Derivation of severity index for rheumatoid arthritis and its association with healthcare outcomes

Abstract

Objectives:

To develop a claims-based severity index for rheumatoid arthritis (RA) using the Veterans Health Administration (VHA) database.

Methods:

Adult patients with at least two RA diagnoses 2 months apart were identified between 10/1/2008–09/30/2009. Patients were required to have at least 12 months continuous health plan enrollment before and after the index date (first RA diagnosis date) for an overall study period of 10/1/2007–09/30/2010. A severity index for rheumatoid arthritis (SIFRA, a proprietary algorithm of SIMR, Inc. [STATinMED Research]) was developed by calculating a weighted sum of 34 RA-related indicators assessed by an expert Delphi panel of six rheumatologists, including laboratory, clinical, and functional status, extra-articular manifestations, surgical history, and medications, during a 1-year pre-index period. Separate SIFRA versions were derived for patients with and without laboratory information. Correlations between SIFRA and previously validated claims-based indexes for RA severity (CIRAS), and other traditional comorbidity indexes were calculated during the pre-index period. The relationship between SIFRA and follow-up healthcare outcomes was also examined using histograms.

Results:

The Spearman’s rank correlations between SIFRA and CIRAS were 0.525 for SIFRA without and 0.539 with laboratory data. The correlations between SIFRA and the Charlson Comorbidity Index (CCI) (0.1503 without, 0.1135 with laboratory data), Elixhauser Index (ELIX) (0.105 without, 0.079 with laboratory data), and Chronic Disease Score (CDS) (0.255 without, 0.239 with laboratory data) were low. Histograms showed that patients in the upper tercile of SIFRA incurred $9123 more all-cause and $1326 more RA-related healthcare costs during the 1-year post-index period than patients in the lower tercile. Using SIFRA in combination with CCI, CDS, or ELIX significantly increased the percentage of variation explained in outcomes measures.

Limitations:

Patients in the VHA database may not represent typical RA patients since the database generally contains older, economically disadvantaged men with a high disease burden. Validity of the score is indirectly based on disease activity score 28 (DAS28), which measures disease activity rather than severity.

Conclusions:

SIFRA was found to have moderate correlations with the previously validated CIRAS score, and demonstrated evidence of being a significant determinant of total and RA-related healthcare costs for RA patients. This study suggests that SIFRA could be an important methodological tool to control for severity in RA-related outcomes research. The algorithm can be applied to any claims dataset.     

Cost-effectiveness of denosumab vs zoledronic acid for prevention of skeletal-related events in patients with solid tumors and bone metastases in the United States

Abstract

Objective:

With increasing healthcare resource constraints, it has become important to understand the incremental cost-effectiveness of new medicines. Subcutaneous denosumab is superior to intravenous zoledronic acid (ZA) for the prevention of skeletal-related events (SREs) in patients with advanced solid tumors and bone metastases. This study sought to determine the lifetime cost-effectiveness of denosumab vs ZA in this setting, from a US managed-care perspective.

Methods:

A lifetime Markov model was developed, with relative rate reductions in SREs for denosumab vs ZA derived from three pivotal Phase 3 trials involving patients with castration-resistant prostate cancer (CRPC), breast cancer, and non-small-cell lung cancer (NSCLC), and bone metastases. The real-world SRE rates in ZA-treated patients were derived from a large commercial database. SRE and treatment administration quality-adjusted life year (QALY) decrements were estimated with time-trade-off studies. SRE costs were estimated from a nationally representative commercial claims database. Drug, drug administration, and renal monitoring costs were included. Costs and QALYs were discounted at 3% annually. One-way and probabilistic sensitivity analyses were conducted.

Results:

Across tumor types, denosumab was associated with a reduced number of SREs, increased QALYs, and increased lifetime total costs vs ZA. The costs per QALY gained for denosumab vs ZA in CRPC, breast cancer, and NSCLC were $49,405, $78,915, and $67,931, respectively, commonly considered good value in the US. Costs per SRE avoided were $8567, $13,557, and $10,513, respectively. Results were sensitive to drug costs and SRE rates.

