SUMMARY BACKGROUND: Breast cancer is a public health problem with both high incidence and cure rates. After treatment, patients are monitored for long periods of time due to the risk of recurrence. Thus, staging and follow-up strategies should consider not only the best results for the patient but also its costs for the public health system. OBJECTIVE: The objective of this study was to quantify the waste of resources on breast cancer follow-up and evaluate its impact on the public health system. METHODS: This is a retrospective analysis of consecutive medical records to identify the intervals between consultations and tests used for staging and during the first 2 years of follow-up of patients with breast cancer treated at a public hospital in Brazil. Data were compared with the guidelines of the main international consensus. RESULTS: Medical records of 60 consecutive patients treated in 2018 were selected, of whom 52 had 2 or more years of follow-up, and 8 had only 1 year of complete follow-up. A total of 34 patients (56.67%) underwent excessive examinations for stating. During follow-up, 125 surplus consultations were performed (33.6%). In this phase, 111 surplus exams were also performed, representing an increase of 100.9%. A total of 423 laboratory tests were performed for 18 patients in the first year and 229 tests for 14 patients in the second year. CONCLUSION: Excessive tests and consultations significantly burdened the Unified Health System without any benefit to patients. Better adherence to staging and follow-up recommendations could reduce costs and optimize the limited resources used in the public health system.

ABSTRACT Objective To evaluate whether intrathecal chemotherapy improves clinical outcomes in patients with meningeal carcinomatosis. Methods This retrospective cohort study included consecutive patients with breast cancer diagnosed with meningeal carcinomatosis. Clinical and treatment data were collected from the patients’ medical charts. The primary outcome was overall survival, and the secondary outcomes were time to neurological deterioration and reporting of clinical benefit. Logistic regression and Cox proportional hazard models adjusted for potential confounders were used to evaluate the clinical response and overall survival, respectively. Results Overall, 109 female patients were included, 50 (45.9%) of whom received intrathecal chemotherapy with methotrexate and dexamethasone. The median treatment duration was 3 weeks (range, 1–13 weeks). Patients treated with intrathecal chemotherapy were more likely to report clinical benefit (74% versus 57.7%, adjusted odds ratio [OR] = 9.0, 95%CI=2.6–30.9, p<0.001). However, there was no difference in the time to neurologic deterioration (hazard ratio [HR] = 0.96, 95%CI= 0.57–1.59, p=0.86). Patients who received intrathecal chemotherapy did not show an increase in overall survival compared with that of patients who did not receive intrathecal chemotherapy (median overall survival = 1.8 months, 95%CI= 1.27–3.0 versus 2.5, 95%CI= 1.9–3.9, adjusted HR = 0.71, 95%CI= 0.41–1.22, p=0.21). There was a significant interaction between intrathecal chemotherapy and systemic treatment, and patients who received systemic therapy without intrathecal chemotherapy had better overall survival than that of the no-treatment group (adjusted HR = 0.38, 95%CI= 0.20–0.70, p=0.002). Conclusion Intrathecal chemotherapy did not increase overall survival or time to neurological deterioration and should not preclude or postpone systemic treatments.

ABSTRACT Objective To assess the incidence of febrile neutropenia without primary granulocyte colony-stimulating factor prophylaxis in patients undergoing chemotherapy with adjuvant docetaxel and cyclophosphamide, and to evaluate the toxicity profile of brand-name docetaxel (Taxotere ® ) and the generic formulation. Methods This retrospective study was conducted using data obtained from electronic medical records of patients treated at a Brazilian cancer center. Patients with breast cancer who underwent adjuvant treatment between January 2016 and June 2019 were selected. Data were analyzed using chi-square and Fisher correlation of variables, and multivariate analyses were adjusted for propensity score. Results A total of 231 patients with a mean age of 55.9 years at the time of treatment were included in the study. The majority (93.9%) had luminal histology, 84.8% were at clinical stage I, and 98.2% had a good performance status. The overall incidence of febrile neutropenia in the study population was 13.4% (31 cases). The use of brand-name docetaxel (Taxotere ® ) was the only factor associated with febrile neutropenia occurrence (OR= 3.55, 95%CI= 1.58-7.94, p=0.002). Conclusion In patients with breast cancer who require treatment with adjuvant docetaxel and cyclophosphamide regimen, the toxicity profile differs between brand-name and generic docetaxel. Regardless of the formulation used, the incidence of febrile neutropenia was less than 20%, which may allow for the omission of primary prophylactic granulocyte colony-stimulating factor use in this setting.

