Objective To study the threshold effect of export trade on internal and external R&D investment in China’s pharmaceutical industry,and to provide reference for some pharmaceutical enterprises to improve the inves...Objective To study the threshold effect of export trade on internal and external R&D investment in China’s pharmaceutical industry,and to provide reference for some pharmaceutical enterprises to improve the investment.Methods The panel data of pharmaceutical industry in 25 provinces and cities in China from 2009 to 2019 were selected to conduct empirical analysis by establishing a threshold regression model,and a better export trade interval was obtained.Results and Conclusion There is a threshold value for the effect of new product export on both internal and external R&D expenditures,and the threshold values are 845.2788 million yuan and 318.4198 million yuan,respectively.There is a single threshold effect of export trade on both internal and external R&D investment in China’s pharmaceutical industry,and the effect of export trade on internal and external R&D investment changes from negative to positive as the export trade develops from low to high.展开更多
Objective To study the content of China’s guiding principles on multiplicity issues in clinical trials,and to provide reference for the revision of China’s relevant guiding principles.Methods Based on ICH E9,the sim...Objective To study the content of China’s guiding principles on multiplicity issues in clinical trials,and to provide reference for the revision of China’s relevant guiding principles.Methods Based on ICH E9,the similarities and differences of the guiding principles of US Food and Drug Administration(FDA),European Medicines Agency(EMA),and National Medical Products Administration(NMPA)on the multiplicity issues in clinical trials were compared one by one.Results and Conclusion In general,NMPA guidelines are based on ICH E9,but in detail,the guidelines of FDA and EMA focus differently on the multiplicity issues.Therefore,NMPA guidelines need to be detailed and comprehensive.NMPA guidelines can be refined by referring to foreign guidelines to improve the practical guiding significance for clinical research and promote the level of domestic clinical trials in line with international standards.展开更多
Objective To analyze the characteristics of breakthrough therapy designation(BTD)and its implementation in China,and to provide reference for the optimization of BTD system.Methods A comparative research method was us...Objective To analyze the characteristics of breakthrough therapy designation(BTD)and its implementation in China,and to provide reference for the optimization of BTD system.Methods A comparative research method was used to study the content and implementation effect of BTD system in China and the relevant policies and implementation of the same procedures of drug regulatory authorities in the United States,Japan and the European Union.Then,the differences in policies and implementation results among these countries were analyzed to provide suggestions for the implementation and optimization of this system in China.Results and Conclusion China’s BTD system is implemented late and a small number of drugs has been approved.At the same time,there are problems such as insufficient guidance and communication from the agency to applicants,a broad application condition,single review mode,and lack of full-time personnel.Both the agencies and the applicants have limited experience due to the short implementation time of BTD system in China.There are still some problems despite we have learned a lot from the experience of other drug regulatory agencies.Therefore,based on our national conditions,we should strengthen the guidance of evaluation agency to applicants,optimize the eligibility criteria of BTD system,introduce the rolling review,and increase the number of professional liaisons,which can accelerate the development and marketing process of drugs with obvious clinical value,and finally to address unmet medical need.展开更多
