RESUMO
Sorafenib is the only chemotherapeutic approved for treatment of advanced hepatocellular carcinoma (HCC). However, its effectiveness in patients with Child-Pugh class B cirrhosis and any moderating effects of health system characteristics are unclear. We examined the survival and cost-effectiveness associated with sorafenib in elderly patients with advanced HCC. We performed an analysis of Medicare beneficiaries with HCC diagnoses from 2007 to 2009. We compared advanced stage patients with HCC (American Joint Committee on Cancer stage III/IV) who received sorafenib within 6 months of diagnosis (and were otherwise untreated) to advanced stage patients with HCC who received no therapy (control). We performed univariate and multivariate analyses to identify predictors of survival. Incremental cost-effectiveness ratios (ICERs) were calculated for sorafenib-treated and control patients. We included 228 sorafenib-treated patients and 870 control patients. The median survival of the sorafenib-treated patients was 150.5 days versus 62 days for control patients. On multivariate analysis, significant predictors of improved survival were treatment with sorafenib (hazard ratio [HR], 0.66; 95% confidence interval [CI], 0.57-0.77), being seen at a National Cancer Institute-designated cancer center (HR, 0.77; 95% CI, 0.62-0.97), and being seen at a transplantation center (HR, 0.77; 95% CI, 0.65-0.93). Predictors of worse survival included stage IV disease (HR, 1.40; 95% CI, 1.24-1.58), decompensated cirrhosis (HR, 1.49; 95% CI, 1.30-1.70), and treatment in an urban setting (HR, 1.45; 95% CI, 1.21-1.73.) Although sorafenib use was associated with a survival benefit (HR, 0.61; 95% CI, 0.47-0.79) among patients with decompensated cirrhosis, the median survival benefit was 31 days, and it was not cost-effective (ICER, $224,914 per life year gained). CONCLUSION: Sorafenib is associated with improved survival in elderly patients with advanced HCC; however, it is not cost-effective among those with hepatic decompensation. (Hepatology 2017;65:122-133).
Assuntos
Antineoplásicos/economia , Antineoplásicos/uso terapêutico , Carcinoma Hepatocelular/tratamento farmacológico , Carcinoma Hepatocelular/mortalidade , Análise Custo-Benefício , Neoplasias Hepáticas/tratamento farmacológico , Neoplasias Hepáticas/mortalidade , Niacinamida/análogos & derivados , Compostos de Fenilureia/economia , Compostos de Fenilureia/uso terapêutico , Idoso , Carcinoma Hepatocelular/economia , Carcinoma Hepatocelular/patologia , Bases de Dados Factuais , Feminino , Humanos , Cirrose Hepática , Neoplasias Hepáticas/economia , Neoplasias Hepáticas/patologia , Masculino , Medicare , Estadiamento de Neoplasias , Niacinamida/economia , Niacinamida/uso terapêutico , Modelos de Riscos Proporcionais , Programa de SEER , Sorafenibe , Taxa de Sobrevida , Estados UnidosRESUMO
AIM: The aim was to evaluate cost-effectiveness of yttrium-90 transarterial radioembolization (TARE) in comparison to sorafenib treatment. PATIENTS & METHODS: A single-center, retrospective, observational study was performed, 166 patients with intermediate-/advanced-stage hepatocellular carcinoma were treated with sorafenib and 19 with TARE. The patients out of the sorafenib group matching the inclusion criteria for TARE, were reassigned to a subgroup SOR3. RESULTS: Mean costs for SOR3 patients amounted to 27,992 per patient, instead for TARE treatment, mean expense per patient was 17,761 (p = 0.028). Overall survival was similar between the two groups, while midterm survival rates (p = 0.012) were significantly higher with TARE treatment. CONCLUSION: TARE causes significantly lower treatment costs than sorafenib with better outcome in midterm survival.
Assuntos
Carcinoma Hepatocelular/tratamento farmacológico , Neoplasias Hepáticas/tratamento farmacológico , Niacinamida/análogos & derivados , Compostos de Fenilureia/administração & dosagem , Radioisótopos de Ítrio/administração & dosagem , Idoso , Carcinoma Hepatocelular/economia , Carcinoma Hepatocelular/patologia , Quimioembolização Terapêutica/economia , Quimioembolização Terapêutica/métodos , Análise Custo-Benefício/economia , Feminino , Humanos , Neoplasias Hepáticas/economia , Neoplasias Hepáticas/patologia , Masculino , Pessoa de Meia-Idade , Niacinamida/administração & dosagem , Niacinamida/economia , Compostos de Fenilureia/economia , Estudos Retrospectivos , Sorafenibe , Radioisótopos de Ítrio/economiaRESUMO
OBJECTIVES: To perform a cost-effectiveness analysis comparing the use of transarterial radioembolization (TARE) with that of sorafenib in the treatment of patients with intermediate or advanced hepatocellular carcinoma (HCC) according to the Barcelona Clinic Liver Cancer staging system. METHODS: Patient-level data were consecutively recorded and collected at three oncology centers in Italy. A propensity score matching was performed to compare patients with similar clinical characteristics who underwent TARE or sorafenib treatment. Clinical data from the matched cohorts were used to populate a Markov model to project, on a lifetime horizon, life years, quality-adjusted life years, and economic outcomes associated with TARE and sorafenib for both intermediate and advanced HCC stages. RESULTS: Starting from data covering 389 and 241 patients who underwent TARE and sorafenib treatment, respectively, the propensity score matching yielded a total of 308 matched patients. For intermediate-stage patients, the model estimated for TARE versus sorafenib an incremental cost-utility ratio of 3,302/QALY (incremental cost-effectiveness ratio of 1,865 per life year gained), whereas for patients in advanced stage TARE dominated (lower costs and greater health improvements) compared with sorafenib. CONCLUSIONS: From an Italian health care service perspective, TARE could be a cost-effective strategy in comparison with sorafenib for patients with intermediate or advanced HCC. The results from forthcoming randomized controlled trials comparing TARE with sorafenib will be able to confirm or reject the validity of this preliminary evaluation. In the meantime, decision makers can use these results to control and coordinate the diffusion of the technology.