SUMMARY OBJECTIVE: Scientific writing in English is a daunting task for non-native English speakers. The challenges of writing in a foreign language are evident in the scientific literature where texts by non-native English-speaking scientists tend to be less clear and succinct, contain grammatical errors, and are often rejected by prestigious journals. METHODS: We conducted a non-systematic review of the most recent literature using the terms “Artificial Intelligence,” “Scientific Writing,” and “Non-English Speaking” to create a narrative review. RESULTS: Artificial intelligence can be a solution to improve scientific writing, especially for non-native English-speaking scientists. Artificial intelligence can assist in the search for pertinent scientific papers, generate summaries, and help with the writing of different sections of the manuscript, including the abstract, introduction, methods, results, and discussion. Artificial intelligence-based programs can correct grammatical errors and improve writing style, both of which are particularly helpful for non-native English speakers. Two artificial intelligence programs that can help with the search for pertinent scientific papers on the internet are Elicit and ResearchRabbit. Scispace Copilot can be used to summarize the retrieved reference. The artificial intelligence software programs such as Grammarly and Paperpal can correct grammatical and spelling errors, while ChatGPT can also restructure sentences and paragraphs, reword text, and suggest appropriate words and phrases. CONCLUSION: Overall, artificial intelligence can be an effective tool to improve the clarity, style, and coherence of scientific writing, helping non-native English-speaking scientists to communicate their research more effectively. OBJECTIVE nonnative non native speakers Englishspeaking speaking succinct journals METHODS nonsystematic systematic Intelligence, Intelligence Writing, Writing NonEnglish Non Speaking RESULTS summaries manuscript abstract introduction methods results discussion intelligencebased based style ResearchRabbit reference paragraphs text phrases CONCLUSION Overall clarity effectively

ABSTRACT Objective Human epidermal growth factor receptor 2 (HER2) overexpression occurs in up to 30% of breast cancer cases. Ado-trastuzumab emtansine (T-DM1) is approved to treat residual HER2-positive breast cancer after neoadjuvant therapy. The aim of this study was to determine the quality-adjusted time with symptoms or toxicity and without symptoms or toxicity (Q-TWiST) of T-DM1 compared to trastuzumab for residual invasive HER2-positive breast cancer. Methods The authors developed an analytical model extracting individual patient data and estimated invasive disease-free survival and overall survival over a 30-year time horizon. Only direct costs from adjuvant treatment were considered as well as relapse treatment from Brazilian and American payer perspectives. Heart events were considered for utility and cost analysis. Results The 30-year projection utilizing the Weibull method estimated a mean invasive disease-free survival of 16.4 years for T-DM1 and 10.4 for Trastuzumab, in addition to a mean overall survival of 18.1 and 15.4 years, respectively. We determined a Q-TWiST gain of 3,812 years for the T-DM1 arm when compared to trastuzumab and an Incremental cost-effectiveness ratio per Q-TWiST of US$ 11,467.65 in the United States and US$ 3,332.73 in Brazil. Conclusion Ado-trastuzumab emtansine is cost-effective from both Brazilian and American perspectives.

