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OBJECTIVES: Economic analysis in type 2 diabetes mellitus (T2DM) requires an assessment of the effect of a wide range of complications. The objective of this article was to identify a set of utility values consistent with the National Institute for Health and Care Excellence (NICE) reference case and to critically discuss and illustrate challenges in creating such a utility set. METHODS: A systematic literature review was conducted to identify studies reporting utility values for relevant complications. The methodology of each study was assessed for consistency with the NICE reference case. A suggested set of utility values applicable to modeling was derived, giving preference to studies reporting multiple complications and correcting for comorbidity. RESULTS: The review considered 21 relevant diabetes complications. A total of 16,574 articles were identified; after screening, 61 articles were assessed for methodological quality. Nineteen articles met NICE criteria, reporting utility values for 20 of 21 relevant complications. For renal transplant, because no articles meeting NICE criteria were identified, two articles using other methodologies were included. Index value estimates for T2DM without complication ranged from 0.711 to 0.940. Utility decrement associated with complications ranged from 0.014 (minor hypoglycemia) to 0.28 (amputation). Limitations associated with the selection of a utility value for use in economic modeling included variability in patient recruitment, heterogeneity in statistical analysis, large variability around some point estimates, and lack of recent data. CONCLUSIONS: A reference set of utility values for T2DM and its complications in line with NICE requirements was identified. This research illustrates the challenges associated with systematically selecting utility data for economic evaluations.
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Complicações do Diabetes/economia , Diabetes Mellitus Tipo 2/economia , Modelos Econômicos , Qualidade de Vida , Análise Custo-Benefício , Nível de Saúde , HumanosRESUMO
OBJECTIVE: The aim of this study was to estimate the cost-effectiveness of atazanavir/ritonavir (atazanavir/r) versus lopinavir/ritonavir (lopinavir/r) in treatment-naïve human immunodeficiency virus-1 (HIV-1) patients in Sweden for whom efavirenz is not suitable. METHODS: A Markov model was developed to predict the lifetime outcomes of atazanavir/r and lopinavir/r in terms of quality-adjusted life years (QALYs) and total costs. The model was structured to focus on treatment lines--how patients progress from first- to second-, and then to third-line treatment. Model inputs were derived directly from clinical trials, such as the CASTLE study (a 96-week head-to-head trial in first-line therapy), and from the Framingham risk-equation. The analysis was conducted from a payer perspective and included extensive scenario and probabilistic sensitivity analyses. RESULTS: The model predicted atazanavir/r to save 0.16 (95% confidence interval (CI) 0.00 to 0.33) QALYs and reduce total costs by -202,896 SEK (95% CI -332,156 to -81,644 SEK) over a lifetime horizon. Probabilistic sensitivity analyses showed that atazanavir/r had a 100% probability to be cost-effective at a willingness to pay of 200,000 SEK per QALY. CONCLUSION: The results indicate that atazanavir/r is cost-saving and more effective compared to lopinavir/r for patients who have previously not been exposed to antiretroviral drugs in Sweden.
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Fármacos Anti-HIV/administração & dosagem , Terapia Antirretroviral de Alta Atividade/métodos , Infecções por HIV/tratamento farmacológico , HIV-1/isolamento & purificação , Oligopeptídeos/administração & dosagem , Piridinas/administração & dosagem , Pirimidinonas/administração & dosagem , Ritonavir/administração & dosagem , Adulto , Fármacos Anti-HIV/economia , Terapia Antirretroviral de Alta Atividade/economia , Sulfato de Atazanavir , Ensaios Clínicos como Assunto , Análise Custo-Benefício , Infecções por HIV/virologia , Humanos , Lopinavir , Modelos Estatísticos , Oligopeptídeos/economia , Piridinas/economia , Pirimidinonas/economia , Anos de Vida Ajustados por Qualidade de Vida , Ritonavir/economia , SuéciaRESUMO
INTRODUCTION: Novel treatment options are needed to improve outcomes in transplant-ineligible relapsed/refractory (R/R) diffuse large B cell lymphoma (DLBCL). This systematic literature review evaluated clinical evidence on treatments for patients with R/R DLBCL ineligible for, or relapsed following, stem cell transplantation. METHODS: We assessed the feasibility of conducting an indirect treatment comparison (ITC) or network meta-analysis (NMA) to evaluate the relative efficacy and safety of polatuzumab vedotin in combination with bendamustine + rituximab versus other relevant treatments. RESULTS: Thirty-seven studies were identified, of which 20 were eligible [seven randomized, controlled trials (RCTs); 13 observational/single-arm trials]. Due to a lack of RCTs, an ITC/NMA summary of the relative efficacy and safety of the treatment options was not possible. Only two of the seven RCTs had positive outcomes. CONCLUSIONS: These findings highlight the paucity of published RCTs to establish the comparative efficacy of treatments for transplant-ineligible R/R DLBCL and lack of standard of care in this setting.
