ObjectivesTo provide a reference for the evaluation procedures of genetic testing technology applicable to China by combining the existing evaluation frameworks and procedures for genetic testing techniques globally, and also put forward design suggestions for the construction of evaluation procedures in China.MethodsThe literature research method was primarily used to summarize different evaluation progress, as well as put forward design suggestions.ResultsAt present, numerous developed countries have organized genetic testing technology evaluation projects. The various evaluation frameworks developed were based on the ACCE or HTA framework. The evaluation and decision-making procedures were similar in general, including topic selection, evaluation implementation, results reporting and making recommendations. However, there still remained difficulties such as limited evidence and uncertainty in decision-making.ConclusionsTo establish the procedures of genetic testing technology applicable in China, the following specific procedures are recommended: selecting target genetic testing technology topics; analyzing necessity and feasibility of target testing technology evaluation; evaluating and reviewing the evidence; applying results and decision-making transformation; developing regular review and revision mechanisms.
With the increasingly prominent contradiction between limited health resources and the growing population, priority setting of health research, as a response, has received widespread attention from health systems worldwide. As the results of priority setting at different levels increase year by year, some questions in the results reporting are also constantly emerging. For example, the process of producing the results is vague, too dependent on individual subjective judgment, the participation of individual stakeholder groups is limited or lack of voice, unable to identify potential conflicts of interest, and so on. It does not only seriously affect the effectiveness and rationality of the results themselves, but also create intangible obstacles to their promotion and adoption. In 2019, BMC Medical Research Methodology published ‘Reporting guideline for priority setting of health research (REPRISE)’, which makes uniform specifications for more comprehensive and consistent reporting of results in priority areas. This paper interpreted the background, formulation process and key contents of the REPRISE guideline, with an aim to promote the application of the reporting guideline in China.
ObjectiveTo systematically review the economics evaluation studies on the early screening or diagnosis of primary immunodeficiency diseases (PID). MethodsWeb of Science, CRD, PubMed, The Cochrane Library, CNKI and WanFang Data databases were electronically searched to collect the economics evaluation studies on the early screening or diagnosis of PID from inception to July 1st, 2022. Two reviewers independently screened literature, extracted data and assessed the risk of bias of included studies, then, a descriptive systematic review was performed. ResultsA total of 10 studies focusing on SCID were included. The results showed that under a relatively high threshold, the early screening and diagnosis of SCID were cost-effective, which can reduce severe infections in patients and treatment costs while improving patient’s survival. ConclusionCurrent evidence shows that early diagnosis of PID can reduce costs and improve benefits. Due to limited quality and quantity of the included studies, more high quality studies are needed to verify above conclusion.
ObjectivesTo initially construct a scientific, reasonable and precision medicine technology value judgment framework suitable for China’s national conditions based on expert consultation method, so as to provide scientific value judgment system support for China's medical insurance decision-making.MethodsThe preliminary evaluation indicator system for precision medicine technology value was established by using literature analysis and expert consultation method, and the direct weighting method was used to determine the indicators weight.ResultsAfter two rounds of expert consultation, an indicator system suitable for the value judgment of precision medicine technology in China was constructed, including 5 primary indicators (health needs, health effects, economics, innovation and suitability) and 14 secondary indicators. Each indicator was weighted according to importance.ConclusionsA set of precision medicine value judgment indicator system suitable for China has been initially established, which lays a certain foundation for further measurement research of the indicator system and provides a scientific basis for medical insurance decision-making.
The main task of Campbell Collaboration is to collaborate with Cochrane Collaboration so as to produce high quality evidence for the social welfare, education, justice and criminal, international development policy and other social sciences. This article systematically introduces Campbell Collaboration, its origins, achievements and development, with the purpose of allowing more scholars understand evidence-based ideas and methods of social science, providing evidence-based methodology basis for China's social policy.
Health technology assessment (HTA) is becoming more and more popular recently. For populations in China that share at least half of the global disease burden of liver cancer, it is extremely vital to give rise to an efficient secondary prevention strategy. The China central government launched liver cancer screening program in rural areas in 2005, and then extended to populations in urban in 2012. The studies of health technology assessment of liver cancer screening are based on available evidence, from an HTA perspective, aims to evaluate performance of liver screening, economic burden and cost-effectiveness and some other issues, in order to raise suggestions for possible directions in research and public health program related to liver cancer screening in China.
Patient-reported outcome measures (PROM) measure attribute studies refer to studies conducted by investigators to validate the measurement attributes of PROM. The consensus-based standards for the selection of health measurement instruments (COSMIN), an international consensus standard for the selection of health measurement instruments, divides this attribute into three aspects: reliability, validity and responsiveness, and adds interpretability as an additional important feature for evaluating PROM. The purpose of this paper is to introduce the verification methods and principles of the three major measurement attributes in the COSMIN consensus, as well as the significance and direction of interpretability evaluation, and to provide international methodological experience and reference for the development of high-quality PROM psychometric attribute verification in China.