ABSTRACT Objective To describe of the translation from English to Portuguese and adaption process of subitems of the Functional Assessment of Cancer Therapy – Multiple Myeloma. Methods In the first phase, translations from English into Portuguese of two subitems of Functional Assessment of Cancer Therapy – Multiple Myeloma were performed. Subsequently, a consensus and back translation were conducted, and, finally, translation and back translations were reviewed by four independent bilingual experts. In the second phase, the translated subitems were applied, along with a questionnaire, to 10 native Portuguese speakers patients with multiple myeloma. Results There was a recognition of the translation process in its first version applied to 10 patients with multiple myeloma, whose reported no difficult to understand the translated and validated instrument. Patients also did not find the content irrelevant or offensive, and they did not suggested changes. Conclusion The subitems of the Functional Assessment of Cancer Therapy – Multiple Myeloma were translated from English into Portuguese following the proposed methodology and there was not need of readjustments. This process allowed this instrument of quality of life, which is widely known to be beneficial in the management of patients with multiple myeloma, to be used among our population.

ABSTRACT Introduction Pulmonary neuroendocrine tumors account for approximately 20% of all primary lung tumors. Few studies summarize the current body of pulmonary neuroendocrine tumors studies worldwide. Objective A quantitative scientometric analysis was conducted to evaluate the development of applications and innovations and to analyze their contribution to various areas of improvement in treatment and diagnosis of pulmonary neuroendocrine tumors. Methods We searched for studies published in the last 20 years in the databases United States National Library of Medicine (PubMed), Scientific Electronic Library Online (SciELO), Scopus, and Web of Science, using the terms ‘pulmonary neuroendocrine tumors’, ‘bronchial neuroendocrine tumors’, ‘bronchial carcinoid tumors’, ‘pulmonary carcinoid’, ‘typical pulmonary carcinoid’, ‘atypical pulmonary carcinoid’, ‘pulmonary carcinoid and diagnosis’, ‘pulmonary carcinoid and treatment’, ‘pulmonary carcinoid and epidemiology’ and ‘pulmonary carcinoid and prognosis’. Results Our results showed the number of publications increased significantly over the study period and was strongly associated with the economic or financial situation of the publications’ countries of origin. We observed a predominance of studies on histological diagnosis compared to treatment, and among the studies related to treatment, a predominance of retrospective studies relative to prospective studies was found. Conclusion Based on the published literature, we concluded research on pulmonary neuroendocrine tumors still seems to be incipient, because it favors studies related to histological characterization of the disease, and therapeutic studies are still predominantly of a retrospective nature.

RESUMO Objetivo Avaliar a gravidade da infecção por COVID-19 em pacientes oncológicos, determinando os aspectos clínicos e epidemiológicos associados ao pior desfecho, seja em termos de mortalidade, necessidade de internação em unidade de terapia intensiva ou ventilação mecânica. Métodos Pacientes com câncer e diagnóstico confirmado por laboratório de COVID-19 foram identificados nos bancos de dados de um hospital oncológico entre 16 de março e 29 de maio de 2020. Os dados coletados incluíram história, idade, sexo e comorbidades dos pacientes, além dos tipos de câncer e do tratamento anticâncer. Resultados Dentre os 105 pacientes analisados, a idade variou de 18 a 92 anos, e 80,9% eram do sexo feminino. Dispneia foi o sintoma inicial mais prevalente entre os que morreram (30,4%). No momento do diagnóstico da infecção, 57,1% apresentavam doença metastática e 60% performance status ruim (Eastern Cooperative Oncologic Group ≥2). A taxa de mortalidade geral foi 40,95% e superior entre os homens e pacientes com baixo nível de performance status (p<0,0001). Conclusão Este coorte é um dos estudos mais robustos do Brasil, descrevendo características clínicas e epidemiológicas de pacientes com câncer e COVID-19. Os achados do estudo alertam para a vulnerabilidade dos pacientes oncológicos na pandemia atual e demonstram alta mortalidade por COVID-19 em pacientes do sexo masculino e com pior performance status. Essa análise pode ajudar a selecionar os pacientes que podem se beneficiar de isolamento rigoroso e até mesmo da interrupção do tratamento, reduzindo a exposição à infecção.

