RESUMO
BACKGROUND: The rate of events such as recurrent heart failure (HF) hospitalization and death are known to dramatically increase directly after HF hospitalization. Furthermore, the number of HF hospitalizations is associated with irreversible long-term disease progression, which is in turn associated with increased event rates. However, cost-effectiveness models of HF treatments commonly fail to capture both the short- and long-term association between HF hospitalization and events. OBJECTIVE: The aim of this study was to provide a decision-analytic model that reflects the short- and long-term association between HF hospitalization and event rates. Furthermore, we assess the impact of omitting these associations. METHODS: We developed a life-time Markov cohort model to evaluate HF treatments, and modeled the short-term impact of HF hospitalization on event rates via a sequence of tunnel states, with transition probabilities following a parametric survival curve. The corresponding long-term impact was modeled via hazard ratios per HF hospitalization. We obtained baseline event rates and utilities from published literature. Subsequently, we assessed, for a hypothetical HF treatment, how omitting the modeled associations (through a simple two-state model) affects incremental quality-adjusted life-years (QALYs). RESULTS: We developed a model that incorporates both short- and long-term impacts of HF hospitalizations. Based on an assumed treatment effect of a 20% risk reduction for HF hospitalization (and associated reductions in all-cause mortality of 15%), omitting the short-term, the long-term, or both associations resulted in a 5%, 1%, and 22% decrease in QALYs gained, respectively. CONCLUSION: For both modeling components, i.e., the short- and long-term implications of HF hospitalization, the impact on incremental outcomes associated with treatment was substantial. Considering these aspects as proposed within this modeling approach better reflects the natural course of this progressive condition and will enhance the evaluation of future HF treatments.
Assuntos
Insuficiência Cardíaca , Análise Custo-Benefício , Insuficiência Cardíaca/terapia , Hospitalização , Humanos , Modelos de Riscos Proporcionais , Anos de Vida Ajustados por Qualidade de VidaRESUMO
This study evaluated the cost-effectiveness of 1 year of romosozumab followed by alendronate versus oral bisphosphonates alone in women with postmenopausal osteoporosis at very high risk for fracture in Canada. Results showed that romosozumab sequenced to alendronate is a cost-effective treatment option, dominating both alendronate and risedronate alone. PURPOSE: To demonstrate the value of romosozumab sequenced to alendronate compared to alendronate or risedronate alone, for the treatment of osteoporosis in postmenopausal women with a history of osteoporotic fracture and who are at very high risk for future fracture in Canada. METHODS: A Markov model followed a hypothetical cohort of postmenopausal osteoporotic women at very high risk for future fractures, to estimate the cost-effectiveness of romosozumab and alendronate compared to oral bisphosphonates alone. A total treatment period of 5 years was assumed. Quality-adjusted life years and costs were estimated for each comparator across health states defined by different types of fragility fractures. RESULTS: Romosozumab/alendronate was associated with a lifetime gain of 0.103 and 0.127 QALYs and a cost reduction of $343 and $3805, relative to alendronate and risedronate, respectively. These results were driven by a reduction of the number of fractures (2561 per 1000 patients, versus 2700 for alendronate and 2724 for risedronate over lifetime). Romosozumab/alendronate had the highest probability of being cost-effective, relative to alendronate and risedronate, at any willingness to pay threshold value. CONCLUSION: Romosozumab/alendronate was associated with reduced costs and greater benefit relative to other comparators. Probabilistic, deterministic, and scenario analyses indicate that romosozumab/alendronate represents the best value for money; the uncertainty analyses are robust, and therefore romosozumab should be considered for reimbursement by public drug plans in Canada .
Assuntos
Conservadores da Densidade Óssea , Osteoporose Pós-Menopausa , Fraturas por Osteoporose , Alendronato/uso terapêutico , Anticorpos Monoclonais , Conservadores da Densidade Óssea/uso terapêutico , Análise Custo-Benefício , Feminino , Humanos , Osteoporose Pós-Menopausa/complicações , Fraturas por Osteoporose/tratamento farmacológico , Fraturas por Osteoporose/epidemiologia , Fraturas por Osteoporose/prevenção & controle , Pós-Menopausa , Anos de Vida Ajustados por Qualidade de Vida , Ácido Risedrônico/uso terapêuticoRESUMO
This study assessed the cost-effectiveness of continued denosumab treatment, compared with discontinuation of denosumab after one dose, for the treatment of postmenopausal osteoporosis in Taiwan, using real-world fracture reduction effectiveness and cost data. Outcomes indicate that continued denosumab treatment produces an incremental cost-effectiveness ratio of USD $16,743 per QALY. PURPOSE: To evaluate the cost-effectiveness of continued denosumab use versus discontinuation after one dose, for the treatment of postmenopausal osteoporosis in Taiwan, using real-world fracture reduction effectiveness and cost data. METHODS: A Markov cohort model was used to evaluate the lifetime costs and QALYs associated with continued denosumab treatment versus discontinuation of treatment after one dose. The evaluation was conducted from the perspective of Taiwan's healthcare system and used a discount rate of 3% per annum. The patient population consisted of postmenopausal women with osteoporosis with a mean age of 77 years who initiated denosumab treatment. Fracture reduction effectiveness data, baseline fracture rates, mortality data, and costs of fracture were informed by Taiwan's National Health Insurance Research Database. RESULTS: Model outcomes showed that continued treatment with denosumab produced an expected gain of 0.042 QALYs and an incremental cost of USD $704, compared with discontinuation of denosumab after one dose. This corresponds to an incremental cost-effectiveness ratio of USD $16,743 per QALY gained. Probabilistic and scenario analysis showed that results are stable to variations in model assumptions and parameters. CONCLUSION: In a real-world setting, at a cost per QALY threshold equivalent to gross domestic product per capita in 2020 in Taiwan (USD $30,038), continued treatment with denosumab in postmenopausal women with osteoporosis is cost-effective compared with treatment discontinuation.
