Cost-effectiveness of teduglutide in adult patients with short bowel syndrome - a European socioeconomic perspective.

BACKGROUND: Short bowel syndrome with intestinal failure (SBS-IF) is a rare but devastating medical condition. An absolute loss of bowel length forces the patients into parenteral support dependency and a variety of medical sequelae, resulting in increased morbidity and mortality. Interdisciplinary treatment may include therapy with the effective but expensive intestinotrophic peptide teduglutide. OBJECTIVES: A time-discrete Markov model was developed to simulate the treatment effect [lifetime costs, quality-adjusted life years (QALYs), and life years (LYs)] of teduglutide plus best supportive care compared with best supportive care alone in patients with SBS-IF. METHODS: The health status of the model was structured around the number of days on PS. Clinical data from 3 data sets were used: 1) an Austrian observational study (base case), 2) pooled observational cohort studies, and 3) a prospective study of teduglutide effectiveness in parenteral nutrition-dependent short bowel syndrome subjects. Direct and indirect costs were derived from published sources. QALYs, LYs, and costs were discounted (3% per annum). RESULTS: Under the base case assumption, teduglutide is associated with costs of 2,296,311 € per patient and 10.78 QALYs (13.74 LYs) over a lifetime horizon. No teduglutide is associated with 1,236,816 € and 2.24 QALYs (8.57 LYs). The incremental cost-utility ratio (ICUR) amounts to 123,945 €. In case of the pooled clinical data set, the ICUR increases to 184,961 €. If clinical data based on the study of teduglutide effectiveness in parenteral nutrition-dependent short bowel syndrome subjects were used, the ICUR increased to 235,612 €. CONCLUSIONS: Teduglutide in treating patients with SBS-IF meets the traditional cost-effectiveness criteria from a European societal perspective. Nevertheless, the varying concentrations of teduglutide efficacy leave a degree of uncertainty in the calculations.

A budget impact analysis of a powered hybrid mattress to prevent pressure ulcers in the Austrian inpatient setting: An original research.

Background and Aims: Pressure ulcer is a considerable health problem and is associated with an increased hospital length of stay (LOS), nursing effort, costs, and reduced quality of life. The aim of this analysis is to compare the economic, clinical, and nursing benefit after the implementation of the powered hybrid mattress Dyna-Form Mercury Advance SMARTcare® in 2019 with the situation in 2017 (rental systems Arjo BariAir® and Arjo TheraKair Visio®, and purchased foam mattresses Arjo Simuflex®) using a single center. Methods: In the framework of a budget impact analysis, a difference-in-difference approach was used to compare pre-implementation longitudinal data (2017) with post-implementation data (2019). The longitudinal data comprise the mattress resource use, patient characteristics, and LOS. The care effort was gathered based on a full survey of all 75 members of the nursing staff using the online tool "LimeSurvey." In this survey, the resource use of the different mattress systems was identified in terms of time (minutes or days). This analysis was performed from the hospital perspective and included direct costs of mattress systems, Austrian diagnostic-related group costs for the hospital stay and nursing staff costs for 2019. Results: Based on 1253 patients "at-risk," the total yearly costs implementing the powered hybrid mattress amounts to 654,248€ compared with 901,469€ when using Arjo products. The budget impact shows a cost advantage of 247,221€. Furthermore, the powered hybrid mattress system leads to fewer nursing-staff time in minutes per year (-1,031,097 min [1,993,204 vs. 3,024,302]); 242€ are saved per patient. Compared with the Arjo products, fewer inpatient cases of pressure ulcers (-44 cases [0 vs. 44]) were documented. Conclusion: Despite the higher total outlay of costs associated with the powered hybrid mattress, the long-term savings potential showed a significant cost advantage per annum for the single center. Therefore, the use of the hybrid mattress leads to considerable economic, clinical, and nursing benefits.

Cost-effectiveness of NT-proBNP-supported screening of chronic heart failure in patients with or without type 2 diabetes in Austria and Switzerland.