ABSTRACT Objective To evaluate the severity of COVID-19 in cancer patients to describe clinical and epidemiological factors associated with poor outcomes (mortality and need of intensive care unit admission or mechanical ventilation). Methods Retrospective data from patients with cancer and laboratory diagnosis of COVID-19, obtained between March 16 and May 29, 2020, were retrieved out of a cancer center database. Data analyzed included patient history, age, sex, comorbidities, types of cancer and anticancer therapy. Results This sample comprised 105 patients aged 18-92 years, 80.9% of whom were females. Dyspnea was the most prevalent initial symptom (30.4%) among patients who died (p<0.0001). Overall, 57.1% of patients had metastatic disease and 60% had poor performance status (Eastern Cooperative Oncologic Group ≥2) at the time of COVID-19 diagnosis. The overall mortality rate was 40.95%. Mortality rates were higher in male patients and those with poor performance status (p<0.0001). Conclusion This cohort is one of the largest Brazilian studies describing clinical and epidemiological features of patients with cancer and concurrent COVID-19. Findings of this study emphasize the vulnerability of cancer patients in the current pandemic, and indicate high mortality from COVID-19 among male cancer patients and cancer patients with poor performance status. This analysis may assist the selection of patients who may benefit from strict isolation and eventual discontinuation of anticancer therapy to reduce exposure to infection.

RESUMO INTRODUÇÃO: Pacientes oncológicos e seus acompanhantes incorrem em custos não cobertos pelo Sistema Único de Saúde (SUS) durante o seu tratamento, com gastos relacionados a transporte, medicações sintomáticas, alimentação, perda de dias de trabalho dentre outros. OBJETIVO: Avaliar os custos incorridos e não cobertos pelo SUS por pacientes com câncer e seus acompanhantes durante a realização do tratamento, oncológico. MÉTODOS: Trata-se de um estudo transversal realizado com 110 pacientes oncológicos em tratamento com quimioterapia ou quimioterapia e radioterapia e 88 acompanhantes, no último mês, antes de sua inclusão no estudo. Correlacionamos os custos dispendidos com variáveis clínicas e sociodemográficas como sexo, idade, raça, estado civil, escolaridade, ocupação, naturalidade, procedência, renda mensal, renda familiar, moradia, comorbidades, tipo de neoplasia e estadiamento. RESULTADOS: Observamos que a média do custo global dos pacientes do estudo foi de R$ 747,92, que corresponde a 78,4% do salário mínimo e a média do custo dos acompanhantes foi de R$ 118,86 reais que equivale a 12,46% do salário mínimo. Dentre todas as variáveis analisadas, a média do custo global mensal dos pacientes correlacionou-se positivamente com a variável ocupação (p=0,021) assim como com a variável procedência (p = 0,038). Para as demais variáveis, não foram detectadas associações significantes. CONCLUSÃO: A correlação positiva encontrada entre ocupação e procedência com custos incorridos e não cobertos por pacientes sugere a criação de programas que viabilizem o pagamento dos custos não cobertos pelo SUS e a descentralização do acesso ao tratamento oncológico podem potencialmente facilitar a aderência do paciente ao tratamento oncológico.

SUMMARY INTRODUCTION: Cancer patients and their caregivers incur costs not covered by the Sistema Único de Saúde – SUS(Unified Health System) during their treatment, with expenses related to transportation, symptomatic medications, food, loss of working days, and others. OBJECTIVE: To evaluate the costs incurred and not covered for cancer patients and their caregivers during cancer treatment at SUS. METHODS: This is a cross-sectional study of 110 cancer patients undergoing chemotherapy, radiotherapy, and 88 caregivers in the last month prior to their inclusion in the study. We correlated costs with clinical and sociodemographic variables such as gender, race, age, marital status, education, occupation, place of birth, origin, monthly income, family income, housing, comorbidities, types of cancer, and staging. RESULTS: We observed that the average cost for study patients was R$ 747.92, which corresponds to 78.4% of the minimum wage, and the average cost for caregivers was R$ 118.86, which is 12.46% of the minimum wage. Among all variables analyzed, the average overall monthly cost for patients was positively correlated with the occupation (p = 0.021) and origin (p = 0.038) variables. For the other variables, no significant associations were detected. CONCLUSION: The positive correlation found between occupation and origin variables with costs incurred and not covered for patients suggests that the creation of programs that enable the payment of costs not covered by SUS and the decentralization of access to cancer treatment could potentially facilitate patients' adherence to cancer treatment.