Assuntos
Conservadores da Densidade Óssea , Osteoporose Pós-Menopausa , Idoso , Análise Custo-Benefício , Denosumab , Feminino , Humanos , Cadeias de Markov , Osteoporose Pós-Menopausa/tratamento farmacológico , Anos de Vida Ajustados por Qualidade de Vida , Taiwan/epidemiologiaRESUMO
BACKGROUND: The 'German Consortium for Hereditary Breast and Ovarian Cancer' (GC-HBOC) offers women with a family history of breast and ovarian cancer genetic counseling. The aim of this modeling study was to evaluate the cost-effectiveness of genetic testing for BRCA 1/2 in women with a high familial risk followed by different preventive interventions (intensified surveillance, risk-reducing bilateral mastectomy, risk-reducing bilateral salpingo-oophorectomy, or both mastectomy and salpingo-oophorectomy) compared to no genetic test. METHODS: A Markov model with a lifelong time horizon was developed for a cohort of 35-year-old women with a BRCA 1/2 mutation probability of ≥ 10%. The perspective of the German statutory health insurance (SHI) was adopted. The model included the health states 'well' (women with increased risk), 'breast cancer without metastases', 'breast cancer with metastases', 'ovarian cancer', 'death', and two post (non-metastatic) breast or ovarian cancer states. Outcomes were costs, quality of life years gained (QALYs) and life years gained (LYG). Important data used for the model were obtained from 4380 women enrolled in the GC-HBOC. RESULTS: Compared with the no test strategy, genetic testing with subsequent surgical and non-surgical treatment options provided to women with deleterious BRCA 1 or 2 mutations resulted in additional costs of 7256 and additional QALYs of 0,43 (incremental cost-effectiveness ratio of 17,027 per QALY; cost per LYG: 22,318). The results were robust in deterministic and probabilistic sensitivity analyses. CONCLUSION: The provision of genetic testing to high-risk women with a BRCA1 and two mutation probability of ≥ 10% based on the individual family cancer history appears to be a cost-effective option for the SHI.
Assuntos
Neoplasias da Mama/genética , Neoplasias da Mama/cirurgia , Testes Genéticos/economia , Programas de Rastreamento/economia , Mastectomia/economia , Modelos Econômicos , Neoplasias Ovarianas/genética , Neoplasias Ovarianas/cirurgia , Salpingo-Ooforectomia/economia , Adulto , Feminino , Alemanha , Humanos , Anos de Vida Ajustados por Qualidade de Vida , RiscoRESUMO
INTRODUCTION: Successful breast conserving cancer surgeries come along with tumor free resection margins and account for cosmetic outcome. Positive margins increase the likelihood of tumor recurrence. Intra-operative fluorescence molecular imaging (IFMI) aims to focus surgery on malignant tissue thus substantially lowering the presence of positive margins as compared with standard techniques of breast conservation (ST). A goal of this paper is to assess the incremental number of surgeries and costs of IFMI vs. ST. METHODS: We developed a decision analytical model and applied it for an early evaluation approach. Given uncertainty we considered that IFMI might reduce the proportion of positive margins found by ST from all to none and this proportion is assumed to be reduced to 10% for the base case. Inputs included data from the literature and a range of effect estimates. For the costs of IFMI, respective cost components were added to those of ST. RESULTS: The base case reduction lowered number of surgeries (mean [95% confidence interval]) by 0.22 [0.15; 0.30] and changed costs (mean [95% confidence interval]) by -663 [-1,584; 50]. A tornado diagram identified the Diagnosis Related Group (DRG) costs, the proportion of positive margins of ST, the staff time saving factor and the duration of frozen section analysis (FSA) as important determinants of this cost. CONCLUSIONS: These early results indicate that IFMI may be more effective than ST and through the reduction of positive margins it is possible to save follow-up surgeries-indicating further health risk-and to save costs through this margin reduction and the avoidance of FSA.
