FDA之声：扩展通道：FDA讲述简化申请流程

 翻译：JULIA 来源：Julia法规翻译

Expanded Access: FDA Describes Efforts to Ease Application Process

扩展通道：FDA讲述简化申请流程

Posted on October 3, 2017 by FDA Voice

By: Scott Gottlieb, M.D.

FDA has a long history of supporting patient access to investigational new treatments. This includes working with drug and devicecompanies through the clinical trial process that may lead to FDA approval ofthe treatment. We also offer expanded access programs that provide investigational drugs and devices to patients with serious conditions (generally prior to product approval), when there is no therapeutic alternative.

FDA在支持患者获得临床试验用新疗法方面有着很久的历史。其中包括与药品和医疗器械生产公司在可能会获得FDA批准的临床试验过程中合作。我们还提供了扩展通道，采用此方式为病情严重且没有替代疗法的患者提供临床试验用药品和器械（通常是在药品批准之前）。

Each year, FDA receives over 1,000 applications for the treatment of patients through expanded access, also known as compassionate use, and the agency authorizes the vast majority (about 99%). FDA recognizes that time is critical for these seriously ill patients who do not have  alternative therapies, and who cannot take part in a clinical trial of an investigational therapy.

每年，通过扩展通道FDA都会收到超过1000起患者治疗申请，这也被称为特许使用，而FDA批准了其中的大部分申请（约为99%）。FDA认识到对于这些严重的病患来说，时间是很宝贵的，他们没有其它的替代治疗，也不能参与临床试验研究的治疗。

Submissions are authorized quickly, often in a matterof days. In the case of emergencies, FDA will typically provide authorization over the phone in a matter of hours. In an effort to eliminate potential hurdles that might delay or even discourage applications, FDA streamlined the expanded access process by introducing a new application form which a physicianmay use to request expanded access for their patient. Form FDA 3926 reduced the number of required information fields and attachments, and is estimated to take only 45 minutes for a physician to complete. Before expanded access can occur, the drug company must decide whether or not to provide the product. FDA cannot require a manufacturer to provide aproduct.

申请一般会很快被批准，通常就是几天时间。如果是紧急情况，FDA一般会通过电话在几个小时之内批准。为了消除患者可能会延误甚至放弃申报的障碍，FDA梳理了扩展通道流程，引入了新的申请表格，医生可以使用该表格来为其病人申请扩展通道。FDA表格3926减少了所需填写的信息数量及附件数量，预计医生只需要花费45分钟填写完成。在没有扩展通道之前，是由制药公司来决定是否提供药品，而FDA不能要求生产商提供药品。

Today, we’re lifting another potential burden for physicians who apply to FDA to use an investigational drug to treat their patient.

今天，我们要减除医生向FDA申请使用临床试验用药品来治疗其患者的另一个潜在负担。

Prior to treating a patient under expanded access, the physician must obtain approval from the Institutional Review Board (IRB) attheir facility. This is an important step to protect the rights, safety andwell-being of human subjects in clinical research – but assembling the full board may cause delays because they may not routinely meet.

在依据扩展通道治疗患者之前，医生必须获得其所在场所伦理委员会（IRB）的批准。这是保护临床研究中人体受试对象的权利、安全和福祉的重要措施—然而要组织完整的伦理委员会可能会导致延误，因为他们可能并不会定期碰头。

As part of a plan to simplify the process forphysicians seeking access to an investigational product to treat their patient, I’m announcing today that just one IRB member – the chair or another appropriate person – can now approve the treatment. I believe the simplified IRB process will facilitate access while still protecting patients.

作为简化医生获取临床试验药品用以治疗其患者的计划的一部分，我今天宣布只需要一位IRB成员---主席或其它适当的人员---即可以批准该类治疗。我相信简化后的IRB流程会有利于获取药品同时保护患者。

More simplifications and clarifications are in the pipeline.

更多的简化措施和澄清说明正在制订中。

We’ve seen some reluctance among companies to provide investigational drugs for expanded access. This may have been due, in part, to uncertainty about how data for adverse events that occur during treatment underexpanded access are viewed by FDA. Companies have voiced concerns that anyapparent negative effects might jeopardize the product’s development.

