特应性皮炎治疗药物临床试验设计的关键技术考虑  

Consideration on the key techniques of clinical trial design for drugs for the treatment of atopic dermatitis

作　　者：葛玉梅 杨靖怡 钱思源 翟云 赵聪 韵文萍 谢松梅 GE Yu-mei;YANG Jing-yi;QIAN Si-yuan;ZHAI Yun;ZHAO Cong;YUN Wen-ping;XIE Song-mei(Center for Drug Evaluation,National Medical Products Administration,Beijing 100022,China)

机构地区：[1]国家药品监督管理局药品审评中心,北京100022

出　　处：《中国新药杂志》2023年第19期1923-1929,共7页Chinese Journal of New Drugs

摘　　要：目的:探讨特应性皮炎治疗药物临床试验设计的技术考虑。方法:通过梳理近年来国内外特应性皮炎治疗药物临床研发情况,调研国内外特应性皮炎治疗药物技术指南进展,基于我国研发和审评的实践经验,形成相关技术考虑。结果与结论:特应性皮炎治疗药物临床试验设计应根据疾病特点、药物特性、研究目的制定整体临床研发计划。应在有前期研究证据支持并能确保受试者安全的前提下,尽早开展儿童患者临床试验。临床试验设计应选择适宜的研究人群,合理设置对照药、疗效和安全性指标、研究周期等一系列关键要素,以助力此类药物科学研发。研究人群定位应基于药物作用机制和药效作用强度选择对应严重程度的患者,疗效考察应全面评估患者皮损、症状、生活质量等改善情况,并关注用于儿童患者以及长期治疗时的疗效和安全性等。Objective:To explore the consideration on the key techniques of clinical trial design of drugs for the treatment of atopic dermatitis.Methods:By summarizing the clinical research status of drugs for the treatment of atopic dermatitis,and investigating the progress of technical guidelines for the drugs in China and abroad in recent years,relevant technical considerations are proposed based on the China,s experience in Drug R&D and the process of review.Results and Conclusion:The design of whole clinical trial of drugs treating atopic dermatitis should be based on the characteristics of the disease and investigational drug,the purpose of the study.Clinical trials for pediatric patients should be conducted as early as possible under the premise of prior research evidence and ensuring the safety of subjects.The appropriate study population should be selected,and a series of key elements,such as control drugs,efficacy and safety indicators,study period,etc.,should be reasonably set up in the clinical trial design,in order to assist the scientific research and the development of the drugs.The positioning of the study population should be based on the drug action mechanism and drug effect intensity,and the patients with corresponding severity should be selected.The efficacy of the study should be comprehensively evaluated to assess the improvement of skin lesions,symptoms,quality of life,etc.,and attention should be paid to the efficacy and safety of the study when used in pediatric patients as well as in long⁃term treatment.

关 键 词：特应性皮炎 临床试验设计 疗效指标 评价量表 获益⁃风险评估 

分 类 号：R976[医药卫生—药品]