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Nadia Quignot,1 Heng Jiang,1 Indraraj Umesh Doobaree,2 Jan Lehmann,3 Ola Ghatnekar4 1Evidence & Access, Certara France, Paris, France; 2Evidence & Access, Certara UK, Sheffield, UK; 3Department of Urology, Städtisches Krankenhaus, Kiel, Germany; 4Ferring Pharmaceuticals, Kastrup, DenmarkCorrespondence: Nadia Quignot, Certara France, 54 Rue de Londres, Paris, 75008, France, Tel +3 318 514 2683, Email nadia.quignot@certara.comBackground: Intermediate and high-risk non-muscle-invasive bladder cancer (NMIBC) is typically managed with transurethral resection of the bladder tumour (TURBT) followed by intravesical Bacillus Calmette–Guérin (BCG) immunotherapy; however, NMIBC patients can become refractory or unresponsive to BCG treatment, and/or progress to muscle-invasive bladder cancer (MIBC). Healthcare resource utilization (HCRU) and costs in these patient populations are high.Methods: A retrospective longitudinal cohort design of adult (≥ 18 years) patients with bladder cancer and BCG treatment (01/01/2012– 31/12/2017) was conducted using data from a representative subset of the German statutory health insurance database. During the follow-up period after last BCG, patients were categorized into subgroups of No further NMIBC treatment, Continuous treatment for NMIBC, or MIBC evidence; HCRU and costs were tabulated for each subgroup and for the entire cohort.Results: A total of 1049 patients met the study inclusion criteria (mean age, 70.9 years; 84.8% male). Across the different subgroups, patients showing MIBC evidence had more than two times higher hospitalization rates compared to the other subgroups. Overall, the entire BCG-treated cohort’s total direct medical cost including hospitalizations, outpatient care and drugs was € 33.9 million and € 9250 per patient-year. Cost for patients with MIBC evidence was much higher, at € 17,983 per patient-year, than patients with No further NMIBC treatment (€ 6617) and patients with Continuous treatment for NMIBC (€ 7786). Across the subgroups, hospitalization was the largest driver of cost and contributed the most to cost for those with MIBC evidence.Conclusion: The overall cost burden of this BCG-treated cohort of 1049 patients is high (€ 38 million whereof 4.1 million are indirect costs) over a mean follow-up of 3.9 years; economic burden is especially substantial for patients who fail BCG treatment and those who progress.Keywords: urinary bladder neoplasm, retrospective study, healthcare cost, intravesical instillation, healthcare resource

Background There are few recent studies on the use of 5-aminosalicylates (5-ASA) as therapy for Crohn’s disease (CD) in routine clinical practice. The aim of this database investigation was to provide real-world evidence on 5-ASA use in CD. Methods Patients with CD, aged ≥18 years when first prescribed 5-ASA (index date) and having received 5-ASA at any time between 01 January 2006 and 07 May 2018, were included for analysis. Outcomes included treatment patterns and resource use. Results Of 21,456 patients with CD, 9492 (44.2%) had been prescribed 5-ASA, with the majority (5606; 59.1%) starting on oral 5-ASA as monotherapy. 58.3% (5537) of patients on 5-ASA did not require dose change, 67.6% (6416) did not require supplementary treatment (e.g., corticosteroids, immunosuppressants, etc.), and 4.6% (436) required a switch to another treatment. Resource use was significantly decreased in the year after vs. year before 5-ASA initiation (including: specialist referrals, hospitalizations and hospital days; all P

