Development and Psychometric validation of the Diabetes Therapy-Related QOL (DTR-QOL) Questionnaire

Abstract

Objective:

We developed and evaluated the psychometric properties of the Diabetes Therapy-Related QOL (DTR-QOL) as a disease-specific, self-administered questionnaire to assess the influence of diabetes treatment on patient QOL, regardless of treatment method.

Methods:

This new questionnaire was developed and validated in a standardized manner: Item development, pilot-testing and psychometric validation. A survey was conducted using the provisional version of the questionnaire, and reliability and validity were evaluated with psychometric testing.

Results:

The provisional version of the questionnaire was generated with 29 items through literature review and pilot testing. For psychometric assessment, analyses were performed on the responses of 284 adult Japanese patients with diabetes. Factor analysis by the principal factor method with promax rotation revealed 4 factors; “burden on social activities and daily activities” (13 items), “anxiety and dissatisfaction with treatment” (8 items), “hypoglycemia” (4 items), and “satisfaction with treatment” (4 items). For reliability, the intraclass correlation was 0.92, and Cronbach’s alpha coefficient was 0.94, indicating adequate test-retest reliability and internal consistency. For known-group validity, there were significant differences in scores for following variables: age, diabetes type, HbA1c, treatment method, glycemic control, hypoglycemia, nocturnal hypoglycemia, concern about weight gain, health status (patient assessment), and degree of communication with physician.

Conclusions:

The DTR-QOL, with good reliability and validity, can assess the influence of diabetes treatment on patient QOL. The DTR-QOL can be used regardless of treatment method that patients receive, and this characteristic enables to detect a difference on patients QOL between treatment methods before and after a switch of treatment. Limitations of this study include representativeness of the patient sample. The relatively small number of patients with type 1 diabetes should be noted. Also, responsiveness of the DTR-QOL has not yet been examined.     

Development and validation of a new instrument to evaluate the ease of use of patient-controlled analgesic modalities for postoperative patients

Abstract

Objective: To describe the development and psychometric evaluation of a questionnaire assessing the ease of use that patients associate with patient-controlled analgesia (PCA) modalities.

Methods: Qualitative interviews were conducted with patients who had experience with intravenous (IV) PCA for postoperative pain management to generate items relevant to the ease of using PCA modalities. The content validity of the resulting questionnaire was examined through follow-up patient interviews, and an expert panel reviewed the questionnaire. Cognitive debriefing interviews were conducted with patients to determine the clarity and content of the instructions, items, and response scales, and the ease of completing the instrument. Psychometric evaluation was performed with patients who had undergone surgery and received IV PCA for postoperative pain management. Item and scale quality and the internal consistency reliability of the questionnaire were assessed. Construct validity was evaluated by examining the relationship between subscales of the questionnaire with patient-reported outcome measures. Known-groups validity was determined by assessing the instrument's ability to differentiate between patients with versus without an IV PCA problem. A potential limitation of this study was the exclusive sampling of patients who had experience with IV PCA.

Results: The Patient Ease-of-Care (EOC) Questionnaire included 23 items in the following subscales: Confidence with Device, Comfort with Device, Movement, Dosing Confidence, Pain Control, Knowledge/Understanding, and Satisfaction. Coefficient alpha reliability estimates were ≥0.66 for Overall EOC (includes all subscales except Satisfaction) and all EOC subscales. Construct validity was supported by the moderate relationship between the Pain Control subscale and measures of pain severity and pain interference; additional evidence of construct validity was provided by correlations of the Confidence with Device subscale, the Satisfaction subscale, and Overall EOC with measures of pain severity, pain interference, and satisfaction. Significant mean score differences were reported between participants with and without IV PCA problems for Overall EOC and for the Comfort with Device, Confidence with Device, Movement, Pain Control, and Satisfaction subscales indicating known-groups validity.

Conclusions: Results provide evidence for the reliability and validity of the Patient EOC Questionnaire as a measure of the ease of use that patients associate with PCA systems and may be useful for evaluating emerging PCA modalities.     

