Utilization and quality assessment of digestive endoscopy in China: results from 5-year consecutive nationwide surveys.

BACKGROUND: Worldwide, the volume and availability of digestive endoscopy have undergone dramatic development in recent years, with increasing attention on quality assurance. We investigated the utilization and quality of digestive endoscopy in China from 2015 to 2019 and developed a quantitative quality evaluation tool for medical institutions. METHODS: We invited all tertiary/secondary hospitals in Chinese mainland to participate in the survey annually. The questionnaires included the personnel, annual volume, and quality indicators of endoscopy. An endoscopy quality index (EQI) was developed based on recorded quality indicators using principal component analysis to determine the relative weight. RESULTS: From 2015 to 2019, 806, 1412, 2644, 2468, and 2541 hospitals were respectively enrolled in this study. The average annual volume of endoscopy increased from 12,445 to 16,206 (1.30-fold) and from 2938 to 4255 (1.45-fold) in tertiary and secondary hospitals, respectively. The most obvious growth was observed in diagnostic colonoscopy (1.44-fold for all hospitals after standardization). The proportion of early cancer among all esophageal and gastric cancers during diagnostic esophagogastroduodenoscopy increased from 12.3% (55,210/448,861) to 17.7% (85,429/482,647) and from 11.4% (69,411/608,866) to 16.9% (107,192/634,235), respectively. The adenoma detection rate of diagnostic colonoscopy increased from 14.9% (2,118,123/14,215,592) to 19.3% (3,943,203/20,431,104). The EQI model included 12 quality indicators, incorporating 64.9% (7.792/12) of the total variance into one comprehensive index. According to the EQI measurements, the quality of endoscopy was higher in tertiary hospitals and hospitals in developed areas with higher volume or more endoscopists than that in other hospitals. CONCLUSIONS: Digestive endoscopy in China has developed considerably in recent years in terms of both volume and quality. The EQI is a promising tool to quantify the quality of endoscopy at different hospitals.

Cost-effectiveness analysis of gene-based therapies for patients with spinal muscular atrophy type I in Australia.

INTRODUCTION: Spinal muscular atrophy (SMA) is an inherited neuromuscular disorder and regarded as one of the most frequent genetic causes of infant mortality. The aim of this study is to develop a cost-effectiveness analysis of AVXS-101 (Onasemnogene Abeparvovec/Zolgensma®) and nusinersen (Spinraza®) for SMA to inform decision-making on reimbursement policies in Australia. METHODS: A Markov model was developed with five health states to evaluate the costs and effects for patients with SMA Type I from a healthcare system perspective over a time-horizon of 100 years. The model parameters were based on clinical trials, parametric distributions, published literature, and Australian registries. One-way and probabilistic sensitivity analysis were performed to appraise the uncertainties of the parameters in the model. A threshold analysis was conducted to estimate the cost of AVXS-101 of being cost-effective. RESULTS: The incremental cost-effectiveness ratio (ICER) of AVXS-101 was $1,808,471 per quality-adjusted life year (QALY) and that of nusinersen was $2,772,798 per QALY, compared to standard of care, respectively. The ICER of AVXS-101 was $1,238,288 per QALY compared to nusinersen. The key drivers influencing on ICERs were costs of using treatments and utility values of sitting and walking independently. CONCLUSION: Both nusinersen and AVXS-101 resulted in health benefits, but they were not cost-effective with a commonly used willingness-to-pay (WTP) threshold of $50,000 per QALY. Developing high-quality clinical data and exploring appropriate WTP thresholds are critical for decision-making on reimbursement policies in the treatment of rare diseases.

Economic evaluation of reproductive carrier screening for recessive genetic conditions: a systematic review.

