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1.
PurposeUnderstanding the value of genetic screening and testing for monogenic disorders requires high-quality, methodologically robust economic evaluations. This systematic review sought to assess the methodological quality among such studies and examined opportunities for improvement.MethodsWe searched PubMed, Cochrane, Embase, and Web of Science for economic evaluations of genetic screening/testing (2013-2019). Methodological rigor and adherence to best practices were systematically assessed using the British Medical Journal checklist.ResultsAcross the 47 identified studies, there were substantial variations in modeling approaches, reporting detail, and sophistication. Models ranged from simple decision trees to individual-level microsimulations that compared between 2 and >20 alternative interventions. Many studies failed to report sufficient detail to enable replication or did not justify modeling assumptions, especially for costing methods and utility values. Meta-analyses, systematic reviews, or calibration were rarely used to derive parameter estimates. Nearly all studies conducted some sensitivity analysis, and more sophisticated studies implemented probabilistic sensitivity/uncertainty analysis, threshold analysis, and value of information analysis.ConclusionWe describe a heterogeneous body of work and present recommendations and exemplar studies across the methodological domains of (1) perspective, scope, and parameter selection; (2) use of uncertainty/sensitivity analyses; and (3) reporting transparency for improvement in the economic evaluation of genetic screening/testing.  相似文献   
2.
目的研究左乙拉西坦治疗小儿癫痫的疗效以及对智力的影响。方法在本院2017年4月-2019年5月收治的小儿癫痫患儿中选取74例开展研究,按照随机数表法分两组观察组和对照组,观察组37例,对照组37例,对照组采用奥卡西平进行治疗,观察组采用左乙拉西坦进行治疗,对比观察组与对照组组的治疗总有效率和智力评分变化。结果观察组与对照组比较,观察组的治疗总有效率较高,智力评分明显较高,两项对比差异有统计学意义(P <0.05)。结论左乙拉西坦治疗小儿癫痫有较好的治疗效果,治疗总有效率较高,且对患儿的智力有明显的改善作用,在实际临床小儿癫痫的治疗中具有较高的运用价值。  相似文献   
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4.

Background

Payers frequently rely on budget impact model (BIM) results to help determine drug coverage policy and its effect on their bottom line. It is unclear whether BIMs typically overestimate or underestimate real-world budget impact.

Objective

We examined how different modeling assumptions influenced the results of 6 BIMs from the Institute for Clinical and Economic Review (ICER).

Study Design

Retrospective analysis of pharmaceutical sales data.

Methods

From ICER reports issued before 2016, we collected estimates of 3 BIM outputs: aggregate therapy cost (ie, cost to treat the patient population with a particular therapy), therapy uptake, and price. We compared these against real-world estimates that we generated using drug sales data. We considered 2 classes of BIM estimates: those forecasting future uptake of new agents, which assumed “unmanaged uptake,” and those describing the contemporaneous market state (ie, estimates of current, managed uptake and budget impact for compounds already on the market).

Results

Differences between ICER's estimates and our own were largest for forecasted studies. Here, ICER's uptake estimates exceeded real-world estimates by factors ranging from 7.4 (sacubitril/valsartan) to 54 (hepatitis C treatments). The “unmanaged uptake” assumption (removed from ICER's approach in 2017) yields large deviations between BIM estimates and real-world consumption. Nevertheless, in some cases, ICER's BIMs that relied on current market estimates also deviated substantially from real-world sales data.