Cost effectiveness of escitalopram versus SNRIs in second-step treatment of major depressive disorder in Sweden

Abstract

Objectives:

Escitalopram is the S-enantiomer of citalopram and is the most discriminating of the selective serotonin reuptake inhibitors (SSRI). The aim of the current analysis was to assess the cost effectiveness of escitalopram versus the serotonin norepinephrine reuptake inhibitors (SNRI) duloxetine and generic venlafaxine as second-step treatment of major depressive disorder.

Methods:

The analysis was based on a decision analytic model. Effectiveness outcomes were quality-adjusted life-years (QALYs) and remission rates; cost outcomes were direct medical costs, including impact of treating adverse events, and indirect costs associated with lost productivity. The analysis was performed from the societal perspective in Sweden over a 6-month timeframe.

Results:

Estimated remission rates showed an incremental effectiveness in favour of escitalopram of 16.4 percentage points compared with both SNRI comparators. The escitalopram strategy was associated with a 0.025 increase in QALYs. Sensitivity analyses demonstrated that the model is robust and that escitalopram remains a cost-effective option when considering future predicted price reductions of generic venlafaxine.

Limitations:

The main limitation in this study was the lack of data available for second-step treatment. The remission rates, which are a key input to the model, were obtained from a relatively small sample of patients on second-step treatment and there are no published relapse rates for second-step treatment. The model also assumed that there was no difference in the adverse event (AE) rates between treatments after the first 8 weeks.

Conclusions:

This cost-effectiveness analysis indicates that, at a willingness-to-pay threshold of £30,000, escitalopram is the most cost-effective second-step treatment option for MDD in Sweden in over 85% cases compared with both venlafaxine and with duloxetine. Benefits for escitalopram included both increased effectiveness and reduced overall costs. The major contributing costs were those associated with productivity loss.

The model was shown to have internal validity and robustness through the use of stochastic simulations and sensitivity analyses, which were conducted around the key efficacy parameters.     

Direct treatment cost of atrial fibrillation in the elderly American population: a Medicare perspective

Abstract

Objective: Although atrial fibrillation (AF) is the most commonly sustained arrhythmia in adults, few studies have examined the direct treatment cost of AF.

Methods: A Medicare database of a 5% random national sample of all beneficiaries was used to identify patients diagnosed with AF in 2003 and to follow them for 1 year after diagnosis. These patients were matched on a 1:1 basis by age, gender and race. The incremental cost of treating AF was calculated with multivariate regression models adjusting for covariates.

Results: In total, 55,260 subjects developed new AF, of which 69% were ≥75 years old, 54% were female and 91% were White. The adjusted mean incremental treatment cost of AF was $14,199 (95% confidence interval $13,201–15,001; p<0.01). Some of this cost was attributable to the incidence of stroke and heart failure at the 1-year post-AF diagnosis. A significantly higher proportion of AF patients experienced stroke (23.1 vs. 13.3%; p<0.01) and heart failure (36.7 vs. 10.4%; p<0.01) compared with Medicare beneficiaries without AF.

Conclusions: Mean incremental direct treatment costs for Medicare beneficiaries with AF were higher than previously reported. Interventions that can reduce the incidence of AF and its complications may also reduce the national economic impact of AF.     

Validation of the Bowel Function Index to detect clinically meaningful changes in opioid-induced constipation

Abstract

Objective: The Bowel Function Index (BFI) is a clinician-administered, patient-reported, 3-item questionnaire to evaluate opioid-induced constipation in cancer and non-cancer chronic pain patients. The objective of the present analysis was to evaluate the psychometric characteristics of the BFI using data from clinical studies of oral prolonged release (PR) oxycodone/naloxone.

Methods: OXN2401 was a multicenter, controlled, randomized, double-blind, parallel-group study including oral PR oxycodone combined with oral PR naloxone as well as oral PR oxycodone combined with corresponding naloxone placebo. OXN3401 and OXN3001 were 12-week multicenter, controlled, randomized, double-blind, parallel-group studies of a fixed combination of oral PR oxycodone/naloxone versus PR oxycodone. In addition, a placebo group was included in study OXN3401. BFI psychometric characteristics (reliability, reproducibility, convergent/known groups validity, and responsiveness) were evaluated.

