The economic burden of tuberous sclerosis complex in UK patients with renal manifestations: a retrospective cohort study in the clinical practice research datalink (CPRD)

Background: Tuberous sclerosis complex (TSC) is a multi-system genetic disorder in which renal manifestations occur in ～50% of children and 80% of adults. Since these often present alongside other manifestations, renal TSC is likely to incur significant costs. This study aims to quantify healthcare resource use (HCRU) and costs for renal TSC patients in the UK.

Methods: TSC patients in the Clinical Practice Research Datalink (CPRD) linked to Hospital Episodes Statistics were identified from January 1987–June 2013. Clinical data were extracted over the entire history and costs were reported over the most recent 3-year period. HCRU was compared with a matched comparator cohort. Incremental costs were reported and the key cost drivers by primary manifestation category were identified by regression modeling.

Results: A total of 79 renal TSC patients were identified with manifestations including chronic kidney disease stage 3–5 (with prevalence increasing with age) and renal angiomyolipoma. Renal TSC patients consistently reported greater HCRU than the comparator. Inpatient hospitalizations were more frequent for renal TSC patients (3.2 vs 1.6), but length of stay was comparable; however, 70.9% of renal TSC patients recorded no kidney-related procedures ever and averaged <1 test per year in the 3-year period. Average costs for renal TSC patients were nearly 3-fold greater than the comparator (£15,162 vs £5672). Costs increased with additional manifestation categories (£3600: only renal; £27,531: renal with ≥4 additional manifestation categories [25% of patients]). Additional nervous system and dermatology/psychiatric manifestations significantly (p?Conclusions: Renal TSC patients have greater HCRU than the general CPRD population, likely to result from progression of renal disease and additional manifestations; however, surveillance for disease progression appears to be deficient. Inadequate monitoring may contribute to a lack of co-ordinated care and increased healthcare-associated costs. Efforts should be made to follow the TSC guidelines to effectively monitor and treat patients.     

Healthcare resource utilization and costs among psoriasis patients treated with biologics,overall and by disease severity

Aims: To describe healthcare resource utilization (HCRU) and costs among biologic-treated psoriasis patients in the US, overall and by disease severity.

Materials and methods: IQVIA PharMetrics Plus administrative claims data were linked with Modernizing Medicine Data Services Electronic Health Record data and used to select adult psoriasis patients between April 1, 2010 and December 31, 2014. Eligible patients were classified by disease severity (mild, moderate, severe) using a hierarchy of available clinical measures. One-year outcomes included all-cause and psoriasis-related outpatient, emergency department, inpatient, and pharmacy HCRU and costs.

Results: This study identified 2,130 biologic-treated psoriasis patients: 282 (13%) had mild, 116 (5%) moderate, and 49 (2%) severe disease; 1,683 (79%) could not be classified. The mean age was 47.6 years; 45.4% were female. Relative to mild psoriasis patients, patients with moderate or severe disease had more median all-cause outpatient encounters (28.0 [mild] vs 32.0 [moderate], 36.0 [severe]), more median psoriasis-related outpatient encounters (6.0 [mild] vs 7.5 [moderate], 8.0 [severe]), and a higher proportion of overall claims for medications that were psoriasis-related (28% [mild] vs 37% [moderate], 34% [severe]). Relative to mild psoriasis patients, patients with moderate or severe disease had higher median all-cause total costs ($37.7k [mild] vs $42.3k [moderate], $49.3k [severe]), higher median psoriasis-related total costs ($32.7k [mild] vs $34.9k [moderate], $40.5k [severe]), higher median all-cause pharmacy costs ($33.9k [mild] vs $36.5k [moderate], $36.4k [severe]), and higher median psoriasis-related pharmacy costs ($32.2k [mild] vs $33.9k [moderate], $35.6k [severe]).

Limitations: The assessment of psoriasis disease severity may not have necessarily coincided with the timing of biologic use. The definition of disease severity prevented the assessment of temporality, and may have introduced selection bias.

