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1.
  • Acosta, C., et al. (författare)
  • Modeling the cost-effectiveness of prolonged-release fampridine for the treatment of walking impairment in patients with multiple sclerosis in Sweden
  • 2021
  • Ingår i: Journal of Medical Economics. - : Informa UK Limited. - 1369-6998 .- 1941-837X. ; 24:1, s. 770-780
  • Tidskriftsartikel (refereegranskat)abstract
    • Aims: To evaluate the cost-effectiveness of adding prolonged-release (PR)-fampridine to best supportive care (BSC) versus BSC alone for the improvement of walking ability in patients with MS. Methods: A cost-utility analysis based on a Markov model was developed to model responders and timed 25-foot walk (T25FW) scores, accumulated costs, and quality-adjusted life-years (QALY) in adults with MS and Expanded Disability Status Scale (EDSS) scores between 4 and 7. The analysis was conducted from a Swedish societal perspective. Results: In the base-case analysis, PR-fampridine plus BSC led to a higher QALY gain than BSC alone. The largest direct cost was professional care provision followed by hospital inpatient stays while the indirect cost was the loss of earnings due to days off work. The incremental cost-effectiveness ratio (ICER) for PR-fampridine plus BSC compared with BSC alone was 57,109 Swedish Kronor (kr)/QALY (€5,607/QALY [1 kr = €0.0981762 on 8 April 2021] and $6,675/QALY [1 kr = $0.116890 on 8 April 2021]). All sensitivity analyses performed resulted in ICERs below 500,000 kr (€49,088 and $58,445). Limitations: Resource use data were not specific to the Swedish market. Conclusions: PR-fampridine represents a cost-effective treatment for MS-related walking impairment in Sweden, due to improvements in patients’ quality of life and reduced healthcare resource utilization. © 2021 Biogen International GmbH. Published by Informa UK Limited, trading as Taylor & Francis Group.
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  • Andersson, Fredrik, et al. (författare)
  • Assessing the impact of nocturia on health-related quality-of-life and utility: results of an observational survey in adults
  • 2016
  • Ingår i: Journal of Medical Economics. - : TAYLOR & FRANCIS LTD. - 1369-6998 .- 1941-837X. ; 19:12, s. 1200-1206
  • Tidskriftsartikel (refereegranskat)abstract
    • Background and aim: The impact of nocturia (getting up at night to void) on health-related quality-of-life (HRQoL) is often under-estimated. This study investigated the relative burden in terms of HRQoL and utilities of nocturia in a real-world setting. Methods: Patient data were collected from two surveys: a nocturia-specific, cross-sectional survey of physicians and their patients (DSP), and a general UK population health survey (HSFE). Utilities (EQ-5D-5L), productivity (Work Productivity and Activity Index), and the impact of nocturia symptoms (Nocturia Impact Diary and Overactive Bladder Questionnaires) were assessed against the number of voids. A robust linear regression model with propensity score weights was used to control for confounding factors in estimating utilities. Results: Physician-recorded data were available from 8,738 patients across the US, Germany, Spain, France, and the UK; of these, 5,335 (61%) included patient-reported outcomes. In total, 6,302 controls were drawn from the two surveys and compared to 1,104 nocturia patients. Deterioration of HRQoL was associated with increasing number of night-time voids (pamp;lt;0.0001). In particular, significant differences were observed between 0-1 and 2 voids (pamp;lt;0.001). The regression model demonstrated that nocturia (amp;gt;= 2 per night) is associated with a modest but significant deterioration in utility of 0.0134 (pamp;lt;0.05). Limitations: The cause of nocturia is multifactorial and the mostly elderly patients may have several concomitant diseases. The authors tried to adjust for the most common ones, but there may be diseases or unknown relationships not included. Conclusions: Nocturia negatively affected HRQoL and patient utility. A clear effect is seen already at two voids per night. Every effort should, therefore, be made to reduce nocturia below the bother threshold of two voids per night.
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  • Andersson, Maria, et al. (författare)
  • Cost effectiveness of benralizumab for severe, uncontrolled oral corticosteroid-dependent asthma in Sweden
  • 2020
  • Ingår i: Journal of Medical Economics. - : TAYLOR & FRANCIS LTD. - 1369-6998 .- 1941-837X. ; 23:8, s. 877-884
  • Tidskriftsartikel (refereegranskat)abstract
    • Aim: We investigated cost effectiveness of benralizumab vs. standard of care (SOC) plus oral corticosteroids (OCS) for patients with severe, eosinophilic OCS-dependent asthma in Sweden. Materials and methods: A three-state, cohort-based Markov model of data from three Phase III benralizumab clinical trials (ZONDA [NCT02075255], SIROCCO [NCT01928771], and CALIMA [NCT01914757]) was used to assess the incremental cost-effectiveness ratio of benralizumab vs. SOC plus OCS. Health outcomes were estimated in terms of quality-adjusted life-years (QALYs). The model included costs and disutilities associated with extrapolated OCS-related adverse events. Patients with severe asthma were defined as those receiving OCS >= 5 mg/day. Results: Benralizumab demonstrated a cost-effectiveness ratio vs. SOC plus OCS of 2018 Swedish Kronor (SEK) 366,855 (euro34,127) per QALY gained, based on increases of 1.33 QALYs and SEK 488,742 (euro45,344) per patient. Benralizumab treatment costs contributed most to incremental costs. The probability of benralizumab's being cost-effective with willingness-to-pay (WTP) thresholds between SEK 429,972 (euro40,000) and SEK 752,452 (euro70,000) ranged from 75% to 99%. Limitations: Potential limitations of these analyses include the use of combined data from three different clinical trials, a one-way sensitivity analysis that did not include mortality and transition estimates, and Observational & Pragmatic Research Institute (OPRI) data from the UK as a proxy of the Swedish health care system. Conclusions: The results of these analyses demonstrate that benralizumab has a high probability of being cost-effective compared with SOC plus OCS for a subgroup of patients with severe, eosinophilic asthma receiving regular OCS treatment and may support clinicians, payers and patients in making treatment decisions.
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  • Bakhai, Ameet, et al. (författare)
  • Development of a health economic model to evaluate the potential benefits of optimal serum potassium management in patients with heart failure.
