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1.

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   
2.

Background

Observational studies of the relative effectiveness of influenza vaccines are essential for public health decision making. Their estimates, however, are subject to bias due to unmeasured confounders. Instrumental variable (IV) methods can control for observed and unobserved confounders.

Methods

We used linked electronic medical record databases in the Veterans Health Administration (VHA) as well as Medicare administrative files to examine the relative vaccine effectiveness (rVE) of high-dose influenza vaccine (HD) versus standard-dose influenza vaccines (SD) in preventing hospitalizations among VHA-enrolled Veterans ≥65?years of age during 5 influenza seasons (2010–2011 through 2014–2015). Using multivariable IV Poisson regression modeling to address unmeasured confounding and bias, we analyzed the data by each season and through longitudinal analysis of all five seasons.

Findings

We included 3,638,924 person–influenza seasons of observation where 158,636 (4%) were among HD vaccine recipients and 3,480,288 (96%) were among SD vaccine recipients. Of the 1,728,562 Veterans, 1,702,824 (98.5%) were male and 1,299,412 (75%) were non-Hispanic white. Based on the longitudinal analysis of all five seasons, the IV-adjusted rVE estimate of HD vs. SD was 10% (95% CI, 8–12%) against all-cause hospitalization; 18% (95% CI, 15–21%) against cardiorespiratory-associated hospitalization; and 14% (95% CI, 6–22%) against influenza/pneumonia-associated hospitalization. The findings by season were similar.

Interpretation

Our analysis of VHA clinical data collected from approximately 1.7 million Veterans 65?years and older during five seasons demonstrates that high-dose influenza vaccine is more effective than standard-dose influenza vaccines in preventing influenza- or pneumonia-associated hospitalizations, cardiorespiratory hospitalizations, and all-cause hospitalizations.  相似文献   
3.
宫腹腔镜联合手术诊治不孕症150例分析   总被引:14,自引:0,他引:14  
目的:探讨宫腹腔镜联合手术在诊治不孕症中的应用。方法:对150例不孕症患者行宫腹腔镜联合手术,对不孕症病因进行诊断,同时行治疗。结果:盆腔粘连和输卵管阻塞是不孕症的主要原因。子宫内膜息肉和正常盆腔占次要比例。子宫内膜异位症和多囊卵巢也是主要病因。150例同时行宫腹腔镜输卵管通液和各种疾病的治疗。联合手术后妊娠率为48.8%。结论:腹腔镜联合手术,在一次麻醉下,可以对不孕的原因全面评价和明确诊断,在诊断同时进行治疗,对不孕症的诊断和治疗有重大的意义,值得推广。  相似文献   
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6.
Instillation of human neutrophil elastase (HNE) into hamster lungs produces milder emphysema but more pulmonary hemorrhage than an equivalent amount of porcine pancreatic elastase (PPE), whether equivalence is determined by elastolytic units or moles. We undertook a study of the mechanisms of these differences. 125I-HNE or 3H-PPE were instilled intratracheally into hamsters. The partitioning of radioactivity between bronchoalveolar lavage fluid (BAL) and lung tissue was similar for HNE and PPE as were the half-lives, 45 and 51 min, respectively, for uncomplexed, enzymatically active HNE and PPE. In BAL there was preferential binding and inactivation of HNE by the hamsters' alpha-1-protease inhibitor (a-1-PI) whereas PPE was preferentially bound by alpha-2-macroglobulin (a-2-M). This was also observed in vitro when HNE and PPE were incubated with plasma from untreated hamsters. Nevertheless, when the sum of the elastase binding capacity of a-1-PI and a-2-M was considered, hamster plasma had similar binding capacities for HNE and PPE. It is known that the enzymatic activity of elastases is inhibited by formation of a stable complex with a-1-PI. On the other hand, elastases bound to a-2-M are protected against a-1-PI inhibition but can free themselves by proteolysis and exhibit elastolytic activity. Preferential inactivation of HNE by a-1-PI may be one mechanism that accounts for the lesser emphysema-inducing potency of HNE than of PPE.  相似文献   
7.
To evaluate the early diastolic peak filling rate of the left ventricle, three groups of children (normal children, patients with aortic valvular stenosis, and patients with aortic coarctation) were examined with the peak filling rate normalized to stroke volume calculated from the mitral valve inflow Doppler recording as the peak E velocity divided by the velocity time integral. The normal value for this index in children was 6.78 +/- 0.99 SV/sec and did not vary with age, weight, body surface area, or heart rate. Compared with normal subjects, both patients with aortic stenosis and patients with coarctation had increased left ventricular mass, but patients with aortic stenosis had decreased normalized peak filling rates (5.3 +/- 0.84 SV/sec, p < 0.01), while patients with coarctation had normal rates (6.79 +/- 0.98 SV/sec, p = 0.97). Compared with patients with aortic coarctation, patients with aortic stenosis had higher Doppler gradients. Thus the Doppler index of peak filling rate normalized to stroke volume is particularly useful in children because it is independent of heart rate, age, weight, and body surface area. Patients with coarctation may have normal peak filling rates normalized to stroke volume despite increased left ventricular mass because of milder obstruction or better coronary artery perfusion compared with that of patients with aortic stenosis.  相似文献   
8.
A case of life-threatening respiratory distress during a Caesarean section under spinal anaesthesia is reported. Possible causes of the event including anaphylactoid reactions and the methods of their diagnosis are discussed. The most likely cause of the episode was felt to be an anaphylactoid reaction to Syntocinon.  相似文献   
9.
Abstract: This paper dicusses the use of esophageal dilatation with a Rigiflex TTS balloon. This method was used 45 times on 11 patients affected by anastomotic or a severe grade peptic esophageal stenosis. Fluoroscopic guidance was used in 36 procedures (80%) without effecting the mean duration of the treatment (12 minutes). The results were considered satisfactory when these goals had been achieved: a) dilatation of the stenosis over 15 mm; b) a dysphagia free-time of more than 6 months. A satisfactory result was achieved in 10 patients (90.9%), without deaths and major complications. 5 patients received 1 dilatation and the other 5 needed, 3-3-4-7–11 procedures respectively to obtain a satisfactory result. On these basis we consider that its great efficacy, security and tolerability depend on the following characteristics of the Rigiflex TTS balloon: 1) “radial” dilatation; 2) the possibility of introducing the balloon through the operative channel of the fiberscope; 3) direct visualization of the stenosis during dilatation. The following disadvantages with this method are: the absence of a tactile sensation of dilatation and the elevated cost of the instrument. We conclude that the Rigiflex TTS balloon is an important alternative to guide-wire techniques, especially for the treatment of severe esophageal strictures.  相似文献   
10.
目的:探讨妊娠肝内胆汁淤积症(ICP)患者外周静脉血清、新生儿脐静脉血清中一氧化氮(NO)、内皮素(ET)、丙二醛(MDA)和超氧化物歧化酶(SOD)含量的变化及在ICP发病中的作用。方法:以ICP组28例为研究组,测定其外周静脉血清及新生儿脐静脉血清中的NO、ET、MDA和SOD,以年龄相近的24例正常孕妇作为对照组。结果:ICP患者的MDA和ET含量较正常晚期妊娠显著增高(P<0.01),ICP患者的NO和SOD含量与对照组相比无显著性差异(P>0.05)。母血清中NO、ET、MDA含量均较新生儿脐静脉血清中的含量高,差异有显著性(P<0.01)。结论:妊娠期体内氧化和抗氧化失衡及ET水平的增高可能与ICP的发生、发展有关。  相似文献   
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