ObjectivesTo provide a useful framework for improved understanding of international value drivers in the decision-making process of medical insurance access, and to explore the value assessment criteria of orphan drugs from stakeholders’ perspective.MethodsPubMed, EMbase, CINAHL Plus, ProQuest, Web of Science, CNKI and WanFang Data databases were electronically searched to collect studies from January 1st, 1983 to December 31st, 2018. Research questions were constructed based on SPIDER model. We established the inclusion and exclusion criteria to filter studies. Study quality was evaluated using the Critical Appraisal Skills Programme (CASP) checklist. A thematic synthesis was undertaken to develop descriptive themes, analytical constructs and third-level themes of value drivers by NVivo 11 software, and confidence in the findings was assessed using the CERQual method.ResultsA total of 10 studies including 20 research countries were included. Fifty descriptive themes were interpreted and embedded within 14 analytical constructs and 3 third-level themes after induction. Specifically, 3 broad themes were disease-related influence factors, which included severity, unmet requirements, disease burden, affected individuals, and moral and ethical considerations; drug-related influence factors, which included safety, efficacy, economics, innovation, quality of evidence; and some external and non-pharmaceutical intrinsic properties factors, which included reimbursement status in other countries or regions, government goals and priorities, confirmed drug supply and impact on environment.ConclusionsIn addition to conventional considerations such as efficacy and pharmacoeconomics, stakeholders are willing to take a broader perspective when in the case of the value assessment of orphan drugs. Comprehensive understanding of these value drivers is important to shape policy and enhance decision-making.
ObjectiveTo systematically review the efficacy and safety of simvastatin and its different doses in the adjunct therapy of chronic obstructive pulmonary disease (COPD).MethodsPubMed, EMbase, Web of Science, The Cochrane Library, CNKI, WanFang Data, CBM and VIP databases were electronically searched to collect randomized controlled trials (RCTs) on adjunct therapy of simvastatin in patients with COPD from inception to May 15th, 2020. Two reviewers independently screened literature, extracted data and assessed risk bias of included studies; then, meta-analysis was performed by using Stata 14.0 software.ResultsA total of 22 RCTs involving 2 377 patients were included. The results of meta-analysis showed that treatment with 20 mg simvastatin could improve FEV1%pred, FEV1/FVC, and reduce inflammatory indexes such as CRP, hs-CRP, IL-8 and TNF-α, while 40 mg failed to improve. Simvastatin could reduce COPD score (CAT), but failed to increase the 6-minute walking distance or alleviate acute exacerbation.ConclusionsCurrent evidence shows that treatment with 20 mg simvastatin can improve pulmonary function, reduce inflammatory index and optimize CAT score in COPD patients, but it cannot increase the 6-minute walking distance and reduce the number of acute exacerbations of COPD. Due to the limited quantity and quality of included studies, the above conclusions are needed to be verified by more high-quality studies.
Objective To evaluate the effectiveness of robot-assisted percutaneous vertebroplasty (PVP) in the treatment of osteoporotic vertebral compression fracture (OVCF) in the elderly. Methods The clinical data of 90 elderly patients with OVCF treated in Sichuan Science City Hospital between June 2019 and March 2021 were retrospectively analyzed. The patients were divided into robot-assisted group (40 cases) and C-arm-assisted group (50 cases). The pre- and post-operative Visual Analogue Scale (VAS) score, pre- and post-operative Oswestry Disability Index (ODI) score, intraoperative cement leakage, intraoperative fluoroscopy frequency, operation time, and the loss of hemoglobin were recorded and compared between the two groups. Results The incidence of intraoperative cement leakage (5% vs. 20%), intraoperative fluoroscopy frequency [(18.3±3.2) vs. (41.3±7.8) times], operation time [(28.3±4.6) vs. (43.2±7.3) min] and the loss of hemoglobin [(7.2±2.0) vs. (15.2±4.4) g/L] of the robot-assisted group were less than those of the C-arm-assisted group (P<0.05). There was no statistically significant difference between the two groups in the decrease of VAS score or ODI score (3.63±1.64 vs. 3.40±1.65, P>0.05; 50.70±4.95 vs. 52.10±6.69, P>0.05). Conclusions Robot-assisted PVP for elderly patients with OVCF can significantly reduce the risk of cement leakage, shorten the operation time, reduce the intraoperative fluoroscopy frequency, and reduce the surgical hidden blood loss. It can be used for clinical promotion and application.