BACKGROUND: Heart failure (HF) is a clinical syndrome with a global burden. Signs and symptoms of HF are nonspecific and often shared with other conditions. The N-terminal prohormone of brain natriuretic peptide (NT-proBNP) serves as a useful biomarker for the diagnosis of HF not only in patients with acute symptoms but also in outpatients with an ambiguous clinical presentation. The aim of the analysis is to evaluate the cost-effectiveness of implementing NT-proBNP in the diagnostic algorithm in patients with/without type 2 diabetes mellitus (T2DM), compared with a diagnosis based primarily on clinical signs or symptoms from the perspective of the Austrian and Swiss healthcare system. METHODS: A time-discrete Markov model was developed to simulate the effect/improvement (lifetime-costs, quality-adjusted life-years [QALYs], and life-years [LYs]) due to an NT-proBNP screening in undetected HF patients. Undetected HF patients are included in the model according to a distribution of New York Heart Association (NYHA) classes. The model considers disease progression by transition of NYHA classes. Undetected patients may remain undetected or be detected with the help of NT-proBNP or symptoms. Patients with known HF exhibit a slower disease progression. The probability of dying is influenced by the respective NYHA class. Direct costs (2021 € or CHF) were derived from published sources. QALYs, LYs, and costs were discounted (3% p.a.). RESULTS: In the per-patient analysis (at age 60 over lifetime), the incremental cost-utility ratio (ICUR)/QALY of NT-proBNP vs. no screening was €3,042 for HF patients in Austria. Considering the total cohort of undetected HF patients (n = 9,377) with the corresponding age structure over a lifetime, the ICUR increases to €4,356. In Switzerland, the per-patient results show an ICUR of CHF 897. Considering the total cohort of undetected HF patients (n = 6,826) the ICUR amounts to CHF 4,513. If indirect costs are considered, NT-proBNP screening becomes the dominant strategy in both countries. CONCLUSION: Overall, the analysis concludes that screening with NT-proBNP is a highly cost-effective or cost-saving diagnostic option for patients with HF, and a sensitivity analysis confirmed these findings.

The cost-of-illness and burden-of-disease of treatment-resistant depression in Austria.

BACKGROUND: Treatment-resistant depression (TRD) in major depressive disorder (MDD) is most commonly defined as the failure to respond to at least two antidepressant (AD) treatments of adequate duration and adherence. While the health care utilization (RU) and costs of patients with MDD are well documented, little is known about patients with TRD. Therefore, the aim of this study was to determine the direct medical RU of complex therapy pathways and to analyze the total cost-of-illness and the burden-of-disease in Austria. METHODS: In order to quantify the cost-of-illness and burden-of-disease of TRD, the analysis was designed with two steps. First, RU data were collected through an extensive survey of Austrian experts and a systematic literature review. Second, direct, indirect, and intangible costs were calculated using the micro-costing method. The results are presented per patient and based on a patient flow for the entire cohort of TRD patients. RESULTS: In Austria, the derived prevalence of TRD is 43,732 patients or 583 per 100,000 population. For 2021, the annual direct costs of TRD were estimated at 345.0 million €. At 684.7 million €, the estimated indirect costs were higher than the direct costs, representing 66.5% of the total cost-of-illness. The average annual cost per TRD patient is 23,547 €, of which direct costs are 7,890 €. Adding the years lived with a disability to the years lost due to premature death attributed to TRD resulted in a total of 29,884 disability-adjusted life years (DALYs) for the Austrian society. CONCLUSION: Although TRD accounts for only 0.7% (range: 0.6%-1%) of the total health care budget, it represents a significant burden-of-disease. In addition, TRD is associated with a high level of lost productivity in the Austrian economy. These findings support efforts to prioritize TRD as a focus area to achieve health-related goals.

Impact of metabolic surgery on cost and long-term health outcome: a cost-effectiveness approach.