Assuntos
Antineoplásicos/economia , Antineoplásicos/uso terapêutico , Carcinoma Hepatocelular/terapia , Embolização Terapêutica/economia , Neoplasias Hepáticas/terapia , Niacinamida/análogos & derivados , Compostos de Fenilureia/economia , Compostos de Fenilureia/uso terapêutico , Idoso , Institutos de Câncer , Carcinoma Hepatocelular/patologia , Estudos de Coortes , Análise Custo-Benefício , Embolização Terapêutica/métodos , Feminino , Humanos , Itália , Estimativa de Kaplan-Meier , Neoplasias Hepáticas/patologia , Masculino , Cadeias de Markov , Pessoa de Meia-Idade , Estadiamento de Neoplasias , Niacinamida/economia , Niacinamida/uso terapêutico , Anos de Vida Ajustados por Qualidade de Vida , Sorafenibe , SobrevidaRESUMO
BACKGROUND AND AIM: Antiviral therapy has been demonstrated to significantly improve the survival in patients with advanced hepatitis B virus (HBV)-related hepatocellular carcinoma (HCC). The aim of the study was to investigate the cost-effectiveness of antiviral therapy in patients with advanced HBV-related HCC treated with sorafenib. METHODS: To conduct the analysis, a Markov model comprising three health states (progression-free survival, progressive disease, and death) was created. The efficacy data were derived from medical records. Cost data were collected based on the Chinese national drug prices. Utility data came from the previously published studies. One-way sensitivity analyses as well as probabilistic sensitivity analyses were performed to explore model uncertainties. RESULTS: In the base-case analysis, addition of antiviral therapy to sorafenib generated an effectiveness of 0.68 quality-adjusted life years (QALYs) at a cost of $25 026.04, while sorafenib monotherapy gained an effectiveness of 0.42 QALYs at a cost of $20 249.64. The incremental cost-effectiveness ratio (ICER) was $18 370.77/QALY for antiviral therapy group versus non-antiviral therapy group. On the other hand, the ICER between the two groups in patients with high or low HBV-DNA load, with or without cirrhosis, normal or elevated alanine aminotransferase/aspartate aminotransferase were $16 613.97/QALY, $19 774.16/QALY, $14 587.66/QALY, $19 873.84/QALY, $17 947.07/QALY, and $18 785.58/QALY, respectively. CONCLUSIONS: Based on the cost-effectiveness threshold ($20 301.00/QALY in China), addition of antiviral therapy to sorafenib is considered to be a cost-effective option compared with sorafenib monotherapy in patients with advanced HBV-related HCC in China from the patient's perspective.
Assuntos
Antineoplásicos/economia , Antineoplásicos/uso terapêutico , Antivirais/economia , Antivirais/uso terapêutico , Carcinoma Hepatocelular/tratamento farmacológico , Carcinoma Hepatocelular/economia , Custos de Medicamentos , Hepatite B/tratamento farmacológico , Hepatite B/economia , Neoplasias Hepáticas/tratamento farmacológico , Neoplasias Hepáticas/economia , Niacinamida/análogos & derivados , Compostos de Fenilureia/economia , Compostos de Fenilureia/uso terapêutico , Adulto , Idoso , Carcinoma Hepatocelular/mortalidade , Carcinoma Hepatocelular/virologia , China , Análise Custo-Benefício , Progressão da Doença , Intervalo Livre de Doença , Feminino , Hepatite B/complicações , Hepatite B/mortalidade , Humanos , Estimativa de Kaplan-Meier , Neoplasias Hepáticas/mortalidade , Neoplasias Hepáticas/virologia , Masculino , Cadeias de Markov , Pessoa de Meia-Idade , Modelos Econômicos , Niacinamida/economia , Niacinamida/uso terapêutico , Qualidade de Vida , Anos de Vida Ajustados por Qualidade de Vida , Estudos Retrospectivos , Sorafenibe , Fatores de Tempo , Resultado do TratamentoRESUMO
UNLABELLED: The purpose was to assess the cost-effectiveness of sorafenib in the treatment of hepatocellular carcinoma (HCC) patients incorporating current prices and the results of the recent published field practice SOraFenib Italian Assessment (SOFIA) study. We created a Markov Decision Model to evaluate, in a hypothetical cohort of Caucasian male patients, aged 67 years with Barcelona Clinic Liver Cancer (BCLC) C HCC, or BCLC B HCC who were unfit or failed to respond to locoregional therapies, well compensated cirrhosis, and with performance status 0-1 according to Eastern Cooperative Oncology Group (ECOG), the cost-effectiveness of the following strategies: (1) full or dose-adjusted sorafenib for BCLC B and C patients together; (2) full or dose-adjusted sorafenib for BCLC B patients; (3) full or dose-adjusted sorafenib for BCLC C patients. Outcomes include quality-adjusted life years (QALYs), costs, and incremental cost-effectiveness ratio (ICER). In the base-case analysis dose-adjusted sorafenib was the most effective of the evaluated strategies. For dose-adjusted sorafenib, QALY was 0.44 for BCLC B and C patients together, 0.44 for BCLC C patients, and 0.38 for BCLC B patients. The ICER of dose-adjusted sorafenib compared with BSC was 34,534 per QALY gained for BCLC B and C patients together, 27,916 per QALY gained for BCLC C patients, and 54,881 per QALY gained for BCLC B patients. Results were sensitive to BSC survival rate, and sorafenib treatment duration. CONCLUSION: In daily practice dose-adjusted, but not full-dose, sorafenib is a cost-effective treatment compared to BSC in intermediate and advanced HCC.
