ObjectiveTo learn the development and implementation of orphan drug policies, in order to provide decision-making references for the establishment of orphan drug policy according with China's national conditions. MethodsWe electronically searched databases including CBM, CNKI, VIP, EMbase, PubMed, Web of Knowledge, National Library of Medicine, CRD database, The Campbell Library, The Cochrane Library and the drug administration websites of USA, Canada, UK, Ireland, the Netherlands, Germany, Spain, France, Australia, New Zealand, China, India, South Korea, Japan, and South Africa to collect studies about orphan drug policy. The search date was up to February 2014. Two reviewers independently screened literature, and extracted data. Then, all included orphan drug policies were summarized and a comparative analysis was performed. ResultsA total of 110 studies were included. USA, Singapore, Japan, Australia, European Union, Chinese Taiwan and South Korea had introduced orphan drugs incentive policies. South Africa, India, Canada, New Zealand and Chinese Hongkong were producing orphan drugs policy frameworks. The main items of orphan drug policy included marketing exclusivity, tax incentives, technical assistance, grant funding, expedite approval process and prolong re-evaluated time. ConclusionIn mainland China, there is no orphan disease management policy. China should establish specific organization and working procedures, promote orphan drug policy related legislative work, clarify the definition and prevalence of orphan diseases, provide incentive mechanism to promote the research and development of orphan drugs, provide enterprises to develop compensation mechanism to safeguard the rights and interests of patients, as well as establish patients register network platform to track the processes of the diseases.
ObjectiveTo systematically review the medication adherence in children with tic disorder to assist in the selection of clinical treatment options and enhance the efficacy of medications for tic disorder.MethodsDatabases including Medline (Ovid), EMbase (Ovid), The Cochrane Library, PsycINFO (EBSCOhost), CINAHL Plus (EBSCOhost), CNKI, WanFang Data and VIP were searched from inception to August 2020, and original studies on medication adherence in children with tic disorder were included. Two researchers independently screened literature, extracted data on the definition of compliance, compliance rate, and factors affecting compliance, and evaluated risk bias of included studies. Systematic review was performed to analyze the status of medication adherence in children with tic disorder.ResultsA total of 12 studies were included, involving seven randomized controlled trials, two case series studies, and three cross-sectional studies. Most studies failed to specify the definition of compliance. The results of cross-sectional studies showed that the proportion of children with good medication compliance was 29.3% to 47.1%. The first-line treatment drugs, tiapride, risperidone, aripiprazole, and clonidine, had relatively good adherence. Medication adherence was affected by drug factors, patient and family factors, and environmental factors.ConclusionsThe adherence rate of medications for tic disorder varies between studies. Few studies have analyzed the factors that affect medication adherence for tic disorder, and some influencing factors are controversial. The first-line treatment drugs, tiapride, risperidone, aripiprazole, and clonidine, have high medication adherence and are recommended for clinical use.
Objective We aimed to develop a self-management assessment scale for children with epilepsy and test its reliability and validity. Methods A research group was established, and the items were revised through literature review, group discussion and pre-investigation, and 280 patients with epilepsy in children were included, and the reliability and validity of the scale were tested. Results 28 items in 4 dimensions were developed to form the scale, namely, knowledge and belief of diseases and medication, compliance of medication and treatment, self-efficacy of medication and obstacles of medication. Confirmatory factor analysis extracted four common factors with characteristic roots greater than 1, and the cumulative variance explanation rate was 65.639%. The factor load of all items is > 0.5. The overall Cronbach’s alpha is 0.880, and the coefficients in seven measurement dimensions are all greater than 0.8. Conclusion The self-management assessment scale for children’s epilepsy drugs has good reliability and validity, and can provide a measuring tool for the drug management of children’s epilepsy diseases.
Objective To compare the 2009 edition national essential medicine list (part of basic medicine and heath institution ) in China and WHO model list of essential medicines for children update in 2010, so as to provide the evidence for model list of essential medicines for children of China. Methods We compared the diference in composition of content, the categories, speciic drugs, formulation, and introduction and sign of list by descriptive analysis. Results (1) WHO model list of essential medicines for children were comprised with core and complimentary lists, which were not in the 2009 edition national essential medicine list of China; (2) The 2009 edition essential medicine lists of China included 20 categories in WHO model list of essential medicines for children ,while lacked of antineoplastic agents, blood products, disinfectants, peritoneal dialysis luid and speciic medicines for neonatal care; (3) he average conincidence rate with WHO model list of essential medicines for children of the same drugs was 52.61%. here were 15 categories in the interval of 20%-80%, which accounted for 75%. he average conincidence rate with 2009 edition national essential medicine list of China was 44.19%. here were 11 categories in the interval of 20%-50%,which accounted for 55%. the same drugs of regulate water, electrolyte and acid-base balance in the two list accounted for more than 80% of WHO model list of essential medicines for children. Drugs of ear, nose and throat were totally diferent. (4) he 2009 edition essential medicine list of China didn’t have speciication and sign of drug for children, age limitation, instruction of list, suitable drug formulations for children such as suspension, syrup, drops, granules, scored tablets, etc. Conclusion 2009 edition national essential medicine list of China can’t meet the demand of children. WHO model list of essential medicines for children which bases on global burden of disease, can not be copied into China. We suggest to reference fromWHO model list of essential medicines for children to carry out selecting essential medicines for children in China, formulating essential medicine list for children in China which bases on burden of children disease and clinical demand in China to improve rationality of drugs in children.
