Sickle cell disease (SCD) is an inherited disorder of hemoglobin, associated with significant morbidity and mortality. Although the disease is most prevalent in Africa, it has been increasingly common in Western countries due to migration. The number of SCD patients in Sweden is unknown and the comprehensive national registries in Sweden provide a unique opportunity to address the objective to assess the burden of SCD. Using Sweden’s national patient registry, the 1-year prevalence of SCD with crisis (at least one registration of ICD-10 code D57.0 during the identification period, 2001-2018) was estimated during a 13-year follow-up period (2006-2018). Costs for hospital care of SCD (any ICD-10 D57) were estimated through hospital remuneration amounts based on diagnosis-related group. Productivity losses due to sick leave or disability for SCD, from the Swedish Social Insurance Agency, were assessed for working-age patients (18-65 years) and costed with Swedish mean salary, plus social security contributions. The 1-year prevalence of SCD with crisis increased during the follow-up period from 139 patients in year 2006 to 260 in 2018. A total of 2,427 inpatients stays were recorded with SCD (ICD-10 D57) as main reason for stay (observed in 2,632 SCD patient years) over the study period. In addition, 7,213 outpatient visits due to SCD were recorded. These stays and visits were estimated to cost 76.4 million (M) Swedish Krona (SEK) and 31.0 M SEK, respectively. Productivity losses due to sick leaves and disability pension amounted to 14.8 M SEK and 68.3 M SEK, respectively. This study demonstrates that SCD with crisis is associated with economic burden to society, health care and patients in Sweden and increasing prevalence and costs over the years of follow up. In total, for the years 2006-2018, the cost of hospital care and productivity losses for SCD amounted to 190.6 M SEK.

Short clinical trial follow-up periods at the time of submission and heterogeneity in outcomes often introduce uncertainty in the health technology assessment of immuno-oncologic (IO) therapies. To address this, some agencies prefer to use conservative methods for survival extrapolation. To evaluate the predictive accuracy of various survival extrapolation methods using data from CheckMate 067, a phase III trial of nivolumab plus ipilimumab (NIVO+IPI) vs either monotherapy in untreated metastatic melanoma. Six different extrapolation models were independently fitted to overall survival (OS) data for the three arms of CheckMate 067 using successive database locks (DBL) at: 28, 40, 52 and 60 months. NICE DSU TSD 14 informed model selection. 60-month follow-up from CheckMate 067, as well as external data for ipilimumab, were used to evaluate the predictive accuracy of the survival models. Extrapolated 20-year mean survival times (20-YR MST) were also compared with a 5% discount rate applied. For the 28-month DBL, standard parametric models, piecewise models and splines underestimated 60-month survival (range -3.8% to -11.7% across methods/arms). Mixture cure models (MCM), parametric mixture models (PMM) and response-based landmark models (RBLM) provided more accurate estimates (range -3.2% to +2.0% across methods/arms). MCM, PMM and RBLM produced closer estimates of 7-year and 10-year OS when compared to the external data for ipilimumab than other methods. 20-YR MSTs were higher for MCM, PMM and RBLM (NIVO+IPI: 6.02-7.02 years) than those for other models (NIVO+IPI: 4.56-5.62 years). Estimates of 20-YR MST for MCM, PMM and RBLM were more consistent across the successive database locks than with other methods. MCM, PMM and RBLM provided accurate and consistent estimates of OS across a range of follow-up periods. Extrapolation models that fail to account for heterogeneity in survival may result in biased assessments of the survival benefit of IO therapies for melanoma.