Assuntos
Carcinoma Hepatocelular/tratamento farmacológico , Carcinoma Hepatocelular/economia , Neoplasias Hepáticas/tratamento farmacológico , Neoplasias Hepáticas/economia , Niacinamida/análogos & derivados , Compostos de Fenilureia/economia , Compostos de Fenilureia/uso terapêutico , Idoso , Antineoplásicos/economia , Antineoplásicos/uso terapêutico , Carcinoma Hepatocelular/mortalidade , Análise Custo-Benefício , Custos de Medicamentos , Humanos , Estimativa de Kaplan-Meier , Neoplasias Hepáticas/mortalidade , Masculino , Cadeias de Markov , Análise Multivariada , Niacinamida/economia , Niacinamida/uso terapêutico , Estudos Prospectivos , Anos de Vida Ajustados por Qualidade de Vida , SorafenibeRESUMO
BACKGROUND: The prognosis for hepatocellular carcinoma (HCC) is dependent upon tumour stage, performance status (PS), severity of underlying liver disease, and the availability of appropriate therapies. The unavailability of sorafenib may have a significantly adverse effect on the prognosis of UK patients with advanced HCC. During the study period, access to sorafenib was at the discretion of local health funding bodies, a process that may delay or deny access to the drug and that remains in place for Wales, Scotland, and Northern Ireland. Here, we attempt to address the impact of this system on patients with advanced HCC in the United Kingdom. METHODS: This is a retrospective study performed in the two largest specialist hepatobiliary oncology units in the United Kingdom. Funding applications were made to local funding bodies for patients with advanced HCC for whom sorafenib was considered appropriate (advanced HCC not suitable for loco-regional therapies, compensated chronic liver disease, PS 0-2). RESULTS: A total of 133 applications were made, of which 57 (43%) were approved and 76 (57%) declined. Demographics and prognostic factors were balanced between the two groups. This cohort had a number of adverse prognostic features: patients were predominantly PS 1-2; the majority had multifocal disease with the largest lesion being >5 cm; and macroscopic vascular invasion, metastases, and AFP >,000 ng ml(-1), were each present in one-third of cases. The median time from application to funding decision was 17 days (range 3-260 days). For the primary 'intention-to-treat' analysis, median overall survival was 4.1 months when funding was declined, and 9.5 months when funding was approved (hazard ratio (HR) 0.48; 95% CI 0.3186-0.7267; P=0.0005). CONCLUSION: These data support the use of sorafenib for patients with advanced HCC as an effective intervention. In the United Kingdom, this applies to a relatively small group of patients, estimated to total â¼800 per year who, unfortunately, do not survive long enough to themselves lobby for the availability of this drug. These data provide a comparison of sorafenib with supportive care and demonstrate the potential detrimental impact on patient outcomes of rationing health-care resources on the basis of cost.
Assuntos
Antineoplásicos/provisão & distribuição , Carcinoma Hepatocelular/tratamento farmacológico , Alocação de Recursos para a Atenção à Saúde/economia , Neoplasias Hepáticas/tratamento farmacológico , Neoplasias Primárias Múltiplas/tratamento farmacológico , Niacinamida/análogos & derivados , Compostos de Fenilureia/provisão & distribuição , Adolescente , Adulto , Idoso , Idoso de 80 Anos ou mais , Antineoplásicos/economia , Antineoplásicos/uso terapêutico , Carcinoma Hepatocelular/mortalidade , Carcinoma Hepatocelular/patologia , Estudos de Coortes , Feminino , Humanos , Neoplasias Hepáticas/mortalidade , Neoplasias Hepáticas/patologia , Masculino , Pessoa de Meia-Idade , Neoplasias Primárias Múltiplas/patologia , Niacinamida/economia , Niacinamida/provisão & distribuição , Niacinamida/uso terapêutico , Compostos de Fenilureia/economia , Compostos de Fenilureia/uso terapêutico , Estudos Retrospectivos , Sorafenibe , Reino Unido , Adulto JovemAssuntos
Carcinoma Basocelular/prevenção & controle , Carcinoma de Células Escamosas/prevenção & controle , Niacinamida/uso terapêutico , Neoplasias Cutâneas/tratamento farmacológico , Neoplasias Cutâneas/prevenção & controle , Carcinoma Basocelular/tratamento farmacológico , Carcinoma Basocelular/epidemiologia , Carcinoma de Células Escamosas/tratamento farmacológico , Carcinoma de Células Escamosas/epidemiologia , Efeitos Psicossociais da Doença , Humanos , Ceratose Actínica , Niacinamida/administração & dosagem , Niacinamida/economia , Prevenção Secundária/métodos , Neoplasias Cutâneas/epidemiologia , Complexo Vitamínico B/efeitos adversos , Complexo Vitamínico B/uso terapêuticoRESUMO
AIM: to evaluate treatment compliance and survival of patients receiving oral sorafenib in Indonesia. METHODS: a prospective cohort trial. Unresectable Hepatocelullar carcinoma patients receiving Sorafenib in NexPAP program were recruited between October 2008 and September 2011. A historical cohort from Cipto Mangunkusumo Hospital, between 1998 and 2000 was selected to serve as control group. Patients in the control group received symptomatic treatment. Survival analysis was done by the Kaplan-Meier survival curve analysis and the log-rank test. Median survival difference between the NexPAP and control group was tested using the Cox-regression hazard analysis. RESULTS: There were 48 patients in the NexPAP group and 40 patients in the control group. Treatment compliance was very good; no patient with drew from the study. Sorafenib generally could be tolerated by the patients. The most common adverse events are mild or moderate hand and foot skin reaction and diarrhea. The median survival was 49 weeks in NexPAP group (95% CI 37.9-60.1) vs. 20 weeks in the control group (95% CI 9.0-31.0). Cox-regression analysis showed that sorafenib significantly prolonged overall survival with a hazard ratio (HR) of 0.339 (95% CI: 0.196-0.584). There was no survival difference between patients with Child-Pugh class A and class B in both NexPAP (median 49 vs. 52 weeks; HR 1.1; 95% CI 0.5-2.3; p=0.855) and control groups (27 vs. 20 weeks; HR 1.1; 95% CI 0.5-2.4; p=0.822). CONCLUSION: Sorafenib patient assistant program in unresectable hepatocellular carcinoma ensured compliance treatment and significantly prolonged overall survival over the historical cohort receiving palliative treatment.
