
在美国食品药品监督管理局公布加速罕见病治疗药物审批的新计划一周后,新的立法正被提出,以重新构想这些药物的研究方式。
One week after the Food and Drug Administration unveiled a new initiative for speeding up the approval of rare diseases treatments, new legislation is being introduced to reimagine the way that those drugs are studied.
这项两党法案将于今日由民主党众议员杰克·奥钦洛斯(Jake Auchincloss)和共和党众议员摩根·格里菲斯(Morgan Griffith)共同提出,旨在让更多患者参与临床试验,并缩短启动试验的时间。所谓的《加速临床试验法案》(Faster Clinical Trials Act)还将鼓励药企在早期药物研发中使用计算机模拟,并解决美国食品药品监督管理局因避免延误而暂停临床试验的一些常见原因。
The bipartisan bill, which will be introduced today by Democratic Rep. Jake Auchincloss and Republican Rep. Morgan Griffith, would make it easier for more patients to participate in clinical trials and reduce the time it takes to get them started. The so-called "Faster Clinical Trials Act" would also encourage drugmakers to use computer simulations in early-stage drug development and address some of the common reasons why the FDA suspends clinical trials to avoid delays.
通常,临床试验会在特定地点召集各类人员。试验结束后,基础设施被拆除,所有人各奔东西,尽管针对特定疾病或治疗类型的许多环节在不同试验之间是相同的。该立法旨在建立一个可重复使用的临床试验场所网络。
Typically, clinical trials assemble various people at specific locations. When the trial ends, the infrastructure is dismantled and everyone goes their separate ways, even though many things are the same from trial to trial for a particular disease or type of treatment. The legislation aims to create a network of reusable clinical trial sites.
“所以你不必每次尝试新药时都重新造轮子,”奥钦洛斯在接受CNBC Cures采访时说。“你已经有了一个平台,现在只需要一个接一个地尝试不同的东西。”格里菲斯表示,该法案将惠及除罕见病患者之外的其他患者,包括他在弗吉尼亚州西南部选区的居民,这些居民希望参与新疗法对黑肺病的研究。他的一些选民居住在距离通常举办临床试验的医疗中心两小时甚至更远的路程。
"So you're not recreating the wheel every time you're trying to do a new drug," Auchincloss said in an interview with CNBC Cures. "You have a platform, and now you're just trying one thing after another." Griffith said the bill would benefit patients beyond those with rare diseases, including residents of his southwest Virginia district who want to participate in studies of new treatments for black lung disease. Some of his constituents live a two-hour drive or longer from medical centers that typically host clinical trials.
“我们正尝试把临床试验带到患者身边,而不是如今把患者带到临床试验现场的模式,”他在采访中说。
"We're trying to bring the clinical trial to the patient instead of the model today of bringing the patient to the clinical trial," he said in an interview.
该立法在食品药品管理局(FDA)宣布自身计划以加速罕见病新疗法研发仅几天后出台。在新的罕见病框架倡议下,该机构表示,将在评估小规模患者群体疗法时更加灵活。FDA的计划将为制药商提供“非常明确的长期适用的行业规则”,前FDA专员斯科特·戈特利布(Scott Gottlieb)在10月19日CNBC《早间快报》(Squawk Box)节目采访中表示。
The legislation comes just days after the FDA announced its own plan to accelerate new treatments for rare diseases. Under the new Rare Disease Framework Initiative, the agency said it will be more flexible in evaluating therapies for small patient populations. The FDA's plan will give drugmakers "very clear rules of the road that are going to last a long time," former FDA Commissioner Scott Gottlieb said in an interview on CNBC's Squawk Box on Oct. 19.
新立法在FDA内设立“临床试验创新中心”,以协调临床试验站点网络。它还对FDA罕见病创新中心提出具体指示,例如授权其就制药商的顾虑进行咨询,并为该机构的审查团队提供帮助。
The new legislation creates a "Center for Clinical Trial Innovation" at the FDA to coordinate networks of clinical trial sites. It also lays out specific instructions to the FDA's Rare Disease Innovation Hub, like giving it the authority to consult with drugmakers about their concerns and provide help to the agency's review teams.
该法案创造了一种情景:患者可能在常规医生办公室获得实验性药物,而无需前往临床试验站点。试验研究人员可以通过视频通话、电子病历或可穿戴技术追踪患者进展。
The bill creates a scenario where patients may get an experimental drug in their regular doctor's office instead of traveling to a clinical trial site. Trial researchers could track their progress through Zoom calls, electronic medical records or wearable technology.