Background:AML affects all ages with an incidence rate of 5 per 100,000, but is much more frequent in older people. The overall lifetime risk of AML is estimated to be 0.5–1%. Long‐term overall survival in younger (age< 60 years) is about 50%, but much worse among older. Although AML therapy is one of the most resource‐intensive cancer treatments, there are few estimates of the resource use and economic burden.Aims:The aim of this study was to estimate the cost of treatment of AML in a population‐based setting, and to identify and obtain in‐depth information on treatment‐resource use and costs associated sick leave (SL) and early retirement (ER) in patients with AML.Methods:This study was a retrospective database study performed on Swedish national data. Adult patients (age>17 years) diagnosed with AML in Sweden between 2007 and 2015 were identified in the Swedish Cancer Registry, along with vital status. Data on resource use were collected from national registers for inpatient‐ and outpatient specialized care and prescribed drugs. Information on diagnostics and treatment was accessed from the Swedish national AML Registry (SwAMLR). Data on SL and ER came from the Swedish Social Insurance Agency (absent days costed with the mean salary in Sweden). Hospital care resource use was costed using diagnosis‐related group (DRG) remunerations, and include cost of inpatient drugs.Mean costs per patient were estimated per month for all cost categories and survival was analysed using Kaplan Meier survival estimations. Expected five‐years costs were then estimated by multiplying mean costs with survival estimates for each month, using the method described by Lin et al. to account for right censoring.Results:A total of 2,954 adult patients diagnosed with AML between 2007 and 2015 were identified. Approximately 48% of the patient population were female and median age at diagnosis was 71 years. Median survival was estimated at 8 months for the overall population. Expected 5‐year cost per patient was estimated to €80,669. Of this, €57,250 were from costs of hospitalizations, €8,936 from outpatient visits, €3,391 from prescribed drugs, and €11,092 from SL/ER.In the SwAMLR, 1,772 intensively treated patients were identified, with a median age of 64 years,. Median survival was estimated at 16.8 months. For this patient population, the expected cost per patient over 5 years was estimated to be € 115,830. Of this, €79,762 were from costs of hospitalizations, €12,965 from outpatient visits, €5,147 from prescribed drugs, and €17,956 from SL/ER.Similarly, patients treated with palliative intent (n = 994) were identified in the SwAMLR. Median age at diagnosis was 81 years. Median survival was estimated at 1.9 months. Expected 5‐year cost per patient was estimated to be €23,291. Of this, €15,783 was from costs of hospitalizations, €4,170 from outpatient visits, €645 from prescribed drugs, and €692 from SL/ER.Summary/Conclusion:Previous studies on the economic burden of AML have been limited to specific patient populations or limited to specific treatment events, such as induction therapy. In contrast, this study describes the AML‐associated costs in a nationwide population and includes direct costs as well as indirect costs of productivity losses.imageWe found that the society's cost of AML is high, despite the fact that there were few high‐cost drugs used during the study period. Also, in patients with intensive treatment, there are significant costs after the intensive treatment period, in part due to a substantial proretrportion still on sick leave during follow‐up.

To estimate the cost of treatment-resource use and costs associated with sick leave (SL) and early retirement (ER) in patients below the age of 60 years with AML undergoing allogenic stem cell transplantation (SCT). This study was a national population-based retrospective database study performed on Swedish data. Patients diagnosed with AML between 2007 and 2015 were identified in the Swedish Cancer Registry, along with vital status. Data on resource use were collected from registers for inpatient- and outpatient specialized care and prescribed drugs. Data on SL and ER came from the Swedish Social Insurance Agency (absent days costed with the mean salary in Sweden). Hospital care resource use was costed using diagnosis-related group (DRG) remunerations, and includes cost of inpatient drugs. The occurrence of SCT was identified based on procedure codes as recorded in the NPR. Mean costs per patient were estimated per month and survival was analysed using Kaplan Meier survival estimations. Expected five-years costs were estimated by multiplying mean costs with survival estimates for each month, using the method described by Lin et al. to account for right censoring. Maximum follow-up was five years. A total of 361 patients under 60 years diagnosed with AML undergoing SCT were identified. Patients could undergo SCT at any stage during the disease (within the 5-year follow-up). The 5-year survival rate was 56.5%. The mean time from diagnosis to SCT was 228 days, the median time to SCT was 154. Expected 5-year cost per patient was estimated to €240,587. Of this, €142,080 were from costs of hospitalizations, €28,156 from outpatient visits, €11,823 from prescribed drugs, and €58,528 from SL/ER. Cost of AML in patients diagnosed below 60 years and undergoing SCT is high. Initially, costs are mainly driven by inpatient-care, while longer-term costs are substantially affected by SL/ER.