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Anticorpos Monoclonais/uso terapêutico , Protocolos de Quimioterapia Combinada Antineoplásica/uso terapêutico , Transplante de Células-Tronco Hematopoéticas/estatística & dados numéricos , Imunoconjugados/uso terapêutico , Linfoma Difuso de Grandes Células B/tratamento farmacológico , Humanos , Linfoma Difuso de Grandes Células B/etiologia , Rituximab/uso terapêutico , Resultado do TratamentoRESUMO
In this study, we assessed the productivity gains associated with the use of obinutuzumab in combination with chemoimmunotherapy (G-chemo) in first-line treatment among follicular lymphoma patients. Health benefits, measured as an increase in progression-free survival, were translated into productivity gains in both paid and unpaid work using gross value added as productivity measure. From 2017 to 2030, 11,870 overall progression-free years can be gained by utilizing obinutuzumab. These progression-free years correspond to undiscounted productivity gains of about 187.9 million in paid work and about 535.9 million in unpaid work. Our study shows that the benefits of the use of obinutuzumab in the first-line treatment of follicular lymphoma extend beyond clinical advantages.
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Anticorpos Monoclonais Humanizados/uso terapêutico , Antineoplásicos Imunológicos/uso terapêutico , Linfoma Folicular/tratamento farmacológico , Adulto , Protocolos de Quimioterapia Combinada Antineoplásica/uso terapêutico , Feminino , Alemanha/epidemiologia , Humanos , Linfoma Folicular/mortalidade , Linfoma Folicular/patologia , Masculino , Pessoa de Meia-Idade , Rituximab/administração & dosagem , Resultado do TratamentoRESUMO
Aim: To evaluate the cost-effectiveness of polatuzumab vedotin (pola) + bendamustine + rituximab (BR) in relapsed/refractory diffuse large B-cell lymphoma based on the GO29365 trial from a US payer's perspective. Materials & methods: A partitioned survival model used progression-free survival and overall survival data from the GO29365 trial. The base case analysis assumed overall survival was informed by progression-free survival; a mixture cure model estimated proportion of long-term survivors. Results: In the base case, pola + BR was cost-effective versus BR at US$35,864 per quality-adjusted life year gained. Probabilistic and one-way sensitivity analyses showed that the findings were robust. Conclusion: Pola + BR is cost-effective versus BR for the treatment of transplant-ineligible relapsed/refractory diffuse large B-cell lymphoma in the US.
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Anticorpos Monoclonais/economia , Antineoplásicos/economia , Cloridrato de Bendamustina/economia , Imunoconjugados/economia , Linfoma Difuso de Grandes Células B/tratamento farmacológico , Rituximab/economia , Adolescente , Adulto , Idoso , Anticorpos Monoclonais/uso terapêutico , Antineoplásicos/uso terapêutico , Análise Custo-Benefício , Humanos , Imunoconjugados/uso terapêutico , Linfoma Difuso de Grandes Células B/mortalidade , Pessoa de Meia-Idade , Taxa de Sobrevida , Resultado do TratamentoRESUMO
The GALLIUM trial compared obinutuzumab (GA101, G)-based chemotherapy followed by G monotherapy (G + chemo) for up to two years to rituximab (R)-based chemotherapy followed by R monotherapy (R + chemo) for up to two years in previously untreated follicular lymphoma (FL) patients. We estimated the cost-effectiveness of G + chemo versus R + chemo utilizing GALLIUM trial data and published literature. G + chemo had increased drug costs (undiscounted: $135,200 versus $127,700 for R + chemo), representing a relative increase of 5.9%. However, this was offset by a $6,400 lower cost for disease progression. G + chemo led to increased quality-adjusted life years (QALYs) relative to R + chemo of 0.81 (95% credible range, [CR]: 0.22-1.37), and the overall discounted incremental cost was $1,900 (95% CR: -$7,400 to $8,900). The incremental cost-effectiveness ratio was â¼$2,300 per QALY gained, and the results were highly robust to sensitivity analyses. Treatment with G + chemo compared to R + chemo is cost-effective in previously untreated FL patients in the US.