Assuntos
Neoplasias da Mama/diagnóstico por imagem , Neoplasias da Mama/cirurgia , Custos de Cuidados de Saúde/estatística & dados numéricos , Margens de Excisão , Mastectomia Segmentar , Imagem Molecular , Imagem Óptica , Cirurgia Assistida por Computador , Benzenossulfonatos/análise , Bevacizumab/análise , Neoplasias da Mama/economia , Neoplasias da Mama/epidemiologia , Ensaios Clínicos Fase I como Assunto/economia , Técnicas de Apoio para a Decisão , Feminino , Corantes Fluorescentes/análise , Secções Congeladas/economia , Alemanha/epidemiologia , Gastos em Saúde/estatística & dados numéricos , Humanos , Indóis/análise , Mastectomia Segmentar/economia , Modelos Teóricos , Imagem Molecular/economia , Duração da Cirurgia , Imagem Óptica/economia , Reoperação/economia , Reoperação/estatística & dados numéricos , Risco , Cirurgia Assistida por Computador/economia , Cirurgia Assistida por Computador/métodosRESUMO
INTRODUCTION: Etelcalcetide is a novel intravenous calcimimetic for the treatment of secondary hyperparathyroidism (SHPT) in haemodialysis patients. The clinical efficacy and safety of etelcalcetide (in addition to phosphate binders and vitamin D and/or analogues [PB/VD]) was evaluated in three phase III studies, including two placebo-controlled trials and a head-to-head study versus the oral calcimimetic cinacalcet. OBJECTIVE: The objective of this study was to develop a decision-analytic model for economic evaluation of etelcalcetide compared with cinacalcet. METHODS: We developed a life-time Markov model including potential treatment effects on mortality, cardiovascular events, fractures, and subjects' persistence. Long-term efficacy of etelcalcetide was extrapolated from the reduction in parathyroid hormone (PTH) in the phase III trials and the available data from the outcomes study in cinacalcet (EVOLVE trial). Etelcalcetide was compared with cinacalcet, both in addition to PB/VD. We applied unit costs averaged from five European countries and a range of potential etelcalcetide pricing options assuming parity price to weekly use of cinacalcet and varying it by a 15 or 30% increase. RESULTS: Compared with cinacalcet, the incremental cost-effectiveness ratio of etelcalcetide was 1,355 per QALY, 24,521 per QALY, and 47,687 per QALY for the three prices explored. The results were robust across the probabilistic and deterministic sensitivity analyses. CONCLUSIONS: Our modelling approach enabled cost-utility assessment of the novel therapy for SHPT based on the observed and extrapolated data. This model can be used for local adaptations in the context of reimbursement assessment.
Assuntos
Cinacalcete/economia , Análise Custo-Benefício/estatística & dados numéricos , Técnicas de Apoio para a Decisão , Hiperparatireoidismo Secundário/economia , Peptídeos/economia , Adolescente , Adulto , Idoso , Idoso de 80 Anos ou mais , Quelantes/economia , Quelantes/uso terapêutico , Cinacalcete/uso terapêutico , Quimioterapia Combinada/economia , Custos de Cuidados de Saúde/estatística & dados numéricos , Humanos , Hiperparatireoidismo Secundário/tratamento farmacológico , Cadeias de Markov , Pessoa de Meia-Idade , Modelos Econômicos , Peptídeos/uso terapêutico , Anos de Vida Ajustados por Qualidade de Vida , Vitamina D/análogos & derivados , Vitamina D/economia , Vitamina D/uso terapêutico , Adulto JovemRESUMO
BACKGROUND: Women with a BRCA1 or BRCA2 mutation are at increased risk of developing breast and/or ovarian cancer. This economic modeling study evaluated different preventive interventions for 30-year-old women with a confirmed BRCA (1 or 2) mutation. METHODS: A Markov model was developed to estimate the costs and benefits [i.e., quality-adjusted life years (QALYs), and life years gained (LYG)] associated with prophylactic bilateral mastectomy (BM), prophylactic bilateral salpingo-oophorectomy (BSO), BM plus BSO, BM plus BSO at age 40, and intensified surveillance. Relevant input data was obtained from a large German database including 5902 women with BRCA 1 or 2, and from the literature. The analysis was performed from the German Statutory Health Insurance (SHI) perspective. In order to assess the robustness of the results, deterministic and probabilistic sensitivity analyses were performed. RESULTS: With costs of 29,434 and a gain in QALYs of 17.7 (LYG 19.9), BM plus BSO at age 30 was less expensive and more effective than the other strategies, followed by BM plus BSO at age 40. Women who were offered the surveillance strategy had the highest costs at the lowest gain in QALYs/LYS. In the probabilistic sensitivity analysis, the probability of cost-saving was 57% for BM plus BSO. At a WTP of 10,000 per QALY, the probability of the intervention being cost-effective was 80%. CONCLUSIONS: From the SHI perspective, undergoing BM plus immediate BSO should be recommended to BRCA 1 or 2 mutation carriers due to its favorable comparative cost-effectiveness.