BACKGROUND: The increase in obesity has become a major public health concern. Morbid obesity is associated with co-morbidities, reduced quality-of-life, and death. Metabolic surgery (MS) is the most effective treatment for obesity. OBJECTIVES: The aim of this study was to evaluate the costs and outcomes of MS compared with no surgery in patients with a body mass index ≥30 kg/m2. SETTING: Health care system, Austria METHODS: A total of 177 patients who underwent MS were documented retrospectively, based on the hospital records of 2 specialized centers in Austria, over a 1-year period. At baseline 26.0% of patients exhibited type 2 diabetes (T2D), 52.5% cardiovascular disease (CVD), 23.2% hyperlipidemia, and 23.7% depression. Following the observation period, a Markov chain simulation model was developed to analyze the long-term consequences of T2D, including diabetic complications, CVD, hyperlipidemia, depression, non-alcoholic steatohepatitis (NASH), myocardial infarction, and stroke, over a total of 20 years. Direct medical costs were expressed in 2017 euros from the payer's perspective. Quality-adjusted life years (QALYs), life years (LYs), and costs were discounted. RESULTS: MS led to costs of €40,427 and 9.58 QALYs (15.58 LYs) per patient over 20 years. No MS was associated with €64,819 and 6.33 QALYs (13.92 LYs). Total cost-savings amounted to €24,392, which offset the cost of the procedure including re-operations. Over 20 years MS saved -6.7 patient-years per patient with T2D, -5.8 patient-years with CVD, -1.5 patient-years with hyperlipidemia, -1.8 patient-years with depression, and -3.8 patient-years with NASH. CONCLUSION: MS is associated with substantial savings in long-term costs, expected health benefits, and reduced onset of complications. MS significantly increases quality of life.

Cost-effectiveness analysis of apixaban compared to other direct oral anticoagulants for prevention of stroke in Austrian atrial fibrillation patients.

OBJECTIVES: Several direct oral anticoagulants (DOACs) have been approved by the European Medicines Agency since 2008. The aim of the present cost-effectiveness-analysis was to analyze apixaban compared to other DOACs and vitamin K antagonists (warfarin) in Austria. METHODS: A cost-utility-model was developed to simulate lifetime-costs and quality-adjusted-life-years of DOACs and warfarin, based on a published Markov-Model and 23 randomized trials with 94,656 atrial-fibrillation (AF) patients. Each year, a patient has a probability of suffering a clinically relevant (extracranial) bleed, an intracranial hemorrhage (ICH), an ischemic stroke or a myocardial infarction (MI), remaining healthy, or deceasing. Direct-costs (2018€) were derived from published sources from the payer's perspective. RESULTS: In the base-case, warfarin had the lowest cost of 12,968 € (95%-CI±593 €) followed by apixaban (15,269 €±661 €), edoxaban (15,534 €±641 €), dabigatran (15,687 €±667 €), and rivaroxaban (17,522 €±764 €). Apixaban had the highest quality-adjusted-life-years estimate at 5.45 (SD, 0.06). In a Monte-Carlo probabilistic sensitivity analysis, apixaban was cost-effective vs. edoxaban, dabigatran, warfarin, and rivaroxaban in 85.6%, 79.0%, 76.4%, and 61.2% of the simulations, respectively. CONCLUSION: In patients with AF and an increased risk of stroke, prophylaxis with apixaban was highly cost-effective from the perspective of the Austrian health-care system.

Health-related quality of life, work productivity and costs related to patients with inflammatory bowel disease in Austria.

OBJECTIVE: Inflammatory-Bowel-Disease (IBD) is a lifelong illness with significant impact on health-related quality of life (HRQoL). The disease-burden causes work productivity impairment, such as sick-leave and restriction of leisure time activities. From a societal perspective, productivity loss often contributes significantly to the total costs. The aim of the study is to analyze the impact of disease-burden on work productivity, daily activities, and HRQoL. METHODS: We performed a survey among Austrian patients with IBD to assess general questions about demography and disease pattern, impaired work productivity and reduced daily activities using the Work Productivity and Activity Impairment questionnaire and HRQoL utilizing the Short Inflammatory Bowel Disease Questionnaire (SIBDQ). Associations were assessed employing linear regressions. The questionnaire was circulated by the Austrian Crohn's Disease/Ulcerative Colitis Association. RESULTS: In total, 510 patients with median age of 40 years completed the questionnaire. Patients had a median disease-duration of 9 years, 46% were treated with biologics and 64% were employed. The median SIBDQ score was 45 (16-69). Patients in self-assessed remission (n = 319) and relapse (n = 96) differed by 18 points on the SIBDQ score (p value < 0.0001). Mean work productivity reduction amounted to 7 h 17 min (95% CI 6:05-8:29) during the last 7 days. Work absenteeism accounted for 4 h 28 min (95% CI 3:25-5:31) and reduced productivity at the workplace (presenteeism) for 2 h 49 min (95% CI 2:31-3:07). During a relapse, 12 h 9 min were lost (p value < 0.0001). Productivity reduction correlated with HRQoL (r = 0.3964). In total, 143 €per patient per week (95% CI 119-167) were lost (7,411 €per year). On average, patients lost 4 h (95% CI 3:44-4:16) of unpaid daily activities; 3 h 17 min the employed subgroup and 5 h 19 min the not employed. CONCLUSION: IBD leads to a substantial burden of disease, which is associated with indirect costs for society related to work ability reduction. HRQoL significantly influences loss of work productivity. Improving HRQoL should be a key factor to reduce disease burden and societal costs.