  • 2018
  • Ingår i: Journal of Medical Economics. - : Informa UK Limited. - 1369-6998 .- 1941-837X. ; 21:12, s. 1172-1182
  • Tidskriftsartikel (refereegranskat)abstract
    • Aims: Patients with heart failure are at increased risk of hyperkalemia, particularly when treated with renin-angiotensin-aldosterone system inhibitor (RAASi) agents. This study developed a model to quantify the potential health and economic value associated with sustained potassium management and optimal RAASi therapy in heart failure patients.Materials and methods: A patient-level, fixed-time increment stochastic simulation model was designed to characterize the progression of heart failure through New York Heart Association functional classes, and predict associations between serum potassium levels, RAASi use, and consequent long-term outcomes. Following internal and external validation exercises, model analyses sought to quantify the health and economic benefits of optimizing both serum potassium levels and RAASi therapy in heart failure patients. Analyses were conducted using a UK payer perspective, independent of costs and utilities related to pharmacological potassium management.Results: Validation against multiple datasets demonstrated the predictive capability of the model. Compared to those who discontinued RAASi to manage serum potassium, patients with normokalemia and ongoing RAASi therapy benefited from longer life expectancy (+1.38 years), per-patient quality-adjusted life year gains (+0.53 QALYs), cost savings (110) pound, and associated net monetary benefit (10,679 pound at 20,000 pound per QALY gained) over a lifetime horizon. The predicted value of sustained potassium management and ongoing RAASi treatment was largely driven by reduced mortality and hospitalization risks associated with optimal RAASi therapy.Limitations: Several modeling assumptions were made to account for a current paucity of published literature; however, ongoing refinement and validation of the model will ensure its continued accuracy as the clinical landscape of hyperkalemia evolves.Conclusions: Predictions generated by this novel modeling approach highlight the value of sustained potassium management to avoid hyperkalemia, enable RAASi therapy, and improve long-term health economic outcomes in patients with heart failure.
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  • Björnerstedt, Jonas, et al. (författare)
  • Validation of overall survival extrapolations made by TLV in the assessment of cost-effectiveness of oncology drugs in Sweden : A pilot study comparing extrapolated and observed life-years gained
  • 2024
  • Ingår i: Journal of Medical Economics. - : Taylor & Francis. - 1369-6998 .- 1941-837X. ; 27:1, s. 193-200
  • Tidskriftsartikel (refereegranskat)abstract
    • Aim: The aim of the study is to assess the accuracy of overall survival (OS) extrapolations in cost-effectiveness analysis made by the Dental and Pharmaceutical Benefits Agency (TLV) to decide on the reimbursement and use of oncology drugs in Sweden.Material and methods: TLV appraisals for oncology drugs were identified during a 5-year period (2013-2017). To be included each appraisal and health economic model must include a TLV base case extrapolation of OS. Further, Kaplan-Meier (KM) estimates on OS from the original and follow-up clinical trials must be available. Potential follow-up trials on OS were identified in ClinicalTrials.gov and the Lund University Libraries databases, and in the Swedish Medical Products Agency (MPA) and the European Medicines Agency (EMA) databases. In cases where the KM estimates were not available, data points from figures published in TLV's appraisals were extracted using the semi-automated tools Digitizelt and WebPlotDigitizer. The accuracy of survival extrapolations was assessed by comparing extrapolated and observed life-years (LYs), using three different measures: 1) difference in LYs between the treatment and control group; 2) LYs in the treatment group, 3) LYs in the control group.Results: We study TLV's preferred OS extrapolations and show that on average they overestimate the observed mean gain in LYs by 17%, and underestimate observed LYs by 5% and 1% in the treatment and control group, respectively.Conclusions: We conclude that it is feasible to validate OS extrapolations by comparing extrapolated and observed life-years. Even if survival extrapolations are reasonably accurate for the treatment and control group, respectively, this may still imply that extrapolations of LYs gained deviates to a larger extent. Follow-up studies on OS should be carried out to an increased extent to be able to validate, update and improve OS extrapolations in cost-effectiveness analysis of oncology drugs.
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  • Blomström Lundqvist, Carina, et al. (författare)
  • Impact of non-adherence to direct oral anticoagulants amongst Swedish patients with non-valvular atrial fibrillation: results from a real-world cost-utility analysis
  • 2022
  • Ingår i: Journal of Medical Economics. - : Taylor & Francis. - 1369-6998 .- 1941-837X. ; 25:1, s. 1085-1091
  • Tidskriftsartikel (refereegranskat)abstract
    • Aims: A third of non-valvular atrial fibrillation (NVAF) patients are non-adherent to direct oral anticoagulants (DOACs). Estimates of the economic value of full adherence and the cost of two types of adherence improving interventions are important to healthcare planners and decision-makers.Methods: A cost-utility analysis estimated the impact of non-adherence over a 20-year horizon, for a patient cohort with a mean age of 77 years, based on data from the Stockholm Healthcare database of NVAF patients with incident stroke between 2011 and 2018. Adherence was defined using a medication possession ratio (MPR) cut-off of 90%; primary outcomes were the number of ischemic strokes and associated incremental cost–utility ratio.Results: Hypothetical comparisons between cohorts of 1,000 patients with varying non-adherence levels and full adherence (MPR >90%) predicted an additional number of strokes ranging from 117 (MPR = 81–90%) to 866 (MPR <60%), and years of life lost ranging from 177 (MPR = 81– 90%) to 1,318 (MPR < 60%; discounted at 3%). Chronic disease co-management intervention occurring during each DOAC prescription renewal and patient education intervention at DOAC initiation will be cost-saving to the health system if its cost is below SEK 143 and SEK 4,655, and cost-effective if below SEK 858 and SEK 28,665, respectively.Conclusion: Adherence improving interventions for NVAF patients on DOACs such as chronic disease co-management and patient education can be cost-saving and cost-effective, within a range of costs that appear reasonable to the Swedish healthcare system.
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  • Bufe, Alexander, et al. (författare)
  • Costs of bleeds relating to acute coronary syndrome therapies.