Conclusions: Biologic-treated patients with moderate or severe psoriasis cost the healthcare system more than patients with mild psoriasis, primarily driven by higher pharmacy costs and more outpatient encounters.     

Economic evaluation among Chinese patients with nosocomial pneumonia caused by methicillin-resistant staphylococcus aureus and treated with linezolid or vancomycin: a secondary,post-hoc analysis based on a Phase 4 clinical trial study

Objective:

To assess cost-effectiveness of linezolid vs vancomycin in treating nosocomial pneumonia caused by methicillin-resistant Staphylococcus aureus (MRSA-NP) in China and the impact of renal failure on healthcare resource utilization (HCRU) and costs.

Methods:

Cost-effectiveness analysis was conducted based on data from the ZEPHyR trial, with efficacy measured by treatment success and costs calculated from HCRU. Confidence intervals (CI) for cost, efficacy and incremental cost-effectiveness ratios (ICER) were calculated by non-parametric bootstrap. Chi-square test was used for renal failure rate and t-test for HCRU/cost comparisons. Impact of renal failure was assessed using regression model.

Results:

Data from 448 patients (1:1 linezolid:vancomycin) were analyzed. More patients treated with linezolid achieved success (55% [95% CI?=?48–62%]) than with vancomycin (45% [38–52%]). Treatment cost were ¥79,551 (95% CI?=?¥72,421–¥86,680) for linezolid vs ¥77,587 (¥70,656–¥84,519) for vancomycin in Beijing, ¥90,995 (¥82,598–¥99,393) vs ¥89,448 (¥81,295–¥97,601) in Guangzhou, ¥82,383 (¥74,956–¥89,810) vs ¥80,799 (¥73,545–¥88,054) in Nanjing and ¥59,413 (¥54,366–¥64,460) vs ¥57,804 (¥52,613–¥62,996) in Xi’an. Per successful treatment, the ICER of linezolid over vancomycin were ¥19,719

(?¥143,553 to ¥320,980) (Beijing), ¥15,532 (?¥185,411 to ¥349,693) (Guangzhou), ¥15,904 (?¥161,935 to ¥314,987) (Nanjing) and ¥16,145 (?¥100,738 to ¥234,412) (Xi’an). From simulations, the majority of linezolid cases had greater efficacy and higher costs and more than one third had greater efficacy and lower costs. More vancomycin patients developed renal failure (15% vs 4%, p?<?0.001). Patients with renal failure had higher cost (Nanjng: ¥100,449 (SD?=?¥65,080) vs ¥74,944 (SD?=?¥49,632), p?=?0.002).

Conclusion:

Linezolid was more cost-effective than vancomycin in treating MRSA-NP from a Chinese payer’s perspective, and associated with less renal failure, HCRU and cost.     

Healthcare utilization and costs among chronic bronchitis patients treated with maintenance medications from a US managed care population

Abstract

Objectives:

This study aimed to examine the real-world healthcare resource utilization (HCRU) and direct costs among chronic bronchitis (CB) patients treated with chronic obstructive pulmonary disease (COPD) maintenance medications.

Methods:

This retrospective analysis utilized administrative claims data from 14 US commercial managed care plans. Eligible patients were ≥40 years old, had ≥2 years of continuous enrollment, ≥1 CB (ICD-9-CM code 491.xx) hospitalization or emergency department (ED) visit or ≥2 office visits between 1/1/2004 and 5/31/2011, and had ≥2 pharmacy fills for COPD medications during follow-up (first fill served as the index date). All-cause and COPD-related HCRU and costs were assessed during follow-up. Multivariate models were utilized to identify predictors of total costs.