Economic impact of industry-sponsored clinical trials of pharmaceutical products in Austria.

Aims: Modern pharmaceutical product development is a long and complex process associated with significant investments by pharmaceutical companies. The innovative pharmaceutical industry accounts for the vast majority of expenditures in clinical trials of potential new pharmaceuticals and therefore generates economic activity within a country. The aim was to assess the far-reaching economic impact of industry-sponsored clinical-trials (ISCTs) of pharmaceutical products for the healthcare system and the national economy.Materials and methods: The study approach was based on three analytical steps. First, a survey among 15 pharmaceutical companies in Austria was conducted to evaluate the annual number of ISCTs subdivided according to trial phase, therapeutic areas and associated employees. Second, the monetary value of treatments performed in ISCTs was calculated based on a sample of clinical-trial protocols. Finally, the macroeconomic impact, measured in terms of value-added and jobs created by the conducted ISCTs, was calculated using Input-Output analysis by applying an extended Leontief-model.Results: The study demonstrated that €116.22 million spent in ISCTs generated a total value added of €144 million, €74 million direct, in 2018. Each year a medical treatment value of €100 million was financed through 463 ISCTs, with an average value of medical treatment of €37,068 per recruited patient. This represents a significant 0.3% of annual current health-expenditures. In summary, each Euro invested by the pharmaceutical industry in ISCTs generates €1.95 for the Austrian economy. ISCTs also created and secured employment in the extent of 2,021 full-time-equivalents, thus resulting in an employment multiplier of 1.66.Conclusions: In conclusion, conducting clinical-trials by pharmaceutical industry-beside its importance in its own domain-results in tangible benefits and a positive macroeconomic impact that contribute to the sustainability of the Austrian healthcare system by complementing its limited resources. Furthermore, it is a non-negligible factor in locational and industrial policy.

Cost-Effectiveness Analysis of Landiolol, an Ultrashort-Acting Beta-Blocker, for Prevention of Postoperative Atrial Fibrillation for the Germany Health Care System.

OBJECTIVES: Landiolol is an ultrashort-acting beta-blocker with high beta-1 receptor affinity and less blood pressure-lowering properties than other beta-blockers available for intravenous use in Germany. The present analysis aimed to determine whether perioperative treatment with landiolol in cardiac surgical patients is cost-effective under the conditions of the German Diagnosis-Related Groups health cost reimbursement system. DESIGN: On the basis of clinical outcome data from a meta-analysis that included 622 patients from 7 randomized controlled trials, a decision-model was developed to determine the cost-effectiveness of landiolol versus standard-of-care (SoC). SETTING: Hospital setting. PARTICIPANTS: Hospital patients undergoing a representative mix of cardiac surgical procedures (MIX-CS) and isolated coronary artery bypass grafting (CABG). INTERVENTIONS: Landiolol versus SoC in prevention of atrial fibrillation immediately after cardiac surgery. MEASUREMENTS AND MAIN RESULTS: The model benefit was expressed in a reduction of postoperative atrial fibrillation (POAF) episodes and reduced complications. The model calculated total inpatient costs over the hospital length of stay. Costs from published sources were used for the German hospital perspective. SoC was associated with POAF rates of 36.0% to 39.2% and 24.4% to 30.1% in the MIX-CS and CABG populations, respectively. Patients with POAF had a higher morbidity and mortality. Estimated total costs for SoC patients in the MIX-CS and CABG groups were 28.792 € and 25.630 €, respectively. Landiolol reduced the incidence of POAF to 12.6% in the MIX-CS and 12.1% in the CABG groups. This was associated with a cost reduction of 2.209 € and 1.470 €. CONCLUSIONS: This analysis suggests that preventing POAF with landiolol is highly cost-effective. Additional studies are needed to assess whether a comparable reduction in POAF and associated cost savings may be achieved using conventional intravenous beta-blockers or amiodarone.

