2022年中国眼科药物临床试验进展

Analysis of the progress of clinical trials for ophthalmic drug in China,2022

目的:了解2022年我国眼科药物临床试验的进展,探讨其与2014~2021年的变化,为新药研发、临床试验的实施提供最新数据参考,为相关决策提供依据。方法:采用横断面研究,检索国家药品监督管理局“药物临床试验登记与信息公示平台”数据库,纳入公示日期为2022-01-01/12-31且适应证为眼病的眼科药物临床试验。描述眼科药物试验整体情况、适应证及试验分期及占比情况、实施效率、主要研究者的地域分布情况。结果:2022年我国眼科药物临床试验共公示55项,占所有临床试验的1.66%,呈稳步增长趋势,以化学药物占比最高为58.18%。开展试验最多的前三个适应证为年龄相关性黄斑变性、近视、干眼。开展基因治疗药物临床试验2项。7个药物品种开展≥2项临床试验,其中以硫酸阿托品、重组抗血管内皮生长因子(VEGF)人源化单克隆抗体开展数量最多,分别为7、5项。试验分期以Ⅰ期、Ⅲ期为主,分别占比36.36%、27.27%。2022年Ⅰ期试验启动耗时为2.72(0.77,3.47)mo,较2014~2021年的启动耗时3.87(3.00,6.30)mo显著缩短(Z=-2.630,P=0.009),BE、Ⅱ、Ⅲ、Ⅳ期与2014~2021年相比均无差异(P>0.05)。结论:2022年我国眼科药物临床试验开展的数量、实施效率均稳步增长。适应证以眼底病、近视和干眼为主。多数新药处于早期研发或临近上市状态。基因治疗药物崭露头角。

AIM:To explore the progress of clinical trials for ophthalmic drugs in China in 2022 and discuss its changes with 2014 to 2021,thus providing the latest data reference for the development of new drug and the implementation of clinical trials,and a basis for decision-making.METHODS:In this cross-sectional study,we retrieved the drug clinical trials registration and information disclosure platform of National Medical Products Administration database.Drug clinical trials for eye diseases registered from January 1 to December 31,2022 were included.Number (proportion) was used to describe the characteristics of clinical trials for ophthalmic drug,the indication,the trial phase,the efficacy and the geographical distribution.RESULTS:A total of 55 clinical trials for ophthalmic drug were included,which accounted for 1.66% of all clinical trials,showing a steady growth trend.Main drug type was chemical drugs with the highest proportion of 58.18% .The top three indications with the most clinical trials were age-related macular degeneration,myopia and dry eye.Two gene drugs emerged in 2022,and 7 drugs carried out ≥2 trials,of which atropine sulfate and recombinant anti-vascular endothelial growth factor (VEGF) humanized monoclonal antibody were the most (7 and 5 respectively).Most trials were in phase I and phase III stages,accounting for 36.36% and 27.27% respectively.The median start-up time of phase I trials in 2022 was 2.72 (0.77,3.47)mo,which was significantly shorter than 3.87 (3.00,6.30)mo of 2014~2021 ( Z = -2.630 ,P =0.009),and there were no significant differences between BE,phase II,III,IV comparing with 2014~2021 ( P >0.05).CONCLUSIONS:In 2022,the number and implementation efficiency of clinical trials for ophthalmic drugs in China increased steadily.The indications are mainly fundus disease,myopia and dry eye.Most new drugs are in the early stage of research and development or close to market.Gene therapy drugs began to emerge.