Limitations:

Differences in pain severity and analgesic use favoring denosumab over ZA were not captured. Mortality was extrapolated from fitted generalized gamma function beyond the trial duration.

Conclusion:

Denosumab is a cost-effective treatment option for the prevention of SREs in patients with advanced solid tumors and bone metastases compared to ZA. The overall value of denosumab is based on superior efficacy, favorable safety, and more efficient administration.     

Cost-minimisation analysis of erlotinib in the second-line treatment of non-small-cell lung cancer: a Brazilian perspective

Abstract

Objective: A cost-minimisation and budget impact analysis of erlotinib versus docetaxel or pemetrexed as second-line treatment for advanced non-small-cell lung cancer (NSCLC).

Methods: Costs and budgetary impacts were estimated from the perspective of a Brazilian private healthcare payer, based on results of the BR.21 study of erlotinib and pivotal trials of docetaxel and pemetrexed. A 126-day timeframe was evaluated, based on the progression-free survival determined for erlotinib in BR.21. A Delphi panel identified local practices and associated costs in Brazil. Other costs accounted for included medical payments, pre- and post-chemotherapy medication and drug administration costs. Multivariate sensitivity analyses were performed, but given the short time frame used, discounting was not applied.

Results: Total costs were R$26,825 for erlotinib, R$42,284 for docetaxel and R$79,841 for pemetrexed. Cost savings with erlotinib were attributable to lower acquisition costs (R$26,795 vs. R$40,217 for docetaxel and R$78,911 for pemetrexed) and lower costs for the management of side effects. Sensitivity analyses confirmed the robustness of the results. The budget impact analysis showed savings with erlotinib in the first year, ranging from R$3 million to R$28 million.

Conclusion: Erlotinib is cost-saving over established chemotherapy in the second-line treatment of advanced NSCLC under the Brazilian private healthcare system.     

A cost-effectiveness analysis of using azacitidine vs. decitabine in treating patients with myelodysplastic syndromes

Abstract

Objective:

Azacitidine and decitabine are used to treat patients with myelodysplastic syndromes (MDS) in the United States (US). This study sought to assess their relative cost-effectiveness.

Design and methods:

The authors developed a cost-effectiveness Markov model (1-month cycles) tracking hypothetical cohorts of MDS patients treated with azacitidine or decitabine over 2 years. The model used a US payer perspective and 2009 costs. Health states modeled included MDS with Transfusion Dependence, MDS with Transfusion Independence, Progression to Acute Myelogenous Leukemia (AML), and Death. Incremental cost-effectiveness outcomes included cost per quality-adjusted life year (QALY), cost per life year (LY), cost per patient-month of transfusion independence, and cost per case of AML progression avoided. One-way sensitivity analyses were performed on key model parameters.

Results:

Compared to decitabine, azacitidine was associated with better survival (1.512 LYs vs 1.292), more QALYs gained (1.041 vs 0.870), more patient-months with transfusion independence (8.328 vs 6.224), and a greater proportion of patients avoiding progression to AML (50.9% vs 28.5%). Total per-patient costs over 2 years for azacitidine were lower than for decitabine ($150,322 vs $166, 212).

Limitations:

To inform and update the model over time, it will be important that randomized or observational clinical studies be conducted to directly compare azacitidine and decitabine, provide new information on how these medicines are used, and on their relative clinical effectiveness.

Conclusion:

Results demonstrate that azacitidine provides greater clinical benefit and costs less than decitabine across all key outcomes. These results accentuate the positive role of azacitidine in providing cost-effective care for MDS.     

Medical resource use and cost of different first-line treatments for metastatic colorectal cancer in Brazil

Abstract

Background: Metastatic colorectal cancer (mCRC) is one of the most common malignancies worldwide. The availability of new chemotherapeutic agents have modified the treatment of mCRC over the years creating the need to evaluate the financial impact of treatment. The aim of this study was to establish and quantify the financial resources needed during the first-line treatment of mCRC in Brazil.

Methods: The authors began by reaching expert consensus using a modified Delphi panel with oncologists working at public and private services in Brazil. Costs were calculated using official databases and the microcosting technique.