Assuntos
Antineoplásicos/uso terapêutico , Carcinoma Hepatocelular/tratamento farmacológico , Neoplasias Hepáticas/tratamento farmacológico , Adesão à Medicação , Niacinamida/análogos & derivados , Compostos de Fenilureia/uso terapêutico , Adulto , Idoso , Idoso de 80 Anos ou mais , Antineoplásicos/efeitos adversos , Antineoplásicos/economia , Feminino , Apoio Financeiro , Humanos , Indonésia , Estimativa de Kaplan-Meier , Masculino , Pessoa de Meia-Idade , Niacinamida/efeitos adversos , Niacinamida/economia , Niacinamida/uso terapêutico , Compostos de Fenilureia/efeitos adversos , Compostos de Fenilureia/economia , Avaliação de Programas e Projetos de Saúde , Modelos de Riscos Proporcionais , Estudos Prospectivos , Sorafenibe , Adulto JovemAssuntos
Indústria Farmacêutica/legislação & jurisprudência , Controle de Medicamentos e Entorpecentes/legislação & jurisprudência , Regulamentação Governamental , Política de Saúde/legislação & jurisprudência , Acessibilidade aos Serviços de Saúde/legislação & jurisprudência , Patentes como Assunto/legislação & jurisprudência , Antineoplásicos/economia , Benzamidas/economia , Custos de Medicamentos , Indústria Farmacêutica/economia , Controle de Medicamentos e Entorpecentes/economia , Medicamentos Genéricos/economia , Política de Saúde/economia , Acessibilidade aos Serviços de Saúde/economia , Humanos , Mesilato de Imatinib , Índia , Niacinamida/análogos & derivados , Niacinamida/economia , Compostos de Fenilureia/economia , Piperazinas/economia , Pirimidinas/economia , SorafenibeRESUMO
Sorafenib is the only Food and Drug Administration (FDA)-approved first-line therapy shown to have survival benefit for patients with advanced hepatocellular carcinoma (HCC). Patients with advanced HCC are often but not exclusively transferred from non-oncologists to oncologists to initiate systemic therapy. The objective of this study was to assess whether sorafenib prescribing by non-oncologists has any impact on utilization, adverse effects, cost or outcome.This was a retrospective cohort study utilizing data from patients prescribed sorafenib for HCC within Veterans Health Administration hospitals with 100% chart abstraction to confirm HCC diagnosis, identify prescribing provider specialty (oncology versus gastroenterology/hepatology), and obtain data required for cancer staging by the Barcelona Clinic Liver Cancer (BCLC) system. The primary outcome was overall survival from the time of sorafenib prescription.A total of 4903 patients who prescribed sorafenib for HCC were identified, for whom 340 patients (6.9%) were prescribed drug by a non-oncologist (Onc). BCLC Stage, age, Child-Turcotte-Pugh score, and comorbidity indices were similar between patients prescribed sorafenib by oncologists and non-oncologists. Oncologists more often discontinued sorafenib due to progression, whereas non-oncologists were more likely to continue sorafenib until death resulting in greater pill utilization and cost. Overall survival in both unadjusted and multivariable models showed no significant impact of prescriber type on survival (222 vs 217 days, Pâ=â.96), confirmed with propensity-matched subcohorts.Similar survival outcomes were observed for patients with HCC prescribed sorafenib by non-oncologists and oncologists, suggesting that non-oncologists with expertise in the management of HCC can safely and effectively administer sorafenib.