  • 2010
  • Ingår i: Journal of Medical Economics. - : Informa Healthcare. - 1369-6998 .- 1941-837X. ; 13:2, s. 236-240
  • Tidskriftsartikel (refereegranskat)abstract
    • Objective: To estimate the costs to manage selected types of bleeding complications in patients with acute coronary syndrome (ACS) treated with antithrombotics, including antiplatelet, and fibrinolytic therapies: decrease in haemoglobin >3 g/dL, puncture site, and bleeding requiring transfusion of blood products.Methods: Retrospective chart reviews of ACS patients experiencing decrease in Hb, puncture site, or bleeding requiring transfusion were conducted in hospitals in France, Germany, Italy, Spain and Sweden to determine the total length of stay (LOS), stay post-bleeding, procedures to identify the extent of bleeding, number and types of transfusion products, patient demographics, reason for hospital admission and disposition upon discharge. Country-specific costs were applied to estimate the average cost per patient by type of bleeding event.Results: Records of 158 ACS patients with the bleeding complications were examined (26.9% decrease in Hb, 35.5% puncture site, and 37.8% transfusion). The average LOS was 10.6 days for decreasing Hb, 7.7 days for puncture site bleeding, and 11.3 days for patients receiving transfusions due to these bleeding events. The average costs per patient ranged between €3,986 and €10,252. The LOS ranged widely both within and across countries.Limitations: The study has a small sample size and costs are confounded by co-morbidities.Conclusions: The estimated costs for decreasing Hb, puncture site bleeding and bleeding resulting in transfusions provide a tool for researchers to conduct economic analyses of bleeding events associated with therapies for ACS. 
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  • Geale, Kirk, et al. (författare)
  • Budget impact analysis of demineralized bone matrix in combination with autograft in lumbar spinal fusion procedures for the treatment of lumbar degenerative disc disease in Spain
  • 2018
  • Ingår i: Journal of Medical Economics. - : Taylor & Francis. - 1369-6998 .- 1941-837X. ; 21:10, s. 977-982
  • Tidskriftsartikel (refereegranskat)abstract
    • Objective: To estimate the budget impact (BI) of introducing local autograft (LA) combined with demineralized bone matrix (LA + DBM) in lumbar spinal fusion (LSF) procedures to treat lumbar degenerative disc disease (LDDD) in Spain.Methods: A decision tree model was developed to evaluate the 4-year BI associated with introducing LA + DBM putty to replace currently available grafting methods, including iliac crest bone graft (ICBG), LA alone, and LA combined with beta-tricalcium phosphate (LA + ceramics), with 30%, 40%, and 30% market shares, respectively. The analysis was conducted for a hypothetical cohort of 100 patients with LDDD receiving LSF, assuming LA + DBM would replace 100% of the standard of care mix. The fusion rates extracted from the literature were validated by an expert panel. Costs ((sic)2017) were obtained from different Spanish sources. Budget impact and incremental cost per successful fusion were calculated from the perspective of the Spanish National Health System (NHS).Results: Over 4 years, replacing currently available options with LA + DBM for 100 patients resulted in an additional cost of (sic)12,330 ((sic)123/patient), and an additional 14 successful fusions, implying a cost of (sic)881 per additional successful fusion. When costs of productivity loss were included, the introduction of LA + DBM resulted in cost savings of (sic)70,294 ((sic)703/patient).Limitations: The lack of high-quality, homogeneous, head-to-head research studying the efficacy of grafting procedures available to patients undergoing LSF, in addition to a lack of long-term follow-up in existing studies. Therefore, the number of fusions occurring within the model's time horizon may be underestimated.Conclusions: Acquisition costs of DBM were partially offset by costs of failed fusions, adverse events and reoperation when switching 100 hypothetical LDDD patients undergoing LSF procedures from standard of care grafting methods to LA + DBM from the perspective of the Spanish NHS. DBM cost was entirely offset when costs of lost productivity were considered.
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  • Hofmarcher, Thomas, et al. (författare)
  • Cost-effectiveness analysis of ferric carboxymaltose in iron-deficient patients with chronic heart failure in Sweden.
  • 2015
  • Ingår i: Journal of Medical Economics. - : Informa Healthcare. - 1941-837X .- 1369-6998. ; 18:7, s. 492-501
  • Tidskriftsartikel (refereegranskat)abstract
    • OBJECTIVE: Iron deficiency is a common but treatable comorbidity in chronic heart failure (CHF) that is associated with impaired health-related quality-of-life (HRQoL). This study evaluates the cost-effectiveness of the intravenous iron preparation ferric carboxymaltose (FCM) for the treatment of iron deficiency in CHF from a Swedish healthcare perspective. METHODS: A cost-effectiveness analysis with a time horizon of 24 weeks was performed to compare FCM treatment with placebo. Data on health outcomes and medical resource use were mainly taken from the FAIR-HF trial and combined with Swedish cost data. An incremental cost-effectiveness ratio (ICER) was calculated as well as the change in per-patient costs for primary care and hospital care. RESULTS: In the FCM group compared with placebo, quality-adjusted life years (QALYs) are higher (difference = 0.037 QALYs), but so are per-patient costs [(difference = SEK 2789 (€303)]. Primary care and hospital care equally share the additional costs, but within hospitals there is a major shift of costs from inpatient care to outpatient care. The ICER is SEK 75,389 (€8194) per QALY. The robustness of the result is supported by sensitivity analyses. CONCLUSIONS: Treatment of iron deficiency in CHF with FCM compared with placebo is estimated to be cost-effective. The ICER in the base case scenario is twice as high as previously thought, but noticeably below SEK 500,000 (€54,300) per QALY, an informal average reference value used by the Swedish Dental and Pharmaceutical Benefits Agency. Increased HRQoL and fewer hospitalizations are the key drivers of this result.
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  • Jakovljevic, Mihajlo, et al. (författare)
  • Comparative financing analysis and political economy of noncommunicable diseases
  • 2019
  • Ingår i: Journal of Medical Economics. - : Informa UK Limited. - 1941-837X .- 1369-6998. ; 22:8, s. 722-727
  • Tidskriftsartikel (refereegranskat)abstract
    • The pandemic of chronic non-communicable diseases (NCDs) poses substantial challenges to the health financing sustainability in high-income and low/middle income countries (LMICs). The aim of this review is to identify the bottle neck inefficiencies in NCDs attributable spending and propose sustainable health financing solutions. The World Health Organization (WHO) introduced the "best buy" concept to scale up the core intervention package against NCDs targeted for LMICs. Population- and individual-based NCD best buy interventions are projected at US$170 billion over 2011-2025. Appropriately designed health financing arrangements can be powerful enablers to scale up the NCD best buys. Rapidly developing emerging nations dominate the landscape of LMICs. Their capability and willingness to invest resources for eradicating NCDs could strengthen WHO outreach efforts in Asia, Africa, and Latin America, much beyond current capacities. There has been a declining trend in international donor aid intended to cope with NCDs over the past decade. There is also a serious misalignment of these resources with the actual needs of recipient countries. Globally, the momentum towards the financing of intersectoral actions is growing, and this presents a cost-effective solution. A budget discrepancy of 10:1 in WHO and multilateral agencies remains in donor aid in favour of communicable diseases compared to NCDs. LMICs are likely to remain a bottleneck of NCDs imposed financing sustainability challenge in the long-run. Catastrophic household health expenditure from out of pocket spending on NCDs could plunge almost 150 million people into poverty worldwide. This epidemiological burden coupled with population ageing presents an exceptionally serious sustainability challenge, even among the richest countries which are members of the Organization for Economic Co-operation and Development (OECD). Strategic and political leadership of WHO and multilateral agencies would likely play essential roles in the struggle that has just begun.