RESUMO OBJETIVO Relacionar os níveis de ansiedade e depressão com os de espiritualidade dos pacientes oncológicos da região do ABC. MÉTODO Estudo transversal realizado nos ambulatórios de oncologia do Centro Universitário ABC. Para avaliação da espiritualidade, foi aplicado o questionário Religiosidade, espiritualidade e crenças pessoais da Organização Mundial da Saúde (SRPB-WHO). Para avaliar os níveis de depressão e ansiedade foi aplicado a Hospital Anxiety and Depression Scale (Hads). Variáveis qualitativas foram descritas por frequência e porcentagem, as quantitativas por média e desvio padrão ou mediana e intervalo. Relações foram feitas por meio do teste de t ou Wilcoxon-Mann-Whitney e correlações pelo teste de Pearson ou Spearman, a depender da normalidade avaliada pelo teste de Shapiro-Wilk. RESULTADO Foram incluídos 99 pacientes; 68% do sexo feminino, mediana de idade 60 (19 a 81); 24% tiveram níveis altos ou limítrofes para ansiedade e 21% para depressão. Foi observada correlação negativa entre os níveis de depressão e espiritualidade (rho=–0,44, p<0,001) e ansiedade e espiritualidade (rho=–0,232, p=0,02). Não foi observada diferença entre os níveis de ansiedade, depressão ou espiritualidade ao estratificar por escolaridade, renda, etnia ou estado civil. Houve correlação positiva entre os níveis de ansiedade e depressão (cor=0,477, p<0,001). CONCLUSÃO A espiritualidade pode ser uma ferramenta complementar a ser utilizada no tratamento do paciente com câncer.

SUMMARY OBJECTIVE To relate anxiety and depression levels to the spirituality levels of oncology patients in the ABC region. METHODS Cross-sectional study performed at the ABC University Center oncology outpatient clinics. For the evaluation of spirituality, the Religiosity, Spirituality, and Personal Beliefs instrument of the World Health Organization (SRPB-WHO) was applied. To evaluate the levels of depression and anxiety, the Hospital Anxiety and Depression Scale (HADS) was applied. Qualitative variables were described by frequency and percentage, and quantitative variables by mean and standard deviation or median and range. Relationships were established using either the T-test or Wilcoxon-Mann-Whitney test and correlations with Pearson or Spearman tests, depending on the normality assessed by the Shapiro-Wilk test. RESULTS We included 99 patients, 68% female, with a median age of 60 years (19 to 81). A total of 24% had high or borderline levels of anxiety and 21% of depression. There was a negative correlation between levels of depression and spirituality (rho = -0.44, p <0.001), and anxiety and spirituality (rho=-0.232, p=0.02). We found no significant difference between levels of anxiety, depression, or spirituality when stratified by schooling, income, ethnicity, or marital status. There was a positive correlation between levels of anxiety and depression (cor = 0.477, p <0.001). CONCLUSION Spirituality can be a complementary tool in the treatment of patients with cancer.