Objectives To evaluate the methodological bias and the reliability of the conclusions of systematic reviews (SRs) on urate-lowering therapy (ULT) for chronic gout. Methods PubMed, EMbase, The Cochrane Library, Epistemonikos, CBM, WanFang Data and CNKI databases were electronically searched to collect published systematic reviews and meta-analyses evaluating urate-lowering drugs in chronic gout from inception to April 8th 2017. Two reviewers independently screened literature, extracted data, assessed the methodological quality of included SRs using the AMSTAR tool, and assessed the quality of the body of evidence for each outcome using the GRADE approach. Results A total of 11 relevant SRs/Mets were included, containing 3 main outcome measures. All these SRs contained allopurinol. Ten SRs contained febuxostat, 3 SRs contained benzbromarone and 1 SR contained probenecid. Ten SRs assessed the risk of bias of included original studies. Eight SRs used the" assessing risk of bias”tool recommended by Cochrane Collaboration for this assessment while 2 used other tools. The assessment results of AMSTAR tool showed: the scores of 4 SRs were ≥9, and the others were ≤8. GRADE results showed: the quality of the evidence of 20 outcomes was low or very low, 10 outcomes was moderate and two outcomes was high. Conclusions Moderate quality evidence shows that febuxostat is beneficial in achieving target serum uric acid levels when comparing to allopurinol, and high quality evidence shows the incidence of gout flares is not significantly different between the two groups. High quality evidence also shows that the safety of febuxostat is better than allopurinol. Evidence of SRs is still insufficient to support the effectiveness and safety of other urate-lowering drugs. It is expected that more scientific and rigorous researches will be performed in the future, for which more high quality evidence will be produced to fill relevant gaps.
ObjectivesTo evaluate the reporting quality of clinical practice guidelines published in Chinese journals in 2017.MethodsCBM, CNKI and WanFang Data databases were searched for articles published in 2017. Two reviewers independently screened literature, extracted data, and evaluated the reporting quality of clinical practice guidelines using the Reporting Items for Practice Guidelines in Healthcare (RIGHT).ResultsOne hundred and seven clinical practice guidelines were included and a total reporting rate of 34.8%±0.1% in RIGHT. Among the seven domains of RIGHT, field on basic information had the highest reporting rate (56.8%) and fields on review and quality assurance had the lowest reporting rate (9.3%).The average reporting rate of RIGHT items of Chinese Science Citation Database (CSCD) articles was lower than non-CSCD [MD=−0.73, 95%CI (−0.78, −0.68)] articles. The average reporting rates of RIGHT items differed between Chinese Medical Association (CMA) journal articles and non-CMA journal articles [MD=2.30, 95%CI (2.26, 2.34)]. The average reporting rates of RIGHT items was lower in guidelines established by associations or institutes [MD=−3.78, 95%CI (−3.83, −3.73)], and was higher reported in Chinese medicine guidelines [MD=21.94, 95%CI (21.91, 21.97)].ConclusionsThe reporting quality of clinical practice guidelines published in journals of mainland China in 2017 is low in general, especially in fields such as review and quality assurance, funding and declaration and management of interests and other information. To improve this phenomena, it is suggested that guideline developers report the guidelines rigorously with international standard.
ObjectiveAntiviral treatments could benefit chronic hepatitis B (CHB) patients with the regression or improvement of liver fibrosis. However, the degree of dynamic change of liver fibrosis for patients who had not received antiviral treatment remained to be studied. The current study aimed to observe the long-term variation of liver stiffness measurement (LSM), virological and biochemical response on patients without standard antiviral therapy.MethodsA total of 220 patients who were diagnosed with chronic HBV infection, who had not reached the standard of antiviral therapy, and completed a follow-up date of over 2 years in the First Affiliated Hospital of Xi’an Jiaotong University from 2012 to 2018 were retrospectively enrolled. According to the changes of LSM in baseline and follow-up period, the patients were divided into regression group, non-progressive group, and progressive group. The virological and biochemical characteristics of each group were analyzed.ResultsAmong the 220 patients, 153 patients (69.5%) had no progress in LSM degree. Alanine aminotransferase (ALT), HBV DNA, and HBsAg in a few patients increased or slightly decreased, while the vast majority remained in a relatively stable state. 89.5% (137/153) of the non-progressive patients were in grade F0. In addition, 58 patients showed spontaneous improvement with a decreasing rate of 0.460 kPa per year. Patients with ALT of 1-2 ULN had a statistically significant decrease in LSM improvement compared to patients with normal ALT. 82.8% of the LSM-improving patients showed baseline LSM of F1-F3. Only 9 patients showed LSM deterioration, however, which could not be explained by virus replication or necroinflammatory activity. ConclusionsFor patients unsatisfying standard antiviral therapy, most patients with baseline LSM of F0 grade fail to progress, and patients with baseline LSM of F1-F3 show a decrease during follow-up, LSM progression occurs in 4.1% of patients.