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Protocolos de Quimioterapia Combinada Antineoplásica/economia , Custos de Medicamentos/estatística & dados numéricos , Linfoma Folicular/tratamento farmacológico , Linfoma Folicular/economia , Adulto , Idoso , Idoso de 80 Anos ou mais , Anticorpos Monoclonais Humanizados/administração & dosagem , Protocolos de Quimioterapia Combinada Antineoplásica/uso terapêutico , Cloridrato de Bendamustina/administração & dosagem , Ciclofosfamida/administração & dosagem , Doxorrubicina/administração & dosagem , Feminino , Seguimentos , Humanos , Linfoma Folicular/patologia , Masculino , Pessoa de Meia-Idade , Prednisona/administração & dosagem , Prognóstico , Anos de Vida Ajustados por Qualidade de Vida , Rituximab/administração & dosagem , Taxa de Sobrevida , Estados Unidos , Vincristina/administração & dosagem , Adulto JovemRESUMO
BACKGROUND: Compliance among patients with schizophrenia is typically poor. Consequently, treatments that are equally efficacious under trial-based conditions but face different compliance rates in clinical practice (e.g. due to adverse-effect profile, ease of use, reputation) may have differences in effectiveness not observed during trials. This study analyses the impact of differences in compliance with atypical antipsychotics using a pharmacoeconomic discrete-event simulation (DES) model, adapted to the Swedish treatment setting. METHODS: An existing 5-year DES model was adapted to reflect a Swedish setting; the analysis was conducted from a third-party payer perspective, with only direct costs included. The two treatment arms were identical except for percentage of compliant patients. Non-compliant patients experienced shorter time between relapses and had inferior symptom control than their compliant counterparts. The difference in compliance rates was varied from 0% to 15%, and incremental costs and effects were recorded and analysed. RESULTS: With a 5%, 10% and 15% difference in compliance rate, incremental effects increased to 0.021, 0.037 and 0.062, respectively, while generating cost savings of Swedish kronor (SEK)31 500, SEK62 000 and SEK104 500, respectively (SEK9.25 = 1, Euro year 2007 values). Hence, each percentage point of compliance gain is predicted to roughly result in a cost saving of SEK6000 and a QALY gain of 0.004. On average, the model predicts that, with a 15% increase in compliance, 0.5 relapses are prevented, the average Positive And Negative Syndrome Scale (PANSS) score decreases by 3.3 points and patients spend 22 fewer days in hospital over 5 years. CONCLUSIONS: The DES model predicts that increases in compliance may lead to considerable cost savings and health improvements. Therefore, when determining the cost effectiveness of a new antipsychotic, efficacy rates from clinical trials should not be taken at face value, but should be interpreted in tandem with expectations concerning compliance, in light of product characteristics such as adverse effects. These results further suggest that efforts to improve compliance among patients with schizophrenia are expected to prove cost effective if compliance gains and the resulting health improvements and cost savings are in balance with the additional costs.
