RESUMEN
INTRODUCTION: Recently, new oncology therapies were developed using a biomarker for patient selection. In the era of cancer genomics, this paradigm is expected to increase. Most cytotoxic chemotherapies and other oncological treatments were historically approved without a biomarker. However, this strategy seems to be less efficient. We reviewed the biomarker-based strategy and its impact in cancer drug development. AREAS COVERED: Oncology drugs approval rates are low and most of the drugs that failed to be approved were in late stages of development. In addition to that, attrition rates are high. The use of biomarkers in drug development has shown higher response rates, longer progression-free survival rates and even higher overall survival rates. Hence, the biomarker-based strategy seems to be associated with more successful drug programs, including a shorter timeline and higher likelihood of success. EXPERT OPINION: Even though the development of biomarker-driven strategies is promising, there are some challenges surrounding this field of study, such as reducing the cost of drug development, enhancing the technique of biomarkers identification (aiming more specific biomarkers and considering tumor heterogeneity) and exploring the role of next-generation sequencing tests in drug development. Also, collaboration between clinicians, scientists and regulatory agencies is fundamental.
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Antineoplásicos/uso terapéutico , Biomarcadores de Tumor , Desarrollo de Medicamentos/métodos , Terapia Molecular Dirigida , Neoplasias/tratamiento farmacológico , Medicina de Precisión/métodos , Antineoplásicos/economía , Antineoplásicos/farmacología , Inteligencia Artificial , Biomarcadores de Tumor/análisis , Biomarcadores de Tumor/antagonistas & inhibidores , Biomarcadores de Tumor/genética , Ensayos Clínicos como Asunto/métodos , Análisis Mutacional de ADN , Aprobación de Drogas , Desarrollo de Medicamentos/tendencias , Secuenciación de Nucleótidos de Alto Rendimiento , Humanos , Inhibidores de Puntos de Control Inmunológico/uso terapéutico , Colaboración Intersectorial , Metaanálisis como Asunto , Técnicas de Diagnóstico Molecular , Terapia Molecular Dirigida/economía , Terapia Molecular Dirigida/métodos , Neoplasias/química , Neoplasias/genética , Selección de Paciente , Factores de Tiempo , Estados Unidos , United States Food and Drug AdministrationRESUMEN
ABSTRACT BACKGROUND: Lung cancer is the fourth most common cancer in Brazil. In the 2000s, better understanding of molecular pathways led to development of epidermal growth factor receptor (EGFR)-targeted treatments that have improved outcomes. However, these treatments are unavailable in most Brazilian public healthcare services (Sistema Único de Saúde, SUS). OBJECTIVE: To assess the potential number of years of life not saved, the budget impact of the treatment and strategies to improve access. DESIGN AND SETTING: Pharmacoeconomic study assessing the potential societal and economic impact of adopting EGFR-targeted therapy within SUS. METHODS: We estimated the number of cases eligible for treatment, using epidemiological data from the National Cancer Institute. We used data from a single meta-analysis and from the Lung Cancer Mutation Consortium (LCMC) study as the basis for assessing differences in patients' survival between use of targeted therapy and use of chemotherapy. The costs of targeted treatment were based on the national reference and were compared with the amount reimbursed for chemotherapy through SUS. RESULTS: There was no life-year gain with EGFR-targeted therapy in the single meta-analysis (hazard ratio, HR, 1.01). The LCMC showed that 1,556 potential life-years were not saved annually. We estimated that the annual budget impact was 125 million Brazilian reais (BRL) with erlotinib, 48 million BRL with gefitinib and 52 million BRL with afatinib. Their incremental costs over chemotherapy per life-year saved were 80,329 BRL, 31,011 BRL and 33,225 BRL, respectively. A drug acquisition discount may decrease the budget impact by 30% (with a 20% discount). A fixed cost of 1,000 BRL may decrease the budget impact by 95%. CONCLUSION: Reducing drug acquisition costs may improve access to EGFR-targeted therapy for lung cancer.