Results:

Treated CB patients (n?=?17,382; 50.6% female; mean age 66.7 (SD?=?11.4) years) had a mean of 7.6 (SD?=?6.3) COPD maintenance medication fills during follow-up. Overall, 32.6% of patients had ≥1 COPD-related inpatient hospitalizations, 12.9% had ≥1 ED visit, and 81.8% had ≥1 office visit. Mean all-cause and COPD-related total costs were $25,747 (SD?=?$51,105) and $12,609 (SD?=?$36,801), respectively, during follow-up. Among the sub-group with ≥1 exacerbation during baseline year, 42.3% had ≥1 COPD-related inpatient hospitalization, 18.5% had ≥1 ED visit, and 88.2% had ≥1 office visit. Mean follow-up all-cause and COPD-related total costs were $29,861 (SD?=?$49,799) and $16,784 (SD?=?$34,170), respectively. The number of baseline exacerbations was a significant predictor of all-cause and COPD-related total costs during follow-up.

Limitations:

This study lacked standard measures of CB severity; however, severity proxies were utilized.

Conclusion:

HCRU and costs among CB patients were substantial during follow-up, despite treatment with COPD maintenance medications. Additional interventions aiming to prevent or reduce HCRU and costs among CB patients warrant exploration.     

Predictors of government subsidized pharmaceutical use in patients with diabetes or cardiovascular disease in a primary care setting: evidence from a prospective randomized trial

Abstract

Objectives:

This study uses data from a prospective randomized controlled trial to estimate predictors of pharmaceutical expenditure in diabetes (DM) or cardiovascular disease (CVD) patients. Identifying drivers of pharmaceutical use and the extent to which they are modifiable may inform cost-effective policy-making.

Methods:

The trial followed 260 patients aged >18 years (mean 68) from three general practices for 12 months. Patients had type 2 diabetes (90 patients) or cardiovascular disease (170 patients). Costs for pharmaceuticals prescribed on the Pharmaceutical Benefits Scheme (PBS) were obtained retrospectively at 12 months. Sociodemographic data and health-related quality-of-life (QoL) were recorded from questionnaires. Clinical measures (including body mass index (BMI), blood pressure, high and low density lipoprotein (LDL), and HbA1c) were also collected.

Results:

Mean pharmaceutical costs for DM patients (AU$4119) was greater than CVD patients (AU$2424). The largest contributor to costs in both groups was pharmaceuticals used for management of conditions other than CVD or DM. QoL (EQ5D) and BMI were significant predictors of costs in both groups. A history of cardiac events, HbA1c, age, and unemployment were significant predictors of costs in the DM group. A diagnosis of heart failure, frequency of hospital admissions, and LDL levels were significant predictors of costs in the CVD group. Roughly one third of total variation of costs can be explained by the regressors in both models.

Limitations:

Generalizability will be limited as data was derived from a trial and the study was not powered for this post-hoc analysis. Missing data imputation and self-reporting bias may also impact on results.

Conclusions:

Factors such as QoL BMI, HbA1c levels, and a history of cardiac events are significant predictors of costs. The results suggest there may be a place for interventions that improve quality-of-life and concurrently reduce pharmaceutical costs in patients with CVD or DM.     

Value of clinical trial narrative data to estimate the costs of adverse event management: a feasibility study

Abstract

Aims: The objective of this feasibility study was to determine the extent to which data from randomized controlled trials (RCTs) may serve as a useful source for collecting health care resource use (HCRU) for the purposes of estimating costs of managing adverse events (AEs), specifically, grade 3–4 nausea and thrombocytopenia, which may be experienced during chemotherapy treatment.

Materials and Methods: The feasibility study was conducted in four steps: (1) HCRU data were extracted from patient narratives in four phase 3 RCTs in non–small cell lung cancer; (2) missing HCRU data were imputed; (3) unit costs were applied to the resulting HCRU data set and costs of managing AEs were estimated; and (4) the overall utility of using RCT data as a source for estimating costs of AEs was evaluated.