我们已经看到了，有些公司并不愿意提供临床用药品给扩展通道。这可能部分因为他们担心FDA会如何看待在扩展通道的治疗中所发生的不良反应数据。公司已经表示担心明显的不良影响可能会阻碍产品的开发。

We recognize that patients receiving expanded accessare usually treated outside of a controlled clinical trial setting. As aresult, they may have more advanced disease than clinical trial participants,be receiving other drugs at the same time, and have other diseases. FDA recognizes that these factors make it more difficult to determine the cause an adverse reaction.

我们知道接受扩展通道治疗的患者通常是在受控临床试验设置以外进行治疗的，因此，他们可能会比临床试验参与者的病情更为严重，可能同时还在使用其它药品，还有其它的疾病。FDA知道这些因素会使得确定不良反应的原因更为困难。

To clarify how adverse event data in these circumstances are viewed, we’ve updated the guidance for industry entitled, ‘Expanded Access to Investigational Drugs for Treatment Use: Questions and Answers’ (questions 25 and 26). The guidance clarifies that suspected adverse reactions must be reported “only if there is evidence to suggest a causal relationship between the drug and the adverse event.”

为了明确在这些情况下产生的不良事件数据要如何看待，我们已经更新了行业指南标题，“临床试验药品扩展通道用于治疗：问答”（问题25和26）。指南中明确说明了可疑的不良反应“只有在有证据显示药品与不良反应之间具有因果关系时”才必须报告。

I’m confident these changes will help to address recent issues raised by the Government Accountability Office (GAO), which said that FDA “should further clarify how adverse event data are used.” We are still evaluating the GAO recommendations to identify other possible ways to respond to their concerns.

我相信这些变化会有助于解决近期GAO所提出的问题，其中说FDA“应进一步明确要如何使用不良反应数据”。我们仍在评估GAO的建议，以找出其它可能的方式来对其关切进行反馈。

We’re committed to helping patients and physicians fully understand the expanded access process. Dedicated staff in the Office of Health and Constituent Affairs and CDER’s Office of Communications, Division of Drug Information, already assist physicians and patients in navigating thissystem. We issued three final guidance documents last year to clarify and explain the process. This past July, we collaborated with the Reagan-UdallFoundation, patient advocacy groups, the pharmaceutical industry, and other federal agencies to launch a new online tool called the Expanded Access Navigator. This includes a directory where companies can submit public links to their expanded access policies, the criteria used by companies to determine whether to make a drug available through expanded access, and contact information. The directory offers patients and physicians a helpful starting point for researching available investigational therapies.

我们承诺要帮助患者和医生全面了解扩展通道程序。卫生和组织事务办公室与CDER的沟通办公室、药物信息中心的专职人员已开始协助医生和患者使用此系统。我们去年签发了三份定稿指南以澄清和解释该流程。在7月，我们与里根-尤德尔基金、患者支持团队、制药企业和其它联邦机构合作，启动了一个新的在线工具，称为扩展通道导航。其中包括了引导公司提交公众链接至其扩展通道的政策、公司用以决定是否通过扩展通道提供其药品的标准，以及联系信息。指导书为患者和医生搜索可用的临床试验用治疗方法提供了一个有用的起始点。

In addition, we’re working with the Reagan-Udall Foundation to expand this new tool. I’m please to announce that Reagan-Udallwill expand its portfolio to include FDA’s Rare Disease Program, with the goal of promoting more expanded access to treatments for rare disorders.

此外，我们还在与里根-尤德尔基金一起扩展此新工具。我很高兴地宣布，里根-尤德尔会扩充其内容，在上面包括FDA的罕见病计划，其目标是促进更多罕见疾病的扩展通道。

Looking ahead, there will be more options for expanded access. FDA will continue our efforts to educate stakeholders on these opportunities and further simplify the process in order to improve the program.

展望未来，扩展通道会有更多的选择。FDA会持续努力让相关人士了解此类机遇，并进一步简化流程改进计划。

Scott Gottlieb, M.D., is Commissioner ofthe U.S. Food and Drug Administration

Follow Commissioner Gottlieb on Twitter @SGottliebFDA