ObjectiveTo explore the optimal conditions of rat model of hyperuricemia (HUA) induced by different doses of potassium oxanate (PO) combined with adenine, and to provide reference for the treatment of HUA.MethodsMale Sprague-Dawley rats (220-240 g body weight) were divided into normal control group, potassium oxanate (1000, 1500 mg/kg) and adenine (0, 50, 100 mg/kg) combined model groups, with 8 rats in each group. After 5 weeks of intragastric administration, blood were collected from tail vein of rats every week, and serum uric acid, creatinine and blood urea nitrogen level were measured. At the 6th week, the changes of the pathological characteristics, expression of inflammatory and fibrosis-related factors in the kidneys were observed.ResultsIn the 1500 mg/kg potassium oxanate combined with 100 mg/kg adenine group, rats died after 2 weeks of molding, and the survival rate at the 6th week was 62.5%; but there was no significant difference between the other groups and the normal control group in survival rate (P>0.05). Compared with the normal group, the level of serum uric acid in each model group increased significantly after 1 week of molding (P<0.05), but recovered to the pre-model level after stopping intragastric administration in week 6. After 5 weeks, in model groups the levels of serum creatinine and blood urea nitrogen were higher than those in the normal control group; and the inflammation and fibrosis-related factors mRNA and protein expression of kidney tissue in model groups increased with the increase of ademine dose, and there was a significant difference in the PO 1 000 mg/kg with adenine 100 mg/kg group, PO 1 500 mg/kg with Adenine 50 mg/kg group compared to the normal control group (P<0.05). The results of renal anatomy and histology testing in rats showed that with the increased of the dosage of PO and adenine in the model groups, the increase of white deposition of renal medulla, tubulointerstitial fibrosis, and tubular epithelial cell necrosis was found, and the glomerular atrophy aggravated. Compared with the indexes in the normal control group, the expression levels of inflammation and fibrosis related genes and proteins in the 50 mg/kg adenine combined with 1 500 mg/kg PO group were higher, and inflammatory cell infiltration and fibrosis were observed, which was consistent with the clinical manifestation of hyperuricemia induced renal injury.ConclusionPO (1500 mg/kg) combined with adenine (50 mg/kg) can establish a stable hyperuricemic nephropathy model in rats.
ObjectiveTo systematically review the efficacy and safety of different tyrosine kinase inhibitors (TKIs) in the treatment of chronic myelocytic leukemia (CML).MethodsPubMed, EMbase, The Cochrane Library, CBM, WanFang Data and CNKI databases were electronically searched to collect randomized controlled trials (RCTs) of nilotinib, dasatinib, flumatinib and imatinib for CML from inception to August, 2020. Two reviewers independently screened literature, extracted data and assessed the risk of bias of included studies; network meta-analysis was then performed using Stata 15.0 software and R 3.4.0 software.ResultsA total of 8 RCTs involving 2 775 patients were included. Compared with other TKIs, flumatinib had higher 3-month early molecular response and 1-year progression free survival, and the incidence of serious side effects was relatively low. Major molecular response and complete cytogenetic response were significantly superior to imatinib, and had the same or similar effects to other second-generation TKIs.ConclusionsCurrent evidence shows that flumartinib in the treatment of CML is obviously superior to imatinib, has the same or similar effect with other second generation TKIs. Due to limited quality and quantity of the included studies, more high quality studies are needed to verify above conclusions.
Objective To systematically review the health economic evaluation studies of medicines for the treatment of acute myeloid leukemia (AML). MethodsThe PubMed, EMbase, Cochrane Library, CBM, CNKI, and WanFang Data, as well as the CRD database specifically for health economics were electronically searched from inception to June 2022, and related journals in the field of health economics and the websites of HTA institutions in various countries were manually searched. The quality of the studies was assessed using the CHEERS checklist. The basic characteristics of health economics evaluation publications were summarized, the quality of model structures and methodologies was assessed and economic evaluation results were compared among different treatments. Results A total of 17 studies were included, and cost-effectiveness analyses were conducted from the perspectives of the health system, patients, the whole society, and medical insurance payers. The economic evaluation models were relatively unified, but there were differences in methods and results reporting, and the quality needed to be improved. The research objects were mainly the comparison of hypomethylating agents, targeted medicine and traditional chemotherapy regimens, as well as the comparison of different chemotherapy combinations and different drug dosages. Conclusion Real-world studies are mainly focused on traditional chemotherapy regimens, and model-based health economic evaluations, such as Markov models, are more frequently applied to newly developed targeted drugs and demethylation drugs. Among all treatments, the chemotherapy regimens including cytarabine, midostaurin, and decitabine are found to be more cost-effective.