Results: 33 nausea and 68 thrombocytopenia AEs met eligibility criteria and were evaluated in this study. Medication usage was recorded as a treatment in 76% of nausea AEs, although only 14% of the instances of medication usage included the minimum data elements required for costing. Platelet transfusions were provided in 24% of thrombocytopenia AEs; however, in only one instance were the minimum data elements recorded. Of nausea and thrombocytopenia AEs, 18% and 72%, respectively, required no missing data assumptions or imputation.

Limitations: Only two AEs were considered, and they may not be representative of all AEs in terms of suitability for use in estimating HCRU and costs of managing AEs. Not all grade 3–4 AEs met the criteria for requiring a patient narrative. HCRU data in the narratives were incomplete.

Conclusions: The usefulness of RCTs for estimating the costs of AEs may be improved by using a standardized form to collect HCRU data for key AEs, including an appropriate level of detail required to estimate costs of managing the AEs.     

Clinical,humanistic, and economic outcomes between drug-eluting stent (DES) and bare metal stent (BMS): 18-month follow-up study

Background: Percutaneous coronary intervention (PCI) is one of the most performed interventions for ischemic heart diseases. In Hong Kong, the total number of patient discharges and deaths for ischemic heart diseases in 2009 was 33,363, including 4,360 deaths. There are over 5,000 cases of PCI yearly. This study aimed to compare clinical, economic, and humanistic outcomes among patients receiving drug-eluting stent (DES) or bare metal stent (BMS) in Hong Kong.

Methods: Patients who received stent implantation between September 15, 2009 and October 11, 2010 in Prince of Wales Hospital, Hong Kong, were recruited and followed for 18 months. Occurrence of major adverse cardiac events (cardiac death, non-fatal MI, TLR and TVR) was employed as the clinical outcome measurements. Improvement in quality-of-life by stent interventions was measured as quality-adjusted life-year (QALY). EQ-5D questionnaire was adopted to assess the QALY gained. Cost-utility analysis and cost-effectiveness analysis for BMS and DES were employed as the economic outcome measurement.

Results: Six hundred and eighty-four patients (DES?=?402; BMS?=?282) were included. From 0–18 months, TLR rate (2.7% vs 3.5%, p?=?.549) and TVR rate (3.7% vs 6.4%, p?=?.111) were lower in the DES group, but without statistical significance. EQ VAS (71.06?±?14.56 vs 71.07?±?16.57, p?=?.998) and utility score (0.81?±?0.17 vs 0.78?±?0.16, p?=?.162) were comparable between DES and BMS group. Overall, the cost per QALY gained was HKD + 1,178,100 and ICER was HKD + 187,000 (1USD?=?7.8 HKD).

Conclusions: No significant difference in TVR, TLR rates, EQ VAS, and utility score was found between the DES and BMS group. The higher cost of index procedure for the DES group was found to be partly offset by reduced cost of follow-up, offering cost-effectiveness in ACS patients, predominantly in STEMI patients. DES was recommended for STEMI patients.     

The cost-effectiveness and budget impact of stepwise addition of bolus insulin in the treatment of type 2 diabetes: evaluation of the FullSTEP trial

Background and aims:

Intensification of basal insulin-only therapy in type 2 diabetes is often achieved through addition of bolus insulin 3-times daily. The FullSTEP trial demonstrated that stepwise addition (SWA) of bolus insulin aspart was non-inferior to full basal-bolus (FBB) therapy and reduced the rate of hypoglycemia. Here the cost-effectiveness and budget impact of SWA is evaluated.

Methods:

Cost-effectiveness and budget impact models were developed to assess the cost and quality-of-life (QoL) implications of intensification using SWA compared with FBB in the US setting. At assessment, SWA patients added one bolus dose to their current regimen if the HbA1c target was not met. SWA patients reaching three bolus doses used FBB event rates. Outcomes were evaluated at trial end and projected annually up to 5 years. Models captured hypoglycemic events, the proportion meeting HbA1c target, and self-measured blood glucose. Event rates and QoL utilities were taken from trial data and published literature. Costs were evaluated from a healthcare-payer perspective, reported in 2013 USD, and discounted (like clinical outcomes) at 3.5% annually. This analysis applies to patients with HbA1c 7.0–9.0% and body mass index <40?kg/m².