Objective To assess the global situation of off-label drug use in outpatient children.Methods A search was conducted in PubMed, EMbase, CBM, CNKI and VIP for collecting studies on off-label drug use in outpatient children aged 0 to 18 years, and the publication language was limited to English and Chinese. The quality assessment was based on Crombie Criteria for cross-sectional studies, and the descriptive analysis was performed to analyze the incidence of off-label drug use at all levels of medical institutes and different age groups, the common types of off-label used drugs, and the constitution ratio of off-label use drugs. Results A total of 20 cross-sectional studies were included, involving 1 933 760 children and 5 262 573 prescriptions. The results of descriptive analysis showed: a) The incidence of off-label drug use in outpatient children in primary care and tertiary care institutes were 19.5% (IQR 13.5%, 24.5%) and 26.0% (IQR 24.0%, 35.0%), respectively; b) The primary studies reported that neonate, infant and adolescent had the highest incidence of off-label drug use, while the other children had the lowest incidence; c) Drugs for respiratory system, anti-infection, sensory organs, and dermatosis were listed as the top 5 frequently used off-label drugs by more than half of the studies; d) The studies with issue of “lack of instruction for children” showed it was exactly the type with highest incidence of off-label use, while other studies without that issue showed the highest incidence was over aged off-label use. Conclusion a) Off-label drug use is common in outpatient children. The incidence of off-label drug use may be higher in tertiary care institutes than primary care institutes, and it is higher in neonate, infant and adolescent rather than other age groups of children; b) Incidence of off-label use may be higher in inpatient children than outpatient ones; c) Drugs for respiratory system, anti-infection, sensory organs, and dermatosis were most common types of off-label use; and d) Off-label use due to both “lack of instruction for children” and “over aged use” is more common. So it suggests that further studies trials should focus on fields in which clinical trials of drugs are totally absent.
ObjectiveTo systematically review the efficacy and safety of deproteinized calf blood extractives (DCBE) for diabetic complications. MethodsPubMed, EMbase, The Cochrane Library, CNKI, WanFang Data, CBM and VIP databases were electronically searched to collect studies on the efficacy and safety of DCBE for diabetic complications from inception to July 8th, 2020. Two reviewers independently screened literature, extracted data and assessed the risk of bias of included studies; then, meta-analysis was performed using RevMan 5.3 software. ResultsA total of 69 studies were included. The results of meta-analysis showed that for diabetic neuropathy, the effect rate of DCBE was superior to control, mecobalamin, vitamin B12, vitamin B1+vitamin B12, and vitamin B2, etc. For diabetic foot, the effect rate of DCBE was superior to control, sensitive antibiotic and compound salvia-miltiorrhiza injection. For diabetic retinopathy, the effect rate of DCBE was superior to control. The incidence of adverse events associated with DCBE was 4.59%. However, there was no significant difference with the control group. ConclusionCurrent evidence shows that DCBE has good efficacy and safety in diabetic neuropathy, diabetic foot, diabetic dry eye, diabetic retinopathy, and other diseases. Due to limited quality and quantity of the included studies, more high quality studies are required to verify the above conclusion.
Objective To study the medication usage in children with crush syndrome in Wenchuan Earthquake. Methods The information of the medications of 7 children with crush syndrome in West China Second University Hospital, Sichuan University was collected by medical history. The cases and times of medication, the total number of drugs, the drug cost per capita and the drug cost per day were calculated by Excel. Results A total of 113 drugs in 19 categories were appl ied to 7 children with crush syndrome. Most children used Vitamins and antibacterial agents. The cost of gamma globul in injection was the highest. Conclusions The rational ity valuation for children medication lacks international vulgate index and needs more basic research.
ObjectiveThis study aims to compare different references for the fetal risk of drugs used in pregnancy to provide evidence for the safety of drug use in pregnancy.MethodsFour drug databases, including Lexicomp, Micromedex, TERIS, and Reprotox, as well as two books of drugs in pregnancy edited by Briggs and Schaefer, were searched. Descriptive analysis was performed regarding the definition of pregnancy recommendations and the specific content of medication.ResultsThe six references employed slightly different approaches to drugs in pregnancy, however, all of them included summaries of the risk in pregnancy, data of crossing the placenta, and human and animal data. The databases of Micromedex, TERIS, and a book edited by Briggs had their risk classification systems for drug use during pregnancy. For specific drugs, the summary of different information in pregnancy was different, the amount and content of listed evidence varied, and there was no evaluation of the quality and relevance of evidence among the references.ConclusionsThere is no consensus on the risk assessment of drugs in pregnancy. Risk classification systems for drugs in pregnancy are still an important method for determining the fetal risk of drugs. The existing references merely list studies of drugs in pregnancy, without comprehensive quality assessment. A methodological study of assessment of the risk of drugs in pregnancy is required.