Torrey Cope关注了FDA最近强调的“仅在中国进行的试验”所面临的挑战。他探察了FDA的顾虑：仅在中国进行的临床试验无法证明这种药物是否适合美国人群，并且根据美国境外试验数据探讨了美国审批的监管请求。

Torrey Cope looks at the recent FDA emphasis on the challenges of a China-only trial strategy. He explores the FDA concern that a China-only clinical trial will be unable to prove that a drug is suitable for the U.S. population, and considers the regulatory requirements for U.S. approvals based on ex-U.S. trial data.

最近，仅在中国进行的临床试验在美国申请批准的事态发展，让许多中国制药公司好奇仅在中国进行的试验是否可能获得美国批准。事实上，可能获批，但这些申请者现在面临着许多挑战。

Recent developments relating to the use of China-only clinical trials for seeking approval in the U.S. have left many Chinese pharma companies wondering whether it’s possible to use China-only trials to get U.S. approval. It is, but those seeking to do so now face a number of challenges.

广义上，美国食品和药物管理局（FDA）希望美国境外数据适用于美国人口和医疗实践。此外，FDA还需要考虑可能会影响一个群体的数据是否适用于另一个群体的各种内在（遗传和生理）和外在（文化和环境）因素。

At the broadest level, the U.S. Food and Drug Administration (FDA) wants to be satisfied that ex-U.S. data are applicable to the U.S. population and medical practice. Among other things, this generally requires consideration of a variety of intrinsic (genetic and physiologic) and extrinsic (cultural and environmental) factors that may influence whether data from one population are applicable to another.

最近，FDA对仅在中国进行的试验数据的态度发生了一些变化。2019年，FDA肿瘤中心负责人理查德·帕兹杜尔博士在AACR会议上发表评论，建议根据实际数据的质量，对仅在中国产生的临床数据的申请持开放态度。

We have recently seen evidence of some changes in the FDA’s attitude towards data from China-only trials. In 2019, the head of the FDA’s oncology centre, Dr. Richard Pazdur, made comments at the AACR conference which suggested an openness to applications with clinical data generated only in China, depending on the quality of the actual data

然而，今年早些时候，FDA在审查一项治疗非小细胞肺癌的药物申请时，就美国批准仅在中国进行的试验方法所面临的挑战，发表了一些声明。这些声明侧重于考虑特定类别和产品的试验设计问题，例如，具有适用于美国人口和医疗实践的对照组和终点，以及与美国患者相比，外国患者有哪些已知和未知特征的潜在差异。

Earlier this year, however, the FDA made statements about the challenges of a China-only approach to U.S. approval in connection with its review of an application for a drug to treat non-small cell lung cancer. These statements focused on the need for consideration of class- and product-specific trial design issues, such as having a comparator arm and end points that are applicable to the U.S. population and medical practice, as well as potential differences in known and unknown characteristics of foreign patients compared to U.S. patients.

非小细胞肺癌药物的例子表明，一旦FDA开始考虑特定产品的特定申请，仅在中国进行的试验数据问题会变得多么复杂，也显示了FDA对仅在中国进行的临床试验数据的看法是如何迅速转变的。在上述例子中，FDA指出，非小细胞肺癌的治疗模式在2019年（帕兹杜尔博士最初发表声明的时间）和2022年（FDA实际开始考虑该领域新疗法的申请）之间发生了巨大变化。

The non-small cell lung cancer drug example demonstrates how complicated the issue of data from China-only trials can become once the FDA begins to consider a specific application for a specific product. It also shows how the FDA’s views on the acceptability of data from China-only clinical trials can change quite quickly. In the above example, the FDA pointed out that the treatment paradigm for non-small cell lung cancer had changed dramatically between 2019 — the time of Dr. Pazdur’s original statement — and 2022 when the FDA actually came to consider an application for a new treatment in that area.

总的来说，在FDA强调美国批准的试验应该反映美国人口种族多样性的情况下，人们提出了这些担忧，并表示强烈希望将多区域临床试验（MRCT）作为全球药物开发的新标准。FDA强调，我们正在推动更多的多样性和更多的MRCT，而单国试验本身就是一种“退步”。

Generally, these concerns are being raised in a climate where the FDA has strongly emphasised that trials for U.S. approval should reflect the diversity of ethnic subgroups in the U.S. population, and has expressed strong interest in making multiregional clinical trials (MRCTs) the new standard for global drug development. The FDA has stressed that because of the push for more diversity and more MRCTs, single country trials are, in its own words, ‘a step backward.’

在这种情况下，我们应该预料到，仅在中国进行的临床试验将会受到美国逐类和逐个产品的审查。防止拒绝的最好方法是在整个研发过程中寻求并仔细考虑FDA的意见，公司还可以通过确保审查相关的法律标准、FDA指南和先例来降低风险。

In such climate, we should expect that applications for U.S. approval based on China-only trials will be scrutinized on a class-by-class and a product-by-product basis. The best way to guard against rejection is to seek and carefully consider FDA input throughout the development process. Companies can also mitigate risk by ensuring they review the relevant legal standards, FDA guidance, and precedents.