RESUMO OBJETIVO Avaliar o conhecimento sobre fatores de risco para câncer em pacientes atendidos nos ambulatórios da Faculdade de Medicina do ABC (FMABC). MÉTODOS Estudo transversal e observacional conduzido em 2019. Foi utilizado o questionário Cancer Risk Awarness Survey do American Institute for Cancer Research com 29 fatores de risco para câncer, sendo 14 fatores comprovadamente causadores de câncer e 15 sem consenso ou evidência científica de causalidade com o câncer, mas que são frequentemente citados pela população. Variáveis qualitativas foram descritas por frequência e porcentagem, e variáveis quantitativas por média e desvio padrão ou mediana e intervalo a depender da normalidade, avaliada pelo teste de Shapiro-Wilk. Estudo realizado de acordo com a Declaração de Helsinque para pesquisa e aprovado pelo Comitê de Ética em Pesquisa. RESULTADOS Foram incluídos 191 pacientes. Mediana de idade 54 (20 a 90), 64% do sexo feminino; 35,6% reportaram tabagismo atual ou anterior; 3,1% consumiam mais que cinco doses de álcool por semana; 56% reportaram sedentarismo; 44% tinham pelo menos um caso de câncer em parentes de até segundo grau. A média de acertos na população analisada foi 12,83 ± 3,06. Foi observada correlação positiva fraca entre renda e número de acertos (rho=0,177, p=0,02). Não foram observadas relações entre o número de acertos e nível de escolaridade, idade, sexo, estado civil, cor ou pacientes com história familiar positiva para câncer. CONCLUSÃO O conhecimento sobre fatores de risco para câncer na população do ABC é baixo, o que pode contribuir para a adoção de comportamentos de risco para a doença.

SUMMARY OBJECTIVE To evaluate the knowledge about risk factors for cancer in patients treated at the ABC Medical School (FMABC). METHODS Cross-sectional observational study conducted in 2019. The American Cancer Institute’s Cancer Risk Awareness Survey questionnaire was used with 29 cancer risk factors, 14 of which were proven to cause cancer and 15 without consensus or scientific evidence of causality with cancer but that are often reminded by most of the population. Qualitative variables were described by frequency and percentage, and quantitative variables by mean and standard deviation or median and range depending on normality, assessed by the Shapiro-Wilk test. The study was conducted in accordance with the Helsinki Declaration for Research and approved by the Research Ethics Committee. RESULTS 191 patients were included. Median age 54 (20 to 90), 64% female. 35.6% reported current or previous smoking. 3.1% consumed alcohol more than 5 drinks/week. 56% reported sedentary lifestyle. 44% had at least 1 case of cancer in relatives up to 2nd degree. The average of correct answers in the analyzed population was 12.83 ± 3.06. A weak positive correlation was observed between income and number of cases (rho = 0.177, p = 0.02). No relationship was observed between the number of correct answers and level of education, age, sex, marital status, race or patients with a positive family history for cancer. CONCLUSION The knowledge about risk factors for cancer in the ABC population is low, which may contribute to the adoption of risk behaviors for the disease.

RESUMO Equipes multidisciplinares têm sido cada vez mais empregadas no tratamento de pacientes com câncer. Este estudo buscou avaliar a percepção de médicos, profissionais e pacientes oncológicos internados em relação à sua interação com a equipe multidisciplinar tanto no setor público quanto no privado. Entrevistamos 18 médicos, 63 profissionais da área da saúde e 120 pacientes oncológicos. Em relação ao trabalho da equipe multidisciplinar, a percepção foi positiva entre os pacientes e os médicos (médias iguais a 89,4% e 66,82%, respectivamente), mas maior entre os pacientes (p<0,001), maior entre as pacientes mulheres do que entre os homens (médias de 77,5% e 85,21%, respectivamente, p<0,001), maior entre os idosos do que entre os adultos (médias de 91,98% e 76,01%, respectivamente, p<0,0001), e maior no setor público do que no setor privado (médias de 83,12% e 70,74%, respectivamente, p<0,0001). Os resultados demonstram que apesar da diferença entre grupos, tanto pacientes como membros dos grupos multidisciplinares apresentaram satisfação com o atendimento multidisciplinar, e alguns grupos, como mulheres idosas cuidadas em serviços públicos, poderão especialmente ter um impacto maior de grupo multiprofissionais.