Results: Demographic data (n=985) were comparable and analyses indicated a high degree of internal consistency (Cronbach's alpha >0.7). Change of less than 5 points in BFI was indicative of high reproducibility. Correlations between BFI item and total scores to stool frequency were statistically significant and in the low-to-moderate range (OXN2401 –0.23 to –0.29, p < 0.001; OXN3401 range –0.26 to –0.40, p < 0.001; OXN3001 –0.14 to –0.15, p < 0.05). Data indicate that a BFI score change of ≥12 points represents a clinically meaningful change in constipation.

Limitations: This publication for validation of BFI only includes data from three clinical trials. However, another publication of an additional specifically designed cross-sectional validation study is in preparation.

Conclusion: The BFI is a valid and reliable instrument for the assessment of opioid-induced constipation in chronic pain patients. Psychometric analyses from clinical trials support the BFI's psychometric properties.     

Cost-effectiveness of rivaroxaban versus warfarin for stroke prevention in non-valvular atrial fibrillation in the Japanese healthcare setting

Abstract

Aims: This article aimed to examine the cost-effectiveness of rivaroxaban in comparison to warfarin for stroke prevention in Japanese patients with non-valvular atrial fibrillation (NVAF), from a public healthcare payer’s perspective.

Materials and methods: Baseline event risks were obtained from the J-ROCKET AF trial and the treatment effect data were taken from a network meta-analysis. The other model inputs were extracted from the literature and official Japanese sources. The outcomes included the number of ischaemic strokes, myocardial infarctions, systemic embolisms and bleedings avoided, life-years, quality-adjusted life-years (QALYs), incremental costs and incremental cost-effectiveness ratio (ICER). The scenario analysis considered treatment effect data from the same network meta-analysis.

Results: In comparison with warfarin, rivaroxaban was estimated to avoid 0.284 ischaemic strokes per patient, to increase the number of QALYs by 0.535 per patient and to decrease the total costs by ¥118,892 (€1,011.11) per patient (1 JPY = 0.00850638 EUR; XE.com, 7 October 2019). Consequently, rivaroxaban treatment was found to be dominant compared to warfarin. In the scenario analysis, the ICER of rivaroxaban versus warfarin was ¥2,873,499 (€24,446.42) per QALY.

Limitations: The various sources of data used resulted in the heterogeneity of the cost-effectiveness analysis results. Although, rivaroxaban was cost-effective in the majority of cases.

Conclusion: Rivaroxaban is cost-effective against warfarin for stroke prevention in Japanese patients with NVAF, giving the payer WTP of 5,000,000 JPY.     

Clinical and economic impact of rivaroxaban on the burden of atrial fibrillation: The case study of Japan

Objectives: Atrial fibrillation (AF) affects an estimated 1.5 million individuals in Japan, increasing their stroke risk and imposing considerable costs on the Japanese healthcare system. To reduce stroke incidence, guidelines recommend using anticoagulants in moderate-to-high risk non-valvular AF (NVAF) patients; however, many patients receive no treatment, aspirin only, or remain poorly-controlled on vitamin K antagonists (VKAs) due to high VKA discontinuation rates and non-adherence to guidelines. A prevalence-based Markov model was developed to estimate the clinical and budgetary impact of treating these patients with Xarelto^TM (rivaroxaban, Bayer AG) in Japan.

Methods: Population, baseline risk of events, and associated management costs were estimated using data from Japanese publications where available. Treatment efficacy and safety were derived from published data and the J-ROCKET AF trial. Drug and physician visit costs were based on data from the Ministry of Health, Labor, and Welfare, the J-ROCKET AF trial, and Japanese clinical guidelines.

Results: This model demonstrates that increased use of rivaroxaban in inadequately-managed NVAF patients could avoid 456 081 non-fatal ischemic strokes (IS) and 76 975 cardiovascular deaths over 10 years in Japan. This clinical benefit offsets the increased incidence of myocardial infarctions and anticoagulant-related bleeding. Decreased event costs could lead to a ¥188.4 billion decrease in net spending over the analysis time horizon.

Conclusions: Introducing rivaroxaban may decrease the burden of NVAF in Japanese society. From a clinical perspective, the reduction in IS and embolic events outweighs the increased risk of anticoagulant-related bleeding; from an economic perspective, reduced event costs offset drug and physician visit costs, resulting in cost savings.     