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Humanos , Costos de la Atención en Salud , Años de Vida Ajustados por Calidad de Vida , Inhibidores de Proteínas Quinasas/economía , Receptores ErbB/economía , Neoplasias Pulmonares/economía , Quinazolinas/economía , Quinazolinas/uso terapéutico , Brasil , Presupuestos , Análisis de Supervivencia , Análisis Costo-Beneficio/economía , Prorrateo de Riesgo Financiero/métodos , Inhibidores de Proteínas Quinasas/uso terapéutico , Terapia Molecular Dirigida/economía , Receptores ErbB/uso terapéutico , Accesibilidad a los Servicios de Salud/economía , Neoplasias Pulmonares/mortalidad , Neoplasias Pulmonares/tratamiento farmacológicoRESUMEN
BACKGROUND: Lung cancer is the fourth most common cancer in Brazil. In the 2000s, better understanding of molecular pathways led to development of epidermal growth factor receptor (EGFR)-targeted treatments that have improved outcomes. However, these treatments are unavailable in most Brazilian public healthcare services (Sistema Único de Saúde, SUS). OBJECTIVE: To assess the potential number of years of life not saved, the budget impact of the treatment and strategies to improve access. DESIGN AND SETTING: Pharmacoeconomic study assessing the potential societal and economic impact of adopting EGFR-targeted therapy within SUS. METHODS: We estimated the number of cases eligible for treatment, using epidemiological data from the National Cancer Institute. We used data from a single meta-analysis and from the Lung Cancer Mutation Consortium (LCMC) study as the basis for assessing differences in patients' survival between use of targeted therapy and use of chemotherapy. The costs of targeted treatment were based on the national reference and were compared with the amount reimbursed for chemotherapy through SUS. RESULTS: There was no life-year gain with EGFR-targeted therapy in the single meta-analysis (hazard ratio, HR, 1.01). The LCMC showed that 1,556 potential life-years were not saved annually. We estimated that the annual budget impact was 125 million Brazilian reais (BRL) with erlotinib, 48 million BRL with gefitinib and 52 million BRL with afatinib. Their incremental costs over chemotherapy per life-year saved were 80,329 BRL, 31,011 BRL and 33,225 BRL, respectively. A drug acquisition discount may decrease the budget impact by 30% (with a 20% discount). A fixed cost of 1,000 BRL may decrease the budget impact by 95%. CONCLUSION: Reducing drug acquisition costs may improve access to EGFR-targeted therapy for lung cancer.
Asunto(s)
Receptores ErbB/economía , Costos de la Atención en Salud , Neoplasias Pulmonares/economía , Inhibidores de Proteínas Quinasas/economía , Años de Vida Ajustados por Calidad de Vida , Brasil , Presupuestos , Análisis Costo-Beneficio/economía , Receptores ErbB/uso terapéutico , Accesibilidad a los Servicios de Salud/economía , Humanos , Neoplasias Pulmonares/tratamiento farmacológico , Neoplasias Pulmonares/mortalidad , Terapia Molecular Dirigida/economía , Inhibidores de Proteínas Quinasas/uso terapéutico , Quinazolinas/economía , Quinazolinas/uso terapéutico , Prorrateo de Riesgo Financiero/métodos , Análisis de SupervivenciaRESUMEN
PURPOSE: As one solution to reducing costs and medical bankruptcies, experts have suggested that patients and physicians should discuss the cost of care up front. Whether these discussions are possible in an oncology setting and what their effects on the doctor-patient relationship are is not known. METHODS: We used the National Comprehensive Cancer Network (NCCN) Guidelines and the eviti Advisor platform to show patients with metastatic breast, lung, or colorectal cancer the costs associated with their chemotherapy and/or targeted therapy options during an oncology consultation. We measured provider attitudes and assessed patient satisfaction when consultations included discussion of costs. RESULTS: We approached 107 patients; 96 (90%) enrolled onto the study, three (3%) asked if they could be interviewed at a later date, and eight (7%) did not want to participate. Only five of 18 oncologists (28%) felt comfortable discussing costs, and only one of 18 (6%) regularly asked patients about financial difficulties. The majority of patients (80%) wanted cost information, and 84% reported that these conversations would be even more important if their co-pays were to increase. In total, 72% of patients responded that no health care professional has ever discussed costs with them. The majority of patients (80%) had no negative feelings about hearing cost information. CONCLUSION: In an era of rising co-pays, patients with cancer want cost-of-treatment discussions, and these conversations do not lead to negative feelings in the majority of patients. Additional training to prepare clinicians for how to discuss costs with their patients is needed.