Objective To discuss the problems existing in the compensation of Chinese clinical trial participants and propose some suggestions for improving their rights.Methods The literature related to the participants’right t...Objective To discuss the problems existing in the compensation of Chinese clinical trial participants and propose some suggestions for improving their rights.Methods The literature related to the participants’right to compensation at home and abroad was searched to study the inadequacy of the compensation right for clinical trial participants in China from four aspects:insurance system,principle of attribution,legal relationship and compensation regulations.Then,some suggestions to improve the participants’right to compensation were proposed.Results and Conclusion China lacks clear legal norms for participants’right to compensation.There are problems such as unclear insurance rules and compensation rules,unclear contractual relationships between parties to clinical trials,and no laws and regulations to rely on for attribution and compensation.China should issue regulatory guidelines related to the right to compensation of participants in clinical trials,so that all parties in clinical trials can have rules to follow if there is the occurrence of injury,which can better protect the rights and interests of the participants.展开更多
目的探讨机器学习算法构建腹部手术术后脓毒症患者死亡风险预测模型的可行性。方法采用病例-对照研究设计方案,从公共重症监护医学信息数据库(Medical Information Mart for Intensive CareⅣ,MIMIC-Ⅳv1.0)中筛选出行腹部手术后发生脓...目的探讨机器学习算法构建腹部手术术后脓毒症患者死亡风险预测模型的可行性。方法采用病例-对照研究设计方案,从公共重症监护医学信息数据库(Medical Information Mart for Intensive CareⅣ,MIMIC-Ⅳv1.0)中筛选出行腹部手术后发生脓毒症的患者,研究终点事件定义为患者入院后90 d内死亡。根据死亡与否将数据集随机拆分为训练数据集(70%)与测试数据集(30%),在训练数据集上基于Logistic回归(logistic regression,LR)、梯度提升树(gradient boosting decision tree,GBDT)、随机森林(random forest,RF)、支持向量机(support vector machine,SVM)和自适应提升(adaptive boosting,AdaBoost)算法构建预测死亡风险模型;在测试数据集上通过受试者工作曲线(receiver operating characteristic curve,ROC)和曲线下面积(area under the ROC curve,AUC)、敏感性、特异性、阳性预测值、阴性预测值、F1分数和准确率来评估模型效能。结果最终986例患者纳入本研究,其中251例(25.5%)患者入院后90 d内死亡,LR、GBDT、RF、SVM及AdaBoost模型的AUC依次为0.852、0.903、0.921、0.940和0.906,其中SVM的AUC最高,预测性能更好,而LR模型效能最差。结论基于GBDT、RF、SVM及AdaBoost这4种算法建立的腹部手术术后脓毒症死亡率预测模型的效能优于传统的LR模型,可能有助于临床决策,改善不良结局。展开更多
基金Research on Innovation and Development Strategy of Pharmaceutical Industry in Liaoning Province(2020lslktyb-095).
文摘Objective To study the threshold effect of export trade on internal and external R&D investment in China’s pharmaceutical industry,and to provide reference for some pharmaceutical enterprises to improve the investment.Methods The panel data of pharmaceutical industry in 25 provinces and cities in China from 2009 to 2019 were selected to conduct empirical analysis by establishing a threshold regression model,and a better export trade interval was obtained.Results and Conclusion There is a threshold value for the effect of new product export on both internal and external R&D expenditures,and the threshold values are 845.2788 million yuan and 318.4198 million yuan,respectively.There is a single threshold effect of export trade on both internal and external R&D investment in China’s pharmaceutical industry,and the effect of export trade on internal and external R&D investment changes from negative to positive as the export trade develops from low to high.
基金supported by the Special Foundation of Research Institute of Drug Regulatory Science,Shenyang Pharmaceutical University(2021jgkx004).
文摘Objective To study the content of China’s guiding principles on multiplicity issues in clinical trials,and to provide reference for the revision of China’s relevant guiding principles.Methods Based on ICH E9,the similarities and differences of the guiding principles of US Food and Drug Administration(FDA),European Medicines Agency(EMA),and National Medical Products Administration(NMPA)on the multiplicity issues in clinical trials were compared one by one.Results and Conclusion In general,NMPA guidelines are based on ICH E9,but in detail,the guidelines of FDA and EMA focus differently on the multiplicity issues.Therefore,NMPA guidelines need to be detailed and comprehensive.NMPA guidelines can be refined by referring to foreign guidelines to improve the practical guiding significance for clinical research and promote the level of domestic clinical trials in line with international standards.
基金Special Fund for Academy of Pharmaceutical Regulatory Sciences of Research Base for Drug Regulatory Science of National Medical Products Administration-Shenyang Pharmaceutical University(2021jgkx004).