Assuntos
Neoplasias da Mama/genética , Genes BRCA1 , Genes BRCA2 , Neoplasias Ovarianas/genética , Anos de Vida Ajustados por Qualidade de Vida , Adulto , Neoplasias da Mama/prevenção & controle , Análise Custo-Benefício , Feminino , Humanos , Mutação , Neoplasias Ovarianas/prevenção & controleRESUMO
AIMS: This study explored the use of a value-based pricing approach for the new calcimimetic etelcalcetide indicated for the treatment of secondary hyperparathyroidism (SHPT) in patients receiving hemodialysis. It used the US payer perspective and applied the cost-effectiveness framework. Because etelcalcetide is an intravenous therapy that can be titrated for individual patients, and because its utilization is yet to be assessed in real world settings, a range of plausible doses were estimated for etelcalcetide to define a range of prices. These were either in relation to the existing oral calcimimetic cinacalcet or compared to no calcimimetic treatment. MATERIALS AND METHODS: The value-based price of etelcalcetide was determined via a Markov model. This model combined data from the etelcalcetide trials and previously published cost-effectiveness models in SHPT, and allowed extrapolation of treatment effects on mortality, cardiovascular events, fracture, and parathyroidectomy. Several dosing scenarios were explored covering the dose ranges of 30.0-64.18 mg per day for cinacalcet and 1.07-3.11 mg per day for etelcalcetide. These included the mean dose from the etelcalcetide trials, the preliminary defined daily dose, and the expected most common dose in real world. An acceptable price range for etelcalcetide was assessed by comparing the incremental cost-effectiveness ratios obtained with the willingness-to-pay threshold range of $100,000-$300,000/quality-adjusted life-years. RESULTS: Cost-effectiveness analysis supported value-based prices for etelcalcetide ranging from $21.15-$49.97/mg vs cinacalcet, and $13.79-$119.45/mg vs no calcimimetics. LIMITATIONS: There is uncertainty around what the real-world dosing will be for etelcalcetide. Another important nuance is that no studies have examined etelcalcetide effects on hard outcomes and, therefore, this modeling exercise relied on an extrapolation approach. CONCLUSIONS: This cost-effectiveness analysis, including scenarios to address uncertainties, allowed estimation of a value-based price range to aid reimbursement decisions in the US.
Assuntos
Calcimiméticos/uso terapêutico , Hiperparatireoidismo Secundário/tratamento farmacológico , Peptídeos/uso terapêutico , Calcimiméticos/economia , Análise Custo-Benefício , Relação Dose-Resposta a Droga , Feminino , Humanos , Falência Renal Crônica/complicações , Falência Renal Crônica/terapia , Masculino , Cadeias de Markov , Peptídeos/economia , Diálise RenalRESUMO
PURPOSE: The goal of this study was to assess and compare the potential clinical and economic value of emerging bone-forming agents using the only currently available agent, teriparatide, as a reference case in patients at high, near-term (imminent, 1- to 2-year) risk of osteoporotic fractures, extending to a lifetime horizon with sequenced antiresorptive agents for maintenance treatment. METHODS: Analyses were performed by using a Markov cohort model accounting for time-specific fracture protection effects of bone-forming agents followed by antiresorptive treatment with denosumab. The alternative bone-forming agent profiles were defined by using assumptions regarding the onset and total magnitude of protection against fractures with teriparatide. The model cohort comprised 70-year-old female patients with T scores below -2.5 and a previous vertebral fracture. Outcomes included clinical fractures, direct costs, and quality-adjusted life years. The simulated treatment strategies were compared by calculating their incremental "value" (net monetary benefit). FINDINGS: Improvements in the onset and magnitude of fracture protection (vs the teriparatide reference case) produced a net monetary benefit of $17,000,000 per 10,000 treated patients during the (1.5-year) bone-forming agent treatment period and $80,000,000 over a lifetime horizon that included 3.5 years of maintenance treatment with denosumab. IMPLICATIONS: Incorporating time-specific fracture effects in the Markov cohort model allowed for estimation of a range of cost savings, quality-adjusted life years gained, and clinical fractures avoided at different levels of fracture protection onset and magnitude. Results provide a first estimate of the potential "value" new bone-forming agents (romosozumab and abaloparatide) may confer relative to teriparatide.
