Objective To establish a pediatric rare disease catalog,analyze the current status of therapeutic drugs and their coverage of the medical insurance in China,and propose strategies to enhance drug accessibility.Methods Pediatric rare diseases were identified from China's two national rare disease catalogs combined with the EU Orphanet database,US FDA orphan drug database,and the Diagnosis and Treatment Standards for Rare Diseases in Children.We created a specialized drug catalog for pediatric rare diseases,then analyzed drug types(ATC classification),pricing,and medical insurance coverage using descriptive statistics based on Yaozhi.com drug bidding prices and the 2024 Drug of List National Basic Medical Insurance(NBMIDL).Drug affordability was assessed through annual treatment cost calculations.Results The national catalogs included 151 pediatric rare diseases(72.95%of listed conditions),spanning 13 disease systems.We identified 94 dedicated orphan drugs(by generic name)for these conditions,among which 43 were approved internationally but unavailable in China.The average unit price per package was 6 113.53 yuan.Overall NBMIDL coverage was 68.83%,but drugs priced above 7 000 yuan per unit had only 7.69%coverage.Annual treatment costs reached 4.54 million for laronidase(mucopolysaccharidosis).Conclusions Critical gaps persist in China's pediatric rare disease treatment landscape,including catalog deficiencies,inadequate coverage for high-cost drugs and insufficient domestic innovation.It is recommended to establish a list of orphan drugs for pediatric rare diseases,accelerate the import of foreign drugs and the local innovative drugs through policy incentives,optimizing medical insurance reimbursement mechanisms for pediatric rare disease drugs to comprehensively improve therapeutic accessibility.

Objective To establish a pediatric rare disease catalog,analyze the current status of therapeutic drugs and their coverage of the medical insurance in China,and propose strategies to enhance drug accessibility.Methods Pediatric rare diseases were identified from China's two national rare disease catalogs combined with the EU Orphanet database,US FDA orphan drug database,and the Diagnosis and Treatment Standards for Rare Diseases in Children.We created a specialized drug catalog for pediatric rare diseases,then analyzed drug types(ATC classification),pricing,and medical insurance coverage using descriptive statistics based on Yaozhi.com drug bidding prices and the 2024 Drug of List National Basic Medical Insurance(NBMIDL).Drug affordability was assessed through annual treatment cost calculations.Results The national catalogs included 151 pediatric rare diseases(72.95%of listed conditions),spanning 13 disease systems.We identified 94 dedicated orphan drugs(by generic name)for these conditions,among which 43 were approved internationally but unavailable in China.The average unit price per package was 6 113.53 yuan.Overall NBMIDL coverage was 68.83%,but drugs priced above 7 000 yuan per unit had only 7.69%coverage.Annual treatment costs reached 4.54 million for laronidase(mucopolysaccharidosis).Conclusions Critical gaps persist in China's pediatric rare disease treatment landscape,including catalog deficiencies,inadequate coverage for high-cost drugs and insufficient domestic innovation.It is recommended to establish a list of orphan drugs for pediatric rare diseases,accelerate the import of foreign drugs and the local innovative drugs through policy incentives,optimizing medical insurance reimbursement mechanisms for pediatric rare disease drugs to comprehensively improve therapeutic accessibility.

Objective:To explore the correlations of brain network functional connectivity (FC) alterations with cerebrospinal fluid (CSF) pathological biomarkers in patients with Alzheimer's disease (AD).Methods:A total of 39 patients with cognitive impairment, admitted to Department of Neurology, Drum Tower Hospital, Affiliated Hospital of Medical School, Nanjing University from January 2020 to December 2022 were recruited; 23 patients were with AD and 16 with non-AD. Clinical data were compared between the 2 groups. Resting-state functional MRI (rs-fMRI) data were collected, and FC differences between brain networks and FC differences within brain networks were compared by independent component analysis. Correlations of FC differences between brain networks and FC differences within brain networks with concentrations of β-amyloid protein 1-42 (Aβ 1-42) and Tau protein in CSF were analyzed. Results:Compared with the non-AD group, AD group had significantly lower Aβ 1-42 in CSF ( P<0.05). Compared with those in the non-AD group, FC alterations between the left frontoparietal network (lFPN) and anterior default mode network (aDMN) and between the visual network (VN) and posterior cingulate cortex (PCC), as well as FC alterations in lFPN, were significantly increased in AD group ( P<0.05). Compared with those in the non-AD group, FC alterations between lFPN and cerebellar network (CEN), and FC alterations in aDMN, sensorimotor network (SMN) and VN were significantly decreased in AD group ( P<0.05). In AD group, FC in SMN was positively correlated with total Tau and phosphorylated-Tau181 in CSF ( P<0.05); FC between VN and PCC was positively correlated with total Tau in CSF ( P<0.05). CSF Aβ 1-42 was positively correlated with FC alterations in aDMN and VN, but negatively correlated with FC in FPN ( P<0.05). Conclusion:In AD patients, characteristic changes in FC within and between multiple brain networks are noted, which are related to changes of Tau protein and Aβ 1-42 in CSF.