Conclusions

This study highlights challenges with forecasting budget impact. In particular, assumptions about uptake and data source selection can greatly influence the accuracy of results.  相似文献   
5.
ObjectivesAs more countries are implementing measures to address Alzheimer’s disease (AD), it is essential to update the available knowledge on the relationship between economic status and mortality in patients with AD. This study examined the influence of economic status on mortality in Japanese individuals with AD using a medical claims dataset.DesignThis was a retrospective cohort study.Setting and ParticipantsMedical claims data from April 2014 to March 2019 were obtained from 13 local cities participating in the Longevity Improvement and Fair Evidence study. The inclusion criteria were patients aged 65 years and older who were newly diagnosed with AD during the study period.MethodsThe outcome was death during the follow-up period. We assessed economic status by household income (middle to high income and low income); data were obtained from the use of the Medical Expenditure Ceiling Application and Standard Copayment Reduction Card (fee reduction card) when receiving an AD diagnosis, as an indicator of low-income status. We performed multivariate Cox proportional hazards analyses to examine the relationship between economic status and mortality; the model was adjusted for age, sex, the Charlson comorbidity index, and antidementia drug use.ResultsWe identified 39,081 newly diagnosed patients with AD from the Longevity Improvement and Fair Evidence study database (mean age, 83.6 years; female, 67.1%). Of these, 3189 individuals were identified as having a low-income status. After adjusting for possible confounders, low-income status was associated with mortality (hazard ratio, 1.95; 95% confidence interval, 1.84–2.07).Conclusions and ImplicationsLow-income status was associated with substantially poorer prognoses in new AD cases, indicating a need for a thorough examination of medical and nursing care services utilized by low-income individuals with AD and to explore improvement strategies.  相似文献   
6.
??Objective    To investigate the characteristics of indirect injury of periodontal membrane caused by maxillofacial impact injury. Methods    The study was carried out at the laboratory of oral and maxillofacial trauma in Fourth Military Medical University??from November 2013 to January 2014. Totally 18 dogs were divided equally into six groups with random allocation??the right mandible of each dog was impacted by a cylindroid iron cap whose weight was 0.26kg??but teeth were not injured directly??the animals were killed 6 h??24 h??3 d??7 d??2 wk and 4 wk later??respectively??the histopathologic manifestations of periodontal membrane of the fourth premolar of right mandible??the second premolar of right mandible and the second premolar of left mandible were observed. Results    The injury of periodontal membrane of the fourth premolar of right mandible was relatively obvious??but the pathologic changes were reversible??periodontal membrane of the second premolar of right mandible was injured slightly??yet no obvious histopathologic change could be observed in periodontal membrane of the second premolar of left mandible. Conclusion    Maxillofacial impact injury can cause indirect injuries of periodontal membrane of adjacent teeth??but the pathologic changes are reversible??traumatic severity decreases as the distance of the tooth to the wound area increases.  相似文献   
7.
Postoperative complications(PC) are a basic health outcome, but no surgery service in the world records and/or audits the PC associated with all the surgical procedures it performs. Most studies that have assessed the cost of PC suffer from poor quality and a lack of transparency and consistency. The payment system in place often rewards the volume of services provided rather than the quality of patients' clinical outcomes. Without a thorough registration of PC, the economic costs involved cannot be determined. An accurate, reliable appraisal would help identify areas for investment in order to reduce the incidence of PC,improve surgical results, and bring down the economic costs. This article describes how to quantify and classify PC using the Clavien-Dindo classification and the comprehensive complication index, discusses the perspectives from which economic evaluations are performed and the minimum postoperative follow-up established, and makes various recommendations. The availability of accurate and impartially audited data on PC will help reduce their incidence and bring down costs. Patients, the health authorities, and society as a whole are sure to benefit.  相似文献   
8.
目的总结并归纳护理干预对急性心肌梗死患者负性情绪的影响。方法本文挑选100例医院急性心肌梗死患者(2017年2月—2018年2月)为对象。按建档时间分组,对照组50例患者(常规护理),分析组50例患者(加用人文性护理)。比较两组对疾病的了解程度及护理前后焦虑、抑郁等负性情绪的评分,随访1年,比较两组复发率。结果护理后分析组对疾病了解程度高于对照组(P<0.05);护理前分析组焦虑、抑郁评分同对照组无显著差异(P>0.05),提示可比;护理后,分析组焦虑、抑郁评分低于对照组(P<0.05);护理后分析组1年内复发率(8.00%)低于对照组(24.00%)(P<0.05)。结论在急性心肌梗死患者的护理中,人文性护理干预可显著改善患者负性情绪,改善患者预后,且可有效降低患者复发率。  相似文献   
9.
Between 23 and 70% of occupants involved in frontal impacts sustain cervical spine injuries, many with neurological involvement. It has been hypothesized that cervical spinal cord compression and injury may explain the variable neurological profile described by frontal impact victims. The goals of the present study, using a biofidelic whole cervical spine model with muscle force replication, were to quantify canal pinch diameter (CPD) narrowing during frontal impact and to evaluate the potential for cord compression. The biofidelic model and a sled apparatus were used to simulate frontal impacts at 4, 6, 8, and 10 g horizontal accelerations of the T1 vertebra. The CPD was measured in the intact specimen in the neutral posture (neutral posture CPD), under static sagittal pure moments of 1.5 Nm (pre-impact CPD), during dynamic frontal impact (dynamic impact CPD), and again under static pure moments following each impact (post-impact CPD). Frontal impact caused significant (P<0.05) dynamic CPD narrowing at C0-dens, C2-C3, and C6-C7. The narrowest dynamic CPD was observed at C0-dens during the 10 g impact and was 25.9% narrower than the corresponding neutral posture CPD. Interpretation of the present results indicate that the neurological symptomatology reported by frontal impact victims is most likely not due to cervical spinal cord compression. Cord compression due to residual spinal instability is also not likely.  相似文献   
10.
恒河猴坐姿着陆冲击内耳损伤的观察   总被引:2,自引:0,他引:2  
为研究机体及各组织器官对冲击力的耐受限值,探讨致伤原因及提出防护措施,利用着陆冲击塔,使实验动物恒河猴的坐姿状态下,进行冲击试验,试验后1h左右处死,作大体解剖观察并取心,肝,脾,肺,颞骨等组织,固定,切片,镜检。同时重点观察了内耳的损伤。结果表明,冲击载荷不大(平台过载值为≤25G)时内脏虽然有轻度损伤,但内耳可避免受员,当冲击力加大到一定程度(平台过载值为30~35G)导致内脏中等损伤时,内耳  相似文献   
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