Introduction and Overview.

The overall aim of this book is to set out the main changes needed in the field of economic and regulatory conditions as a consequence of these rapid developments in oncology. The traditional approaches of health economics, like health economic evaluation, health technology assessment (HTA), modeling methods, assessing value, pricing techniques, are bound to be altered in the contributions to this book. It is understandable that with the life-threatening diagnosis of cancer the new treatment options need to be accompanied by the best available health economic tools. This pertains to well-implemented decision rules concerning willingness to pay, incremental cost-effectiveness ratio thresholds, equity, patient access, end of life criteria, etc. Their application differs with regard to the usual health economic analyzes implemented in other treatment areas. Overstating a bit one could ask whether we need a strongly modified concept of oncology economics?

Approaches to Capturing Value in Oncology.

This article sets out to describe different value frameworks in the field of new developments in oncology. Since the costs of new oncological therapies follow a steep path, their implementation and financing demand a thorough assessment. This is an ambitious task due to the complex nature of oncological treatments within overall health policy. Five value frameworks were reviewed: European Society for Medical Oncology (ESMO) Magnitude of Clinical Benefit Scale, American Society of Clinical Oncology (ASCO) Value Framework (version 2.0), National Comprehensive Cancer Network (NCCN) Evidence Blocks, Memorial Sloan Kettering Cancer Center DrugAbacus, and the Institute for Clinical and Economic Review Value Assessment Framework. They are all based on a large set of criteria. However, all these frameworks differ considerably in their outcomes. Among the main differences one has to cite are the inclusion of costs and the use of different outcomes, as well as the fact that they address different target stakeholders, etc. Despite these shortcomings, the value frameworks serve the necessity to introduce more rationality in health decision making seen from the perspective of physicians, patients, and financing bodies.

Cost-utility analysis of alemtuzumab in comparison with interferon beta, fingolimod, and natalizumab treatment for relapsing-remitting multiple sclerosis in Austria.

BACKGROUND: Multiple sclerosis (MS), a chronic progressive, demyelinating, inflammatory disease, affects 2.5 million people worldwide. Approximately 63% of cases are classified as relapsing-remitting MS (RRMS) at the time of diagnosis. The aim of this cost-utility analysis is to evaluate alemtuzumab vs interferon beta (intramuscular [IM] interferon beta-1a, subcutaneous [SC] interferon beta-1a, SC interferon beta-1b, and SC pegylated interferon beta-1a) in previously treated, and vs SC interferon beta-1a, fingolimod, and natalizumab in untreated RRMS patients to determine the incremental cost-effectiveness ratio among the treatment alternatives as prices, the route, and the frequency of administration of considered products vary significantly. METHODS: The primary outcome was the modeled incremental cost-effectiveness ratio (ICER; €/quality-adjusted life-year [QALY] gained). Markov modeling with a 10-year time horizon was carried out. During each 3-month cycle, patients maintained the Expanded Disability Status Scale (EDSS) score or experienced progression, developed secondary progressive MS (SPMS), or showed EDSS progression in SPMS; experienced relapses; suffered from an adverse event (AE); changed treatment; or died. A published network meta-analysis (NMA) was used for indirect comparison. The possibility of a therapy switch was considered. Clinical input data and resource utilization data were derived from the literature. Costs were extracted from price lists published in Austria and were calculated from the payer's perspective. RESULTS: In treatment naïve patients, alemtuzumab is associated with costs of €132,663 and 5.25 QALYs in a 10-year time horizon. Costs for SC interferon beta amount to €164,159 and generate 4.85 QALYs. Also, in the pre-treated patients, alemtuzumab dominated comparators by accumulating higher total QALYs (4.88) and lower total costs (€137.409) compared to interferon beta-1a (€200.133), fingolimod (€240.903), and natalizumab (€247.758). CONCLUSION: The analysis shows that alemtuzumab is a cost-saving alternative to treat RRMS in pre-treated and therapy naïve patients. From the patient perspective, alemtuzumab improves quality-of-life.