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  • Jendle, Johan, 1963-, et al. (författare)
  • Real-world cost-effectiveness of insulin degludec in type 1 and type 2 diabetes mellitus from a Swedish 1-year and long-term perspective
  • 2020
  • Ingår i: Journal of Medical Economics. - : Informa Healthcare. - 1369-6998 .- 1941-837X. ; 23:11, s. 1311-1320
  • Tidskriftsartikel (refereegranskat)abstract
    • Background and Aims: The ReFLeCT study demonstrated that switching to insulin degludec from other basal insulins was associated with reductions in glycated hemoglobin and hypoglycemic events in type 1 (T1D) and type 2 diabetes (T2D), and reductions in insulin doses in T1D. The aim of the present analysis was to assess the short- and long-term cost-effectiveness of switching to insulin degludec in Sweden.Methods: Short-term outcomes were evaluated over 1 year in a Microsoft Excel model, while long-term outcomes were projected over patient lifetimes using the IQVIA CORE Diabetes Model. Cohort characteristics and treatment effects were sourced from the ReFLeCT study. Costs (in 2018 Swedish krona [SEK]) encompassed direct medical expenditure and indirect costs from loss of workplace productivity. In the long-term analyses, patients were assumed to receive insulin degludec or continue prior insulin therapy (primarily insulin glargine U100) for 5 years, before all patients intensified to once-daily degludec and mealtime aspart.Results: Switching to insulin degludec was associated with improved quality-adjusted life expectancy of 0.04 and 0.02 quality-adjusted life years (QALYs) over 1 year, and 0.16 and 0.08 QALYs over patient lifetimes, in T1D and T2D. Combined costs in T1D and T2D were estimated to be SEK 1 249 lower and SEK 1 181 higher over the short term, and SEK 157 258 and SEK 2 114 lower over the long term. Benefits were due to lower insulin doses in T1D, reduced rates of hypoglycemia and lower incidences of diabetes-related complications. Insulin degludec was associated with an incremental cost-effectiveness ratio of SEK 64 298 per QALY gained for T2D over 1 year and considered dominant for T1D and T2D in all other comparisons.Conclusions: Insulin degludec was projected to be cost-effective or dominant versus other basal insulins for the treatment of T1D and T2D in Sweden.
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  • Jendle, Johan, 1963-, et al. (författare)
  • Willingness-to-pay for benefits associated with basal insulin treatment of type 2 diabetes
  • 2012
  • Ingår i: Journal of Medical Economics. - Oxfordshire, United Kingdom : Taylor & Francis. - 1369-6998 .- 1941-837X. ; 15:2, s. 261-263
  • Tidskriftsartikel (refereegranskat)abstract
    • Data from a 20-week trial comparing insulin detemir and neutral protamine Hagedorn (NPH) insulin in insulin-naïve people with type 2 diabetes were analyzed using willingness-to-pay (WTP) data, a proxy for patient preference. The advantages of insulin detemir relative to NPH insulin with respect to a lower hypoglycemia rate and less weight gain were associated with a value of €27.87 per month.
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  • Jendle, Johan, 1963-, et al. (författare)
  • Willingness to pay for diabetes drug therapy in type 2 diabetes patients : based on LEAD clinical programme results
  • 2012
  • Ingår i: Journal of Medical Economics. - Oxfordshire, United Kingdom : Taylor & Francis. - 1369-6998 .- 1941-837X. ; 15:Suppl 2, s. 1-5
  • Tidskriftsartikel (refereegranskat)abstract
    • Objective: The purpose of this study was to investigate the preferences of people with diabetes for liraglutide vs other glucose lowering drugs, based on outcomes of clinical trials.Methods: Willingness to pay (WTP) for diabetes drug treatment was assessed by combining results from a recent WTP study with analysis of results from the Liraglutide Effect and Action in Diabetes (LEAD) programme. The LEAD programme included six randomised clinical trials with 3967 participants analysing efficacy and safety of liraglutide 1.2 mg (LEAD 1-6 trials), rosiglitazone (LEAD 1 trial), glimepiride (LEAD 2-3 trials), insulin glargine (LEAD 5 trial), and exenatide (LEAD 6 trial). The WTP survey used discrete choice experimental (DCE) methodology to evaluate the convenience and clinical effects of glucose lowering treatments.Results: People with type 2 diabetes were prepared to pay an extra €2.64/day for liraglutide compared with rosiglitazone, an extra €1.94/day compared with glimepiride, an extra €3.36/day compared with insulin glargine, and an extra €0.81/day compared with exenatide. Weight loss was the largest component of WTP for liraglutide compared with rosiglitazone, glimepiride, and insulin glargine. Differences in the administration of the two drugs was the largest component of WTP for liraglutide (once daily anytime) compared with exenatide (twice daily with meals). A limitation of the study was that it was based on six clinical trials where liraglutide was the test drug, but each trial had a different comparator, therefore the clinical effects of liraglutide were much better documented than the comparators.Conclusions: WTP analyses of the clinical results from the LEAD programme suggested that participants with type 2 diabetes were willing to pay appreciably more for liraglutide than other glucose lowering treatments. This was driven by the relative advantage of weight loss compared with rosiglitazone, glimepiride, and insulin glargine, and administration frequency compared with exenatide.