Own health state utility values (HSUV, “experience-based utility”) is important for economic valuation and other purposes. There is only one preference-based EQ-5D value set derived from direct patient experience. Furthermore, the impact of interactions between EQ-5D dimensions on HSUV is poorly understood. Therefore we constructed a value set using direct patient experience and explored the impact of interactions between health states. The ICUROS was a multinational prospective observational study on the consequences of fragility fracture. In the study, patients who sustained a fragility fracture completed EQ-5D-3L and Time-trade-off (TTO) questionnaires for five time points: before fracture (recall), within two weeks after fracture, and at 4, 12 and 18 months after fracture. We derived a value set by fitting a random intercept linear model with TTO as the dependent variable and ‘moderate’ (Level 2, “L2”) or ‘severe’ impairment (Level 3, “L3”) in each EQ-5D dimension as dummy variables. We also explored the potential impact of interactions between impairment in dimensions using rigorous Lasso regression with cluster robust standard errors. In total 4,873 patients reported 17,779 EQ-5D and TTO pairs. Mean (SD) age at study enrolment was 71 (11) years and 81% were women. In the random intercept model, all coefficients had the expected sign and were statistically significant (p<0.001). The resulting value set was: 0.92 - 0.03xMobillityL2 - 0.08xMobillityL3 - 0.12xSelf-careL2 - 0.14xSelf-careL3 - 0.02xUsual ActivitiesL2 - 0.03xActivitiesL3 - 0.02xPain/DiscomfortL2 - 0.10xPain/DiscomfortL3 - 0.05xAnxiety/DepressionL2 ´- 0.11xAnxiety/DepressionL3. The Lasso regression indicated that interactions between mobility and self-care, anxiety/depression and self-care, and anxiety/depression and usual activities may be important. This experience-based EQ-5D-3L value set had consistently ordered and statistically significant coefficients, demonstrating face validity. Furthermore, the results show that explicit modelling of interactions may improve the predictive power of EQ-5D value sets. Compared to value sets based on hypothetical health, the decrements associated with impaired health were generally smaller.

This study estimated the cost-effectiveness of pharmacological fracture prevention as prescribed in the five largest European countries (EU5) using the IOF reference cost-effectiveness model. Pharmacological fracture prevention as prescribed in clinical practice was cost-saving (provided more QALYs at lower costs) compared to no treatment in each of the EU5. To estimate the real-world cost-effectiveness of pharmacological fracture prevention as prescribed in the five largest European countries by population size: France, Germany, Italy, Spain, and the United Kingdom (UK) (collectively EU5). We analyzed sales data on osteoporosis drugs in each of the EU5 to derive a hypothetical intervention that corresponds to the mix of osteoporosis medication prescribed in clinical practice. The costs for this treatment mix were obtained directly from the sales data, and the efficacy of the treatment mix was estimated by weighing the treatment-specific fracture risk reductions from a published meta-analysis. Subsequently, we estimated the cost-effectiveness using costs per quality adjusted life year (QALY) of the intervention compared to no treatment in each of the EU5 using the International Osteoporosis Foundation (IOF) reference cost-effectiveness model. The model population comprised postmenopausal women, mean age 72 years with established osteoporosis (T-score ≤ − 2.5) among whom 23.6% had a prevalent vertebral fracture. The model was populated with country-specific data from the literature. Pharmacological fracture prevention as prescribed in clinical practice was cost-saving (provided more QALYs at lower costs) compared to no treatment in each country. The findings were robust in scenario analyses. Pharmacological fracture prevention as prescribed in clinical practice is cost-saving in each of the EU5. Because of the under-diagnosis and under-treatment of post-menopausal osteoporosis, from a health economic perspective, further cost-savings may be reached by expanding treatment to those at increased risk of fracture currently not receiving any treatment.

This study used data from the International Costs and Utilities Related to Osteoporotic fractures Study (ICUROS) to estimate the quality of life (QoL) impact of fracture. Hip, vertebral, and distal forearm fractures incur substantial QoL losses. Hip and vertebral fracture results in markedly impaired QoL for at least 18 months.