Assuntos
Antineoplásicos/uso terapêutico , Carcinoma Hepatocelular/tratamento farmacológico , Neoplasias Hepáticas/tratamento farmacológico , Niacinamida/análogos & derivados , Compostos de Fenilureia/uso terapêutico , Padrões de Prática Médica/estatística & dados numéricos , Idoso , Antineoplásicos/economia , Custos de Medicamentos , Prescrições de Medicamentos/estatística & dados numéricos , Feminino , Gastroenterologistas/estatística & dados numéricos , Humanos , Masculino , Oncologia/estatística & dados numéricos , Pessoa de Meia-Idade , Niacinamida/economia , Niacinamida/uso terapêutico , Compostos de Fenilureia/economia , Estudos Retrospectivos , Sorafenibe , Estados UnidosRESUMO
Hepatocellular carcinoma (HCC) presentation is heterogeneous necessitating a variety of therapeutic interventions with varying efficacies and associated prognoses. Poor prognostic patients often undergo non-curative palliative interventions including transarterial chemoembolization (TACE), sorafenib, chemotherapy, or purely supportive care. The decision to pursue one of many palliative interventions for HCC is complex and an economic evaluation comparing these interventions has not been done. This study evaluates the cost-effectiveness of non-curative palliative treatment strategies such as TACE alone or TACE+sorafenib, sorafenib alone, and non-sorafenib chemotherapy compared with no treatment or best supportive care (BSC) among patients diagnosed with HCC between 2007 and 2010 in a Canadian setting. Using person-level data, we estimated effectiveness in life years and quality-adjusted life years (QALYs) along with total health care costs (2013 US dollars) from the health care payer's perspective (3% annual discount). A net benefit regression approach accounting for baseline covariates with propensity score adjustment was used to calculate incremental net benefit to generate incremental cost-effectiveness ratio (ICER) and uncertainty measures. Among 1,172 identified patients diagnosed with HCC, 4.5%, 7.9%, and 5.6%, received TACE alone or TACE+sorafenib, sorafenib, and non-sorafenib chemotherapy clone, respectively. Compared with no treatment or BSC (81.9%), ICER estimates for TACE alone or TACE+sorafenib was $6,665/QALY (additional QALY: 0.47, additional cost: $3,120; 95% CI: -$18,800-$34,500/QALY). The cost-effectiveness acceptability curve demonstrated that if the relevant threshold was $50,000/QALY, TACE alone or TACE+sorafenib, non-sorafenib chemotherapy, and sorafenib alone, would have a cost-effectiveness probability of 99.7%, 46.6%, and 5.5%, respectively. Covariates associated with the incremental net benefit of treatments are age, sex, comorbidity, and cancer stage. Findings suggest that TACE with or without sorafenib is currently the most cost-effective active non-curative palliative treatment approach to HCC. Further research into new combination treatment strategies that afford the best tumor response is needed.
Assuntos
Carcinoma Hepatocelular/tratamento farmacológico , Análise Custo-Benefício , Neoplasias Hepáticas/tratamento farmacológico , Idoso , Idoso de 80 Anos ou mais , Carcinoma Hepatocelular/economia , Quimioembolização Terapêutica/economia , Quimioembolização Terapêutica/métodos , Terapia Combinada , Feminino , Humanos , Estimativa de Kaplan-Meier , Neoplasias Hepáticas/economia , Masculino , Pessoa de Meia-Idade , Estadiamento de Neoplasias , Niacinamida/análogos & derivados , Niacinamida/economia , Niacinamida/uso terapêutico , Cuidados Paliativos/economia , Compostos de Fenilureia/economia , Compostos de Fenilureia/uso terapêutico , Sorafenibe , Resultado do TratamentoRESUMO
BACKGROUND: Lenvatinib (Lenvima®) and sorafenib (Nexavar®) are the two most recently Food and Drug Administration-approved drugs for treating radioiodine-refractory differentiated thyroid cancer (RR-DTC). Both demonstrated superior progression-free survival over placebo in their respective Phase III clinical trials. This study compared the cost-effectiveness of the two treatments with placebo from a limited societal perspective. METHODS: A Markov model was developed to estimate the costs and health benefits for treatment of RR-DTC. The probabilities and survival rates were obtained from two Phase III trials: the SELECT trial comparing lenvatinib to placebo, and the DECISION trial comparing sorafenib to placebo. A bimonthly cycle length and half-cycle correction were used for a lifetime time horizon. Medical costs and utility data were obtained from RedBook, Healthcare Cost and Utilization Project, and the published literature. All costs were adjusted to US$2015, discounted at 3% annually. Then second-order Monte Carlo simulation with distributions was conducted to obtain the acceptability curve to address the uncertainty around model inputs. RESULTS: In the base case, lenvatinib was the most cost-effective treatment compared to sorafenib (incremental cost-effectiveness ratio [ICER] = $25,275/quality-adjusted life year [QALY]) and placebo (ICER = $40,869). Sorafenib is also cost-effective compared to placebo (ICER = $64,067/QALY). The treatment decisions were found to be sensitive to the treatment costs and the health utility associated with lenvatinib and its side effects. The acceptability curve showed lenvatinib optimal 80% of time at WTP of $100,000/QALY. CONCLUSIONS: This study suggests that lenvatinib is the optimally cost-effective treatment for RR-DTC, although both lenvatinib and sorafenib are cost-effective compared to placebo.