Psychometric evaluation of Global Evaluation of Treatment Effectiveness: a tool to assess patients with moderate-to-severe allergic asthma

Summary

Recent trials of treatment for patients with moderate to severe allergic (immunoglobulin E-mediated) asthma have included a Global Evaluation of Treatment Effectiveness (GETE), which is completed by both the patient and the physician. The current analyses were designed to explore some psychometric properties of this measure using secondary analysis of trial data.

The results found some evidence to support the psychometric properties of the measure, and some evidence that was more equivocal. The GETE had low to moderate rates of missing data. There was also a high level of agreement between the two measures. The five levels of the GETE were significantly differentiated in terms of symptom scores, use of rescue medication and forced expiratory volume (physician version only), and health-related quality of life (Asthma Quality of Life Questionnaire).

The GETE is a simple measure of perceived treatment effectiveness that reflects clinical outcomes and health-related quality of life, and is not burdensome for patients or physicians.     

Further validation of the psychometric properties of the Bowel Function Index for evaluating opioid-induced constipation (OIC)

Abstract

Objective:

The BFI (Bowel Function Index) is a 3-item questionnaire for assessing opioid-induced constipation (OIC). The aim of this study was to contribute to the validation of the psychometric properties of the BFI by confirming a constipation threshold, and through correlation with other validated tools: KESS (Knowles Eccersley Scott Symptom) score and generic (12-Item Short Form Health Survey, SF-12) and specific (Patient Assessment of Constipation–Quality of Life, PAC-QoL) quality-of-life scores.

Methods:

A survey on opioid-requiring cancer-patients was carried out in France. A questionnaire was filled out for all patients that recorded their demographic characteristics, an assessment of their constipation using BFI and KESS scores, and included a self-assessment of quality-of-life using PAC-QoL and SF-12. Correlation of BFI with KESS, PAC-QoL, and SF-12 was investigated.

Results:

Five hundred and twenty patients participated in the entire data collection with no loss. BFI was shown to be statistically correlated (r?=?0.571; p?<?0.0001) with the KESS score and matches up with PAC-QoL and to a lesser extent with the SF-12 generic quality-of-life questionnaire. A BFI threshold of 27–29 to discriminate constipated from non-constipated patients was confirmed.

Key limitations:

This cross-sectional study in a selected population of cancer pain patients has validated the psychometric properties of the BFI. Further confirmation of the validity of the BFI could be sought through the use of longitudinal studies, and larger populations, such as non-cancer pain patients treated with opioids.

Conclusion:

This study contributes to the validation of the psychometric properties of the BFI. It confirms the BFI as an easy-to-use tool to assess constipation and its impact on quality-of-life in chronic pain patients.     

Temporal pattern and costs of rehospitalization in atrial fibrillation/atrial flutter patients with one or more additional risk factors

Abstract

Objectives:

The ATHENA study showed that use of dronedarone reduced rates of first cardiovascular (CV) hospitalization in atrial fibrillation/flutter (AF/AFL) patients. AF is associated with high costs to payers, which are driven by high rates of hospitalization. This retrospective cohort study examined readmission patterns and costs to US payers in real-world AF/AFL patients with ≥1 additional risk factor (ARF).

Methods:

Patients hospitalized (January 2005–March 2008) with AF/AFL as primary diagnosis and having ≥1 year of health coverage, before and after their first (index) admission, were identified in the PharMetrics Patient-Centric database. As in the ATHENA study, patients had to be ≥75 years of age or ≥70 years, with ≥1 ARF. Rehospitalization patterns (all-cause, all CV-related [including AF/AFL] and AF/AFL-related alone) were examined over 1 year post-index, and costs of index vs later AF/AFL admissions compared.

Results:

The study included 3498 patients (mean 80 [SD 7.6] years; 42.4% men). Over 1 year, 1389 patients (39.7%) were rehospitalized for any cause (mean 1.7 [SD 1.3] events/patient), with 1223 patients (35.0%) undergoing CV-related (mean 1.6 [SD 1.0] events/patient) and 935 (26.7%) undergoing AF-related rehospitalization (mean 1.4 [SD 0.8] events/patient). Common causes of CV-related readmissions (primary diagnosis) were AF/AFL (47.5%), congestive heart failure (CHF) (9.9%), coronary artery disease (7.4%), and stroke/transient ischemic attack (6.2%). Readmission rates at 3 months were 16.2% (all-cause), 14.3% (all CV-related including AF/AFL), and 10.5% (AF/AFL-related alone). AF/AFL readmissions (primary diagnosis) were longer than initial hospitalizations (mean total 6.9 [SD 12.9] vs 4.3 [SD 5.1] days, p?<?0.0001) and more costly (median $1819 [25th percentile $1066, 75th percentile $5623] vs $1707 [25th percentile $1102, 75th percentile $4749]).

