【奥译言】FDA在临床试验招募过程中纳入女性的关键问题上的开发

2018-05-25 12:17 阅读数：4864 标签：

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2018年5月16日 Ana Mulero

美国食品和药物管理局（FDA）正在从两个关键问题——招募和为治疗性别差异而设计的新产品上着手寻求打破阻碍妇女临床试验数据可用性的障碍。

在本周三由FDA的女性健康办公室（OWH）主持的辩论会上，FDA委员Scott Gottlieb强调该机构正在采取一些双管齐下的方法，并说明“多年来对这些问题的忽视，以及偶尔对于这些问题的不理解，使得我们在进行医疗保健时出现了不一致。”

在这方面已经取得了一些进展。Gottlieb说到，举个例子，2015年参与到末期临床试验的研究受试者中52%为女性，相较于2000年的39%已经有所提升。

他还补充说到，OWH从其1994年建立开始，已经为371个项目提供了4000多万美元的研究资金，此举说明了FDA针对妇女健康和性别差异这些特定问题上所做的工作。这项研究对FDA了解不成比例的女性影响和某些药物是否存在性别特异性效果至关重要。

然而，在新政策和主动权方面，仍有一些有待改进的领域。

为了争取女性纳入到临床试验中，FDA正在全国范围内和国际上采取其他的措施，包括近启动的临床试验中的多样化妇女项目。此外，FDA正致力于预测模型，并通过一个名为“孕妇药物暴露的风险评估项目”的多站点研究合作来将母亲与其孩子的健康记录联系起来。

Gottlieb 补充说到，预测模型在临床试验中不暴露于药物的情况下对反应进行预测，提供了一个研究孕妇接触药物的机会。

今年3月，FDA还发布了科学和伦理方面的指导草案，以帮助研究申办者“明智地将孕妇纳入临床试验”。

Gottlieb说：“我们不希望这些领域中监管方面的不确定性，或者缺乏监管能力，成为创新和投资的障碍。”

另一个步骤是涉及到妇女卫生技术战略协调登记网络的建立，其作为4月份发布的FDA医疗器械安全行动计划的一部分——将数据到器械注册中，以便更好地为试验设计提供信息。

根据FDA的说法，登记网络致力于“在女性特定的临床领域提供更多完整的证据，例如子宫肌瘤、骨盆底紊乱、女性长效、可逆避孕和绝育。

这场辩论的重点是参与心血管临床试验，因为这至少是一个尽管在女性中相对普遍但招募量不足的临床研究领域。

此外，根据Gottlieb的说法，心血管疾病已经超过了乳腺癌，成为女性死亡的主要原因。

由FDA官员引导的并在上周发表于《美国心脏病学会杂志》的一项新的心血管药物治疗的临床研究分析表明，尽管女性“在治疗高血压和心房纤维性颤动中表现良好，且肺动脉高压中比例较高”，但其在心衰、冠状动脉疾病和急性冠脉综合征方面的表现有明显下降。

“观察到的药物有效性和安全性方面的性别差异小”，该项研究的作者补充说到，“考虑到男性和女性在心血管疾病的病理生理学、临床表现和效果方面的差异，女性的充分参与对于研究治疗反应中可能存在的性别差异非常重要”。

英语原文

FDA Hones In On Critical Issues With Recruitment, Inclusion of Women in Clinical Trials

Posted 16 May 2018 | By Ana Mulero

The US Food and Drug Administration (FDA) is looking to break down barriers hindering the availability of clinical trial data on women by focusing on two critical issues—enrollment and new products designed for treating conditions that are gender-unique.

FDA Commissioner Scott Gottlieb highlighted some of the actions the agency is pursuing as part of the two-pronged approach during a Wednesday debate hosted by FDA’s Office of Women’s Health (OWH), noting that the “years of neglect of these issues, and at times ignorance of them, have left us with disparities when it comes to the delivery of healthcare.”

There has been some progress on this front already. For example, 52% of human study subjects in late stage clinical trials in 2015 were women—up from 39% in 2000, Gottlieb said.

OWH has provided more than $40 million in research funding for 371 projects to inform FDA’s work on issues specific to women’s health and gender differences since its 1994 creation, he added. This research is critical in FDA’s understanding of the conditions that disproportionately affect women and whether some drugs have gender-specific effects.

However, there are still several areas of improvements to be address with new policies and initiatives.

Other steps FDA is taking to push for inclusion of women in trials, nationally and internationally, involve the recently launched Diverse Women in Clinical Trials Initiative. Also, FDA is investing in predictive modeling and linking health records on mothers and their babies via a multi-site research collaboration called the Medication Exposure in Pregnancy Risk Evaluation Program.

Predictive modeling offers the opportunity to study maternal exposure to drugs to anticipate response without exposure during a clinical trial, Gottlieb added.

In March, FDA also issued draft guidance on scientific and ethical considerations to aid study sponsors on the “judicious inclusion of pregnant women in clinical trials.”

“We don’t want regulatory uncertainty or lack of regulatory capacity in these areas to be a barrier to innovation and investment,” Gottlieb said.

Another step relates to the Women’s Health Technologies Strategically Coordinated Registry Network being established as part of FDA’s Medical Device Safety Action Plan—released in April—for the incorporation of data in device registries to better inform trial designs.

The registry network is intended to “provide more complete evidence in clinical areas that are unique to women, such as uterine fibroids, pelvic floor disorders, and female long-acting, reversible contraception and sterilization,” according to FDA.

The debate was focused on participation in cardiovascular clinical trials as this at least one area of clinical research in which there is under-enrollment of women, despite these types of conditions being relatively prevalent among women.

Further, cardiovascular disease has surpassed breast cancer as the leading cause of death among women, according to Gottlieb.

A new analysis on clinical studies of cardiovascular medications—conducted by FDA officials and published in the Journal of the American College of Cardiology last week—found that while women were “well represented in trials of drugs for hypertension and atrial fibrillation, and overrepresented for pulmonary arterial hypertension,” representation significantly dropped for trials of heart failure, coronary artery disease and acute coronary syndrome.

“Minimal gender differences in drug efficacy and safety profiles were observed,” the study authors added. “Given differences in pathophysiology, clinical presentation, and outcomes of cardiovascular disease in men and women, adequate participation of women is important to allow examination of possible gender differences in treatment response.”

内容来自：Regulatory Affairs Professionals Society (RAPS)

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