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  • Landfeldt, E., et al. (författare)
  • Assessment of face validity of a disease model of nonsense mutation Duchenne muscular dystrophy: a multi-national Delphi panel study
  • 2022
  • Ingår i: Journal of Medical Economics. - : Informa UK Limited. - 1369-6998 .- 1941-837X. ; 25:1, s. 808-816
  • Tidskriftsartikel (refereegranskat)abstract
    • Objective The objective of this study was to assess the face validity of a disease model evaluating the cost-effectiveness of ataluren for the treatment of nonsense mutation Duchenne muscular dystrophy (nmDMD). Methods This was a Delphi panel study comprising of physicians with first-hand experience of ataluren for the treatment of nmDMD. Consensus was sought for previously unvalidated model data, including patient health status and quality of life measured using the Health Utility Index (HUI), mortality, informal caregiving, and the expected benefit of early ataluren treatment across four states: (1) ambulatory, (2) non-ambulatory, not yet requiring ventilation support, (3) non-ambulatory, night-time ventilation support, and (4) non-ambulatory, full-time ventilation support. Results Nine experts from five countries participated in the Delphi panel. Consensus was obtained for all questions after three panel rounds (except for two HUI-questions concerning hand function [dexterity]). Consensus HUI-derived utilities for state (1) were 1.0000 for ataluren on top of best supportive care (BSC) and 0.7337 for BSC alone. Corresponding estimates for state (2) were 0.3179 and 0.2672, for state (3) 0.1643 and 0.0913, and for state (4) -0.0732 and -0.1163. Consensus mortality rates for states (1), (2), and (3) were 4%, 13%, and 33%, and life expectancy in state (4) was agreed to be 3 years. Panelists further agreed that two informal caregivers typically provide day-to-day care/support to patients with nmDMD, and that starting treatment with ataluren at 2 versus 5 years of age would be expected to delay loss of ambulation by an additional 2 years, and initiation of night-time and full-time ventilation support by an additional 3 years, respectively. Limitations The main limitation concerns the size of the Delphi panel, govern primarily by the rarity of the disease. Conclusion This study confirms the face validity of key clinical parameters and assumptions underlying the ataluren cost-effectiveness model.
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  • Lindblad, Anders, et al. (författare)
  • Clinical, economic, and societal burden of cystic fibrosis and the impact of the CFTR modulator, lumacaftor/ivacaftor : an assessment using linked registry data in Sweden
  • 2024
  • Ingår i: Journal of Medical Economics. - : Taylor & Francis. - 1369-6998 .- 1941-837X. ; 27:1, s. 897-906
  • Tidskriftsartikel (refereegranskat)abstract
    • AimsWe aimed to describe the clinical, economic, and societal burdens of cystic fibrosis (CF) and impact of CF transmembrane conductance regulator modulator (CFTRm) treatment on people with CF, caregivers, and healthcare systems.Material and methodsThis retrospective study used linked real-world data from Swedish national population-based registries and the Swedish CF Quality Registry to assess clinical, economic, and societal burden and CFTR impact in CF. Records from people with CF and a ten-fold control population without CF matched by sex, birth year, and location were compared during 2019. Outcomes for a subset aged >6 years initiating lumacaftor/ivacaftor (LUM/IVA) in 2018 were compared 12 months pre- and post-treatment initiation.ResultsPeople with CF (n = 743) had >10 times more inpatient and outpatient specialist visits annually vs controls (n = 7406). Those aged >18 had an additional 77·7 (95% CI: 70·3, 85·1) days of work absence, at a societal cost of €11,563 (95% CI: 10,463, 12,662), while caregivers of those aged <18 missed an additional 6.1 (5.0, 7.2) workdays. With LUM/IVA treatment, people with CF (n = 100) had significantly increased lung function (mean change in ppFEV1 [3·8 points; 95% CI: 1·1, 6·6]), on average 0·5 (95% CI: −0·8, −0·2) fewer pulmonary exacerbations and 45·2 (95% CI: 13·3, 77·2) fewer days of antibiotics. Days of work lost by caregivers of people with CF aged <18 decreased by 5·4 days (95% CI: 2·9, 7·9).ConclusionCF is associated with a high clinical economic and societal burden in Sweden. Improvements in clinical status observed in people with CF treated with LUM/IVA were reflected in reduced caregiver and societal burden.
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  • Liu, Jia, et al. (författare)
  • Cost-effectiveness of brentuximab vedotin for relapsed or refractory systemic anaplastic large-cell lymphoma in China
  • 2022
  • Ingår i: Journal of Medical Economics. - : Taylor & Francis. - 1369-6998 .- 1941-837X. ; 25:1, s. 99-107
  • Tidskriftsartikel (refereegranskat)abstract
    • Objectives: Systemic anaplastic large-cell lymphoma (sALCL) is a rare hematological malignancy with poor prognosis, which is associated with a significant economic burden. This study aimed to evaluate the cost-effectiveness of brentuximab vedotin (BV) in comparison to conventional chemotherapy in patients with relapsed/refractory sALCL, from a Chinese healthcare perspective.Methods: A partitioned survival model with three health states (progression-free survival, post-progression survival, and death) was adapted to compare BV against chemotherapy. Comparator represented a basket of commonly used chemotherapies in China. Two cohorts in each arm were estimated, representing patients receiving no transplant and autologous stem cell transplant (ASCT) after BV or chemotherapy. Clinical data was obtained from the pivotal phase-II trial (NCT00866047) for BV and also from the literature for a comparator. Resource use items covered drug acquisition and administration; concomitant medications; ASCT; treatment of adverse events; and long-term follow-up. Cost parameters were based on Chinese sources. Outcomes were measured in quality-adjusted life-years (QALYs). Both costs and effects were discounted at 5% according to Chinese guidelines. The impact of uncertainty was evaluated using deterministic and probabilistic sensitivity analyses.Results: The incremental cost-effectiveness ratio (ICER) for BV vs. chemotherapy was $9,610 (¥62,084) per QALY in the base case. The main model driver was superior progression-free and overall survival benefits of BV. The ICERs were relatively robust in the majority of sensitivity analyses, ranging around ±10% of the base case. Under the conventional decision thresholds (1–3 times of Chinese per capita GDP), the probability of BV being cost-effective ranged from 56 to 100%. Limitations of the study included the lack of comparative data from the trial and the small and heterogeneous sample due to its disease nature.Conclusions: BV may be a cost-effective treatment vs. chemotherapy in treating relapsed or refractory systemic anaplastic large-cell lymphoma in China.
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30.
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31.
  • Nuijten, M, et al. (författare)
  • Corrigendum
  • 2018
  • Ingår i: Journal of medical economics. - : Informa UK Limited. - 1941-837X .- 1369-6998. ; 21:9, s. 845-845
  • Tidskriftsartikel (refereegranskat)
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32.