Limitations:

This study excluded patients with pre-existing CHF, did not require electrocardiogram confirmation of AF/AFL diagnosis, and did not distinguish between paroxysmal, persistent, and permanent AF.

Conclusions:

AF/AFL patients with ≥1 ARF have high readmission rates. AF/AFL-related readmissions incur higher costs than the initial AF/AFL admissions.     

The cost-effectiveness of dulaglutide versus insulin glargine for the treatment of type 2 diabetes mellitus in Japan

Aims: Dulaglutide is a new once weekly glucagon-like peptide-1 (GLP-1) receptor agonist administered via a disposable auto-injection pen for the management of type 2 diabetes mellitus (T2DM). The objective of this study was to estimate the cost-effectiveness of dulaglutide vs insulin glargine for the management of T2DM from a Japanese healthcare perspective, in accordance with recently approved Japanese Cost-Effectiveness Guidelines.

Methods: The IQVIA CORE Diabetes Model (version 9) was used to estimate the long-term costs and effects of treatment with dulaglutide and insulin glargine. Direct comparative data from the Araki 2015 trial (NCT01584232) was used to inform the analysis. Costs associated with treatment and complications were derived from Japanese sources wherever possible and inflated to 2015 Japanese Yen (JPY). Utilities were based upon a European systematic review of diabetes utilities and adjusted for use in a Japanese population. One-way and probabilistic sensitivity analyses (OWSA and PSA) were conducted on all inputs and key modeling assumptions.

Results: Dulaglutide 0.75?mg was associated with higher quality-adjusted life years (QALYs), life years (LYs), and total costs, compared to insulin glargine, resulting in an incremental cost-effectiveness ratio (ICER) of 416,280 JPY/QALY gained. Treatment with dulaglutide increased the time alive and free from diabetes-related complications by 4 months. OWSA and PSA indicated that results were robust to plausible variations in input parameters and modeling assumptions.

Limitations: Key limitations of this study are similar to other cost-utility analyses of diabetes, including the extrapolation of short-term clinical trial data into lifelong durations. In addition, due to the lack of robust published Japanese data, some values were derived from non-Japanese sources.

Conclusions: This analysis suggests that dulaglutide 0.75?mg may be a cost-effective treatment alternative to insulin glargine for patients with T2DM in Japan.     

Cost-effectiveness of targeted screening for the identification of patients with atrial fibrillation: evaluation of a machine learning risk prediction algorithm

Abstract

Aims: As many cases of atrial fibrillation (AF) are asymptomatic, patients often remain undiagnosed until complications (e.g. stroke) manifest. Risk-prediction algorithms may help to efficiently identify people with undiagnosed AF. However, the cost-effectiveness of targeted screening remains uncertain. This study aimed to assess the cost-effectiveness of targeted screening, informed by a machine learning (ML) risk prediction algorithm, to identify patients with AF.

Methods: Cost-effectiveness analyses were undertaken utilizing a hybrid screening decision tree and Markov disease progression model. Costs and outcomes associated with the detection of AF compared traditional systematic and opportunistic AF screening strategies to targeted screening informed by a ML risk prediction algorithm. Model analyses were based on adults ≥50?years and adopted the UK NHS perspective.

Results: Targeted screening using the ML risk prediction algorithm required fewer patients to be screened (61 per 1,000 patients, compared to 534 and 687 patients in the systematic and opportunistic strategies) and detected more AF cases (11 per 1,000 patients, compared to 6 and 8?AF cases in the systematic and opportunistic screening strategies). The targeted approach demonstrated cost-effectiveness under base case settings (cost per QALY gained of £4,847 and £5,544 against systematic and opportunistic screening respectively). The targeted screening strategy was predicted to provide an additional 3.40 and 2.05 QALYs per 1,000 patients screened versus systematic and opportunistic strategies. The targeted screening strategy remained cost-effective in all scenarios evaluated.