Objective:Clarify the correlation between the Centralized Drug Volume-based Purchasing Policy,changes in physical&mental health status,and health inequalities among the older adults,as well as exploring whether this correlation differs between urban and rural areas so as to determine the role played by the total cost of healthcare.Methods:Based on CFPS 2018 and 2020,DID and PSM-DID were used to verify the impact of the Centralized Drug Volume-based Purchasing Policy on physical&mental health status and health inequalities of the elderly in urban and rural China.Three-step method and Sobel test were used to verify the mediating effect of total medical costs.Results:The pilot effect of the Centralized Drug Volume-based Purchasing Policy was first realized among the urban elderly population,promoting their physical and mental health through the intermediary mechanism of reducing the total medical costs,but for the time being did not show a significant correlation with health inequalities within this group.Among the rural elderly population,none of the three dependent variables was significant(P＞0.1).Conclusion:The underlying logic of the Centralized Drug Volume-based Purchasing Policy is right,but its future reform should focus on the rural elderly group,and consider the fairness of the effect within the group and between urban and rural areas,so as to improve the health level of the whole society in the longer term.

Objective:Clarify the correlation between the Centralized Drug Volume-based Purchasing Policy,changes in physical&mental health status,and health inequalities among the older adults,as well as exploring whether this correlation differs between urban and rural areas so as to determine the role played by the total cost of healthcare.Methods:Based on CFPS 2018 and 2020,DID and PSM-DID were used to verify the impact of the Centralized Drug Volume-based Purchasing Policy on physical&mental health status and health inequalities of the elderly in urban and rural China.Three-step method and Sobel test were used to verify the mediating effect of total medical costs.Results:The pilot effect of the Centralized Drug Volume-based Purchasing Policy was first realized among the urban elderly population,promoting their physical and mental health through the intermediary mechanism of reducing the total medical costs,but for the time being did not show a significant correlation with health inequalities within this group.Among the rural elderly population,none of the three dependent variables was significant(P＞0.1).Conclusion:The underlying logic of the Centralized Drug Volume-based Purchasing Policy is right,but its future reform should focus on the rural elderly group,and consider the fairness of the effect within the group and between urban and rural areas,so as to improve the health level of the whole society in the longer term.

Objective:Clarify the correlation between the Centralized Drug Volume-based Purchasing Policy,changes in physical&mental health status,and health inequalities among the older adults,as well as exploring whether this correlation differs between urban and rural areas so as to determine the role played by the total cost of healthcare.Methods:Based on CFPS 2018 and 2020,DID and PSM-DID were used to verify the impact of the Centralized Drug Volume-based Purchasing Policy on physical&mental health status and health inequalities of the elderly in urban and rural China.Three-step method and Sobel test were used to verify the mediating effect of total medical costs.Results:The pilot effect of the Centralized Drug Volume-based Purchasing Policy was first realized among the urban elderly population,promoting their physical and mental health through the intermediary mechanism of reducing the total medical costs,but for the time being did not show a significant correlation with health inequalities within this group.Among the rural elderly population,none of the three dependent variables was significant(P＞0.1).Conclusion:The underlying logic of the Centralized Drug Volume-based Purchasing Policy is right,but its future reform should focus on the rural elderly group,and consider the fairness of the effect within the group and between urban and rural areas,so as to improve the health level of the whole society in the longer term.