ObjectiveTo evaluate the scientificity, transparency and applicability of the Chinese consensuses on urological diseases published in 2021. MethodsPubMed, CBM, CNKI, WanFang Data databases and related websites were electronically searched to collect Chinese consensuses on urological diseases from January 1 to December 31, 2021. Each consensus was scored with the scientific, transparent, and applicable rating (STAR) tools, and analyzed by using descriptive methods. ResultsA total of 28 Chinese consensuses were included. The STAR scores ranged from 9.9 to 32.3 with a mean of 17.3±6.3. The included consensus had a high score ratio in the items such as listing participants and institutions, providing identifiable recommendations, explaining the precautions for implementation of recommendations, having corresponding references for recommendations, and reporting future research directions. However, only 14.3% reported the methodology of consensus formation, and the record of consensus process and consideration of patient preferences, values and costs were not noted. No consensus reported responsibilities of panel specialists in the method section or included experts in the field of guideline methodology or evidence-based medicine. No consensus reported detailed information in managing conflicts of interest. Few reported no affection by funding. No consensus reported methods for collecting and selecting clinical questions, or evaluating, summarizing and grading evidence. Research gaps were not reported in a clear or standardized way. ConclusionAs a medical guidance document, expert consensus still plays an important role now in China. The quality of consensus on urological diseases can be further improved in methods of consensus formation, working groups, conflicts of interest, funding, accessibility, clinical questions, retrieval and evaluation of evidence, research gap, etc.
ObjectiveTo summarize the evaluation tools for the implementation effect of clinical practice guidelines (CPGs), to inform and support the evidence-based development of a general tool for the evaluation of implementation effect of CPGs. MethodsSeven biomedical literature databases, including PubMed, Embase and CNKI, and two academic websites, were searched from establishment to June 2022. Theoretical and empirical research on the evaluation tools of the implementation effect of CPGs were included. Two researchers independently screened literature and extracted data according to the inclusion and exclusion criteria. Based on the Implementation Science RE-AIM theoretical framework, an evaluation framework and a list of alternative items of the implementation effect of the CPGs were initially drawn up by thematic synthesis methods. ResultsA total of 208 articles were included, and 8 mature evaluation tools related to the implementation effect of guidelines were selected. Current research on the evaluation of the implementation effect of CPGs mainly focused on single diseases, with concern of the cognition, attitude and compliance of users to CPGs, and the process of the implementation of CPGs and factors affecting the implementation effect of CPGs. There were limitations such as a lack of evaluation on the terminal effect of the implementation of CPGs, the rare use of mature theoretical frameworks, the use of single evaluation perspectives, and inclusion of limited evaluation dimensions. The initial proposed evaluation framework contains a list of alternative items with 6 primary indicators, 12 secondary indicators and 41 tertiary indicators. ConclusionCurrently, there is a lack of a comprehensive, multi-perspective, mature theory based, general tool for the evaluation of implementation effect of CPGs. The framework and the list of alternative items for the evaluation of implementation effect of CPGs based on the implementation science RE-AIM theory can inform and support the development of a tool for the evaluation of implementation effect of CPGs.
Osteoarthritis research society international (OARSI) guidelines for the non-surgical management of knee, hip, and polyarticular osteoarthritis in 2019 contained some recommendations in treating patients with osteoarthritis (OA) who are considering non-surgical treatment. Compared with domestic consensus on diagnosis and treatment for OA, this clinical practice guideline (CPG) prepared by OARSI had some advantages in terms of methodology selection and recommendation. Therefore, it is necessary to interpret this CPG to accelerate the understanding and dissemination of the CPG. The ultimate aims were: to strengthen the normative and understanding of non-surgical treatment of OA; to enhance the understanding of clinicians for this CPG in treating OA; to accelerate the development of guideline development methodologies in China; to provide methodological guidance for the development of CPG based on the current situation in China.
ObjectiveTo evaluate the reliability and validity of Guideline Implementation Success Assessment Tool (A-GIST). MethodsWith the guideline for the prevention and treatment of type 2 diabetes mellitus in China (2020 edition) as the target guideline, health care providers and patients from different hospitals across the country were investigated by questionnaire using A-GIST. Spearman-Brown coefficient and Cronbach's α coefficient were used to evaluate the split-half reliability and internal consistency reliability, while the structural validity, convergent validity and discriminant validity were investigated by confirmatory factor analysis based on structural equation. ResultsThe internal consistency reliability and split half reliability coefficients of the whole tool and each dimension ranged from 0.650 to 0.986. The scale-level content validity index (S-CVI/Ave) of content validity was 0.846. Confirmatory factor analysis showed that, the χ2/df of two sections of the tool were 8.695 and 6.123, respectively. The root mean square residual (RMR), the standard root mean square residual (SRMR) and the root mean square error of approximation (RMSEA) were under or almost under the threshold. Besides, the goodness-of-fit index (GFI) of them were 0.901 and 0.822, the adjusted goodness-of-fit index (AGFI) were 0.836 and 0.787, and the parsimonious normed fit index (PNFI) were 0.545 and 0.788, respectively. ConclusionGuideline Implementation Success Assessment Tool (A-GIST) was proved to be valid and reliable, and it shows that it is necessary to optimize the items under the dimensions of maintenance and evaluation of diagnosis and treatment effect in the future.