“其理念是让患者尽可能留在原地,”杜克大学杜克-马戈利斯健康政策研究所(Duke-Margolis Institute for Health Policy)创始主任马克·麦克莱兰(Mark McClellan)表示,该研究所协助制定了该立法。“无需出行即可入组试验、服药、接受监测并获取随访数据。”该立法在未来五年内向FDA拨款最高每年7500万美元。
"The idea is to make it as easy as possible for the patient to stay right where they are," said Mark McClellan, founding director of the Duke-Margolis Institute for Health Policy at Duke University, which helped craft the legislation. "To enroll in the trial, take the drug and be monitored and get the follow-up data without having to travel." The legislation directs as much as $75 million in annual funding to the FDA over the next five years.
临床试验网络将优先关注罕见病和慢性病。目标是将试验成本和入组参与者所需时间至少降低30%。奥钦克洛斯(Auchincloss)将其比作囊性纤维化基金会在20世纪90年代为研究该疾病而建立的开创性临床试验模式。
The clinical trial networks would prioritize rare and chronic diseases. The goal is to cut the cost of trials and the time it takes to enroll participants by at least 30%. Auchincloss compares it to the pioneering clinical trial model that the Cystic Fibrosis Foundation built in the 1990s to study the disease.
“这样你就不用每次研发新药时都从零开始。你有了平台,现在只是一件接一件地尝试。”美国众议员杰克·奥钦克洛斯(民主党-马萨诸塞州)说。
So you're not recreating the wheel every time you're trying to do a new drug. You have a platform, and now you're just trying one thing after another."
“点位护理”临床试验的理念已有十多年历史。美国退伍军人事务部于2010年开展了首次此类试验,对比两种糖尿病患者的治疗方案。如今,这一理念在澳大利亚已被广泛采用。但在美国,它并未获得多大进展,因为美国研究药物的系统原本就是为在特定临床试验场所进行而设计的。新技术现在使得将药物研究带到患者家门口成为可能。
Rep. Jake Auchincloss, D-Mass. The idea of "point-of-care" clinical trials has been around for more than a decade. The Department of Veterans Affairs ran the first one in 2010 by comparing two treatment regimens for patients with diabetes. Today it's used widely in Australia. But it hasn't gained much traction in the United States, where the system for studying drugs was designed to take place at specific clinical trial sites.
这一理念可能有利于罕见病患者。通过扩大点位护理试验,寻找患者可能会更容易——这是罕见病面临的常见问题,此类疾病可能只影响几十人。此外,许多罕见病患者家庭花费数年时间建立数据库,即自然病史研究,以便在试验中作为对照组,而不是给患者使用安慰剂,这往往不切实际且不道德。根据该法案,这些数据库可以利用电子健康记录来建立。
New technology now makes it possible to bring drug studies to a patient's doorstep. The idea could benefit rare disease patients. By expanding point-of-care trials, it could be easier to find patients—a common problem for rare diseases that may only affect a few dozen people. Also, many families of rare disease patients spend years creating databases, known as natural history studies, so they can be used as control groups in trials instead of giving patients a placebo, which is often impractical and unethical.
其目标是让患者尽可能轻松地留在原地。患者无需出行,即可入组试验、服药、接受监测并获取随访数据。”杜克大学杜克-马戈利斯健康政策研究所创始所长马克·麦克莱兰说。该方法也有局限。密集型治疗,如基因疗法,仍需在学术医学中心进行,麦克莱兰表示。但新的科学进展——如体内基因编辑——使患者能够当天出院。如果他们正在参加临床试验,可以回家并接受远程监测,他说。
Under the bill, those databases could be created with electronic health records. The idea is to make it as easy as possible for the patient to stay right where they are. To enroll in the trial, take the drug and be monitored and get the follow-up data without having to travel." Mark McClellanFounding Director, Duke-Margolis Institute for Health Policy at Duke University There are limits to the approach. Intensive treatments, like gene therapies, will still need to be given at academic medical centers, McClellan said. But new scientific advances – like in vivo gene editing – allow patients to leave the hospital the same day. If they're in a clinical trial, they could go home and be monitored remotely, he said.
格里菲斯表示,该法案可能要到明年才会提交表决。奥钦克洛斯办公室表示,他们正与跨党派参议员合作,计划今年晚些时候在参议院提出一项类似法案。
Griffith said the bill likely won't move forward for a vote until next year. Auchincloss's office said that they are working with a bipartisan group of senators to introduce a similar measure in the Senate later this year.
奥钦克洛斯表示,他预计众议院法案将获得通过,因为它能吸引广泛人群——从希望加快并降低临床试验成本的制药公司和患者团体,到希望更多获得实验性治疗的“让美国再次健康”运动支持者。“这里面每个人都能找到喜欢的东西,”奥钦克洛斯说。
Auchincloss said he expects the house measure will pass because it appeals to a wide range of people — from drugmakers and patient groups that want faster and cheaper clinical trials, to followers of the MAHA movement who want greater access to experimental treatments. "There's something in here for everyone to like," Auchincloss said.