Results:

SWA was associated with improved QoL and reduced costs compared with FBB. Improvement in QoL and cost reduction were driven by lower rates of hypoglycemia. Sensitivity analyses showed that outcomes were most influenced by the cost of bolus insulin and QoL impact of symptomatic hypoglycemia. Budget impact analysis estimated that, by moving from FBB to SWA, a health plan with 77,000 patients with type 2 diabetes, of whom 7.8% annually intensified to basal-bolus therapy, would save USD 1304 per intensifying patient over the trial period.

Conclusions:

SWA of bolus insulin should be considered a beneficial and cost-saving alternative to FBB therapy for the intensification of treatment in type 2 diabetes.     

Health-related quality-of-life in patients after elective surgery for abdominal aortic aneurysm

Abstract

Purpose:

The purpose of this study was to describe the health-related quality-of-life (QoL) in patients after elective surgery for abdominal aortic aneurysm (AAA) compared to a normal population and to study the association between QoL and number of years since surgery.

Methods:

All Danish men who underwent elective surgery for AAA at the age of 65 or more in the period from 1989–2007 in Denmark were invited to participate in the survey. Of 722 patients, 375 were alive and 328 (87%) responded. The instruments EQ-5D (European Quality of life), EQ-VAS and SF-12 (Short Form Health Survey) were applied for measuring health-related QoL. Multiple regression analysis was used to study the association between QoL and number of years since AAA surgery.

Results:

A significantly poorer QoL was found in patients having had AAA surgery compared to the normal population as measured with the SF-12 and the EQ-VAS, but not with EQ-5D. A negative association between QoL and years following surgery was found with EQ-VAS and SF-12 (PCS), but not with the other instruments.

Discussion:

Factors such as selection bias because of mortality and non-response may have resulted in an over-estimate of the QoL in patients having had AAA surgery, thus the difference in QoL compared to the normal population was probably under-estimated. The cross-section design was inefficient for the study of the association between QoL and years since surgery, and EQ-5D may be an insensitive instrument for measuring QoL in AAA patients after surgery.

Limitations:

The main limitation of the study was the cross-sectional design. Males with a higher risk of death were under-represented in the sample.

Conclusion:

A poorer quality-of-life was found in patients having had elective AAA surgery compared to the normal population.     

Overall survival,costs, and healthcare resource use by line of therapy in Medicare patients with newly diagnosed metastatic urothelial carcinoma

Aims: Medicare patients with metastatic or surgically unresectable urothelial carcinoma (mUC) often receive platinum-based chemotherapy as first line of therapy (LOT), but invariably progress, requiring additional LOTs and healthcare resource use (HCRU). To better understand the evolving mUC treatment landscape, the economic burden of chemotherapy-based mUC treatments among US Medicare patients was estimated.

Methods: Newly diagnosed Medicare patients with mUC were identified from the Surveillance, Epidemiology, and End Results (SEER)-Medicare database. Patients were followed from diagnosis to death, disenrollment, or end of study to characterize LOTs (first [LOT1], second [LOT2], and third or greater [LOT3+]). Kaplan-Meier methods were used to estimate overall survival (OS) by LOT. HCRU and mean costs were reported over the follow-up period, LOT duration, and maximum LOT received.