Cost-utility analysis of fracture risk assessment using microRNAs compared with standard tools and no monitoring in the Austrian female population.

BACKGROUND: Osteoporosis poses an immense burden to the society in terms of morbidity, mortality and financial cost. To reduce this burden, it is essential to accurately assess the individual patient's fracture risk and, where indicated, to initiate appropriate treatment that reduces fracture probability. Current screening and monitoring approaches include utilization of FRAX®, a web-based country-specific fracture risk assessment tool, and bone mineral density measurement by Dual Energy X-ray Absorptiometry (DXA). Recently, microRNAs have been recognized as important regulators of bone physiology and potential biomarkers for fracture risk assessment and monitoring. A fracture risk assessment tool based on microRNAs (osteomiR™ test) is currently being developed. The aim of this study was to estimate the cost-effectiveness of fracture risk screening, monitoring, and resulting treatment decisions for the Austrian female population using the osteomiR™ test compared with DXA, with FRAX®, or with no screening/monitoring. METHODS: A cost-utility-model was developed to simulate long-term consequences of Austrian women from age 50 over lifetime or death with respect to osteoporosis. Markov-modelling techniques were used to calculate health state transitions of fracture incidence according to risk groups (high, intermediate, low). High-risk patients receive medical treatment. Probabilities were derived via systematic-literature-review; direct costs (2015, €) from published sources from the payer's perspective. Results evaluate the incremental cost-effectiveness ratios (ICER) for osteomiR™ against the comparators, gains or losses of fractures, life years (LYs), quality-adjusted life years (QALYs), and direct costs. QALYs, life years (LYs) and costs were discounted (3% p.a). RESULTS: Fracture risk assessment and monitoring using the osteomiR™ test reduces fracture incidence compared with no monitoring, DXA alone, or FRAX® alone. In the per-patient analysis, the ICER/QALY of osteomiR™ vs. no-monitoring was 13,103 €, vs. FRAX® 37,813 €, and vs. DXA -19,605 €, indicating that costs can be saved while gaining QALYs. Considering the total cohort over lifetime, the osteomiR™ test can avoid 57,919 fractures compared with DXA, 31,285 fractures compared with FRAX® and 133,394 fractures compared with no monitoring. Sensitivity analysis confirmed the robustness of these findings. CONCLUSION: Fracture risk assessment and monitoring using the osteomiR™ test dominates DXA-strategy and constitutes a cost-effective alternative to FRAX®, and no-monitoring, respectively.

Congenital toxoplasmosis in Austria: Prenatal screening for prevention is cost-saving.

BACKGROUND: Primary infection of Toxoplasma gondii during pregnancy can be transmitted to the unborn child and may have serious consequences, including retinochoroiditis, hydrocephaly, cerebral calcifications, encephalitis, splenomegaly, hearing loss, blindness, and death. Austria, a country with moderate seroprevalence, instituted mandatory prenatal screening for toxoplasma infection to minimize the effects of congenital transmission. This work compares the societal costs of congenital toxoplasmosis under the Austrian national prenatal screening program with the societal costs that would have occurred in a No-Screening scenario. METHODOLOGY/PRINCIPAL FINDINGS: We retrospectively investigated data from the Austrian Toxoplasmosis Register for birth cohorts from 1992 to 2008, including pediatric long-term follow-up until May 2013. We constructed a decision-analytic model to compare lifetime societal costs of prenatal screening with lifetime societal costs estimated in a No-Screening scenario. We included costs of treatment, lifetime care, accommodation of injuries, loss of life, and lost earnings that would have occurred in a No-Screening scenario and compared them with the actual costs of screening, treatment, lifetime care, accommodation, loss of life, and lost earnings. We replicated that analysis excluding loss of life and lost earnings to estimate the budgetary impact alone. Our model calculated total lifetime costs of €103 per birth under prenatal screening as carried out in Austria, saving €323 per birth compared with No-Screening. Without screening and treatment, lifetime societal costs for all affected children would have been €35 million per year; the implementation costs of the Austrian program are less than €2 million per year. Calculating only the budgetary impact, the national program was still cost-saving by more than €15 million per year and saved €258 million in 17 years. CONCLUSIONS/SIGNIFICANCE: Cost savings under a national program of prenatal screening for toxoplasma infection and treatment are outstanding. Our results are of relevance for health care providers by supplying economic data based on a unique national dataset including long-term follow-up of affected infants.

