Aligning approaches to rheumatoid arthritis drug development: a comparison of FDA and EMA guidance.

We examined the EMA's Guidelines on Clinical Trials of Medicinal Products for the Treatment of rheumatoid arthritis and the FDA's draft guidance rheumatoid arthritis Development of Drugs for Treatment, together with relevant cross-indication FDA guidelines and labelling decisions in rheumatoid arthritis in the last two decades.

我们检查了EMA关于类风湿性关节炎治疗药物临床试验的指南和FDA的类风湿性关节炎治疗药物开发的草案指导，以及过去二十年中相关的跨适应症FDA指南和类风湿性关节炎的标签决策。

The key aspects of trial design, including study populations, endpoints, safety requirements, and statistical considerations were analysed.

对试验设计的关键方面进行了分析，包括研究人群、终点、安全要求和统计考虑因素。

The findings were reviewed and interpreted by a panel of rheumatologists, who proposed strategies for aligning regulatory expectations.

这些发现由一组风湿病学家审查并提出了解读，他们建议了与监管预期保持一致的策略。

The FDA and EMA agree on several core principles such as the use of composite clinical endpoints, validated patient-reported outcomes, the importance of preventing structural joint damage, need for adequate long-term safety exposure, and limits on placebo duration.

FDA和EMA在几个核心原则上达成一致，例如使用复合临床终点、验证患者报告的结果、预防结构性关节损伤的重要性、需要充分的长期安全性暴露，以及对安慰剂持续时间的限制。

The EMA, however, favours remission or low disease activity as the preferred primary endpoints, generally requires two pivotal trials in distinct rheumatoid arthritis populations, and background methotrexate unless contraindicated.

然而，EMA更倾向于将缓解或低疾病活动度作为首选的主要终点，通常要求在不同的类风湿性关节炎人群中进行两项关键试验，并且除非有禁忌症，否则需要使用背景甲氨蝶呤。

The FDA offers more flexibility in primary endpoint selection, allows for a single pivotal trial if supported by strong confirmatory evidence, and places greater emphasis on early dose-response characterisation.

美国食品药品监督管理局（FDA）在主要终点选择上提供了更多的灵活性，如果得到强有力的确认性证据支持，允许仅进行一项关键试验，并且更加重视早期剂量反应特征的描述。

Greater alignment between FDA and EMA requirements could help reduce duplication, streamline global data interpretation, and speed patient access to effective rheumatoid arthritis therapies globally.

FDA与欧洲药品管理局（EMA）要求之间的更大一致性可能有助于减少重复工作，简化全球数据解读，并加快患者全球范围内有效类风湿关节炎治疗药物的获取。