The objective of this study was to systematically identify and assess cancer data sources that collect patient-level data in the five Nordic countries: Denmark, Finland, Iceland, Norway and Sweden. A comprehensive review of literature was conducted using the Embase and MEDLINE bibliographic databases. The searches were conducted in November 2016, limiting records to the past five years. Records were included only if a Nordic based cancer related data source was mentioned in the title or abstract. Administrative health registries were not considered in this study. Altogether, 4797 records were screened by title and abstract. In addition, targeted internet searches and a review of grey literature were conducted. In total, 1592 records were included and further reviewed. The majority, 87%, of included studies reported epidemiological and clinical main outcomes, whereas treatment patterns were reported in 4%, patient-reported outcomes in 4%, and resource use related outcomes in 2%. The remaining 3% of studies reported other outcomes, such as validity assessment. Altogether, 195 cancer data sources were identified out of which 84 were in Sweden, 55 in Denmark, 27 in Norway, 12 in Finland, 6 in Iceland, and 11 covering multiple Nordic countries. The types of data sources identified were national and specified cancer registries including quality registries in cancer (n=100), biobanks (n=38), prospective cohort studies (n=34), cancer screening registries (n=13), and other registries that collect patient-level data in cancer (n=10). A multitude of real world cancer data sources was identified in the Nordic countries. These results strongly emphasize the available opportunities of utilizing Nordic real world data in cancer, on the one hand, to meet payer requirements in post authorization treatment effectiveness, safety and resource use, and on the other hand, to pursue innovative research combining the rich data from biobanks, cancer registries and population-based administrative health databases.

To describe Swedish patients treated with long-acting injection (LAI) antipsychotics in terms of demographics and disease severity and to estimate measures of resource utilization and persistence. Patients with a diagnosis of schizophrenia (ICD 10 code F20.X) in the National Patient Register were identified through filled prescriptions for olanzapine (OLAI), paliperidone (PLAI), or risperidone (RLAI) from the Swedish Prescribed Drug Register between 2006 through 2013. We assessed the mean annual rate of hospitalizations (all-cause and for psychiatric reasons) 1 year prior and 1 year after initiation of the LAI antipsychotic treatment. Persistence was investigated using non-parametric survival analysis. During 2006-2013, 210 patients initiated OLAI, 437 PLAI, and 1,652 RLAI (mean age: 44, 47, and 45 years, respectively; 35%-41% female). Mean (SD) annual rate of hospitalizations 1 year before treatment initiation was 2.49 (2.53), 1.78 (2.47), and 1.78 (2.08) for all-cause and 1.81 (2.05), 1.25 (2.06), and 1.16 (1.56) for psychiatric reasons for OLAI, PLAI, and RLAI, respectively. Mean (SD) for all-cause and psychiatric hospitalizations 1 year after treatment initiation was 1.30 (2.45) and 0.99 (1.95), 0.98 (1.85) and 0.76 (1.43), and 1.02 (1.95) and 0.72 (1.49), respectively, corresponding to a decrease of 48% and 45% (OLAI), 45% and 39% (PLAI), and 43% and 38% (RLAI). The probability (survivor function [95% CI]) of being persistent 1 year after treatment initiation was 0.66 (0.58-0.73), 0.71 (0.66-0.76), and 0.46 (0.43-0.49) for OLAI, PLAI, and RLAI, respectively. Swedish patients with schizophrenia treated with a LAI antipsychotic have high hospital resource use, especially in the year prior to LAI initiation. LAI treatment was associated with a substantial reduction in hospitalizations across all groups. In the year following treatment initiation, the largest numerical reduction in hospitalizations was observed with OLAI. Persistence to OLAI treatment was not inferior to persistence with PLAI or RLAI.

This report describes the epidemiology, economic burden and treatment of osteoporosis in Switzerland.

This report describes epidemiology, burden, and treatment of osteoporosis in each of the 27 countries of the European Union (EU27).