Assuntos
Antineoplásicos/uso terapêutico , Modelos Econômicos , Niacinamida/análogos & derivados , Compostos de Fenilureia/uso terapêutico , Inibidores de Proteínas Quinases/uso terapêutico , Qualidade de Vida , Quinolinas/uso terapêutico , Neoplasias da Glândula Tireoide/tratamento farmacológico , Idoso , Antineoplásicos/efeitos adversos , Antineoplásicos/economia , Diferenciação Celular , Ensaios Clínicos Fase III como Assunto , Ensaios Clínicos Controlados como Assunto , Análise Custo-Benefício , Custos de Medicamentos , Feminino , Custos de Cuidados de Saúde , Humanos , Masculino , Pessoa de Meia-Idade , Método de Monte Carlo , Gradação de Tumores , Niacinamida/efeitos adversos , Niacinamida/economia , Niacinamida/uso terapêutico , Compostos de Fenilureia/efeitos adversos , Compostos de Fenilureia/economia , Inibidores de Proteínas Quinases/efeitos adversos , Inibidores de Proteínas Quinases/economia , Quinolinas/efeitos adversos , Quinolinas/economia , Sorafenibe , Análise de Sobrevida , Neoplasias da Glândula Tireoide/economia , Neoplasias da Glândula Tireoide/patologia , Carga Tumoral/efeitos dos fármacos , Estados UnidosRESUMO
OBJECTIVE: Stereotactic body radiotherapy (SBRT) has been shown to improve overall survival in patients with advanced hepatocellular carcinoma. This study aimed to assess the cost-effectiveness of SBRT compared to sorafenib which is the only drug for advanced hepatocellular carcinoma. METHODS: A Markov decision-analytic model was performed to compare the cost-effectiveness of SBRT and sorafenib for unresectable advanced hepatocellular carcinoma. Patients transitioned between three health states: stable disease, progression disease and death. We calculated the data on cost from the perspective of our National Health Insurance Bureau. Sensitivity analyses were conducted to determine the impact of several variables. RESULTS: The incremental cost effectiveness ratio (ICER) for sorafenib compared to SBRT was NT$3,788,238 per quality-adjusted life year gained (cost/QALY), which was higher than the willingness to pay threshold of Taiwan according to WHO's guideline. One-way sensitivity analysis revealed that the utility of progression disease for the sorafenib treatment, utility of progression free survival for SBRT, utility of progression free survival for sorafenib, utility of PFS to progression disease for SBRT and transition probability of progression disease to dead for SBRT were the most sensitive parameters in all cost scenarios. The Monte-Carlo simulation demonstrated that the probability of cost-effectiveness at a willingness to pay threshold of NT$ 2,213,145 per QALY was 100 % and 0 % chance for SBRT and sorafenib. CONCLUSION: This study indicated that SBRT for advanced hepatocellular carcinoma is cost-effective at a willingness to pay threshold as defined by WHO guideline in Taiwan.
Assuntos
Carcinoma Hepatocelular/tratamento farmacológico , Carcinoma Hepatocelular/radioterapia , Neoplasias Hepáticas/tratamento farmacológico , Neoplasias Hepáticas/radioterapia , Niacinamida/análogos & derivados , Compostos de Fenilureia/uso terapêutico , Radiocirurgia/métodos , Idoso , Análise Custo-Benefício , Tomada de Decisões , Progressão da Doença , Intervalo Livre de Doença , Feminino , Humanos , Masculino , Cadeias de Markov , Método de Monte Carlo , Niacinamida/economia , Niacinamida/uso terapêutico , Compostos de Fenilureia/economia , Probabilidade , Anos de Vida Ajustados por Qualidade de Vida , Radiocirurgia/economia , Ensaios Clínicos Controlados Aleatórios como Assunto , Sorafenibe , TaiwanRESUMO
OBJECTIVE: To calculate sustainable generic prices for 4 tyrosine kinase inhibitors (TKIs). BACKGROUND: TKIs have proven survival benefits in the treatment of several cancers, including chronic myeloid leukaemia, breast, liver, renal and lung cancer. However, current high prices are a barrier to treatment. Mass production of low-cost generic antiretrovirals has led to over 13 million people being on HIV/AIDS treatment worldwide. This analysis estimates target prices for generic TKIs, assuming similar methods of mass production. METHODS: Four TKIs with patent expiry dates in the next 5 years were selected for analysis: imatinib, erlotinib, lapatinib and sorafenib. Chemistry, dosing, published data on per-kilogram pricing for commercial transactions of active pharmaceutical ingredient (API), and quotes from manufacturers were used to estimate costs of production. Analysis included costs of excipients, formulation, packaging, shipping and a 50% profit margin. Target prices were compared with current prices. Global numbers of patients eligible for treatment with each TKI were estimated. RESULTS: API costs per kg were $347-$746 for imatinib, $2470 for erlotinib, $4671 for lapatinib, and $3000 for sorafenib. Basing on annual dose requirements, costs of formulation/packaging and a 50% profit margin, target generic prices per person-year were $128-$216 for imatinib, $240 for erlotinib, $1450 for sorafenib, and $4020 for lapatinib. Over 1 million people would be newly eligible to start treatment with these TKIs annually. CONCLUSIONS: Mass generic production of several TKIs could achieve treatment prices in the range of $128-$4020 per person-year, versus current US prices of $75161-$139,138. Generic TKIs could allow significant savings and scaling-up of treatment globally, for over 1 million eligible patients.
Assuntos
Antineoplásicos/economia , Comércio , Saúde Global/economia , Neoplasias/tratamento farmacológico , Inibidores de Proteínas Quinases/economia , Proteínas Tirosina Quinases/antagonistas & inibidores , Antineoplásicos/uso terapêutico , Indústria Farmacêutica/economia , Cloridrato de Erlotinib/economia , Cloridrato de Erlotinib/uso terapêutico , Humanos , Mesilato de Imatinib/economia , Mesilato de Imatinib/uso terapêutico , Lapatinib , Niacinamida/análogos & derivados , Niacinamida/economia , Niacinamida/uso terapêutico , Compostos de Fenilureia/economia , Compostos de Fenilureia/uso terapêutico , Inibidores de Proteínas Quinases/uso terapêutico , Quinazolinas/economia , Quinazolinas/uso terapêutico , SorafenibeRESUMO
BACKGROUND: This study aimed to investigate the pharmaco-economic implications of FOLFOX4 or sorafenib for advanced hepatocellular carcinoma in China. METHODS: To conduct the analysis, we performed a Markov model to simulate the process of advanced HCC treated with sorafenib or FOLFOX4. Clinical data were obtained from the ORIENTAL trial and the EACH trial. Incremental cost-effectiveness ratio was regarded as the primary outcome in the analysis. One-way sensitivity analysis as well as probabilistic sensitivity analysis was performed to explore the impact of essential variables on the results of the analysis. RESULTS: Treatment with sorafenib provided an effectiveness gain of 0.3935 quality-adjusted life year at an average cost of $18,748.00, whereas chemotherapy of FOLFOX4 brought 0.3808 quality-adjusted life year at a cost of $6876.02. The incremental cost-effectiveness ratio of FOLFOX4 versus sorafenib was $934,801.57/QALY. In a probabilistic sensitivity analysis based on a Monte Carlo simulation of 1000 items, the probabilities of FOLFOX4 and sorafenib being cost-effective were 100% and 0% using a willingness-to-pay threshold of $20,301.00 per quality-adjusted life year. CONCLUSIONS: FOLFOX4 chemotherapy is likely to be a cost-effective option compared with sorafenib in the treatment of advanced hepatocellular carcinoma in China.