Assuntos
Conservadores da Densidade Óssea/uso terapêutico , Osteoporose Pós-Menopausa/tratamento farmacológico , Fraturas por Osteoporose/prevenção & controle , Idoso , Anticorpos Monoclonais/economia , Anticorpos Monoclonais/uso terapêutico , Conservadores da Densidade Óssea/economia , Análise Custo-Benefício , Denosumab/economia , Denosumab/uso terapêutico , Feminino , Humanos , Modelos Teóricos , Osteoporose Pós-Menopausa/economia , Fraturas por Osteoporose/economia , Proteína Relacionada ao Hormônio Paratireóideo/economia , Proteína Relacionada ao Hormônio Paratireóideo/uso terapêutico , Pós-Menopausa , Anos de Vida Ajustados por Qualidade de Vida , Risco , Teriparatida/economia , Teriparatida/uso terapêuticoRESUMO
BACKGROUND: The evidence concerning the cost-effectiveness of UGT1A1*28 genotyping is ambiguous and does not allow drawing valid conclusions for Germany. This study evaluates the cost-effectiveness of UGT1A1 genotyping in patients with metastatic colorectal cancer undergoing irinotecan-based chemotherapy compared to no testing from the perspective of the German statutory health insurance. MATERIAL AND METHODS: A decision-analytic Markov model with a life time horizon was developed. No testing was compared to two genotype-dependent therapy strategies: 1) dose reduction by 25%; and 2) administration of a prophylactic G-CSF growth factor analog for homozygous and heterozygous patients. Probability, quality of life and cost parameters used in this study were based on published literature. Deterministic and probabilistic sensitivity analyses were performed to account for parameter uncertainties. RESULTS: Strategy 1 dominated all remaining strategies. Compared to no testing, it resulted in only marginal QALY increases (0.0002) but a cost reduction of 580 per patient. Strategy 2 resulted in the same health gains but increased costs by 10 773. In the probabilistic analysis, genotyping and dose reduction was the optimal strategy in approximately 100% of simulations at a threshold of 50 000 per QALY. Deterministic sensitivity analysis shows that uncertainty for this strategy originated primarily from costs for irinotecan-based chemotherapy, from the prevalence of neutropenia among heterozygous patients, and from whether dose reduction is applied to both homozygotes and heterozygotes or only to the former. CONCLUSION: This model-based synthesis of the most recent evidence suggests that pharmacogenetic UGT1A1 testing prior to irinotecan-based chemotherapy dominates non-personalized colon cancer care in Germany. However, as structural uncertainty remains high, these results require validation in clinical practice, e.g. based on a managed-entry agreement.
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Protocolos de Quimioterapia Combinada Antineoplásica/economia , Biomarcadores Tumorais/genética , Neoplasias Colorretais/economia , Glucuronosiltransferase/genética , Qualidade de Vida , Protocolos de Quimioterapia Combinada Antineoplásica/uso terapêutico , Camptotecina/administração & dosagem , Camptotecina/análogos & derivados , Neoplasias Colorretais/tratamento farmacológico , Neoplasias Colorretais/genética , Análise Custo-Benefício , Fluoruracila/administração & dosagem , Seguimentos , Genótipo , Alemanha , Heterozigoto , Homozigoto , Humanos , Seguro Saúde , Irinotecano , Leucovorina/administração & dosagem , Estadiamento de Neoplasias , Prognóstico , Anos de Vida Ajustados por Qualidade de Vida , Taxa de SobrevidaRESUMO
Despite the increasing availability of routine data, no analysis method has yet been presented for cost-of-illness (COI) studies based on massive data. We aim, first, to present such a method and, second, to assess the relevance of the associated gain in numerical efficiency. We propose a prevalence-based, top-down regression approach consisting of five steps: aggregating the data; fitting a generalized additive model (GAM); predicting costs via the fitted GAM; comparing predicted costs between prevalent and non-prevalent subjects; and quantifying the stochastic uncertainty via error propagation. To demonstrate the method, it was applied to aggregated data in the context of chronic lung disease to German sickness funds data (from 1999), covering over 7.3 million insured. To assess the gain in numerical efficiency, the computational time of the innovative approach has been compared with corresponding GAMs applied to simulated individual-level data. Furthermore, the probability of model failure was modeled via logistic regression. Applying the innovative method was reasonably fast (19 min). In contrast, regarding patient-level data, computational time increased disproportionately by sample size. Furthermore, using patient-level data was accompanied by a substantial risk of model failure (about 80 % for 6 million subjects). The gain in computational efficiency of the innovative COI method seems to be of practical relevance. Furthermore, it may yield more precise cost estimates.
Assuntos
Efeitos Psicossociais da Doença , Modelos Logísticos , Modelos Econômicos , Modelos Estatísticos , Coleta de Dados , Alemanha , Humanos , Doença Pulmonar Obstrutiva Crônica/economiaRESUMO
The aim of this study was to evaluate the inter-rater reliability of the Phillips-checklist, a proposed framework for the quality assessment of modeling studies. Six raters evaluated nine modeling studies from three different medical specialties. Intra-class correlation (ICC) and corresponding variance components were estimated from these studies. Raters were asked to comment on their experience with the framework. While overall the mean inter-rater reliability showed no significant rater-effect (ICC = 0.69, p = 0.064), there was - presumably as a result of a lower study variability - a significant rater effect for clopidogrel only (p < 0.001). The framework allowed a more structured methodological assessment but several items remained unclear. Regarding the quality assessment of modeling studies with the proposed framework, the rater variability is similar or even higher than variability because of studies or residual effects. Several scoring items can and should be improved to ease interpretation.
