible. They also sought a formal, written explanation for why the agency had withdrawn a proposed regulation in October 2018 that would have required certain clinical trial sponsors to promptly report suspected data falsification to the agency. “In light of AveXis’ alarming actions, we also seek to know whether FDA plans to re-issue such regulation,” they wrote. Photo: bpperry, Getty Images Promoted The impact of artificial intelligence on outcomes based contracting Discover insights from decision makers on the trends and challenges of frequently used OBC types. Read the whitepaper. ICON Promoted The U.S. has fantastic health care, the problem is… In part 1 of an executive interview series, CEO and physician Wyatt Decker discusses his perspectives on today’s challenges and opportunities for reinventing health care. Optum How can payers use automation to improve data accuracy and reduce costs? A health technology company wants to help payers improve the accuracy of their data through automation. Learn how. MedCity News Join Us at MedCity INVEST Digital Health 2019 MedCity INVEST Digital Health , held September 17, 2019 in Minneapolis, will unite active investors with corporate business development executives to facilitate investment opportunities with the most innovative companies from the Digital Health sector. Reserve your place.>> AI & Product Launches: Meeting the Need for Speed In this post, ZS’s experts discuss how AI can be leveraged in pharmaceutical companies for product launches and how AI can make product launches more effective and collaborative. ZS Associates
五名民主党参议员(其中两名是总统候选人)呼吁美国食品和药物管理局( Food and Drug Administration )负责人采取行动,此前上周有消息称,全球最大的制药商之一已向该局提交基因治疗申请的批准文件,提交了操纵数据。在上周五发给代理 FDA 专员 Ned Sharpless 的信中,两位参议员写道,上周发生的丑闻是,该机构披露了瑞士制药商诺华(诺华(Novartis))基因治疗佐尔甘斯曼( Zolgensma )的某些动物检测数据受到操纵的指控,但该公司直到获得批准后才通知 FDA 。他们强调了他们所说的制药业的“特权和贪婪”。这封信是由 Sen 签署的。Dick Durbin , Illinois , Tammy Baldwin ,威斯康辛, Bernie Sanders , Vermont , Elizabeth Warren , Massachusetts , Richard Blumenthal ,康涅狄格州。他们写道:“一家制药公司将向联邦监管机构提供被操纵的数据,以便将其产品推向市场、获取联邦补贴,并对其药品收取美国历史上最高的费用,这是不合理的。”“ FDA 不能容忍这种贪婪。”Zolgensma 最初是由 AveXis 公司开发的,诺华(Novartis)公司去年以87亿美元的价格收购了它。5月, Zolgensma 获得了 FDA 的批准,用于脊柱肌肉萎缩,并获得了210万美元的定价。诺华(Novartis)公司的一位发言人指出,该公司上周发布的一份声明,站在治疗的立场上。然而,更大的问题不是 Zolgensma 的利益风险状况。事实上, FDA 官员已经建议继续上市。相反,据称该公司在3月就知道了数据操纵问题,但直到6月底才告诉该机构,这是 Zolgensma 批准的一个多月之后。该公司表示,在3月中旬获悉这一问题后,启动了一项两部分的调查。在上周与分析师举行的电话会议上,当被问及新闻何时登上办公桌时,首席执行官瓦丝•纳拉希姆汉( Vas Narasimhan )没有直接回答,不过这位发言人在5月份发来的一封电子邮件中写道。参议员们指出,由美国国立卫生研究院资助的联邦研究为佐尔甘马的发展做出了贡献,该公司获得了纳税人资助的福利,旨在加快快速通道、突破和优先审查指定等审批程序。作为回应,他们敦促该机构利用其权力追究 AveXi 的责任,包括通过刑事、民事和监管行动。美国食品和药物管理局上周曾表示,可能会受到民事和刑事处罚。他们还要求获得一份正式的书面解释,解释该机构为何在2018年10月撤销了一项拟议的法规,该法规要求某些临床试验赞助商立即向该机构报告可疑的数据造假。“鉴于 AveXis 的惊人举动,我们还试图了解 FDA 是否计划重新发布此类法规,”他们写道。图片: bpperry , Getty Images 促进人工智能对结果的影响基于订约决策者对常用 OBC 类型的趋势和挑战的了解。阅读白皮书。ICON Promoted 美国拥有出色的医疗保健,问题在于……在高管访谈系列的第1部分, CEO 和医生 Wyatt Decker 讨论了他对当今挑战和重塑医疗保健的机会的看法。付款人如何使用自动化来提高数据准确性和降低成本?一家健康科技公司希望通过自动化帮助付款人提高数据的准确性。学习如何。MedCity News 加入我们在 MedCity INVESTT Digital Health 2019 MedCity INVESTT Digital Health 于2019年9月17日在明尼阿波利斯举行的会议,将积极的投资者与企业业务发展主管团结起来,与数字健康领域最具创新性的公司一起为投资机会提供便利。保留你的位置.>> AI & Product Launches :满足速度需求在本文中, ZS 的专家讨论了如何在制药公司中利用 AI 进行产品发布,以及 AI 如何使产品发布更有效和协作。ZS Associates