Budesonide with multi-matrix technology (MMX) is an oral corticosteroid, shown to have high topical activity against ulcerative colitis (UC) while maintaining low systemic bioavailability with few adverse events. The aim of this study was to evaluate the cost-effectiveness of budesonide MMX versus commonly used corticosteroids, in the second-line treatment of active mild-to-moderate UC in the Netherlands.An eight-state Markov model with an 8 week cycle length captured remission, four distinct therapy stages, hospitalization, possible colectomy and mortality. Remission probability for budesonide MMX was based on the CORE-II study. Population characteristics were derived from the Dutch Inflammatory Bowel Disease South Limburg cohort (n = 598) and included patients with proctitis (39%), left-sided (42%) and extensive disease (19%). Comparators (topical budesonide foam and enema, oral budesonide and prednisolone) were selected based on current Dutch clinical practice. Treatment effects were evaluated by network meta-analysis using a Bayesian framework. Cost-effectiveness analysis was performed over a 5 year time horizon from a societal perspective, with costs, health-state and adverse event utilities derived from published sources. Outcomes were weighted by disease extent distribution and corresponding comparators.Budesonide MMX was associated with comparable quality-adjusted life year (QALY) gain versus foam and oral formulations (+0.01 QALYs) in the total UC population, whilst being cost-saving (EUR 366 per patient). Probabilistic sensitivity analysis evaluated an 86.6% probability of budesonide MMX being dominant (cost-saving with QALY gain) versus these comparators. Exploratory analysis showed similar findings versus prednisolone.Differing definitions of trial end-points and remission across trials meant indirect comparison was not ideal. However, in the absence of head-to-head clinical data, these comparisons are reasonable alternatives and currently offer the only comparison of second-line UC treatments.In the present analysis, budesonide MMX was shown to be cost-effective versus comparators in the total UC population, for the second-line treatment of active mild-to-moderate UC in the Netherlands.

Background: Stroke is one of the major causes of disability in the adult population and represents a heavy social and economic burden. Currently available therapeutic tools to support the recovery of impaired brain functions are quite limited. Animal studies have demonstrated that neuronal replacement and partial reconstruction of neural circuitry or modulation of the recovery process is possible with cell transplantation in the damaged adult brain. Stem cell therapy (SCT) may promote functional recovery also in stroke patients, thereby improving quality of life and reducing costs. Our aim was to estimate the potential societal value of SCT in stroke patients. Methods: We created a decision-analytic model in Microsoft Excel 2010 to assess life-long costs and quality-adjusted life years (QALYs) of SCT versus standard care for stroke patients from a societal perspective. The model structure consisted of 7 health states in accordance with the modified Rankin Scale (mRS). We modeled for age (55, 65, and 75 years), functional status at discharge (mRS 2, 3, and 4), effectiveness of SCT (50 and 25% increase in the probability to improve 1 mRS grade), mode of stem cell administration, risk of recurrent stroke, complications of intervention, and use of immunosuppressive drugs. The difference between an assumed societal willingness to pay for a QALY gain in Sweden (110,400 USD) and the cost per QALY gain resulting from the model was interpreted as the value of SCT. Results: Increased survival (1.06 life years) and improved functional status gave rise to an estimated gain of 1.34 QALY in a cohort of patients aged 55 with mRS 2 at hospital discharge. Although the SCT intervention increased costs by 64,014 USD (excluding cost of stem cells), the costs of intervention were offset mainly by decreased productivity losses. In total, the intervention saved 19,055 USD, i.e., at a price of 19,055 USD for stem cells, the SCT would be cost neutral. The societal value of SCT was 166,500 USD. Conclusions: The application of the health-economic model to Sweden shows that in younger stroke patients with moderate disability, the societal value of SCT given a zero price of stem cells is 166,500 USD. Although the transplantation itself is more costly, SCT offers potential for cost offset and cost savings in a long-term perspective by reducing the disability after stroke. The therapy appeared cost effective under a wide range of assumptions. Hence, further research and development in stem cells suitable for stroke therapy could potentially produce great value to society.

A long-term cost-effectiveness model for early decision-making and estimation of outcomes of novel therapeutic procedures for Parkinson's disease (PD) was developed based on the Hoehn and Yahr (HY) stages of PD. Results provided support for model validity. Model application to a future dopamine cell replacement therapy indicated long-term cost offsets and gains in quality-adjusted life years (QALYs) in early onset PD (HY III-IV), as compared to standard drug therapy. The maximum price premium (i.e., profit or compensation for developmental costs) for the intervention to remain cost-effective was estimated to EURO12000-64000 according to cost-per-QALY thresholds of EURO38000-70000 and depending on whether all or only medical direct costs are considered. The study illustrates the value of early health economic modeling and the described model shows promise as a means to estimate outcomes and aid decision-making regarding novel interventions for PD. (c) 2006 Elsevier Ltd. All rights reserved.