Assuntos
Lista de Checagem , Técnicas de Apoio para a Decisão , Atenção à Saúde/métodos , Modelos Teóricos , Atenção à Saúde/economia , Humanos , Variações Dependentes do Observador , Estudos Prospectivos , Reprodutibilidade dos TestesRESUMO
BACKGROUND: Lung cancer is among the top causes of cancer-related deaths. Epidermal growth factor receptor (EGFR)-tyrosine kinase inhibitors can increase progression-free survival compared with standard chemotherapy in patients with EGFR mutation-positive advanced non-small cell lung cancer (NSCLC). OBJECTIVE: The aim of the study was to evaluate the cost-effectiveness of EGFR mutation analysis and first-line therapy with erlotinib for mutation-positive patients compared with non-individualized standard chemotherapy from the perspective of German statutory health insurance. METHODS: A state transition model was developed for a time horizon of 10 years (reference year 2014). Data sources were published data from the European Tarceva versus Chemotherapy (EURTAC) randomized trial for drug efficacy and safety and German cost data. We additionally performed deterministic, probabilistic and structural sensitivity analyses. RESULTS: The individualized strategy incurred 0.013 additional quality-adjusted life-years (QALYs) and additional costs of 200, yielding an incremental cost-effectiveness ratio (ICER) of 15,577/QALY. Results were most sensitive to uncertainty in survival curves and changes in utility values. Cross-validating health utility estimates with recent German data increased the ICER to about 58,000/QALY. The probabilistic sensitivity analysis indicated that the individualized strategy is cost-effective, with a probability exceeding 50 % for a range of possible willingness-to-pay thresholds. LIMITATIONS: The uncertainty of the predicted survival curves is substantial, particularly for overall survival, which was not a primary endpoint in the EURTAC study. Also, there is limited data on quality of life in metastatic lung cancer patients. CONCLUSIONS: Individualized therapy based on EGFR mutation status has the potential to provide a cost-effective alternative to non-individualized care for patients with advanced adenocarcinoma. Further clinical research is needed to confirm these results.
Assuntos
Adenocarcinoma/economia , Análise Mutacional de DNA/economia , Receptores ErbB/genética , Cloridrato de Erlotinib/economia , Neoplasias Pulmonares/economia , Medicina de Precisão/economia , Inibidores de Proteínas Quinases/economia , Adenocarcinoma/tratamento farmacológico , Adenocarcinoma/genética , Adenocarcinoma/mortalidade , Adenocarcinoma de Pulmão , Análise Custo-Benefício , Receptores ErbB/antagonistas & inibidores , Cloridrato de Erlotinib/administração & dosagem , Cloridrato de Erlotinib/uso terapêutico , Alemanha , Humanos , Neoplasias Pulmonares/tratamento farmacológico , Neoplasias Pulmonares/genética , Neoplasias Pulmonares/mortalidade , Modelos Econômicos , Mutação , Invasividade Neoplásica , Inibidores de Proteínas Quinases/administração & dosagem , Inibidores de Proteínas Quinases/uso terapêutico , Ensaios Clínicos Controlados Aleatórios como AssuntoRESUMO
Coronary heart disease (CHD) is a major cause of death and important driver of health care costs. Recent German health care reforms have promoted integrated care contracts allowing statutory health insurance providers more room to organize health care provision. One provider offers KardioPro, an integrated primary care-based CHD prevention program. As insurance providers should be aware of the financial consequences when developing optional programs, this study aims to analyze the costs associated with KardioPro participation. 13,264 KardioPro participants were compared with a propensity score-matched control group. Post-enrollment health care costs were calculated based on routine data over a follow-up period of up to 4 years. For those people who incurred costs, KardioPro participation was significantly associated with increased physician costs (by 33%), reduced hospital costs (by 19%), and reduced pharmaceutical costs (by 16%). Overall costs were increased by 4%, but this was not significant. Total excess costs per observation year were 131 per person (95% confidence interval: [-36.5; 296]). Overall, KardioPro likely affected treatment as the program increased costs of physician services and reduced costs of hospital services. Further effects of substituting potential inpatient care with increased outpatient care might become fully apparent only over a longer time horizon.
Assuntos
Doenças Cardiovasculares/prevenção & controle , Prestação Integrada de Cuidados de Saúde/economia , Custos de Cuidados de Saúde/estatística & dados numéricos , Idoso , Doenças Cardiovasculares/economia , Análise Custo-Benefício , Feminino , Alemanha , Custos Hospitalares/estatística & dados numéricos , Humanos , Seguro Saúde , Masculino , Pessoa de Meia-Idade , Atenção Primária à Saúde/economia , Atenção Primária à Saúde/estatística & dados numéricos , Pontuação de PropensãoRESUMO
PURPOSE: Lynch syndrome (LS) screening among patients with newly diagnosed colorectal cancer can decrease mortality in their affected first-degree relatives. In Germany, it is not yet clinical practice and the cost-effectiveness of different testing strategies is unknown. METHODS: We developed a decision-analytic model to analyze the cost-effectiveness of LS screening from the perspective of the German Statutory Health Insurance system. A total of 22 testing strategies considering family-history assessment, analysis of tumor samples (i.e., immunohistochemistry (IHC), microsatellite instability, and BRAF mutation testing) and genetic sequencing were analyzed. Life-years gained in relatives by closed-meshed colonoscopy and aspirin prophylaxis were estimated by Markov models. Uncertainty was assessed deterministically and probabilistically. RESULTS: On average, detected mutation carriers gained 0.52 life-years (undiscounted: 1.34) by increased prevention. Most strategies were dominated, with three exceptions: family assessment by the Bethesda criteria followed by IHC and BRAF testing and genetic sequencing; IHC and BRAF testing and genetic sequencing; and direct sequencing of all index patients. Their incremental cost-effectiveness was [euro ]77,268, [euro ]253,258, and [euro ]4,188,036 per life-year gained, respectively. CONCLUSION: The results were less favorable than those of previous models. Chemoprevention appears to provide comparably low additional benefit and improves cost-effectiveness only slightly.
