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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   
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Introduction

Physician communication impacts patient outcomes. However, communication skills, especially around difficult conversations, remain suboptimal, and there is no clear way to determine the validity of entrustment decisions. The aims of this study were to 1) describe the development of a simulation-based mastery learning (SBML) curriculum for breaking bad news (BBN) conversation skills and 2) set a defensible minimum passing standard (MPS) to ensure uniform skill acquisition among learners.

Innovation

An SBML BBN curriculum was developed for fourth-year medical students. An assessment tool was created to evaluate the acquisition of skills involved in a BBN conversation. Pilot testing was completed to confirm improvement in skill acquisition and set the MPS.

Outcomes

A BBN assessment tool containing a 15-item checklist and six scaled items was developed. Students' checklist performance improved significantly at post-test compared to baseline (mean 65.33%, SD = 12.09% vs mean 88.67%, SD = 9.45%, P < 0.001). Students were also significantly more likely to have at least a score of 4 (on a five-point scale) for the six scaled questions at post-test. The MPS was set at 80%, requiring a score of 12 items on the checklist and at least 4 of 5 for each scaled item. Using the MPS, 30% of students would require additional training after post-testing.

Comments

We developed a SBML curriculum with a comprehensive assessment of BBN skills and a defensible competency standard. Future efforts will expand the mastery model to larger cohorts and assess the impact of rigorous education on patient care outcomes.  相似文献   
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We present the case of 7‐year‐old African American girl with loose anagen syndrome. Although this is a common cause of hair loss in Caucasian children, and there have been reports of cases occurring in dark‐skinned children of North African and Middle Eastern descent, to our knowledge there have been no cases reported in black children of sub‐Saharan African ancestry. We present this case to broaden the differential diagnosis of hair loss in African Americans.  相似文献   
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Purpose: To study the prevalence and time of onset of ocular complications in intermediate uveitis (IU) and to assess their effects on visual outcome in short-term and long-term follow-up.

Methods: A retrospective cohort study of patients with IU who had a minimal follow-up of one year.

Results: 96 patients (174 eyes, 70% females) were included with a mean age at presentation of 30 years. Children constituted 38% of all patients. Mean follow- up was 64.9 months. Pars planitis was the most common form followed by sarcoidosis and multiple sclerosis. Cystoid macular edema (CME), cataract, epiretinal membrane and posterior synechiae were the most prevalent complications. Posterior synechiae, band keratopathy, cataract and papillitis at presentation were associated with worse presenting visual acuity (VA). Of the late-onset complications, glaucoma/ocular hypertension (OHT) was the most significantly associated with worse long-term VA. Most sight-threatening complications (namely CME and glaucoma) were diagnosed at presentation while late complications predominantly affected the posterior segment and included among others peripheral retinal elevations, vasoproliferative tumors, and vitreous hemorrhage. Median LogMAR VA improved at short-term and long-term follow-up, from 0.2 to 0.1 (p<0.001). Subgroup analysis revealed significant VA improvement for eyes with all the different complications except for eyes with glaucoma/OHT.

Conclusion: IU is a chronic low-grade uveitis that may be associated with protean early and late complications of the anterior or posterior segments or both. Optimal treatment regimens are imperative for the strict control of inflammation and proper management of complications thus allowing a favorable long-term prognosis.  相似文献   

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Quality of Life Research - Health-related quality of life (HRQoL) is an important indicator of population health, yet no age-specific trend analyses in HRQoL have been conducted with a nationally...  相似文献   
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Osteoporosis is a condition in which the bones are thinner and more liable to fracture. It is commoner in women and in people over 50. It cannot be cured so the focus is on prevention, which means identifying and addressing risk factors such as obesity, low vitamin D, chronic inflammation and prolonged steroid medication. Chronic urticaria (hives, CU) is an inflammatory condition, so one might expect it to be linked with osteoporosis, but that has never been investigated. If people with CU are indeed more likely to develop osteoporosis, they could be advised about preventative measures. To study this, doctors from Israel identified 11,944 patients diagnosed with CU in a large medical database covering 4.5 million people. A potential difficulty was that people with CU are more likely to be female, obese and to have been treated with systemic steroids, all of which also increase the risk of osteoporosis. Therefore for each CU patient they studied 5 age- and sex-matched control patients (people of the same age and sex but without CU) and their analyses took into account other known risk factors for osteoporosis. During a 16 year period 8.7% of the patients with CU were diagnosed with osteoporosis compared with 6.8% of the controls. They concluded that CU is a small but significant additional risk factor for osteoporosis. An accompanying editorial cautions against basing conclusions on routine health records which may not have all the information required. Nonetheless, doctors treating chronic urticaria might bear in mind the risk of osteoporosis in their patients, and counsel accordingly.  相似文献   
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