The American College of Rheumatology and the US Food and Drug Administration co-sponsored a public meeting in May 2022 about challenges in the clinical development of drugs for rheumatoid arthritis (RA) and psoriatic arthritis (PsA), focusing on innovative clinical trial designs, outcome measures, and data collection methods. Recommendations include early dose-ranging studies and use of active comparators. Challenges and opportunities in assessing long-term safety by leveraging real-world data from electronic health records (EHRs) and claims data are discussed, along with insights from European registries and the evolving role of real-world evidence and artificial intelligence in regulatory evaluations. Endpoints for assessing disease activity and outcome measures used in RA and PsA trials are explored, emphasizing challenges in defining remission, assessing clinical response, and evaluating structural progression. The need for outcome measures that better reflect treatment targets and the potential of advanced imaging in future trials are highlighted. Challenges with placebo-controlled trials in RA are discussed and use of non-inferiority clinical trial design, in which new drugs are evaluated with active comparators, is proposed. Pragmatic trials in RA and PsA, employing decentralized approaches, are highlighted for their real-world relevance and administrative efficiencies. Strategies for identifying at-risk populations for RA and the challenges of using EHRs and insurance claims data in drug development are discussed. Registry data and digital health technologies show promise in bridging the gap between clinical trials and real-world effectiveness.

中文翻译：

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美国风湿病学会与食品药品管理局峰会：2022 年 5 月 17-18 日会议摘要


美国风湿病学会和美国食品药品监督管理局于 2022 年 5 月共同主办了一次公开会议，讨论类风湿关节炎 (RA) 和银屑病关节炎 (PsA) 药物临床开发的挑战，重点关注创新的临床试验设计、结果措施和数据收集方法。建议包括早期剂量范围研究和使用活性比较剂。讨论了利用电子健康记录 (EHR) 和索赔数据中的真实数据来评估长期安全性的挑战和机遇，以及来自欧洲登记处的见解以及真实世界证据和人工智能在监管评估中不断变化的作用。探讨了 RA 和 PsA 试验中使用的评估疾病活动和结果指标的终点，强调定义缓解、评估临床反应和评估结构进展方面的挑战。强调需要更好地反映治疗目标的结果测量以及先进成像在未来试验中的潜力。讨论了 RA 中安慰剂对照试验的挑战，并提出了使用非劣效性临床试验设计，其中使用活性比较药物来评估新药。 RA 和 PsA 的务实试验采用分散方法，因其现实世界的相关性和管理效率而受到重视。讨论了识别 RA 高危人群的策略以及在药物开发中使用 EHR 和保险索赔数据的挑战。注册数据和数字健康技术有望缩小临床试验与现实世界有效性之间的差距。