  • Ohsfeldt, Robert L., et al. (författare)
  • Cost-effectiveness of rosuvastatin 20 mg for the prevention of cardiovascular morbidity and mortality : a Swedish economic evaluation of the Jupiter trial
  • 2012
  • Ingår i: Journal of Medical Economics. - : Informa Healthcare. - 1369-6998 .- 1941-837X. ; 15:1, s. 125-133
  • Tidskriftsartikel (refereegranskat)abstract
    • OBJECTIVE:This study estimated the long-term health outcomes, healthcare costs, and cost-effectiveness of rosuvastatin 20 mg therapy in primary prevention of major cardiovascular disease (CVD) in a Swedish population.METHODS:Based on data from the JUPITER trial, long-term CVD outcomes with rosuvastatin vs no active treatment were estimated for patients with an elevated baseline CVD risk (Framingham CVD score >20%, sub-population of JUPITER population) and for a population similar to the total JUPITER population. Using a decision-analytic model, trial CVD event rates were combined with epidemiological and cost data specific for Sweden. First and subsequent CVD events and death were estimated over a lifetime perspective. The observed relative risk reduction was extrapolated beyond the trial duration. Incremental effectiveness was measured as life-years gained (LYG) and quality-adjusted life-years (QALYs) gained.RESULTS:Treating 100,000 patients with rosuvastatin 20 mg was estimated to avoid 14,692 CVD events over the lifetime (8021 non-fatal MIs, 3228 non-fatal strokes, and 4924 CVD deaths) compared to placebo. This translated into an estimated gain of 42,122 QALYs and 36,865 total life years (LYG). Rosuvastatin was both more effective and less costly over a lifetime perspective, and rosuvastatin is subsequently a dominant alternative compared to no treatment in the assessed population. Using the overall JUPITER population, rosuvastatin was dominant for the lifetime horizon. In the sensitivity analysis, rosuvastatin was the dominant treatment strategy over a 20-year time horizon, and cost-effective with an incremental cost-effectiveness ratio (cost per QALY) of SEK 1783 over a 10-year time horizon.LIMITATIONS:Some model inputs were derived from literature or other data sources, but uncertainty was controlled by sensitivity analyses.CONCLUSIONS:Results indicate that rosuvastatin 20 mg treatment is a cost-effective option vs no-treatment in patients with Framingham CVD risk >20% in Sweden and might even be cost saving if taking a long-term perspective.
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33.
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34.
  • Olofsson, Sara, et al. (författare)
  • Preferences for improvements in attributes associated with basal insulin : a time trade-off and willingness-to-pay survey of a diabetic and non-diabetic population in Sweden
  • 2016
  • Ingår i: Journal of Medical Economics. - : Informa UK Limited. - 1369-6998 .- 1941-837X. ; 19:10, s. 945-958
  • Tidskriftsartikel (refereegranskat)abstract
    • Objective: Apart from improved health outcomes, treatment convenience per se may have a value to individuals. This is sometimes referred to as process utility and can be estimated in terms of willingness-to-pay (WTP) or quality-adjusted life-years (QALYs). Previous research has produced multiple studies on QALY gains and WTP estimates of insulin-related attributes. There are, however, significant variations between studies, and it is not clear to what extent the value is a reflection of the true preferences or a consequence of the methodological approach. The aim of this study is to estimate the preferences for treatment attributes associated with basal insulin (administration frequency, administration flexibility, and treatment-induced weight gain) using both QALYs—elicited using time trade-off (TTO) and WTP—among a sample of the Swedish general population and among a sample of the Swedish diabetes population. Methods: Data was collected using web-based surveys which were distributed to members of internet panels. The WTP survey presented five hypothetical scenarios with an offer to pay the incremental cost to receive basal insulin with improved attributes. The TTO survey presented six hypothetical scenarios where the respondent could choose between living for the rest of his/her life with diabetes and receiving treatment with a basal insulin with certain attributes or live for a shorter time with full health. The scenarios were combined with either a basal or a basal–bolus treatment regimen. Results from the TTO analysis were translated into monetary estimates using a threshold value of SEK500,000 per QALY. Results: In total, 2012 responses were included. The ratings of the attributes were almost identical, irrespective of method for the general population, while it differed to some extent for the diabetes population. The methods produced the same value for flexibility, but the estimates generated with the TTO approach were higher for one less injection and avoided weight gain. The general population assigned a higher utility gain to convenience attributes, while the diabetes population assigned a higher utility gain to avoiding weight gain. Limitations: About a quarter of the respondents did not accept the scenario in the WTP survey, i.e. protesters. Conclusions: The ranking of the attributes was generally independent of evaluation method, but the TTO method resulted in similar or higher values compared to the WTP method.
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35.
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36.
  • Ridderstråle, Martin, et al. (författare)
  • Cost-effectiveness of insulin detemir compared with NPH insulin in people with type 2 diabetes in Denmark, Finland, Norway, and Sweden.
  • 2013
  • Ingår i: Journal of Medical Economics. - : Informa Healthcare. - 1941-837X .- 1369-6998.
  • Tidskriftsartikel (refereegranskat)abstract
    • Abstract Objective: To assess the cost-effectiveness of insulin detemir compared with Neutral Protamine Hagedorn (NPH) insulin when initiating insulin treatment in people with type 2 diabetes mellitus (T2DM) in Denmark, Finland, Norway, and Sweden. Methods: Efficacy and safety data were derived from a 20-week multi-centre randomized controlled head-to-head clinical trial comparing insulin detemir and NPH insulin in insulin naïve people with T2DM, and short-term (1-year) cost effectiveness analyses were performed. As no significant differences in HbA(1c) were observed between the two treatment arms, the model was based on significant differences in favour of insulin detemir in frequency of hypoglycaemia (Rate-Ratio = 0.52; CI = 0.44-0.61) and weight gain (Δ = 0.9 kg). Model outcomes were measured in Quality Adjusted Life Years (QALYs) using published utility estimates. Acquisition costs for insulin and direct healthcare costs associated with non-severe hypoglycaemic events were obtained from National Health Service public sources. One-way and probabilistic sensitivity analyses were performed. Results: Based on lower incidence of non-severe hypoglycaemic events and less weight gain, the QALY gain from initiating treatment with insulin detemir compared with NPH insulin was 0.01 per patient per year. Incremental cost-effectiveness ratios for the individual countries were: Denmark, Danish Kroner 170,852 (€22,933); Finland, €28,349; Norway, Norwegian Kroner 169,789 (€21,768); and Sweden, Swedish Krona 226,622 (€25,097) per QALY gained. Possible limitations of the study are that data on hypoglycaemia and relative weight benefits from a clinical trial were combined with hypoglycaemia incidence data from observational studies. These populations may have slightly different patient characteristics. Conclusions: The lower risk of non-severe hypoglycaemia and less weight gain associated with using insulin detemir compared with NPH insulin when initiating insulin treatment in insulin naïve patients with type 2 diabetes provide economic benefits in the short-term. Based on cost/QALY threshold values, this represents good value for money in the Nordic countries. Using a short-term modelling approach may be conservative, as reduced frequency of hypoglycaemia and less weight gain may also have positive long-term health-related implications.