Results: The panel reached consensus on six regimens used in the first-line treatment of mCRC, as well as the resources involved in the administration of these regimens. All the regimens contain either fluorouracil (5-FU)/leucovorin or capecitabine, combined with either oxaliplatin or irinotecan. The analysis showed that, when compared with intravenous 5-FU/leucovorin, the cost of capecitabine was offset by administration costs.

Conclusion: The panel concluded that regimens containing capecitabine, especially capecitabine plus oxaliplatin (XELOX) are less expensive than those containing 5-FU/leucovorin. Given the comparable efficacy and good tolerability of the XELOX regimen, it may be an attractive choice for the first-line treatment of Brazilian patients with mCRC.     

Global health resource utilization associated with pacemaker complications

Aim: To estimate health resource utilization (HRU) associated with the management of pacemaker complications in various healthcare systems.

Methods: Electrophysiologists (EPs) from four geographical regions (Western Europe, Australia, Japan, and North America) were invited to participate. Survey questions focused on HRU in the management of three chronic pacemaker complications (i.e. pacemaker infections requiring extraction, lead fractures/insulation breaches requiring replacement, and upper extremity deep venous thrombosis [DVT]). Panelists completed a maximum of two web-based surveys (iterative rounds). Mean, median values, and interquartile ranges were calculated and used to establish consensus.

Results: Overall, 32 and 29 panelists participated in the first and second rounds of the Delphi panel, respectively. Consensus was reached on treatment and HRU associated with a typical pacemaker implantation and complications. HRU was similar across regions, except for Japan, where panelists reported the longest duration of hospital stay in all scenarios. Infections were the most resource-intensive complications and were characterized by intravenous antibiotics days of 9.6?13.5 days and 21.3?29.2 days for pocket and lead infections respectively; laboratory and diagnostic tests, and system extraction and replacement procedures. DVT, on the other hand, was the least resource intensive complication.

Limitations: The results of the panel represent the views of the respondents who participated and may not be generalizable outside of this panel. The surveys were limited in scope and, therefore, did not include questions on management of acute complications (e.g. hematoma, pneumothorax).

Conclusions: The Delphi technique provided a reliable and efficient approach to estimating resource utilization associated with chronic pacemaker complications. Estimates from the Delphi panel can be used to generate costs of pacemaker complications in various regions.     

A discrete choice experiment to assess patients’ preferences for HIV treatment in the rural population in Colombia

Abstract

Aim: To elicit patients’ preferences for HIV treatment of the rural population in Colombia.

Methods: A discrete choice experiment (DCE), conducted in a HIV clinic in Bogotá, was used to examine the trade-off between five HIV treatment attributes: effect on life expectancy, effect on physical activity, risk of moderate side-effects, accessibility to clinic, and economic costs to access controls. Attributes selection was based on literature review, expert consultation and a focus group with six patients. An efficient experimental design was used to define two versions of the questionnaire with each of 12 choice sets and a dominance task was added to check reliability. A mixed logit model was then used to analyse the data and sub-group analyses were conducted on the basis of age, gender, education, and sexual preference.

Results: A total of 129 HIV patients were included for analysis. For all treatment attributes, significant differences between at least two levels were observed, meaning that all attributes were significant predictors of choice. Patients valued the effect on physical activity (conditional relative importance of 27.5%) and the effect on life expectancy (26.0%) the most. Sub-group analyses regard age and education showed significant differences: younger patients and high educated patients valued the effect on physical activity the most important, whereas older patients mostly valued the effect on life expectancy and low educated patients mostly valued the accessibility to clinic.

Limitations: One potential limitation is selection bias, as only patients from one HIV clinic were reached. Additionally, questionnaires were partly administered in the waiting rooms, which potentially led to noise in the data.

Conclusions: This study suggests that all HIV treatment characteristics included in this DCE were important and that HIV patients from rural Colombia valued short-term efficacy (i.e. effect on physical activity) and long-term efficacy (i.e. effect on life expectancy) the most.     

A discrete choice experiment to assess patients’ preferences for HIV treatment in the urban population in Colombia

Abstract

Aim: This study aimed to assess patients’ preferences for HIV treatment in an urban Colombian population.