Assuntos
Protocolos de Quimioterapia Combinada Antineoplásica/economia , Carcinoma Hepatocelular/tratamento farmacológico , Análise Custo-Benefício , Neoplasias Hepáticas/tratamento farmacológico , Niacinamida/análogos & derivados , Compostos de Fenilureia/economia , Protocolos de Quimioterapia Combinada Antineoplásica/uso terapêutico , China , Estudos de Coortes , Feminino , Fluoruracila/economia , Fluoruracila/uso terapêutico , Humanos , Leucovorina/economia , Leucovorina/uso terapêutico , Masculino , Pessoa de Meia-Idade , Método de Monte Carlo , Niacinamida/economia , Niacinamida/uso terapêutico , Compostos Organoplatínicos/economia , Compostos Organoplatínicos/uso terapêutico , Compostos de Fenilureia/uso terapêutico , Anos de Vida Ajustados por Qualidade de Vida , SorafenibeRESUMO
INTRODUCTION: The Oncology field is characterised by a steady increase in demand and a consistent launching of innovative and expensive products. Therefore, cost-effectiveness analysis can contribute as a significant decision-making tool by elucidating the most economically efficient ways to satisfy compelling health needs. AREAS COVERED: The scope of this study is to estimate the cost-effectiveness of axitinib versus sorafenib, for the second-line treatment of renal cell carcinoma. A literature review for evidence synthesis was performed and a probabilistic Markov Model was employed to simulate disease progression. This study will also assess Value of Information. EXPERT OPINION: Compared to sorafenib, axitinib resulted in an incremental cost of 87,936 euro per quality adjusted life year. The probability of axitinib to being cost-effective at the willingness-to-pay threshold of 60,000 euro was 13%, while the corresponding probability of being cost-effective at the highest recommended willingness-to-pay threshold of 100,000 euro was 69.9%. Uncertainty was primarily attributed to the price of the product, the utility values, the progression-free survival and to a lesser degree to the overall survival. Axitinib can be considered as a cost-effective therapeutic option for second-line treatment of renal cell carcinoma.
Assuntos
Antineoplásicos/economia , Carcinoma de Células Renais/economia , Imidazóis/economia , Indazóis/economia , Neoplasias Renais/economia , Inibidores de Proteínas Quinases/economia , Antineoplásicos/uso terapêutico , Axitinibe , Carcinoma de Células Renais/tratamento farmacológico , Análise Custo-Benefício , Progressão da Doença , Intervalo Livre de Doença , Humanos , Imidazóis/uso terapêutico , Indazóis/uso terapêutico , Neoplasias Renais/tratamento farmacológico , Cadeias de Markov , Modelos Econômicos , Niacinamida/análogos & derivados , Niacinamida/economia , Niacinamida/uso terapêutico , Compostos de Fenilureia/economia , Compostos de Fenilureia/uso terapêutico , Inibidores de Proteínas Quinases/uso terapêutico , Anos de Vida Ajustados por Qualidade de Vida , Sorafenibe , IncertezaRESUMO
OBJECTIVE: Sorafenib has been shown to significantly improve the overall survival of patients with advanced hepatocellular carcinoma (HCC). This study aimed to assess the cost-effectiveness of sorafenib as a first-line treatment for patients with advanced HCC. MATERIALS AND METHODS: To carry out the analysis, we collected the data on the efficacy and safety of patients treated with sorafenib from medical records and follow-up of these patients. A Markov model comprising three health states (progression-free survival, progressive disease, and death) was created to simulate the process of advanced HCC. We calculated the data on cost from the perspective of Chinese patients. Sensitivity analyses were also carried out to explore the impact of several essential variables. RESULTS: Overall, 94 patients with advanced HCC were included in our study: 70 in the Child-Pugh A group and 24 in the Child-Pugh B group. The median overall survival was 8.0 months (95% confidence interval: 7.21-8.50). In general, treatment with sorafenib was estimated to increase costs by $18,251.84 compared with best supportive care, with a gain of 0.18 quality-adjusted life years (QALYs). Thus, the incremental cost-effective ratio was $101,399.11/QALY for sorafenib versus best supportive care. In addition, in patients with Child-Pugh A liver function, the total costs and effectiveness were $20,643.06 and 0.48 QALYs, respectively, whereas in the Child-Pugh class B group, the total costs and effectiveness were $15,844.33 and 0.28 QALYs. CONCLUSION: On the basis of the commonly accepted willingness-to-pay threshold ($20,301.00/QALY in China), sorafenib is not a cost-effective option as a first-line treatment for patients with advanced HCC.