Cost-effectiveness of continuous subcutaneous apomorphine in the treatment of Parkinson's disease in the UK and Germany.

BACKGROUND: Parkinson's disease (PD) is the second most common neurodegenerative disease, affecting ∼ 5.2 million people worldwide. Continuous subcutaneous apomorphine (CSAI) represents an alternative treatment option for advanced PD with motor fluctuation. The purpose of this analysis was to estimate the cost-effectiveness of CSAI compared with Levodopa/carbidopa intestinal gel (LCIG), Deep-Brain-Stimulation (DBS) and Standard-of-care (SOC). METHODS: A multi-country Markov-Model to simulate the long-term consequences, disease progression (Hoehn & Yahr stages 3-5, percentage of waking-time in the OFF-state), complications, and adverse events was developed. Monte-Carlo simulation accounted for uncertainty. Probabilities were derived from RCT and open-label studies. Costs were estimated from the UK and German healthcare provider's perspective. QALYs, life-years (LYs), and costs were projected over a life-time horizon. RESULTS: UK lifetime costs associated with CSAI amounts to £78,251.49 and generates 2.85 QALYs and 6.28 LYs (€104,500.08, 2.92 QALYs and 6.49 LYs for Germany). Costs associated with LCIG are £130,011.34, achieves 3.06 QALYs and 6.93 LYs (€175,004.43, 3.18 QALYs and 7.18 LYs for Germany). The incremental-cost per QALY gained (ICER) was £244,684.69 (€272,914.58). Costs for DBS are £87,730.22, associated with 2.75 QALYs and 6.38 LYs (€105,737.08, 2.85 QALYs and 6.61 LYs for Germany). CSAI dominates DBS. SOC associated UK costs are £76,793.49; 2.62 QALYs and 5.76 LYs were reached (€90,011.91, 2.73 QALYs and 6 LYs for Germany). CONCLUSIONS: From a health economic perspective, CSAI is a cost-effective therapy and could be seen as an alternative treatment to LCIG or DBS for patients with advanced PD.

Socio-economic aspects of the testing for antibodies in MS-patients under interferon therapy in Austria: a cost of illness study.

BACKGROUND: According to EU-guidelines testing patients on interferon-beta (IFNb) for the presence of neutralising antibodies (NAb) is recommended; IFNb treatment efficacy of NAb-positive patients equals that of placebo-treated patients. Economic impact of NAb testing in MS patients has not been explored yet. The aim of this analysis is to estimate the impact of NAb testing in RRMS-patients on Austria׳s health-care-system. METHODS: A decision-analytic model over 5 years was performed. The cost effectiveness of NAb testing versus no testing was evaluated. The model considers switching after relapse and withdrawal. All direct costs are based on Austrian data from 2013 and were discounted at 5% per year. The efficacy outcome measure was "relapse free". Clinical data and resource use were determined by literature. RESULTS: Total costs for all Austrian MS-patients on IFNb-therapy with testing amount to 187,554,021€ over 5 years; without testing is 175,091,300 €. Costs per relapse avoided over 5 years were 90,075€ in the NAb testing arm, and 99,535€ in the no NAb test arm, resulting in a difference of 9460€ in favour of routine NAb testing. Considering all 3590 IFNb-treated patients 2082 relapses can be avoided in the NAb testing arm versus 1759 in the no-testing arm within 5 years. Testing for NAb leads to costs per relapse avoided of 18,015€ per year versus 19,907€ when no tests are done. CONCLUSION: The results suggest that NAb testing reduces relapses and associated costs.