SUMMARY Multidisciplinary teams are increasingly employed to treat cancer patients. This study aimed to evaluate the perception of physicians, other health care professionals, and hospitalized oncological patients regarding the multidisciplinary teams of the public and private sector. In total, 18 doctors were interviewed; 63 health professionals and 120 cancer patients. Satisfaction with the work of the multidisciplinary team was positive among patients and physicians (averages of 89.4% and 66.82% respectively), but higher among patients (p <0.0001), among women rather than men (averages of 77.5% and 85.21% respectively, p < 0.0001), elderly individuals in comparison with adults (averages of 91.98% and 76.01% respectively, p <0.0001), and in the public sector in comparison with the private sector (averages of 83.12 and 70.74 respectively, p < 0.0001). The results demonstrate that despite the difference between groups, patients and members of multidisciplinary groups were satisfied with multidisciplinary care, and some groups, such as elderly women from public services, may especially benefit from multi-professional groups.

RESUMO Objetivo: O câncer de pulmão é um importante problema de saúde pela sua alta incidência e mortalidade. O tratamento da doença metastática melhorou após o conhecimento de vias moleculares tumorais. Contudo, a terapia-alvo está indisponível para muitos pacientes do Sistema Único de Saúde (SUS). Nosso objetivo foi avaliar a relação custo-efetividade de erlotinibe, gefitinibe e afatinibe vs. quimioterapia no tratamento do câncer de pulmão não pequenas células no contexto do SUS. Métodos: Foram desenvolvidos modelos analíticos distintos baseados em dados da literatura. Os desfechos foram apresentados em quality-adjusted life years (QALY, anos de vida ajustados pela qualidade) e incremental cost-effectiveness ratio (ICER, relação custo-efetividade incremental). Todos os custos relacionados ao tratamento e terapias de suporte foram incluídos nos modelos. Resultados: No primeiro modelo, dados de estudos retrospectivos apontaram 2,01 anos de vida salvos e uma média de ganho de QALY de 1,169. O ICER variou entre R$ 48.451,29 (gefitinibe) e R$ 85.559,22 (erlotinibe). No segundo modelo, dados de uma meta-análise evidenciaram −0,01 ano de vida salvos e uma média de ganho de QALY de 0,178. O ICER foi de R$ 27.028,30 (gefitinibe) a R$ 75.203,26 (erlotinibe). Conclusões: Não existe um modelo analítico ideal para o SUS. Contudo, diferentes cenários disponíveis na literatura mostram que a terapia-alvo com o uso dessas drogas é custo-efetiva. A adoção de descontos nos preços dos medicamentos melhorará a relação custo-efetividade do tratamento.

ABSTRACT Objective: Lung cancer is an important health problem due to its high incidence and mortality. The treatment of metastatic disease improved after the molecular pathways of cancer came to be known. However, targeted therapy is unavailable to many patients treated within the Brazilian Sistema Único de Saúde (SUS, Unified Health Care System). Our objective was to assess the cost-effectiveness of erlotinib, gefitinib, and afatinib versus that of chemotherapy for the treatment of non-small cell lung cancer in the context of the SUS. Methods: Different analytical models were developed based on data in the literature. The outcomes were presented in quality-adjusted life-years (QALYs) and incremental cost-effectiveness ratios (ICERs) per QALY gained. All costs related to treatment and supportive therapies were included in the models. Results: In one model, data from retrospective studies showed 2.01 life-years saved and a mean QALY gain of 1.169. The ICER per QALY gained ranged from R$48,451.29 (for gefitinib) to R$85,559.22 (for erlotinib). In another model, data from a meta-analysis showed −0.01 life-years saved and a mean QALY gain of 0.178. The ICER per QALY gained ranged from R$27,028.30 (for gefitinib) to R$75,203.26 (for erlotinib). Conclusions: There is no ideal analytical model for the SUS. However, targeted therapy with EGFR-tyrosine kinase inhibitors has been shown to be cost-effective in various scenarios. The adoption of drug price discounts will improve the cost-effectiveness of treatment.

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.