Methods: A Discrete Choice Experiment (DCE) was conducted. Urban Colombian HIV patients were asked to repetitively choose between two hypothetical treatments that differ in regard to five attributes ‘effect on life expectancy’, ‘effect on physical activity’, ‘risk of moderate side effects, ‘accessibility to clinic’ and ‘economic cost to access controls’. Twelve choice sets were made using an efficient design. A Mixed Logit Panel Model was used for the analysis and subgroup analyses were performed according to age, gender, education level and sexual preference.

Results: A total of 224 HIV patients were included. All attributes were significant, indicating that there were differences between at least two levels of each attribute. Patients preferred to be able to perform all physical activity without difficulty, to have large positive effects on life expectancy, to travel less than 2?h, to have lower risk of side-effects and to have subsidized travel costs. The attributes ‘effect on physical activity’ and ‘effects on life expectancy’ were deemed the most important. Sub-analyses showed that higher educated patients placed more importance on the large positive effects of HIV treatment, and a more negative preference for subsidized travel cost (5% level).

Limitations: A potential limitation is selection bias as it is difficult to make a systematic urban/rural division of respondents. Additional, questionnaires were partly administered in the waiting rooms, which potentially led to some noise in the data.

Conclusions: Findings suggests that short-term efficacy (i.e. effect on physical activity) and long-term efficacy (i.e. effect on life expectancy) are the most important treatment characteristics for HIV urban patients in Colombia. Preference data could provide relevant information for clinical and policy decision-making to optimize HIV care.     

Cost-effectiveness analysis of atypical long-acting antipsychotics for treating chronic schizophrenia in Finland

Abstract

Objective:

In Finland, regional rates of schizophrenia exceed those in most countries, impacting the healthcare burden. This study determined the cost-effectiveness of long-acting antipsychotic (LAI) drugs paliperidone palmitate (PP-LAI), olanzapine pamoate (OLZ-LAI), and risperidone (RIS-LAI) for chronic schizophrenia.

Method:

This study adapted a decision tree analysis from Norway for the Finnish National Health Service. Country-specific data were sought from the literature and public documents, guided by clinical experts. Costs of health services and products were retrieved from literature sources and current price lists. This simulation study estimated average 1-year costs for treating patients with each LAI, average remission days, rates of hospitalization and emergency room visits and quality-adjusted life-years (QALY).

Results:

PP-LAI was dominant. Its estimated annual average cost was €10,380/patient and was associated with 0.817 QALY; OLZ-LAI cost €12,145 with 0.810 QALY; RIS-LAI cost €12,074 with 0.809 QALY. PP-LAI had the lowest rates of hospitalization, emergency room visits, and relapse days. This analysis was robust against most variations in input values except adherence rates. PP-LAI was dominant over OLZ-LAI and RIS-LAI in 77.8% and 85.9% of simulations, respectively. Limitations include the 1-year time horizon (as opposed to lifetime costs), omission of the costs of adverse events, and the assumption of universal accessibility.

Conclusion:

In Finland, PP-LAI dominated the other LAIs as it was associated with a lower cost and better clinical outcomes.     

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.     

Preferred reporting items for studies mapping onto preference-based outcome measures: The MAPS statement

Abstract

‘Mapping’ onto generic preference-based outcome measures is increasingly being used as a means of generating health utilities for use within health economic evaluations. Despite publication of technical guides for the conduct of mapping research, guidance for the reporting of mapping studies is currently lacking. The MAPS (MApping onto Preference-based measures reporting Standards) statement is a new checklist, which aims to promote complete and transparent reporting of mapping studies. The primary audiences for the MAPS statement are researchers reporting mapping studies, the funders of the research, and peer reviewers and editors involved in assessing mapping studies for publication.