Background: Societal decision makers increasingly emphasise their need for evidence based economic analyses to make reimbursement decisions. Objective: To analyse the cost utility of adalimumab, on both incremental cost and incremental quality adjusted life years (QALYs), versus traditional disease modifying antirheumatic drugs and the other tumour necrosis factor (TNF) antagonists suitable for submission to the Swedish LFN (Pharmaceutical Benefit Board). Methods: Swedish unit costs and treatment guidelines from a lifetime perspective were implemented. A mathematical model, incorporating data from seven trials, simulated the experiences of 10 000 hypothetical patients with moderate to severe rheumatoid arthritis (RA). The primary outcome measure—QALYs—was derived from utility values calculated from a relationship between the Health Assessment Questionnaire (HAQ) Disability Index (DI) and Health Utility Index-III (HUI-3) from adalimumab trial results. The model followed the progression of HAQ-DI through a number of treatments in a sequence accounting for mortality, drug and monitoring costs, and other direct costs. Results: When using ACR50 as a response threshold for determining successful treatment, adalimumab plus methotrexate showed the greatest number of QALYs gained (2.3 from one study and 2.1 from the pooled results of two trials). The etanercept plus methotrexate strategy yielded QALY gains similar to the pooled adalimumab results. Except for the infliximab strategy, the costs results were between €35 000 and €42 000, a range normally considered cost effective in other European countries. Conclusion: Adalimumab appears to be cost effective for the treatment of moderate to severe RA. The results suggest that adalimumab is at least as cost effective as other TNF antagonists.

Objective: To evaluate the cost effectiveness of a multidisciplinary team including a pharmacist for systematic medication review and reconciliation from admission to discharge at hospital among elderly patients (the Lund Integrated Medicines Management (LIMM)) in order to reduce drug-related readmissions and outpatient visits. Method: Published data from the LIMM project group were used to design a probabilistic decision tree model for evaluating tools for (1) a systematic medication reconciliation and review process at initial hospital admission and during stay (admission part) and (2) a medication report for patients discharged from hospital to primary care (discharge part). The comparator was standard care. Inpatient, outpatient and staff time costs (Euros, 2009) were calculated during a 3-month period. Dis-utilities for hospital readmissions and outpatient visits due to medication errors were taken from the literature. Results: The total cost for the LIMM model was €290 compared to €630 for standard care, in spite of a €39 intervention cost. The main cost offset arose from avoided drug-related readmissions in the Admission part (€262) whereas only €66 was offset in the Discharge part as a result of fewer outpatient visits and correction time. The reduced disutility was estimated to 0.005 quality-adjusted life-years (QALY), indicating that LIMM was a dominant alternative. The probability that the intervention would be cost-effective at a zero willingness to pay for a gained QALY compared to standard care was estimated to 98%. Conclusions: The LIMM medication reconciliation (at admission and discharge) and medication review was both cost-saving and generated greater utility compared to standard care, foremost owing to avoided drug-related hospital readmissions. When implementing such a review process with a multidisciplinary team, it may be important to consider a learning curve in order to capture the full advantage.