INTRODUCTION: Autosomal recessive (AR) and x-linked (XL) conditions are rare but collectively common which impact millions of people globally on morbidity, mortality and costs. Advanced medical technologies allow prospective parents to make informed reproductive decisions to avoid having affected children. Economic evaluations targeting on reproductive carrier screening (RCS) for AR and/or XL conditions have been conducted, but there has not been a systematic review in this area. AREAS COVERED: A systematic search of economic evaluations for RCS was undertaken using the following databases - EMBASE, MEDLINE and SCOPUS. The search strategy was designed to capture full economic evaluations related to RCS since 1990. This review followed the Preferred Reporting Items for Systematic Reviews and Meta-Analysis (PRISMA) strategy. The included 23 studies adopted various types of methodologies to conduct economic evaluations. The majority of studies examined a single condition. The various clinical strategies and screened conditions caused the different cost-effectiveness conclusions in the published studies. EXPERT OPINION: Establishing a validated and practical clinical strategy of RCS and investigating the cost-effectiveness of multiple conditions in one economic evaluation are critical for implementing RCS in the future. Further economic evaluations are essential to provide evidence-based practice for decision-makers.

Modelling a cost-effective vaccination strategy for the prevention of varicella and herpes zoster infection: A systematic review.

BACKGROUND: Varicella zoster virus (VZV) and its re-emergence as herpes zoster (HZ) is associated with significant morbidity and mortality. While studies show that VZV vaccination is effective in reducing VZV incidence, many decision makers have not added VZV to their vaccination schedule, largely due to uncertainty surrounding the effect of VZV vaccination on HZ incidence (exogenous boosting, EB), and the cost-effectiveness (CE) of vaccination. METHODS: A systematic review was conducted to identify the current published evidence of CE of VZV vaccination strategies where both VZV and HZ incidence were modelled. RESULTS: Six studies (one published in 2003 and five between 2010 and 2019), were identified with all conducting cost-utility analysis using a dynamic transmission modelling approach and assuming EB. All predicted that mass infant VZV vaccination would rapidly reduce VZV incidence, but HZ incidence would increase. Compared with no-vaccination, the CE of VZV vaccination strategies ranged from higher costs and poorer outcomes (dominated), towards CE (incremental cost-effectiveness ratios of between $7,000 to $61,000 USD), or lower cost and better outcomes (dominant). However, without EB, HZ incidence immediately dropped below pre-vaccination levels making VZV vaccination quickly CE and/or dominant to a no vaccination strategy. CONCLUSIONS: Current models are sensitive to assumptions of EB suggesting that future studies consider an agent-based modelling approach to address the individual nature of variables that determine the infectiousness of VZV.

Suppression of mTOR Signaling Pathways in Skeletal Muscle of Finishing Pigs by Increasing the Ratios of Ether Extract and Neutral Detergent Fiber at the Expense of Starch in Iso-energetic Diets.

Three iso-energetic and iso-nitrogenous diets were fed to finishing pigs for 28 days to investigate the mammalian target of rapamycin (mTOR) and ubiquitin-proteasome signaling pathways of skeletal muscle by altering compositions of dietary energy sources. Diet A, diet B, and diet C contained 44.1%, 37.6%, and 30.9% starch; 5.9%, 9.5%, and 14.3% ether extract (EE); and 12.6%, 15.4%, and 17.8% neutral detergent fiber (NDF), respectively. An increase of mRNA expression of MuRF1 (1.09 ± 0.10 vs 1.00 ± 0.08) and MAFbx (1.10 ± 0.06 vs 1.00 ± 0.11) and a decrease of concentrations of plasma insulin (8.2 ± 0.8 vs 10.8 ± 1.2) and glucose (5.76 ± 0.12 vs 6.43 ± 0.33) together with mRNA expression of IRS (0.78 ± 0.19 vs 1.01 ± 0.05) and Akt (0.92 ± 0.01 vs 1.00 ± 0.05) were observed in pigs fed diet C compared with diet A. Protein levels of total and phosphorylated mTOR (0.31 ± 0.04 vs 0.48 ± 0.03 and 0.39 ± 0.01 vs 0.56 ± 0.02), 4EBP1 (0.66 ± 0.06 vs 0.90 ± 0.09 and 0.60 ± 0.12 vs 0.84 ± 0.09), and S6K1 (0.66 ± 0.01 vs 0.89 ± 0.01 and 0.48 ± 0.03 vs 0.79 ± 0.02) were decreased; however, total and phosphorylated AMPK (0.96 ± 0.06 vs 0.64 ± 0.04 and 0.97 ± 0.09 vs 0.61 ± 0.09) were increased in pigs fed diet C compared with diet A. In conclusion, diet C suppressed the mTOR pathway and accelerated the ubiquitin-proteasome pathway in skeletal muscle of finishing pigs.