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37.
  • Sigurdardottir, Valgerdur, et al. (författare)
  • Cost-effectiveness analysis of bimekizumab for the treatment of active psoriatic arthritis in Sweden
  • 2023
  • Ingår i: Journal of Medical Economics. - : Taylor & Francis. - 1369-6998 .- 1941-837X. ; 26:1, s. 1190-1200
  • Tidskriftsartikel (refereegranskat)abstract
    • Aims: To evaluate the cost-effectiveness of bimekizumab, an inhibitor of IL-17F and IL-17A, against biologic and targeted synthetic disease-modifying antirheumatic drugs (DMARD) for psoriatic arthritis (PsA) from the Swedish healthcare system perspective.Materials and methods: A Markov model was developed to simulate the clinical pathway of biologic [b] DMARD-naive or tumor necrosis factor inhibitor experienced [TNFi-exp] PsA patients over a lifetime horizon. Treatment response was incorporated as achievement of the American College of Rheumatology 50% (ACR50) and Psoriasis Area and Severity Index 75% (PASI75) response, and changes in the Health Assessment Questionnaire-Disability Index (HAQ-DI) score. The efficacy of bimekizumab was obtained from the BE OPTIMAL (bDMARD-naive) and BE COMPLETE (TNFi-experienced) trials while a network meta-analysis (NMA) informed the efficacy of the comparators. Resource use and drug costs were obtained from published studies and databases of drug retail prices in Sweden. A willingness-to-pay threshold of euro50,000 per quality-adjusted life year (QALY) was applied.Results: In bDMARD-naive patients, bimekizumab achieved greater QALYs (14.08) than with all comparators except infliximab (14.22), dominated guselkumab every 4 and 8 weeks, ixekizumab, secukinumab 300 mg, ustekinumab 45 mg and 90 mg, and was cost-effective against risankizumab, tofacitinib, upadacitinib and TNFis, except adalimumab biosimilar. In TNFi-experienced patients, bimekizumab led to greater QALYs (13.56) than all comparators except certolizumab pegol (13.84), and dominated ixekizumab and secukinumab 300 mg while being cost-effective against all other IL-17A-, IL-23- and JAK inhibitors.Limitations: An NMA informed the comparative effectiveness estimates. Given gaps in evidence of disease management and indirect costs specific to HAQ-DI scores, and sequential clinical trial evidence in PsA, non-PsA cost data from similar joint conditions were used, and one line of active treatment followed by best supportive care was assumed.Conclusions: Bimekizumab was cost-effective against most available treatments for PsA in Sweden, irrespective of prior TNFi exposure
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38.
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39.
  • Steen Carlsson, Katarina, et al. (författare)
  • Cost-effectiveness of add-on treatments to metformin in a Swedish setting: liraglutide vs sulphonylurea or sitagplitin.
  • 2014
  • Ingår i: Journal of Medical Economics. - : Informa Healthcare. - 1941-837X .- 1369-6998. ; :Jun 26, s. 1-12
  • Tidskriftsartikel (refereegranskat)abstract
    • Abstract Objective: To evaluate long-run cost-effectiveness in a Swedish setting for liraglutide compared with sulphonylureas (glimepiride) or sitagliptin, all as add-on to metformin for patients with type 2 diabetes insufficiently controlled with metformin in monotherapy. Methods: The IHE Cohort Model of Type 2 Diabetes was used to evaluate clinical and economic outcomes from a societal perspective. Model input data were obtained from two clinical trials, the Swedish National Diabetes Register and the literature. Cost data reflected year 2013 price level. The robustness of results was checked with one-way-sensitivity analysis and probability sensitivity analysis. Results: The cost per QALY gained for liraglutide (1.2 mg) compared to SU (glimepiride 4 mg), both as add-on to metformin, ranged from SEK 226,000 to SEK 255,000 in analyzed patient cohorts. The cost per QALY for liraglutide (1.2 mg) vs sitagliptin (100 mg) as second-line treatment was lower, ranging from SEK 149,000 to SEK 161,000. Costs of preventive treatment were driving costs, but there was also a cost offset from reduced costs of complications of ∼20%. Notable cost differences were found for nephropathy, stroke, and heart failure. The predicted life expectancy with liraglutide increased the cost of net consumption for liraglutide. Limitations: The analysis was an ex-ante analysis using model input data from clinical trials which may not reflect effectiveness in real-world clinical practice in broader patient populations. This limitation was explored in the sensitivity analysis. The lack of specific data on loss of production due to diabetes complications implied that these costs may be under-estimated. Conclusions: Treatment strategies with liraglutide 1.2 mg improved the expected quality-of-life and increased costs when compared to SU and to sitagliptin for second-line add-on treatments. The cost per QALY for liraglutide was in the range considered medium by Swedish authorities.
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40.
  • Stålhammar, Jan, et al. (författare)
  • Resource utilization and cost of heart failure associated with reduced ejection fraction in Swedish patients
  • 2012
  • Ingår i: Journal of medical economics. - : Informa UK Limited. - 1941-837X .- 1369-6998. ; 15:5, s. 938-946
  • Tidskriftsartikel (refereegranskat)abstract
    • AIM:The purpose of this study was to assess healthcare utilization and costs for heart failure patients with reduced ejection fraction (HF-REF) in Sweden.METHODS AND RESULTS:This was a retrospective, population-based cohort study of patients diagnosed with HF-REF during a period of 18 months at 31 primary care centers in Uppsala County, Sweden. Data was obtained from computerized records from these centers, the Swedish Patient Registry, the Swedish Prescription Registry, the Cause of Death Registry, and a local echocardiography registry maintained by the Department of Physiology, Uppsala University Hospital. Main outcome measures were cardiovascular and heart-failure-related hospitalizations, outpatient visits, medication utilization, mortality (all-cause, cardiovascular, and heart-failure), and healthcare costs for HF-REF patients. During the index period, 252 heart failure patients had a left ventricular ejection fraction measurement ≤ 40% and were categorized as having HF-REF. More than half of the patients had ≥ 1 cardiovascular or heart failure-related hospitalization. On average, patients had >2 such hospitalizations annually. They also averaged ∼1 cardiovascular or heart-failure-related outpatient visit per year. All-cause mortality was high: 15.9% patients died within 1 year after the index date. The mean annual cost per patient for heart-failure-related hospitalizations was SEK 72,613 (EUR 7610). In contrast, annual prescription costs were low, on average 3% of total cost (SEK 3503, EUR 367 per patient)LIMITATIONS:The main limitations of this study include a short follow-up time and small sample size. Also, certain data were missing, such as echocardiograms (available for only 28% of patients), and information on patients' New York Heart Association (NYHA) functional class, validity period for prescriptions or the units of medication prescribed, and medication dosing. Furthermore, the overall mortality could have been under-estimated, as only the primary cause of death was included in the analysis.CONCLUSIONS:The main burden associated with HF-REF is related to hospitalizations for heart-failure events. Effective treatment options that decrease hospitalization rates could reduce patients' suffering and potentially offer considerable cost savings.