A de novo list of 29 candidate reporting items and accompanying explanations was created by a working group comprised of six health economists and one Delphi methodologist. Following a two-round, modified Delphi survey with representatives from academia, consultancy, health technology assessment agencies and the biomedical journal editorial community, a final set of 23 items deemed essential for transparent reporting, and accompanying explanations, was developed. The items are contained in a user friendly 23 item checklist. They are presented numerically and categorised within six sections, namely: (i) title and abstract; (ii) introduction; (iii) methods; (iv) results; (v) discussion; and (vi) other. The MAPS statement is best applied in conjunction with the accompanying MAPS explanation and elaboration document.

It is anticipated that the MAPS statement will improve the clarity, transparency and completeness of reporting of mapping studies. To facilitate dissemination and uptake, the MAPS statement is being co-published by seven health economics and quality of life journals, and broader endorsement is encouraged. The MAPS working group plans to assess the need for an update of the reporting checklist in five years’ time.     

Cost effectiveness of tyrosine kinase inhibitor therapy in metastatic gastrointestinal stromal tumors

Abstract

Background:

Tyrosine kinase inhibitors (TKIs) such as imatinib mesylate have revolutionized the treatment of primary unresectable and/or metastatic gastrointestinal stromal tumors (GISTs), providing durable disease control and extended survival. Although most patients eventually progress on therapy, dose escalation has been shown to benefit some patients. Sunitinib, a multitargeted kinase inhibitor is effective against imatinib-resistant or intolerant GIST patients. Although the cost of TKI therapy in GIST is high, no other effective systemic treatment options exist.

Objective:

Review pharmacoeconomic studies to determine the cost effectiveness (CE) of 1st- and 2nd-line TKI therapies in GIST.

Methods:

A literature review using Medline and PubMed databases was conducted to identify published economic analyses of TKI therapy in GIST. Key results from these studies were analyzed.

Results:

Six pharmacoeconomic studies were identified, including three analyses of 1st-line imatinib and three analyses of 2nd-line sunitinib. These studies employed various time horizons and discount rates and modeled CE from a number of different perspectives. Most of the pharmacoeconomic studies reviewed used survival as their efficacy endpoint, projecting outcomes beyond available data to model CE. Analyses of 2nd-line sunitinib using survival additionally faced the challenge of adjusting for the effect of placebo crossover to active treatment in the pivotal phase III study. Most studies used Markov techniques with a range of transition probabilities.

Conclusions:

Published pharmacoeconomic studies of 1st- and 2nd-line TKI therapy for advanced GIST employ various time horizons, discount rates, and different CE models. Consequently, these differences make comparisons between studies difficult. Studies of 1st-line imatinib concluded that imatinib was cost effective in advanced, metastatic GIST. Likewise, based on data reviewed here, 2nd-line sunitinib appears to be cost effective in patients with advanced GIST who are intolerant/resistant to imatinib. Key limitations of this review included inconsistency among the studies evaluated with regard to methodologies, countries of origination (currency and healthcare systems), and patient demographics.     

Feasibility of using administrative claims data for cost-effectiveness analysis of a clinical trial

Objective: This study was performed retrospectively to determine if Medicare claims data could be used to evaluate the cost effectiveness, from a payer perspective, of different radiation treatment schedules evaluated in a national clinical trial.

Methods: Medicare costs from all providers and all places of service were obtained from the Centers for Medicare & Medicaid Services for patients treated in the period 1992–1996 on Radiation Therapy Oncology Group 90-03, and combined with data on outcomes from the trial.

Results: Of the 1,113 patients entered, Medicare cost data and clinical outcomes were available for 187 patients. Significant differences in tolerance of treatment and outcome were noted between patients with Medicare data included in the study and patients without Medicare data, and non-Medicare patients excluded from it. Ninety-five percent confidence ellipses on the incremental cost-effectiveness scatterplots crossed both axes, indicating non-significant differences in cost effectiveness between radiation treatment schedules.

Conclusions: Claims data permit estimation of cost effectiveness, but Medicare data provide inadequate representation of results applicable to patients from the general population.

Keywords:     

Evaluation of cost savings with ferric carboxymaltose in anemia treatment through its impact on erythropoiesis-stimulating agents and blood transfusion: French healthcare payer perspective

Abstract

Objective:

To evaluate the economic impact of intravenous iron (in the form of intravenous iron preparation of ferric carboxymaltose) in three different clinical settings of iron deficiency anemia: chemotherapy-induced anemia in breast cancer, chemotherapy-induced anemia in digestive cancer, and perioperative anemia in knee and hip surgery.