Objective:Clarify the correlation between the Centralized Drug Volume-based Purchasing Policy,changes in physical&mental health status,and health inequalities among the older adults,as well as exploring whether this correlation differs between urban and rural areas so as to determine the role played by the total cost of healthcare.Methods:Based on CFPS 2018 and 2020,DID and PSM-DID were used to verify the impact of the Centralized Drug Volume-based Purchasing Policy on physical&mental health status and health inequalities of the elderly in urban and rural China.Three-step method and Sobel test were used to verify the mediating effect of total medical costs.Results:The pilot effect of the Centralized Drug Volume-based Purchasing Policy was first realized among the urban elderly population,promoting their physical and mental health through the intermediary mechanism of reducing the total medical costs,but for the time being did not show a significant correlation with health inequalities within this group.Among the rural elderly population,none of the three dependent variables was significant(P＞0.1).Conclusion:The underlying logic of the Centralized Drug Volume-based Purchasing Policy is right,but its future reform should focus on the rural elderly group,and consider the fairness of the effect within the group and between urban and rural areas,so as to improve the health level of the whole society in the longer term.

Objective:Clarify the correlation between the Centralized Drug Volume-based Purchasing Policy,changes in physical&mental health status,and health inequalities among the older adults,as well as exploring whether this correlation differs between urban and rural areas so as to determine the role played by the total cost of healthcare.Methods:Based on CFPS 2018 and 2020,DID and PSM-DID were used to verify the impact of the Centralized Drug Volume-based Purchasing Policy on physical&mental health status and health inequalities of the elderly in urban and rural China.Three-step method and Sobel test were used to verify the mediating effect of total medical costs.Results:The pilot effect of the Centralized Drug Volume-based Purchasing Policy was first realized among the urban elderly population,promoting their physical and mental health through the intermediary mechanism of reducing the total medical costs,but for the time being did not show a significant correlation with health inequalities within this group.Among the rural elderly population,none of the three dependent variables was significant(P＞0.1).Conclusion:The underlying logic of the Centralized Drug Volume-based Purchasing Policy is right,but its future reform should focus on the rural elderly group,and consider the fairness of the effect within the group and between urban and rural areas,so as to improve the health level of the whole society in the longer term.

Objective:Clarify the correlation between the Centralized Drug Volume-based Purchasing Policy,changes in physical&mental health status,and health inequalities among the older adults,as well as exploring whether this correlation differs between urban and rural areas so as to determine the role played by the total cost of healthcare.Methods:Based on CFPS 2018 and 2020,DID and PSM-DID were used to verify the impact of the Centralized Drug Volume-based Purchasing Policy on physical&mental health status and health inequalities of the elderly in urban and rural China.Three-step method and Sobel test were used to verify the mediating effect of total medical costs.Results:The pilot effect of the Centralized Drug Volume-based Purchasing Policy was first realized among the urban elderly population,promoting their physical and mental health through the intermediary mechanism of reducing the total medical costs,but for the time being did not show a significant correlation with health inequalities within this group.Among the rural elderly population,none of the three dependent variables was significant(P＞0.1).Conclusion:The underlying logic of the Centralized Drug Volume-based Purchasing Policy is right,but its future reform should focus on the rural elderly group,and consider the fairness of the effect within the group and between urban and rural areas,so as to improve the health level of the whole society in the longer term.

Objective:Clarify the correlation between the Centralized Drug Volume-based Purchasing Policy,changes in physical&mental health status,and health inequalities among the older adults,as well as exploring whether this correlation differs between urban and rural areas so as to determine the role played by the total cost of healthcare.Methods:Based on CFPS 2018 and 2020,DID and PSM-DID were used to verify the impact of the Centralized Drug Volume-based Purchasing Policy on physical&mental health status and health inequalities of the elderly in urban and rural China.Three-step method and Sobel test were used to verify the mediating effect of total medical costs.Results:The pilot effect of the Centralized Drug Volume-based Purchasing Policy was first realized among the urban elderly population,promoting their physical and mental health through the intermediary mechanism of reducing the total medical costs,but for the time being did not show a significant correlation with health inequalities within this group.Among the rural elderly population,none of the three dependent variables was significant(P＞0.1).Conclusion:The underlying logic of the Centralized Drug Volume-based Purchasing Policy is right,but its future reform should focus on the rural elderly group,and consider the fairness of the effect within the group and between urban and rural areas,so as to improve the health level of the whole society in the longer term.