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41.
  • Stålhammar, Jan, et al. (författare)
  • The burden of preserved ejection fraction heart failure in a real-world Swedish patient population
  • 2014
  • Ingår i: Journal of Medical Economics. - : Informa Healthcare. - 1369-6998 .- 1941-837X. ; 17:1, s. 43-51
  • Tidskriftsartikel (refereegranskat)abstract
    • OBJECTIVES: To evaluate resource use and associated costs in patients with a diagnosis of heart failure with preserved ejection fraction (HF-PEF) in Sweden.METHODS: This retrospective study identified real-world patients with an ICD-10 diagnosis code for heart failure (I50) for the period between July 1, 2005 and December 31, 2006 from electronic medical records of primary care centers in Uppsala County Council, and in the Swedish patient registry data. Patients were categorized as having HF-PEF (left ventricle ejection fraction [LVEF] > 50%) during the index period. The study assessed medication utilization, outpatient visits, hospitalizations, and associated healthcare costs, as well as the incidence rates and time to all-cause and heart failure mortality following the index period.RESULTS: The study included 137 HF-PEF patients with a mean age of 77.1 (SD = 9.1) years. Over 50% of HF-PEF patients were female and hypertensive. Nearly all patients received ≥ 1 medication post-index. Patients had an average of 1.5 heart failure related hospitalizations per follow-up year. The average annual per patient cost for the management of a HF-PEF patient was found in Sweden to be Swedish Krona (SEK) 108,246 (EURO [EUR] 11,344). Hospitalizations contributed to more than 80% of the total cost. All-cause mortality over the 18-month study period was 25.5%, and more than 50% of these deaths occurred within 1 year of index.LIMITATIONS: Due to the limitations of registry data, it is not possible to confirm the HF diagnosis, and therefore the accuracy of registry records must be assumed. Other factors such as short follow-up time, the study-mandated LVEF assessment, and a lack of drug duration data may also have an impact on the study results.CONCLUSIONS: All-cause mortality was high in the HF-PEF population, with more than half of patients dying within 1 year of study follow-up. Study results also indicate that 60% of HF-PEF patients have ≥ 1 hospitalization during follow-up. Hospitalizations, especially heart failure related admissions, represent a substantial proportion of the total healthcare burden of patients with HF-PEF in Sweden.
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42.
  • Thangavelu, Tharshini, et al. (författare)
  • Micro-costing of genetic diagnostics in acute leukemia in Sweden : from standard-of-care to whole-genome sequencing
  • 2024
  • Ingår i: Journal of Medical Economics. - : Informa UK Limited. - 1369-6998 .- 1941-837X. ; 27:1, s. 1053-1060
  • Tidskriftsartikel (refereegranskat)abstract
    • Aims and backgroundWhole-genome sequencing (WGS) is increasingly applied in clinical practice and expected to replace standard-of-care (SoC) genetic diagnostics in hematological malignancies. This study aims to assess and compare the fully burdened cost ('micro-costing') per patient for Swedish laboratories using WGS and SoC, respectively, in pediatric and adult patients with acute lymphoblastic leukemia (ALL) and acute myeloid leukemia (AML).MethodsThe resource use and cost details associated with SoC, e.g. chromosome banding analysis, fluorescent in situ hybridization, and targeted sequencing analysis, were collected via activity-based costing methods from four diagnostic laboratories. For WGS, corresponding data was collected from two of the centers. A simulation-based scenario model was developed for analyzing the WGS cost based on different annual sample throughput to evaluate economy of scale.ResultsThe average SoC total cost per patient was 2,465 for pediatric AML and 2,201 for pediatric ALL, while in adults, the corresponding cost was 2,458 for AML and 1,207 for ALL. The average WGS cost (90x tumor/30x normal; sequenced on the Illumina NovaSeq 6000 platform) was estimated to 3,472 based on an annual throughput of 2,500 analyses, however, with an annual volume of 7,500 analyses the average cost would decrease by 23% to 2,671.ConclusionIn summary, WGS is currently more costly than SoC, however the cost can be reduced by utilizing laboratories with higher throughput and by the expected decline in cost of reagents. Our data provides guidance to decision-makers for the resource allocation needed when implementing WGS in diagnostics of hematological malignancies.
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43.
  • Walter, Evelyn, et al. (författare)
  • Cost-effectiveness of continuous subcutaneous apomorphine in the treatment of Parkinson's disease in the UK and Germany
  • 2015
  • Ingår i: Journal of Medical Economics. - : Informa Healthcare. - 1941-837X .- 1369-6998. ; 18:2, s. 155-165
  • Tidskriftsartikel (refereegranskat)abstract
    • Background: Parkinson's disease (PD) is the second most common neurodegenerative disease, affecting similar to 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 pound and generates 2.85 QALYs and 6.28 LYs ((sic)104,500.08, 2.92 QALYs and 6.49 LYs for Germany). Costs associated with LCIG are 130,011.34 pound, achieves 3.06 QALYs and 6.93 LYs ((sic)175,004.43, 3.18 QALYs and 7.18 LYs for Germany). The incremental-cost per QALY gained (ICER) was 244,684.69 pound ((sic)272,914.58). Costs for DBS are 87,730.22 pound, associated with 2.75 QALYs and 6.38 LYs ((sic)105,737.08, 2.85 QALYs and 6.61 LYs for Germany). CSAI dominates DBS. SOC associated UK costs are 76,793.49; pound 2.62 QALYs and 5.76 LYs were reached ((sic)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.
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