Results: Among 1,873 eligible patients with mUC (median age?=?77?years; median follow-up?=?7.5?months), 1,035 (55%) received no chemotherapy. Among chemotherapy-treated patients, 61% had LOT1 only, 25% had LOT1 and LOT2 only, and 14% had LOT3+. Median OS was 8.1?months, range was 4.3 (untreated) to 29.8 (LOT3+) months. HCRU frequency increased with additional LOTs. Mean cumulative per-patient cost was $82,912 for all patients, increasing with additional LOTs (untreated?=?$57,207; LOT1?=?$99,213; LOT2?=?$125,190; LOT3+?=?$163,884). Mean per patient per month cost was $18,827 for all patients, decreasing with increasing number of LOTs received (untreated?=?$27,211; LOT1?=?$9,601; LOT2?=?$7,325; LOT3+?=?$6,017).

Limitations: Potential for treatment misclassification when using the algorithm defining LOTs and non-generalizability of results to younger patients.

Conclusions: Over 50% of Medicare patients with mUC received no chemotherapy. Among chemotherapy-treated patients, most received only one LOT. Additional LOTs led to higher mean costs and HCRU, but as patients were followed longer, monthly costs decreased. As treatments evolve to include immuno-oncology agents, these findings provide a clinically relevant economic benchmark for mUC treatment across different traditional LOTs.     

Costs of hospital events in patients with metastatic colorectal cancer

Abstract

Background:

In the last decade, the number of new agents, including monoclonal antibodies, being developed to treat metastatic colorectal cancer (mCRC) increased rapidly. While improving outcomes, these new treatments also have distinct and known safety profiles with toxicities that may require hospitalizations. However, patterns and costs of hospitalizations of toxicities of these new ‘targeted’ drugs are often unknown.

Objective:

This study aimed to estimate the costs of hospital events associated with adverse events specified in the ‘Special Warnings and Precautions for Use’ section of the European Medicinal Agency Summary of Product Characteristics for bevacizumab, cetuximab, and panitumumab, in patients with mCRC.

Methods:

From the PHARMO Record Linkage System (RLS), patients with a primary or secondary hospital discharge code for CRC and distant metastasis between 2000–2008 were selected and defined as patients with mCRC. The first discharge diagnosis defining metastases served as the index date. Patients were followed from index date until end of data collection, death, or end of study period, whichever occurred first. Hospital events during follow-up were identified through primary hospital discharge codes. Main outcomes for each event were length of stay and costs per hospital admission.

Results:

Among 2964 mCRC patients, 271 hospital events occurred in 210 patients (mean [SD] duration of follow-up: 34 [31] months). The longest mean (SD) length of stay per hospital admission were for stroke (16 [33] days), arterial thromboembolism (ATE) (14 [21] days), wound-healing complications (WHC), acute myocardial infarction (AMI), congestive heart failure (CHF), and neutropenia (all 9 days; SD 5–15). Highest mean (SD) costs per admission were for stroke (€13,500 [€28,800]), ATE (€13,300 [€18,800]), WHC (€10,800 [€20,500]).

Limitations:

Although no causal link could be identified between any specific event and any specific treatment, data from this study are valuable for pharmacoeconomic evaluations of newer treatments in mCRC patients.

Conclusions:

Inpatient costs for events in mCRC patients are considerable and vary greatly.     

Treatment experience,burden, and unmet needs (TRIBUNE) in Multiple Sclerosis study: the costs and utilities of MS patients in The Netherlands

Abstract

Background:

Multiple sclerosis (MS) is an important, highly disabling neurological disease, common among young adults in The Netherlands. Nevertheless, only a few studies to date have measured the burden imposed by MS on society in The Netherlands.

Objectives:

To estimate the cost and quality-of-life associated with MS in The Netherlands, while focusing on the burden of relapses and increasing disease severity.

Methods:

MS patients in The Netherlands (n?=?263) completed a web-based questionnaire which captured information on demographics, disease characteristics and severity (Expanded Disability Status Scale [EDSS]), co-morbidities, relapses, resource consumption, utilities, fatigue and activities of daily living (ADL).