Methods:

The economic model compared the usual therapeutic strategies of anemia without intravenous iron and strategies including intravenous iron, in each of the three clinical settings selected. Costs related to anemia treatment by erythropoiesis-stimulating agents (ESA), blood transfusion, and intravenous iron were estimated and compared inside each setting. Cost savings were calculated from the French healthcare payer perspective. Data included in the economic model were obtained from scientific literature, public health agencies, and medical experts.

Results:

The most prominent annual cost savings were observed in chemotherapy-induced anemia in breast cancer (€997 and €360 per patient for metastatic and non-metastatic breast cancers, respectively; global cost saving, €33.6 million). This large impact of intravenous iron on costs was mainly explained by both a lower number of women treated and lower ESA dosing. Mean annual cost saving in digestive cancers and knee and hip surgery were estimated to €168 and €216 per patient and global cost savings of €7.5 and €12.1 million, respectively. Overall, annual cost savings in these three settings were estimated to €53 million including €39 million for ESA cost savings. Sensitivity analysis showed that strategies including intravenous iron remained cost-effective even with wide variations in the assumptions, particularly for cost savings on ESA.

Limitations:

Economic model based on literature data and expert opinions.

Conclusions:

The present economic model suggests that use of intravenous iron, according to recommendations of international guidelines, is cost saving, particularly in chemotherapy-induced anemia in breast cancers.     

Understanding the effects on HR-QoL of treatment for overactive bladder: a detailed analysis of EQ-5D clinical trial data for mirabegron

Abstract

Background:

Analysis of EQ-5D data often focuses on changes in utility, ignoring valuable information from other parts of the instrument. The objective was to explore how the utility index, EQ-5D profile, and EQ-VAS captured change in clinical trials of mirabegron, a new treatment for overactive bladder (OAB).

Data:

Data were pooled from three phase III clinical trials that investigated the efficacy and safety of mirabegron vs placebo. Tolterodine ER 4?mg was included as an active control in one study: (1) placebo, mirabegron 50?mg and 100?mg, and tolterodine 4?mg ER; (2) placebo, mirabegron 50?mg and 100?mg; (3) placebo, and mirabegron 25?mg and 50?mg. Data were collected at baseline, week 4, 8, and 12.

Methods:

Analyses were performed on full analysis and modified intention to treat (ITT) data sets using UK utilities. Analysis controlled for relevant patient characteristics. Analysis of Covariance identified changes from baseline at each time point in utilities and EQ-VAS. Areas Under the Curve were estimated to summarize inter-temporal differences in effect. EQ-5D profile data were analysed using the Paretian Classification of Health Change.

Results:

In modified ITT analyses, mirabegron 50?mg was superior to tolterodine 4?mg in changes from baseline utilities after 12 weeks (p?<?0.05); similarly, AUC results showed mirabegron 50?mg to be superior to tolterodine (p?<?0.05) and placebo (p?<?0.05) with the benefit already apparent at 4 weeks (p?<?0.05). EQ-VAS more consistently indicated superior outcomes: all three mirabegron doses showed statistically significant greater effectiveness compared to tolterodine at 12 weeks. Individual EQ-5D dimensions and the overall profile showed no significant differences between study arms.

Conclusion:

Mirabegron showed quicker and superior improvement in HR-QoL compared to tolterodine 4?mg ER. A limitation of the study is that EQ-5D was a secondary outcome in the pivotal trials, which were not powered to measure differences on EQ-5D.     

Converting EORTC QLQ-C30 scores to utility scores in the brigatinib ALTA study

Abstract

Aims: Health utilities summarize a patient’s overall health status. This study estimated utilities based on the European Organization for Research and Treatment of Cancer Quality of Life Questionnaire Core-30 (QLQ-C30), a widely used measure of health-related quality-of-life (HRQoL) in oncology, using published mapping algorithms.

