目前开展的真实世界研究(real world study,RWS)仍存在诸多局限性,而未能在卫生技术评估方面充分发挥应有的作用。因此,有必要改进传统RWS设计,以产生高质量的医学证据。策略之一是仿照随机对照试验(randomized controlled trial,RCT)...目前开展的真实世界研究(real world study,RWS)仍存在诸多局限性,而未能在卫生技术评估方面充分发挥应有的作用。因此,有必要改进传统RWS设计,以产生高质量的医学证据。策略之一是仿照随机对照试验(randomized controlled trial,RCT)开展观察性研究(又称RCT仿真或模拟研究),既借鉴了RCT设计优势,增强了因果推断的强度,又保留了RWS的代表性,可以作为打通药品上市前与上市后证据链的桥梁,以期为医疗决策提供合理的证据支撑。仿照RCT开展观察性研究分为两步:第一步,基于临床问题构建一个目标试验,参照目标试验关键特征构建相应的RWS方案(包括纳排标准、治疗策略、分配程序、随访、结局定义、因果对比和统计分析策略),特别注意减小模拟差异和控制相关偏倚;第二步,使用真实世界数据(real world data,RWD)按照既定研究方案进行数据分析,得到相应结果。展开更多
Objective To analyze clinical feature and information of medication to explore the risk signals of preparations containing Psoraleae Fructus(BGZP)related with hepatobiliary adverse drug reactions(ADR),in order to rein...Objective To analyze clinical feature and information of medication to explore the risk signals of preparations containing Psoraleae Fructus(BGZP)related with hepatobiliary adverse drug reactions(ADR),in order to reinforce pharmacovigilance.Methods A retrospective study was conducted based on hepatobiliary ADR related with BGZP from the China Adverse Drug Reaction Monitoring System in years from January 2012 to December 2016.Serious and general ADRs were analyzed and assessed.Results There were 355 cases of hepatobiliary ADR related to BGZP.Both the amount of cases and the proportion of serious ADR showed an increasing growth by years(P<0.05).It was found that 10.43%of 355 cases may be involved with irrational drug use,including overdose,repeated medication,and combination of multiple drugs.There were 190 cases which used BGZP(non-combination),and they were mainly for common in diseases caused by abnormal immune activation(accounting for 40.53%of the total cases).Especially at the age group with the most cases with age of 41–50 years,the cases associated with immunological diseases of female were obviously more than that of male(P<0.05).The latency of hepatobiliary ADR related to BGZP ranged from 1 to 386 days,and the median latency was 27.5 days,along with the range of cumulative dose(0.45–520.02 g)as well as the daily dose(0.09–2.64 g/d)after the conversion.Conclusions Cases of hepatobiliary ADR related to BGZP showed significant individual differences,and there was no correlation between drug usage duration and dosage and the occurrence of hepatobiliary ADR.It may be similar with idiosyncratic drug-induced liver injury,and recommended that BGZP should be used with more caution under monitoring liver function,especially in female patients with immunological diseases.展开更多
文摘目前开展的真实世界研究(real world study,RWS)仍存在诸多局限性,而未能在卫生技术评估方面充分发挥应有的作用。因此,有必要改进传统RWS设计,以产生高质量的医学证据。策略之一是仿照随机对照试验(randomized controlled trial,RCT)开展观察性研究(又称RCT仿真或模拟研究),既借鉴了RCT设计优势,增强了因果推断的强度,又保留了RWS的代表性,可以作为打通药品上市前与上市后证据链的桥梁,以期为医疗决策提供合理的证据支撑。仿照RCT开展观察性研究分为两步:第一步,基于临床问题构建一个目标试验,参照目标试验关键特征构建相应的RWS方案(包括纳排标准、治疗策略、分配程序、随访、结局定义、因果对比和统计分析策略),特别注意减小模拟差异和控制相关偏倚;第二步,使用真实世界数据(real world data,RWD)按照既定研究方案进行数据分析,得到相应结果。
基金Supported by the National Natural Science Foundation of China(No.81630100)Beijing Nova Program(No.Z181100006218001)Project of China PLA General Hospital(No.2019MBD-023)。
文摘Objective To analyze clinical feature and information of medication to explore the risk signals of preparations containing Psoraleae Fructus(BGZP)related with hepatobiliary adverse drug reactions(ADR),in order to reinforce pharmacovigilance.Methods A retrospective study was conducted based on hepatobiliary ADR related with BGZP from the China Adverse Drug Reaction Monitoring System in years from January 2012 to December 2016.Serious and general ADRs were analyzed and assessed.Results There were 355 cases of hepatobiliary ADR related to BGZP.Both the amount of cases and the proportion of serious ADR showed an increasing growth by years(P<0.05).It was found that 10.43%of 355 cases may be involved with irrational drug use,including overdose,repeated medication,and combination of multiple drugs.There were 190 cases which used BGZP(non-combination),and they were mainly for common in diseases caused by abnormal immune activation(accounting for 40.53%of the total cases).Especially at the age group with the most cases with age of 41–50 years,the cases associated with immunological diseases of female were obviously more than that of male(P<0.05).The latency of hepatobiliary ADR related to BGZP ranged from 1 to 386 days,and the median latency was 27.5 days,along with the range of cumulative dose(0.45–520.02 g)as well as the daily dose(0.09–2.64 g/d)after the conversion.Conclusions Cases of hepatobiliary ADR related to BGZP showed significant individual differences,and there was no correlation between drug usage duration and dosage and the occurrence of hepatobiliary ADR.It may be similar with idiosyncratic drug-induced liver injury,and recommended that BGZP should be used with more caution under monitoring liver function,especially in female patients with immunological diseases.