文摘Objective To analyze the characteristics of breakthrough therapy designation(BTD)and its implementation in China,and to provide reference for the optimization of BTD system.Methods A comparative research method was used to study the content and implementation effect of BTD system in China and the relevant policies and implementation of the same procedures of drug regulatory authorities in the United States,Japan and the European Union.Then,the differences in policies and implementation results among these countries were analyzed to provide suggestions for the implementation and optimization of this system in China.Results and Conclusion China’s BTD system is implemented late and a small number of drugs has been approved.At the same time,there are problems such as insufficient guidance and communication from the agency to applicants,a broad application condition,single review mode,and lack of full-time personnel.Both the agencies and the applicants have limited experience due to the short implementation time of BTD system in China.There are still some problems despite we have learned a lot from the experience of other drug regulatory agencies.Therefore,based on our national conditions,we should strengthen the guidance of evaluation agency to applicants,optimize the eligibility criteria of BTD system,introduce the rolling review,and increase the number of professional liaisons,which can accelerate the development and marketing process of drugs with obvious clinical value,and finally to address unmet medical need.
基金Liaoning Pharmaceutical Industry Innovation and Development Strategy Research and Funding Project(2020lslktyb-095)National Medical Products Administration-Special Fund of Drug Regulatory Research Institute of Shenyang Pharmaceutical University(2021jgkx004)+1 种基金Shenzhen Maternity&Child Healthcare Hospital Science Foundation(2022(73))Shenzhen Health Economics Society Research Fund Project(202333).
文摘Objective To discuss the problems existing in the compensation of Chinese clinical trial participants and propose some suggestions for improving their rights.Methods The literature related to the participants’right to compensation at home and abroad was searched to study the inadequacy of the compensation right for clinical trial participants in China from four aspects:insurance system,principle of attribution,legal relationship and compensation regulations.Then,some suggestions to improve the participants’right to compensation were proposed.Results and Conclusion China lacks clear legal norms for participants’right to compensation.There are problems such as unclear insurance rules and compensation rules,unclear contractual relationships between parties to clinical trials,and no laws and regulations to rely on for attribution and compensation.China should issue regulatory guidelines related to the right to compensation of participants in clinical trials,so that all parties in clinical trials can have rules to follow if there is the occurrence of injury,which can better protect the rights and interests of the participants.
文摘目的探讨机器学习算法构建腹部手术术后脓毒症患者死亡风险预测模型的可行性。方法采用病例-对照研究设计方案,从公共重症监护医学信息数据库(Medical Information Mart for Intensive CareⅣ,MIMIC-Ⅳv1.0)中筛选出行腹部手术后发生脓毒症的患者,研究终点事件定义为患者入院后90 d内死亡。根据死亡与否将数据集随机拆分为训练数据集(70%)与测试数据集(30%),在训练数据集上基于Logistic回归(logistic regression,LR)、梯度提升树(gradient boosting decision tree,GBDT)、随机森林(random forest,RF)、支持向量机(support vector machine,SVM)和自适应提升(adaptive boosting,AdaBoost)算法构建预测死亡风险模型;在测试数据集上通过受试者工作曲线(receiver operating characteristic curve,ROC)和曲线下面积(area under the ROC curve,AUC)、敏感性、特异性、阳性预测值、阴性预测值、F1分数和准确率来评估模型效能。结果最终986例患者纳入本研究,其中251例(25.5%)患者入院后90 d内死亡,LR、GBDT、RF、SVM及AdaBoost模型的AUC依次为0.852、0.903、0.921、0.940和0.906,其中SVM的AUC最高,预测性能更好,而LR模型效能最差。结论基于GBDT、RF、SVM及AdaBoost这4种算法建立的腹部手术术后脓毒症死亡率预测模型的效能优于传统的LR模型,可能有助于临床决策,改善不良结局。