Results:

Most patients included in the study were receiving treatment for MS (76% of the sample). The mean cost per patient per year increased with worsening disability and was estimated at €30,938, €51,056, and €100,469 for patients with mild (EDSS 0–3), moderate (EDSS 4–6.5), and severe (EDSS 7–9) disability, respectively. The excess cost of relapses was estimated at €8195 among relapsing-remitting patients with EDSS score ≤5. The quality-of-life of patients decreased with disease progression and existence of relapses.

Conclusions:

The cost of MS in The Netherlands was higher compared to the results of previous studies. The TRIBUNE study provides an important update on the economic burden of MS in The Netherlands in an era of more widespread use of disease-modifying therapies. It explores the cost of MS linked to relapses and disease severity and examines the impact of MS on additional health outcomes beyond utilities such as ADL and fatigue.

Conclusions:

Study limitations:     

Hospitalization rates in patients switched from oral anti-psychotics to aripiprazole once-monthly: final efficacy analysis

Abstract

Objective:

To compare hospitalization rates in patients with schizophrenia treated prospectively with aripiprazole once-monthly 400?mg (AOM 400; an extended-release injectable suspension) vs the same patients’ retrospective rates with their prior oral anti-psychotic therapy.

Research design and methods:

Multi-center, open-label, mirror-image, naturalistic study in a community setting in North America. Patients who required a change in treatment and/or would benefit from long-acting injectable anti-psychotic therapy were treated prospectively for 6 months with AOM 400. Retrospective data on hospitalization rates were obtained.

Clinical trial registration:

ClinicalTrials.gov: NCT01432444.

Main outcome measures:

The proportion of patients with ≥1 psychiatric inpatient hospitalization with oral anti-psychotic therapy examined retrospectively (months –4 to –1 before oral conversion) and after switching to AOM 400 (months 4–6 after initiating AOM 400).

Results:

Psychiatric hospitalization rates were significantly lower when patients were treated with AOM 400 compared with oral anti-psychotic therapy both in the 3-month primary efficacy sample (2.7% [n?=?9/336] vs 27.1% [n?=?91/336], respectively; p?<?0.0001) and in the total sample (6-month prospective rate: 8.8% [n?=?38/433] vs 6-month retrospective rate: 38.1% [n?=?165/433]; p?<?0.0001). Discontinuations due to adverse events (AEs) during cross-titration were lower in patients cross-titrated on oral aripiprazole for >1 and <4 weeks (2.9% [n?=?7/239]) compared with patients cross-titrated for ≤1 week (10.4% [n?=?5/48]). The most common treatment-emergent AEs during the prospective treatment phase were insomnia (6.7% [n?=?29/431]) and akathisia (6.5% [n?=?28/431]). Patient-rated injection-site pain decreased from the first injection to the last visit.

Conclusions:

In a community setting, patients with schizophrenia demonstrated significantly lower psychiatric hospitalization rates after switching from their prior oral anti-psychotic therapy to AOM 400. Patients served as their own control, and thus an active control group was not included in this study. Confounding factors, such as insurance coverage and availability of hospital beds, were not examined here and deserve further consideration.     

Economic burden associated with inadequate antidepressant medication management among patients with depression and known cardiovascular diseases: insights from a United States–based retrospective claims database analysis

Abstract

Aims: The current study examined the association between insufficient major depressive disorder (MDD) care and healthcare resource use (HCRU) and costs among patients with prior myocardial infarction (MI) or stroke.

Methods: This was a retrospective study conducted using the MarketScan Claims Database (2010–2015). The date of the first MI/stroke diagnosis was defined as the cardiovascular disease (CVD) index date and the first date of a subsequent MDD diagnosis was the index MDD date. Adequacy of MDD care was assessed during the 90 days following the index MDD date (profiling period) using 2 measures: dosage adequacy (average fluoxetine equivalent dose of ≥20?mg/day for nonelderly and ≥10?mg/day for elderly patients) and duration adequacy (measured as the proportion of days covered of 80% or higher for all MDD drugs). Study outcomes included all-cause and CVD-related HCRU and costs which were determined from the end of the profiling period until the end of study follow-up. Propensity-score adjusted generalized linear models (GLMs) were used to compare patients receiving adequate versus inadequate MDD care in terms of study outcomes.