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Antipsicóticos , Adesão à Medicação/estatística & dados numéricos , Modelos Econométricos , Esquizofrenia/tratamento farmacológico , Antipsicóticos/economia , Antipsicóticos/uso terapêutico , Redução de Custos , Efeitos Psicossociais da Doença , Análise Custo-Benefício , Custos Diretos de Serviços/estatística & dados numéricos , Custos de Medicamentos/estatística & dados numéricos , Farmacoeconomia , Pesquisa sobre Serviços de Saúde , Hospitalização/economia , Humanos , Reembolso de Seguro de Saúde/economia , Valor Preditivo dos Testes , Anos de Vida Ajustados por Qualidade de Vida , Recidiva , Fatores de Risco , Comportamento de Redução do Risco , Esquizofrenia/economia , Esquizofrenia/prevenção & controle , Índice de Gravidade de Doença , Suécia , Fatores de TempoRESUMO
BACKGROUND: Conducting an adequately powered survival study in idiopathic pulmonary fibrosis (IPF) is challenging due to the rare nature of the disease and the need for extended follow-up. Consequently, registration trials of IPF treatments have not been designed to estimate long-term survival. OBJECTIVE: To predict life expectancy for patients with IPF receiving pirfenidone versus best supportive care (BSC) in a population that met the inclusion criteria of patients enrolled in the ASCEND and CAPACITY trials. METHODS: Kaplan-Meier survival data for pirfenidone and BSC were obtained from randomized controlled clinical studies (CAPACITY, ASCEND), an open-label extension study (RECAP), and the Inova Fairfax Hospital database. Data from the Inova registry were matched to the inclusion criteria of the CAPACITY and ASCEND trials. Life expectancy was estimated by the area under the curve of parametric survival distributions fit to the Kaplan-Meier data. RESULTS: Mean (95% confidence interval) life expectancy was calculated as 8.72 (7.65-10.15) years with pirfenidone and 6.24 (5.38-7.18) years with BSC. Therefore, pirfenidone improved life expectancy by 2.47 (1.26-4.17) years compared with BSC. In addition, treatment with pirfenidone recuperated 25% of the expected years of life lost due to IPF. Sensitivity analyses found that results were sensitive to the choice of parametric survival distribution, and alternative piecewise and parametric approaches. CONCLUSIONS: This analysis suggests that this population of patients with IPF has an improved life expectancy if treated with pirfenidone compared with BSC. DISCLOSURES: This study was funded by InterMune International AG, a wholly owned Roche subsidiary since 2014. Fisher was previously employed by InterMune UK, a wholly owned Roche subsidiary, until July 2015. He is currently employed by FIECON, which has received funding from F. Hoffmann-La Roche for consulting services. Nathan has received consulting fees from Roche-Genentech and Boehringer Ingelheim. He is also on the speakers' bureau for Roche-Genentech and Boehringer Ingelheim and has received research funding from both companies. Hill was previously employed by InterMune UK until October 2014. Hill and Marshall are employees of MAP BioPharma, which has received funding from F. Hoffmann-La Roche for consulting services. Dejonckheere and Thuresson are employees of F. Hoffmann-La Roche. Maher has received grants, consulting fees, and speaker fees from GlaxoSmithKline and UCB, and grants from Novartis. He has also received consulting fees and speaker fees from AstraZeneca, Bayer, Biogen Idec, Boehringer Ingelheim, Cipla, Lanthio, InterMune International AG, F. Hoffmann-La Roche, Sanofi-Aventis, and Takeda. Maher is supported by a National Institute for Health Research Clinician Scientist Fellowship (NIHR Ref: CS: -2013-13-017). Study concept and design were contributed by Fisher, Hill, Marshall, and Dejonckheere. Fisher, Nathan, and Thuresson collected the data, along with Hill and Marshall. Data interpretation was performed by Fisher, Maher, Nathan, and Dejonckheere. The manuscript was written primarily by Fisher, along with Maher and Dejonckheere, and revised by Fisher and Maher, along with the other authors.
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Anti-Inflamatórios não Esteroides/uso terapêutico , Fibrose Pulmonar Idiopática/tratamento farmacológico , Fibrose Pulmonar Idiopática/mortalidade , Internacionalidade , Expectativa de Vida/tendências , Piridonas/uso terapêutico , Adulto , Idoso , Idoso de 80 Anos ou mais , Feminino , Seguimentos , Humanos , Fibrose Pulmonar Idiopática/diagnóstico , Masculino , Pessoa de Meia-Idade , Valor Preditivo dos Testes , Taxa de Sobrevida/tendências , Resultado do TratamentoRESUMO
INTRODUCTION: For patients with late-stage (metastatic) breast cancer, the impact of treatment on health-related quality of life is a key factor in decision-making. A systematic review was conducted to identify health state utility values (HSUVs) for late-stage breast cancer, derived using methods preferred by health technology assessment (HTA) agencies, by treatment line. The aim was to generate a list of HSUVs, that could help to justify the values used to populate cost-utility models. Areas covered: Ten electronic databases, international congress websites and online HSUV databases were searched (January 1995-May 2014) for HSUVs for adults with late-stage breast cancer that had been derived from methods favoured by HTA agencies. Publications were included only if they reported studies that originated HSUVs. Expert commentary: Large numbers of HSUVs are available for late-stage breast cancer in the published literature. Contrary to expectations, the HSUVs reported in the literature vary greatly for some health states. As a result, the choice of HSUV can have considerable implications for the outcomes of economic evaluations. Standardization of HSUV methodology is expected to reduce variability; however, further research is recommended for assessing the sensitivity of generic preference-based measures in late-stage (metastatic) breast cancer.