Limitations: The analysis relied on assumptions that include the extended period of patient life span and the lack of consideration for treatment discontinuations/switching, as well as the assumption that the ML risk-prediction algorithm will identify asymptomatic AF.

Conclusions: Targeted screening using a ML risk prediction algorithm has the potential to enhance the clinical and cost-effectiveness of AF screening, improving health outcomes through efficient use of limited healthcare resources.     

Co-calibrating quality-of-life scores from three pulmonary disorders: implications for comparative-effectiveness research

Background Efficient use of health resources requires accurate outcome assessment. Disease-specific patient-reported outcome (PRO) measures are designed to be highly relevant to patients with a specific disease. They have advantages over generic PROs that lack relevance to patient groups and miss crucial impacts of illness. It is thought that disease-specific measurement cannot be used in comparative effectiveness research (CER). The present study provides further evidence of the value of disease-specific measures in making valid comparisons across diseases.

Methods The Asthma Life Impact Scale (ALIS, 22 items), Living with Chronic Obstructive Pulmonary Disease (LCOPD, 22 items) scale, and Cambridge Pulmonary Hypertension Outcome Review (CAMPHOR, 25 items) were completed by 140, 162, and 91 patients, respectively. The three samples were analyzed for fit to the Rasch model, then combined into a scale consisting of 58 unique items and re-analyzed. Raw scores on the three measures were co-calibrated and a transformation table produced.

Results The scales fit the Rasch model individually (ALIS Chi² probability value (p-Chi²)?=?0.05; LCOPD p-Chi^2?=^?0.38; CAMPHOR p-Chi^2?=^?0.92). The combined data also fit the Rasch model (p-Chi^2?=^?0.22). There was no differential item functioning related to age, gender, or disease. The co-calibrated scales successfully distinguished between perceived severity groups (p?<?0.001).

Limitations The samples were drawn from different sources. For scales to be co-calibrated using a common item design, they must be based on the same theoretical construct, be unidimensional, and have overlapping items.

Conclusions The results showed that it is possible to co-calibrate scores from disease-specific PRO measures. This will permit more accurate and sensitive outcome measurement to be incorporated into CER. The co-calibration of needs-based disease-specific measures allows the calculation of γ scores that can be used to compare directly the impact of any type of interventions on any diseases included in the co-calibration.     

The cost-effectiveness and monetary benefits of dabigatran in the prevention of arterial thromboembolism for patients with non-valvular atrial fibrillation in the Netherlands

Background: Atrial fibrillation (AF) causes a significant health and economic burden to the Dutch society. Dabigatran was proven to have at least similar efficacy and a similar or better safety profile when compared to vitamin K antagonists (VKAs) in preventing arterial thromboembolism in patients with AF.

Objective: To evaluate the cost-effectiveness and monetary benefit of dabigatran vs VKAs in Dutch patients with non-valvular AF. Value-based pricing considerations and corresponding negotiations on dabigatran will be explicitly considered.

Methods: The base case economic analysis was conducted from the societal perspective. Health effects and costs were analysed using a Markov model. The main model inputs were derived from the RE-LY trial and Dutch observational data. Univariate, probabilistic sensitivity, and various scenario analyses were performed.

Results: Dabigatran was cost saving compared to VKAs. A total of 4,552 QALYs were gained, and €13,892,288 was saved in a cohort of 10,000?AF patients. The economic value of dabigatran was strongly related to the costs of VKA control that are averted. Notably, dabigatran was cost saving compared to VKAs if annual costs of VKA control exceeded €159 per person, or dabigatran costs were below €2.81 per day.

Conclusion: Dabigatran was cost saving compared to VKAs for the prevention of atrial thromboembolism in patients with non-valvular AF in the Netherlands. This result appeared robust in the sensitivity analysis. Furthermore, volume based reduction of the price in the Netherlands will further increase the monetary benefits of dabigatran.     

Hospitalization expenses of acute ischemic stroke patients with atrial fibrillation relative to those with normal sinus rhythm

Background and objective: Atrial fibrillation (AF) is a risk factor for acute ischemic stroke (AIS). In mainland China, little is known of the hospitalization expenses of AIS patients with AF compared to those with normal sinus rhythm (SR). This study compared the itemized expenses of AIS patients with or without AF in a hospital in Huizhou City.