Results: Of 1,568 CVD patients who were treated for MDD, 937 (59.8%) were categorized as receiving inadequate MDD care. Results from the GLMs suggested that patients receiving inadequate MDD care had 14% more all-cause hospitalizations, 4% more all-cause outpatient visits, 17% more CVD-related outpatient visits, 13% more CVD-related emergency room (ER) visits, higher per patient per year CVD-related hospitalization costs ($21,485 vs. $17,756), higher all-cause outpatient costs ($2,820 vs. $2,055), and higher CVD-related outpatient costs ($520 vs. $434) compared to patients receiving adequate MDD care.

Limitations: Clinical information such as depression severity and frailty, which are potential predictors of adverse CVD outcomes, could not be ascertained using administrative claims data.

Conclusions: Among post-MI and post-stroke patients, inadequate MDD care was associated with a significantly higher economic burden.     

Validation of economic and health outcomes simulation model of type 2 diabetes mellitus (ECHO-T2DM)

Abstract

Objective:

This study constructed the Economic and Health Outcomes Model for type 2 diabetes mellitus (ECHO-T2DM), a long-term stochastic microsimulation model, to predict the costs and health outcomes in patients with T2DM. Naturally, the usefulness of the model depends upon its predictive accuracy. The objective of this work is to present results of a formal validation exercise of ECHO-T2DM.

Methods:

The validity of ECHO-T2DM was assessed using criteria recommended by the International Society for Pharmacoeconomics and Outcomes Research/Society for Medical Decision Making (ISPOR/SMDM). Specifically, the results of a number of clinical trials were predicted and compared with observed study end-points using a scatterplot and regression approach. An F-test of the best-fitting regression was added to assess whether it differs statistically from the identity (45°) line defining perfect predictions. In addition to testing the full model using all of the validation study data, tests were also performed of microvascular, macrovascular, and survival outcomes separately. The validation tests were also performed separately by type of data (used vs not used to construct the model, economic simulations, and treatment effects).

Results:

The intercept and slope coefficients of the best-fitting regression line between the predicted outcomes and corresponding trial end-points in the main analysis were ?0.0011 and 1.067, respectively, and the R² was 0.95. A formal F-test of no difference between the fitted line and the identity line could not be rejected (p?=?0.16). The high R² confirms that the data points are closely (and linearly) associated with the fitted regression line. Additional analyses identified that disagreement was highest for macrovascular end-points, for which the intercept and slope coefficients were 0.0095 and 1.225, respectively. The R² was 0.95 and the estimated intercept and slope coefficients were 0.017 and 1.048, respectively, for mortality, and the F-test was narrowly rejected (p?=?0.04). The sub-set of microvascular end-points showed some tendency to over-predict (the slope coefficient was 1.095), although concordance between predictions and observed values could not be rejected (p?=?0.16).

Limitations:

Important study limitations include: (1) data availability limited one to tests based on end-of-study outcomes rather than time-varying outcomes during the studies analyzed; (2) complex inclusion and exclusion criteria in two studies were difficult to replicate; (3) some of the studies were older and reflect outdated treatment patterns; and (4) the authors were unable to identify published data on resource use and costs of T2DM suitable for testing the validity of the economic calculations.

Conclusions:

Using conventional methods, ECHO-T2DM simulated the treatment, progression, and patient outcomes observed in important clinical trials with an accuracy consistent with other well-accepted models. Macrovascular outcomes were over-predicted, which is common in health-economic models of diabetes (and may be related to a general over-prediction of event rates in the United Kingdom Prospective Diabetes Study [UKPDS] Outcomes Model). Work is underway in ECHO-T2DM to incorporate new risk equations to improve model prediction.