Assuntos
Neoplasias Colorretais Hereditárias sem Polipose/diagnóstico , Neoplasias Colorretais Hereditárias sem Polipose/genética , Análise Custo-Benefício , Detecção Precoce de Câncer/economia , Detecção Precoce de Câncer/métodos , Testes Genéticos/economia , Técnicas de Apoio para a Decisão , Atenção à Saúde , Alemanha , Heterozigoto , Humanos , Cadeias de Markov , Mutação , Aceitação pelo Paciente de Cuidados de Saúde , Medicina de Precisão/economia , Medicina de Precisão/métodosRESUMO
OBJECTIVE: The aim of this study was to assess the cost effectiveness of the novel fixed-dose anticoagulant rivaroxaban compared with the current standard of care, warfarin, for the prevention of stroke in patients with atrial fibrillation (AF). METHODS: A Markov model was constructed to model the costs and health outcomes of both treatments, potential adverse events, and resulting health states over 35 years. Analyses were based on a hypothetical cohort of 65-year-old patients with non-valvular AF at moderate to high risk of stroke. The main outcome measure was cost per quality-adjusted life-year (QALY) gained over the lifetime, and was assessed from the German Statutory Health Insurance (SHI) perspective. Costs and utility data were drawn from public data and the literature, while event probabilities were derived from both the literature and rivaroxaban's pivotal ROCKET AF trial. RESULTS: Stroke prophylaxis with rivaroxaban offers health improvements over warfarin treatment at additional cost. From the SHI perspective, at baseline the incremental cost-effectiveness ratio of rivaroxaban was
Assuntos
Anticoagulantes/uso terapêutico , Fibrilação Atrial/tratamento farmacológico , Rivaroxabana/uso terapêutico , Acidente Vascular Cerebral/prevenção & controle , Idoso , Anticoagulantes/administração & dosagem , Anticoagulantes/economia , Fibrilação Atrial/complicações , Fibrilação Atrial/economia , Análise Custo-Benefício , Inibidores do Fator Xa/administração & dosagem , Inibidores do Fator Xa/economia , Inibidores do Fator Xa/uso terapêutico , Alemanha , Hemorragia/economia , Hemorragia/epidemiologia , Humanos , Cadeias de Markov , Anos de Vida Ajustados por Qualidade de Vida , Fatores de Risco , Rivaroxabana/administração & dosagem , Rivaroxabana/economia , Acidente Vascular Cerebral/economia , Acidente Vascular Cerebral/etiologia , Varfarina/administração & dosagem , Varfarina/economia , Varfarina/uso terapêuticoRESUMO
BACKGROUND: Fractures are one of the most costly consequences of falls in elderly patients in nursing homes. OBJECTIVES: To compare the cost-effectiveness of a 'multifactorial fracture prevention program' provided by a multidisciplinary team with 'no prevention' in newly admitted nursing home residents. METHODS: We performed a cost-utility analysis using a Markov-based simulation model to establish the effectiveness of a multifaceted fall prevention program from the perspective of statutory health insurance (SHI) and long-term care insurance (LCI). The rate of falls was used to estimate the clinical and economic consequences resulting from hip and upper limb fractures. Robustness of the results was assessed using deterministic and probabilistic sensitivity analyses. RESULTS: Compared to no prevention a multifactorial prevention program for nursing home residents resulted in a cost-effectiveness ratio of 21,353 euro per quality-adjusted life-year. The total costs for SHI/LCI would result in 1.7 euro million per year. Results proved to be robust following deterministic and probabilistic sensitivity analyses. CONCLUSION: Multifactorial fracture prevention appears to be cost-effective in preventing fractures in nursing home residents. Since the results were based on the number of falls further research is required to confirm the results.
Assuntos
Fraturas Ósseas/economia , Fraturas Ósseas/prevenção & controle , Instituição de Longa Permanência para Idosos/organização & administração , Casas de Saúde/organização & administração , Educação de Pacientes como Assunto/economia , Educação de Pacientes como Assunto/organização & administração , Acidentes por Quedas/economia , Acidentes por Quedas/prevenção & controle , Idoso de 80 Anos ou mais , Simulação por Computador , Análise Custo-Benefício , Avaliação Geriátrica , Alemanha , Fraturas do Quadril , Instituição de Longa Permanência para Idosos/economia , Humanos , Cadeias de Markov , Modelos Econométricos , Casas de Saúde/economia , Anos de Vida Ajustados por Qualidade de Vida , Extremidade SuperiorRESUMO
The Institute for Quality and Efficiency in Health Care (IQWiG) developed-in a consultation process with an international expert panel-the efficiency frontier (EF) approach to satisfy a range of legal requirements for economic evaluation in Germany's statutory health insurance system. The EF approach is distinctly different from other health economic approaches. Here, we evaluate established tools for assessing and communicating parameter uncertainty in terms of their applicability to the EF approach. Among these are tools that perform the following: (i) graphically display overall uncertainty within the IQWiG EF (scatter plots, confidence bands, and contour plots) and (ii) communicate the uncertainty around the reimbursable price. We found that, within the EF approach, most established plots were not always easy to interpret. Hence, we propose the use of price reimbursement acceptability curves-a modification of the well-known cost-effectiveness acceptability curves. Furthermore, it emerges that the net monetary benefit allows an intuitive interpretation of parameter uncertainty within the EF approach. This research closes a gap for handling uncertainty in the economic evaluation approach of the IQWiG methods when using the EF. However, the precise consequences of uncertainty when determining prices are yet to be defined.