Methods: Patients hospitalized for AIS from March 2014 to March 2015 were enrolled, including 73 with AF and 751 with normal SR. Stroke severity was scored using the National Institutes of Health Stroke Scale (NIHSS). Non-parametric statistical tests were used to determine differences in hospital expenses between the two groups, of which influencing factors were analyzed using single factor and multiple stepwise linear regression analyses.

Results: Medicine was the predominant expense during hospitalization of all AIS patients. Patients with AF incurred significantly higher expenses for medicine, bed, treatments, examinations, laboratory tests, and nursing than patients with normal SR (p?p?p?Conclusion: AIS patients with AF incurred higher expenses during hospitalization compared with those with normal SR, due to greater stroke severity, higher rates of pulmonary infection and congestive heart failure, and longer hospital stays.     

Cost-effectiveness of natalizumab vs fingolimod for the treatment of relapsing-remitting multiple sclerosis: analyses in Sweden

Abstract

Objective:

To assess the cost-effectiveness of natalizumab vs fingolimod over 2 years in relapsing-remitting multiple sclerosis (RRMS) patients and patients with rapidly evolving severe disease in Sweden.

Methods:

A decision analytic model was developed to estimate the incremental cost per relapse avoided of natalizumab and fingolimod from the perspective of the Swedish healthcare system. Modeled 2-year costs in Swedish kronor of treating RRMS patients included drug acquisition costs, administration and monitoring costs, and costs of treating MS relapses. Effectiveness was measured in terms of MS relapses avoided using data from the AFFIRM and FREEDOMS trials for all patients with RRMS and from post-hoc sub-group analyses for patients with rapidly evolving severe disease. Probabilistic sensitivity analyses were conducted to assess uncertainty.

Results:

The analysis showed that, in all patients with MS, treatment with fingolimod costs less (440,463 Kr vs 444,324 Kr), but treatment with natalizumab results in more relapses avoided (0.74 vs 0.59), resulting in an incremental cost-effectiveness ratio (ICER) of 25,448 Kr per relapse avoided. In patients with rapidly evolving severe disease, natalizumab dominated fingolimod. Results of the sensitivity analysis demonstrate the robustness of the model results. At a willingness-to-pay (WTP) threshold of 500,000 Kr per relapse avoided, natalizumab is cost-effective in >80% of simulations in both patient populations.

Limitations:

Limitations include absence of data from direct head-to-head studies comparing natalizumab and fingolimod, use of relapse rate reduction rather than sustained disability progression as the primary model outcome, assumption of 100% adherence to MS treatment, and exclusion of adverse event costs in the model.

Conclusions:

Natalizumab remains a cost-effective treatment option for patients with MS in Sweden. In the RRMS patient population, the incremental cost per relapse avoided is well below a 500,000 Kr WTP threshold per relapse avoided. In the rapidly evolving severe disease patient population, natalizumab dominates fingolimod.     

A real-world,multi-site,observational study of infusion time and treatment satisfaction with rheumatoid arthritis patients treated with intravenous golimumab or infliximab

Objectives: To assess real-world infusion times for golimumab (GLM-IV) and infliximab (IFX) for rheumatoid arthritis (RA) patients and factors associated with treatment satisfaction.

Methods: An observational study assessed infusion time including: clinic visit duration, RA medication preparation and infusion time, and infusion process time. Satisfaction was assessed by a modified Treatment Satisfaction Questionnaire for Medication (patient) and study-specific questionnaires (patient and clinic personnel). Comparative statistical testing for patient data utilized analysis of variance for continuous measures, and Fisher’s exact or Chi-square test for categorical measures. Multivariate analysis was performed for the primary time endpoints and patient satisfaction.