Assuntos
Antineoplásicos , Carcinoma Hepatocelular/tratamento farmacológico , Carcinoma Hepatocelular/economia , Análise Custo-Benefício , Neoplasias Hepáticas/tratamento farmacológico , Neoplasias Hepáticas/economia , Niacinamida/análogos & derivados , Compostos de Fenilureia , Adulto , Idoso , Antineoplásicos/economia , Antineoplásicos/uso terapêutico , Carcinoma Hepatocelular/diagnóstico , Carcinoma Hepatocelular/mortalidade , China , Progressão da Doença , Feminino , Humanos , Estimativa de Kaplan-Meier , Neoplasias Hepáticas/diagnóstico , Neoplasias Hepáticas/mortalidade , Masculino , Pessoa de Meia-Idade , Estadiamento de Neoplasias , Niacinamida/economia , Niacinamida/uso terapêutico , Compostos de Fenilureia/economia , Compostos de Fenilureia/uso terapêutico , Estudos Retrospectivos , Sorafenibe , Resultado do TratamentoRESUMO
BACKGROUND: Limited data exist on the economic consequences of implementing targeted therapy (TT) for metastatic renal cell carcinoma (RCC) in a real-world setting. OBJECTIVE: To analyze health care and productivity costs for TT implementation in a national cohort of patients. DESIGN, SETTING, AND PARTICIPANTS: Costs were measured per patient per year during a 2-yr follow-up during 2002-2005 (immunotherapy only) and 2006-2009 (TT implementation). All Danish patients with a diagnosis code for RCC and a procedure code for TT or immunotherapy were linked to the Danish National Patient Registry (contains information on all contacts with primary and secondary health sector). Health care and productivity costs were retrieved from the Danish case-mix system and Coherent Social Statistics, respectively. Drug costs were calculated separately from procedure codes and retail prices. OUTCOME MEASUREMENTS AND STATISTICAL ANALYSIS: Generalized linear models were used to analyze costs adjusted for age, gender, and civil status. RESULTS AND LIMITATIONS: A total of 439 patients were included for 2006-2009 and 192 for 2002-2005. Comparison of the health care cost per patient per year between 2006-2009 and 2002-2005 revealed lower inpatient costs (11 899 vs 19 944, adjusted relative risk [RR] 0.64), higher outpatient costs (14 308 vs 6209, RR 2.39), lower radiotherapy costs (194 vs 633, RR 0.31), higher radiology costs (676 vs 191, RR 3.73), and higher separately calculated drug costs (12 040 vs 3103, RR 3.82, all p<0.001) for the former. Total health care costs per patient per year did not significantly differ (27 676 vs 27 856, RR 1.05, p=0.5) between the two periods. Income from employment did not significantly differ between 2006-2009 and 2002-2005 (RR 1.11, p=0.11) and costs associated with loss of productivity were 7852 and 8265, respectively. CONCLUSIONS: A different pattern of health care costs were observed but total health care costs per patient per year did not significantly differ after implementation of TT for patients with mRCC. PATIENT SUMMARY: In this nationwide study, we found changes in the pattern of health care costs for patients with metastatic kidney cancer after implementation of targeted therapy compared to an immunotherapy control period; however, total health care costs and income from employment were without significant changes.
Assuntos
Inibidores da Angiogênese/economia , Antineoplásicos/economia , Carcinoma de Células Renais/economia , Custos de Cuidados de Saúde , Fatores Imunológicos/economia , Neoplasias Renais/economia , Inibidores de Proteínas Quinases/economia , Sistema de Registros , Adulto , Idoso , Idoso de 80 Anos ou mais , Assistência Ambulatorial/economia , Inibidores da Angiogênese/uso terapêutico , Antineoplásicos/uso terapêutico , Bevacizumab/economia , Bevacizumab/uso terapêutico , Carcinoma de Células Renais/tratamento farmacológico , Carcinoma de Células Renais/secundário , Custos e Análise de Custo , Dinamarca , Custos de Medicamentos , Eficiência , Emprego/economia , Everolimo/economia , Everolimo/uso terapêutico , Feminino , Fluoruracila/economia , Fluoruracila/uso terapêutico , Hospitalização/economia , Humanos , Fatores Imunológicos/uso terapêutico , Indóis/economia , Indóis/uso terapêutico , Interferon-alfa/economia , Interferon-alfa/uso terapêutico , Interleucina-2/economia , Interleucina-2/uso terapêutico , Neoplasias Renais/tratamento farmacológico , Neoplasias Renais/patologia , Masculino , Pessoa de Meia-Idade , Metástase Neoplásica/tratamento farmacológico , Niacinamida/análogos & derivados , Niacinamida/economia , Niacinamida/uso terapêutico , Compostos de Fenilureia/economia , Compostos de Fenilureia/uso terapêutico , Inibidores de Proteínas Quinases/uso terapêutico , Pirróis/economia , Pirróis/uso terapêutico , Radiografia/economia , Radioterapia/economia , Sirolimo/análogos & derivados , Sirolimo/economia , Sirolimo/uso terapêutico , Sorafenibe , SunitinibeRESUMO
The objective of this study is to assess the cost effectiveness of sorafenib as a second line treatment of advanced renal cell carcinoma compared to standard best supportive care (BSC) in Cyprus. A probabilistic Decision analytic Markov Model was created to simulate disease progression and data from landmark trials were used. Actual local costs were set according to current guidelines in Cyprus. The incremental cost per quality adjusted life year of sorafenib versus BSC was 102,059. The probability of sorafenib to be cost effective at the threshold of 60,000 was 0%. Total costs were sensitive to the price of product, its effectiveness and to a lesser degree to the utility values. Sorafenib demonstrated superior clinical effectiveness compared to BSC, but it's not cost effective under current willingness to pay threshold. Its orphan status along with solidarity principle may justify reimbursement on an individual patient basis.