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Neoplasias da Mama/patologia , Tomada de Decisões , Nível de Saúde , Adulto , Neoplasias da Mama/terapia , Análise Custo-Benefício , Feminino , Humanos , Modelos Econômicos , Metástase Neoplásica , Estadiamento de Neoplasias , Qualidade de Vida , Avaliação da Tecnologia BiomédicaRESUMO
BACKGROUND: Glucagon-like peptide-1 (GLP-1) receptor agonists are indicated for improvement of glycemic control in adults with type 2 diabetes. Cost is one aspect of treatment to be considered, in addition to clinical benefits, when selecting optimal therapy for a patient. The objective of this study was to estimate the average dose usage and real world daily cost of the GLP-1 receptor agonists, exenatide twice daily and liraglutide once daily, in Germany, the Netherlands, and the UK. METHODS: Administrative databases were used to source the data from longitudinal records of dispensed prescriptions. Data were extracted from the IMS Longitudinal Prescription database which captures details of prescriptions dispensed in pharmacies. Information on the dispensed quantity of each product was used to estimate average daily usage per patient. Daily dose usage was multiplied by the public price per unit to estimate daily cost. RESULTS: The dispensed volume in Germany corresponded to a mean dispensed daily dose of 16.81 µg for exenatide twice daily and 1.37 mg for liraglutide (mean daily cost 4.02 and 4.54, respectively). In the Netherlands, average dispensed daily doses of 17.07 µg and 1.49 mg were observed for exenatide twice daily and liraglutide (mean daily cost 3.05 and 3.97, respectively). In the UK, the mean dispensed volume corresponded to a daily usage of 20.49 µg for exenatide twice daily and 1.50 mg for liraglutide (mean daily cost £2.53 and £3.28, respectively). CONCLUSION: Estimates of average daily dispensed doses of GLP-1 receptor agonists derived from pharmacy data in real world settings corresponded to the dosing recommendation of the summaries of product characteristics. Nevertheless, the mean daily cost of exenatide twice daily was lower than that of liraglutide in Germany, the Netherlands, and the UK. Such estimates can be used to inform health care decision-makers on the real world usage and cost of medications effective in achieving glycemic control in patients with type 2 diabetes.
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OBJECTIVES: The main objectives were to estimate the cost-effectiveness and budget impact of indacaterol (a once-daily, long-acting-beta2-agonist) compared with 1) salmeterol/fluticasone, 2) formoterol/budesonide, and 3) tiotropium for the treatment of chronic obstructive pulmonary disease in Colombia. METHODS: A Markov model was utilized to simulate the progressive course of chronic obstructive pulmonary disease, distinguished by forced expiratory volume in 1 second predicted according to the four Global Initiative for Chronic Obstructive Lung Disease severity stages by using prebronchodilation values. Efficacy was based on the initial improvement in forced expiratory volume in 1 second, taken from either a network meta-analysis (salmeterol/fluticasone and formoterol/budesonide) or a randomized controlled trial (tiotropium). Colombian direct costs and life tables were incorporated in the adaptation, and analysis was performed from a health care payer perspective, discounting future costs (presented as US dollars) and benefits at 5%. A budget impact model was built to estimate the cost impact of indacaterol in Colombia over 3 and 5 years. RESULTS: Indacaterol was found to be dominant (i.e., less costly and more effective) against both salmeterol/fluticasone and formoterol/budesonide per life year and quality-adjusted life-year gained after a 5-year time horizon. The average cost saving against salmeterol/fluticasone and formoterol/budesonide was US $411 and US $909 per patient, respectively. All probabilistic sensitivity analysis simulations indicated indacaterol to be less costly than salmeterol/fluticasone and formoterol/budesonide. Indacaterol was more effective and more costly than tiotropium, corresponding to an incremental cost-utility ratio of US $2584 per quality-adjusted life-year. CONCLUSIONS: The results indicate that by replacing salmeterol/fluticasone or formoterol/budesonide with indacaterol, there are possible cost savings for the Colombian health care system. This was demonstrated by both cost-effectiveness and budget impact models.