Results: One hundred and fifty patients were enrolled from six US sites (72 GLM-IV, 78 IFX). The majority of patients were female (80.0%) and Caucasian (88.7%). GLM-IV required fewer vials per infusion (3.7) compared to IFX (4.9; p?=?.0001). Clinic visit duration (minutes) was shorter for GLM-IV (65.1) compared to IFX (153.1; p?<?.0001), as was total infusion time for RA medication (32.8 GLM-IV, 119.5 IFX; p?<?.0001) and infusion process times (45.8 GLM-IV, 134.1 IFX; p?<?.0001). Patients treated with GLM-IV reported higher satisfaction ratings with infusion time (p?<?.0001) and total visit time (p?=?.0003). Clinic personnel reported higher satisfaction with GLM-IV than IFX specific to medication preparation time, ease of mixing RA medication, frequency of patients requiring pre-medication, and infusion time.

Limitations: Findings may not be representative of care delivery for all RA infusion practices or RA patients.

Conclusions: Shorter overall clinic visit duration, infusion process, and RA medication infusion times were observed for GLM-IV compared to IFX. A shorter duration in infusion time was associated with higher patient and clinic personnel satisfaction ratings.     

Treatment of candidemia with echinocandins: data on hospital resource use from a real world setting

Abstract

Objective:

Real-world data on patients treated with echinocandins for candidemia are limited. This study examined the effect of three echinocandin-based treatment regimens on resource utilization in patients with Candida infection.

Research design and methods:

A retrospective cohort study of patients hospitalized between 2005 and 2010 with a blood culture positive for Candida. Length of stay (LOS) following AF initiation (post-AF LOS) and total days with AF treatment were compared in patients treated with three different echinocandin regimens: patients with echinocandin only, patients who received fluconazole prior to an echinocandin (fluconazole-echinocandin), and patients who received an echinocandin prior to fluconazole (echinocandin-fluconazole). Generalized linear models were used to adjust for confounders.

Results:

A total of 647 patients met inclusion criteria. Patients treated with echinocandin only were more acutely ill, having more organ dysfunction and sepsis. Unadjusted post-AF LOS was significantly greater in the groups that received both echinocandin and fluconazole (mean, 13.1 days for echinocandin-only vs 25.5 and 21.2 days for fluconazole-echinocandin and echinocandin-fluconazole groups, respectively, p?<?0.001). These groups also had a higher total number of days with AF orders. These differences remained after multivariate adjustment and in survivor-only analyses. Compared with echinocandin-only treatment, the average marginal effect of fluconazole-echinocandin and echinocandin-fluconazole regimens were associated with significantly longer adjusted post-AF LOS (by 7.2 days and 9.3 days, respectively, p?<?0.001) and significantly more adjusted total AF days (by 5.3 days for fluconazole-echinocandin and 6.5 days for echinocandin-fluconazole patients, p?<?0.001). Limitations included lack of visibility to specific reasons for therapy changes.

Conclusions:

Fluconazole before or after echinocandin was associated with significantly greater resource utilization than echinocandin use alone.     

Regression analysis of indicating multiple incremental cost-effectiveness ratios for non-small cell lung cancer treatment

Objectives:

The value of a health technology can be measured in terms of cost and benefit on two-dimensional co-ordinates. This study is to quantitatively analyze the correlation and to conduct a regression on the X-Y plane constituted by cost and QALYs (quality-adjusted life years) associated with the first line treatment, the maintenance treatment, and the second line treatment for non-small cell lung cancer (NSCLC).

Methods:

The cost-effectiveness data of the cost and QALYs were extracted, with respect to the three categories of the NSCLC treatment, from the CEA Registry at Tufts Medical Center, regarding the literature published from 2000–2011. As a result, 44 QALY-cost ratios were identified.

Results:

Based on those extracted data, the correlation and regression analyses were performed by mathematical model using log and square-root functions. The plotted ratios stratified by the three stages for the NSCLC treatment were visually grouped into three clusters. There were statistically significant differences among the correlation coefficients of the cluster. In regression, the log model was found to be better fitted than the square-root model; formulating QALY?=??1.12?+?0.16 log(Cost), ?1.99?+?0.28 log(Cost), and ?0.69?+?0.10 log(Cost) for the first line, the maintenance, and the second line treatment, respectively. Monetary units were standardized to 2008 US dollars.

Conclusion:

A good methodological potential was confirmed so as to assess the Incremental Cost Effectiveness Ratio (ICER) variations, considering stratification by multiple factors such as disease and treatment categories. This study has certain limitations, such as the small number of included articles and the stratification, not reflecting a factor of new genetic findings.