Materials and methods: Data were from the Anaplastic Lymphoma Kinase (ALK) in Lung Cancer Trial of brigatinib (ALTA; NCT02094573), an open-label, international, phase 2 study. ALTA evaluated the efficacy and safety of two randomized dosing regimens of brigatinib in patients with locally advanced or metastatic ALK?+?non-small cell lung cancer (NSCLC) that had progressed on prior therapy with crizotinib. QLQ-C30 scores were mapped to European Quality-of-Life-5 Dimensions (EQ-5D) utility scores using two published algorithms (Khan et al. for EQ-5D-5L; Longworth et al. for EQ-5D-3L). The impact of brigatinib treatment on health utilities over time was assessed.

Results: The analysis included 208 subjects. Mean baseline utility scores for both algorithms ranged between 0.60???0.71 and increased to 0.78 by cycle 5. Utility improvements were sustained during most of the treatment, before disease progression. Minor variations were observed between utility scores; Khan et al. estimates were approximately 0.01 or 0.02 points lower than Longworth et al. estimates.

Limitations: Algorithms considered were limited to those available in the published literature at the time of the study. This utility analysis was exploratory, and the ALTA trial did not include an internal control group (i.e. standard of care) and was not powered to detect differences in QoL/utility outcomes between treatment arms.

Conclusions: Converting QLQ-C30 scores into utilities in trials using established mapping algorithms can improve evaluation of medicines from the patient perspective. Both algorithms suggested that brigatinib improved health utility in crizotinib-refractory ALK?+?NSCLC patients, and improvements were maintained during most of the treatment.

Clinicaltrials.gov identifier: NCT02094573     

Ostomy appliance prices in Europe

Abstract

Objective: This article aims to compare market prices (i.e., third-party reimbursement and patient co-payment) of one-piece and two-piece colostomy, ileostomy and ureterostomy appliances in Belgium, Denmark, England and the Netherlands in 2005.

Methods: Data were collected through contacts with health authorities, health insurance companies, manufacturers, industry associations and distributors. The price difference between Belgium and another country was expressed as a proportion of the Belgian price.

Results: A total of 64 out of the 72 ostomy appliance products considered were cheaper in Belgium. Prices of one-piece colostomy appliances and two-piece ileostomy appliances were consistently lower in Belgium. The highest prices of ostomy appliances were observed in the Netherlands. Sixteen out of 20 products and 21 out of 25 products were more expensive in Denmark and England, respectively, than in Belgium. Colostomy appliances were more expensive in England than in Belgium.

Conclusions: Market prices varied substantially between countries, indicating that manufacturers adapt their pricing strategy to the policy environment existing in the ostomy appliance market of each country. Also, there appears to be scope for reducing prices in some countries.     

The economic impact of galantamine vs placebo: An analysis based on functional capacity in a Swedish cohort study

Abstract

Objective:

To analyse the economic impact of galantamine, based on basic activities of daily living (ADL).

Methods:

Data were derived from Swedish patients enrolled in a 6-month placebo-controlled trial of galantamine (GAL-INT-1; n?=?80), and from the Kungsholmen–Nordanstig Project, a longitudinal study of 919 elderly persons in Sweden. Basic ADL were assessed using the Katz’ Index of Independence in Activities of Daily Living (ADL) (number of ADL lost [dependency in 0, 1–2, 3–4, or 5–6 ADL]). Costs were appraised based on regression analysis and on costs directly linked to ADL. Six-month costs for galantamine and placebo were calculated.

Results:

In the regression analyses, each increase in a Katz stage was associated with an annual cost increase of SEK 81,415–83,683 (～€8000). Results were similar using stage-specific costs. Overall, there was a small, non-significant numerical cost benefit for galantamine indicating cost neutrality.

Limitations:

The small number of Swedish patients in the GAL-INT-1 study, which was not powered for economic outcomes, limits the statistical power of the analysis. In addition, long-term outcomes are difficult to assess in persons with dementia because of practical and logistical problems.

Conclusions:

The benefits of galantamine in patients with AD can be achieved with no increase in cost. Combined with positive effects in terms of outcome, treatment with galantamine can be regarded as cost-effective using a cost–consequence approach.