Assuntos
Pessoal Administrativo , Seguro Saúde/organização & administração , Comunicação , Análise Custo-Benefício , Economia Médica/estatística & dados numéricos , Alemanha , Custos de Cuidados de Saúde/estatística & dados numéricos , Política de Saúde/economia , Humanos , Seguro Saúde/economia , Seguro Saúde/legislação & jurisprudência , Seguro Saúde/estatística & dados numéricos , Modelos Teóricos , IncertezaRESUMO
BACKGROUND: Cardiovascular disease is the leading cause of morbidity and mortality in the developed world. To reduce this burden of disease, a German sickness fund ('Siemens-Betriebskrankenkasse', SBK) initiated the prevention programme 'KardioPro' including primary (risk factor reduction) and secondary (screening) prevention and guideline-based treatment. The aim of this study was to assess the effectiveness of 'KardioPro' as it is implemented in the real world. METHODS: The study is based on sickness fund routine data. The control group was selected from non-participants via propensity score matching. Study analysis was based on time-to-event analysis via Cox proportional hazards regression with the endpoint 'all-cause mortality, acute myocardial infarction (MI) and ischemic stroke (1)', 'all-cause mortality (2)' and 'non-fatal acute MI and ischemic stroke (3)'. RESULTS: A total of 26,202 insurants were included, 13,101 participants and 13,101 control subjects. 'KardioPro' enrollment was associated with risk reductions of 23.5% (95% confidence interval (CI) 13.0-32.7%) (1), 41.7% (95% CI 30.2-51.2%) (2) and 3.5% (hazard ratio 0.965, 95% CI 0.811-1.148) (3). This corresponds to an absolute risk reduction of 0.29% (1), 0.31% (2) and 0.03% (3) per year. CONCLUSION: The prevention programme initiated by a German statutory sickness fund appears to be effective with regard to all-cause mortality. The non-significant reduction in non-fatal events might result from a shift from fatal to non-fatal events.
Assuntos
Doenças Cardiovasculares/mortalidade , Doenças Cardiovasculares/prevenção & controle , Interpretação Estatística de Dados , Pessoal de Saúde/normas , Prevenção Primária , Avaliação de Programas e Projetos de Saúde , Estudos de Casos e Controles , Feminino , Humanos , Inibidores de Hidroximetilglutaril-CoA Redutases/uso terapêutico , Seguro Saúde , Masculino , Pessoa de Meia-Idade , Prognóstico , Pontuação de Propensão , Estudos Retrospectivos , Fatores de Risco , Taxa de Sobrevida , Fatores de TempoRESUMO
BACKGROUND: Cardiovascular diseases are the main cause of death worldwide, making their prevention a major health care challenge. In 2006, a German statutory health insurance company presented a novel individualised prevention programme (KardioPro), which focused on coronary heart disease (CHD) screening, risk factor assessment, early detection and secondary prevention. This study evaluates KardioPro in CHD risk subgroups, and analyses the cost-effectiveness of different individualised prevention strategies. METHODS: The CHD risk subgroups were assembled based on routine data from the statutory health insurance company, making use of a quasi-beta regression model for risk prediction. The control group was selected via propensity score matching based on logistic regression and an approximate nearest neighbour approach. The main outcome was cost-effectiveness. Effectiveness was measured as event-free time, and events were defined as myocardial infarction, stroke and death. Incremental cost-effectiveness ratios comparing participants with non-participants were calculated for each subgroup. To assess the uncertainty of results, a bootstrapping approach was applied. RESULTS: The cost-effectiveness of KardioPro in the group at high risk of CHD was 20,901 per event-free year; in the medium-risk group, 52,323 per event-free year; in the low-risk group, 186,074 per event-free year; and in the group with known CHD, 26,456 per event-free year. KardioPro was associated with a significant health gain but also a significant cost increase. However, statistical significance could not be shown for all subgroups. CONCLUSION: The cost-effectiveness of KardioPro differs substantially according to the group being targeted. Depending on the willingness-to-pay, it may be reasonable to only offer KardioPro to patients at high risk of further cardiovascular events. This high-risk group could be identified from routine statutory health insurance data. However, the long-term consequences of KardioPro still need to be evaluated.