Purpose: To map health outcome related variables from a national register, not part of any validated instrument, with EQ-5D weights among stroke patients. Methods: We used two cross-sectional data sets including patient characteristics, outcome variables and EQ-5D weights from the national Swedish stroke register. Three regression techniques were used on the estimation set (n = 272): ordinary least squares (OLS), Tobit, and censored least absolute deviation (CLAD). The regression coefficients for “dressing“, “toileting“, “mobility”, “mood”, “general health” and “proxy-responders” were applied to the validation set (n = 272), and the performance was analysed with mean absolute error (MAE) and mean square error (MSE). Results: The number of statistically significant coefficients varied by model, but all models generated consistent coefficients in terms of sign. Mean utility was underestimated in all models (least in OLS) and with lower variation (least in OLS) compared to the observed. The maximum attainable EQ-5D weight ranged from 0.90 (OLS) to 1.00 (Tobit and CLAD). Health states with utility weights

Introduction: Little is known regarding healthcare costs for HIV/AIDS patients in the era of highly active antiretroviral therapy (HAART) and subgroups of patients according to the severity and progression of HIV infection in Sweden. The objective of this study is therefore to describe the direct medical resource use and cost of healthcare for HIV patients at a university clinic in Sweden. Methods: A patient registry database for HIV treatment at the Department of Infectious Diseases, Sahlgrenska University Hospital, between 2000 and 2005 provided information on patient characteristics, antiretroviral drugs and dosages, tests and diagnostic procedures, outpatient visits and inpatient stays. The review used publicly available unit costs with a county council perspective, expressed in 2006 Euros. Results: Two hundred and eighty-five patients with a mean age of 38 years in 2000 (64% men) were followed for 1368 patient-years. They had a mean (median) of 6.3 (0) inpatient days, 4.1 (3.7) physician visits, 4.2 (3.8) nurse visits, 2.6 (0.7) counsellor visits and 11.5 (7.7) tests and diagnostic procedures per patient-year. Only 12 deaths were recorded during the study period, and the proportion of treated patients with successful treatment (HIV-RNA

ObjectiveAtrial fibrillation (AF) is an important risk factor for stroke. It is prevalent in approximately one-fourth of stroke patients, and predictive of worse outcomes. This study aimed to analyze the effect of AF on stroke-related inpatient costs among first-ever stroke patients in Sweden.MethodsHospitalizations and death records were monitored for 3 years in 6611 first-ever stroke patients. For stroke as primary diagnosis, inpatient costs were calculated on the basis of length of stay at different wards. For stroke as secondary diagnosis, costs were based on diagnosis-related groups.ResultsPatients with AF (24% of all patients) were older (80 years vs. 73 years), had a higher prevalence of hypertension (49% vs. 41%) and/or diabetes (22% vs. 19%), higher risk of experiencing a restroke, and higher case fatality rate (43% vs. 25%) than patients without AF. The average cost per patient over 3 years was €9004, with no statistically significant difference between AF and non-AF patients. However, a multiple regression analysis showed that the presence of AF resulted in higher costs after considering a number of background factors. Among patients surviving the index event, AF patients had on average €818 higher inpatient costs over 3 years than non-AF patients (€10,192 vs. €9374, P < 0.01). The difference in costs was highest for patients aged

Objectives:To estimate the excess cost of stroke in Sweden and the potential costs that could be avoided by preventing first-ever strokes.Methods:We adopted the incidence approach for estimating the present value of both direct and indirect costs. Data on mortality, stroke recurrence, and inpatient care were estimated from a national register of patient data with a four-year follow-up period. To estimate costs for social services, we used survey data on living conditions before stroke onset and at three and at twenty-four months. Costs for outpatient visits, rehabilitation, drugs, and production losses due to premature death and early retirement were estimated on the basis of both published and nonpublished sources. Lifetime costs were based on life tables adjusted for excess mortality of stroke, and costs in year 4 were extrapolated to subsequent years.Results:The present value direct cost for an average stroke patient is SEK513,800 (US$56,024 or Euro60,825). The corresponding indirect cost is SEK125,110 (US$13,640 or Euro14,810). Almost 45 percent of the direct costs were attributable to social services. Women had higher costs than men, and costs for survivors increased with age due to social services.Conclusions:With an incidence of 213 first-ever strokes per 100,000 individuals, the total excess direct and indirect cost of stroke would be SEK12.3 billion (approximately US$1.3 billion or Euro1.5 billion). Hence, there are large potential cost offsets both in the health-care sector and in the social service sector if the incidence of first-ever stroke could be reduced.