来源：《自然》
原文刊登日期：2022年5月13日
Nearly a year after the US Food and Drug Administration (FDA) gave the green light to a controversial drug to treat Alzheimer’s disease, lawmakers are attempting to amend the process that led to its approval.
近一年前，美国食品和药物管理局(FDA)批准了一种有争议的治疗阿尔茨海默氏症的药物，立法者正试图修改导致该药物获得批准的程序。
The House Committee on Energy and Commerce, which oversees drug safety and biomedical research, announced last week that it hopes to grant the FDA greater authority to rescind accelerated approvals if a company fails to complete follow-up studies on the treatment in a reasonable amount of time.
监督药物安全和生物医学研究的众议院能源和商业委员会上周宣布，如果一家公司未能在合理的时间内完成治疗的后续研究，它希望赋予FDA更大的权力，以撤销已经授予的加速批准。
The provision, which was introduced as part of an FDA funding reauthorization bill, likely to be passed before September, comes on the heels of the agency’s 2021 approval of aducanumab, an antibody drug shown to reduce the accumulation of plaques in the brain associated with the progression of Alzheimer’s. Despite a nearly unanimous vote against the approval by an independent panel of experts, the agency fast-tracked the drug, which was developed by Biogen, a biotechnology company based in Massachusetts. Three advisory-panel members resigned in protest against the decision, and the approval is the subject of multiple investigations by federal regulators.
这项规定是可能会在9月前通过的FDA资助再授权法案的一部分，它紧随2021年FDA批准aducanumab后被提出，aducanumab是一种抗体药物，可以减少与阿尔茨海默病进展相关的大脑斑块的积累。尽管一个独立的专家小组几乎全票反对批准，FDA还是快速批准了由马萨诸塞州生物技术公司百健(Biogen)开发的这种药物。三名顾问小组成员辞职以抗议这一决定，联邦监管机构对这一批准进行了多项调查。
Aducanumab is not the only reason that this drug-approval pathway is coming under fire: since its inception, the programme has led to 279 treatments reaching the market, with nearly two-thirds in the past decade alone. The programme’s increasing popularity signals a shift away from its original intent, says Diana Zuckerman, president of the National Center for Health Research. “Accelerated approval started out as a special programme for a small number of drugs, and now most cancer drugs are going through accelerated or some other expedited pathway,” she says.
Aducanumab并不是这一药物批准途径受到抨击的唯一原因：自该项目启动以来，已经有279种疗法进入市场，这其中有近三分之二是在过去十年进入市场的。国家健康研究中心主席戴安娜·扎克曼表示，该项目越来越受欢迎，标志着该项目偏离了其初衷。她说：“加速审批最初是针对少数药物的一个特殊项目，现在大多数癌症药物都在使用加速审批或其他一些加快审批的途径。”
Companies, moreover, have been slow to produce the follow-up studies promised as part of the approval process. The FDA has limited power to compel them to provide the data, but the legislative proposal — which could still change significantly as it wends its way through the House of Representatives and the Senate — could grant it more authority to do so.
此外，制药公司迟迟未能完成批准过程中承诺的后续研究。FDA在强迫制药公司提供数据方面的权力有限，但9月前的立法提案——在众议院和参议院通过的过程中仍可能发生重大变化——可能会赋予FDA更大的权力来这样做。
Days before his appointment in February, FDA commissioner Robert Califf pledged to make accelerated-approval reform a priority for the agency. Researchers who spoke to Nature agree that reforms are needed to protect the integrity of the programme, and that the proposed legislation is a good start. But they also recommended more agency oversight and other changes that would further prevent pharmaceutical firms from abusing this route to the market.
FDA局长罗伯特•卡利夫在2月份被任命前几天承诺，将快速审批改革作为该机构的优先事项。接受《自然》杂志采访的研究人员同意，需要进行改革以保护该项目的公正性，而拟议中的立法是一个良好的开端。但他们也建议加强机构监管和其他改革，以进一步防止制药公司滥用这条进入市场的途径。
“Instead of the drug companies living up to and working to ensure that they are employing the accelerated-approval pathway as intended, we have too many that are willing to take advantage of the loopholes where they can find them,” says David Mitchell, president of Patients for Affordable Drugs, who serves as a consumer representative on the independent panel that reviews cancer drugs for the FDA.
“制药公司没有做到并努力确保他们按照预期使用加速批准途径，而是有太多的制药公司愿意利用漏洞，只要他们能够找到。”平价药物患者协会主席大卫·米切尔说，他是FDA审查抗癌药物的独立委员会的消费者代表。