Cost analysis: treatment of chemotherapy-induced anemia with erythropoiesis-stimulating agents in five European countries.

OBJECTIVE: Cost-analysis comparing darbepoetin-alfa (DARB), epoetin-alfa (EPO-A), and epoetin-beta (EPO-B) for treatment of chemotherapy-induced anemia in Belgium concluded that costs for DARB-treated patients were significantly lower than costs for EPO-A- or EPO-B-treated patients. The objective of the present study was to extend the Belgian analysis to Austria, France, Italy, Portugal, and Spain, estimating differences in costs between erythropoiesis-stimulating agents (ESAs) in each country. METHODS: Differences in epidemiology and treatment patterns between countries were adjusted using data from Eurostat, national cancer registries, IMS sales data, and reimbursement and treatment guidelines. Belgian unit costs were replaced with country-specific costs. Costs were analyzed using a mixed-effects model stratifying for propensity score quintiles. RESULTS: All populations were comparable to the Belgian population in terms of age, gender, ESA, and blood transfusions use. After adjusting for country-specific chemotherapy use and cancer incidence, total management costs per patient (Euro, 2010) were 19-26% (France, Spain) lower with DARB compared with EPO-A (p < 0.0001) and 20-36% (Portugal, Austria) compared with EPO-B (p < 0.01). Anemia-related costs with DARB were between 12% (Portugal; p = 0.0235) and 38% (Italy; p < 0.0001) lower compared with EPO-A (p < 0.01; all remaining countries), and between 13% (Austria; p = 0.064) and 19% (Portugal; p = 0.0028) lower compared with EPO-B (p < 0.05; all remaining countries except Italy; p = 0.0935). LIMITATIONS: Not all differences could be accounted for by a lack of country-specific data; however, the potential under- and over-estimation of costs should be similar for all three ESAs. CONCLUSIONS: These findings are in line with the Belgian analysis. In all countries, total and anemia-related costs were lowest in patients receiving DARB vs EPO-A or EPO-B. This study demonstrates the feasibility of adapting real-life country-specific data to other settings, adjusting for differences in patients' characteristics and treatment strategies. These findings should be valuable in healthcare decision-making in oncology patients treated in each of the countries studied.

Cost-effectiveness of palivizumab for respiratory syncytial virus infection in high-risk children, based on long-term epidemiologic data from Austria.

OBJECTIVE: To assess the cost-effectiveness of palivizumab, a monoclonal antibody against respiratory syncytial virus (RSV), in infants at high risk for severe RSV lower respiratory tract infection, such as premature infants, infants with bronchopulmonary dysplasia, and those with congenital heart disease, based on long-term epidemiologic data from Austria. METHODS: A decision-tree model was used, and the analysis was based on a lifetime follow-up investigating cost-effectiveness of palivizumab versus no RSV infection prevention. The primary perspective of the study was that of the healthcare system, the second that of society. Cost and effects were discounted by 5%. The base case analysis included only direct medical costs, and a scenario analysis included various indirect costs. RESULTS: Analyses were based on epidemiologic data on a total of 1579 children hospitalized because of RSV lower respiratory tract infection during 16 seasons. The incremental cost-effectiveness ratio for the first outcome measure (life years gained) amounted to discounted costs of €34,956 (for all preterm infants), €35,056 (for < 33 weeks' gestational age [wGA] infants), €35,233 (for 33-35 wGA infants), €35,611 (for infants with bronchopulmonary dysplasia), and €8956 (for infants with congenital heart disease). Use of palivizumab compared with no prophylaxis had an incremental cost-utility ratio of €26,212, €26,292, €24,392, €24,654, and €8484, respectively, per quality-adjusted life years. Results from the society perspective were more cost-effective in all study populations. An additional scenario analysis with 7 injections for the 33 to 35 wGA group revealed cost-effectiveness as well. CONCLUSIONS: Our results based on nationwide long-term epidemiologic data suggest that palivizumab is cost-effective in prevention of RSV disease in high-risk infants.