Malignant melanoma in Portuguese adult population: a scoping review of the real-world evidence.

PURPOSE: Malignant melanoma is an aggressive cancer, and there is a notable dearth on epidemiology, clinical and treatment characterization within the Portuguese population. We performed a scoping review to identify real-world evidence studies focused in Portuguese adult patients with malignant melanoma. METHODS: A comprehensive search was conducted. After screening, we described the studies by design, sample size, geographics, setting, population, and outcomes reported. RESULTS: The search yielded 54 studies, mainly retrospective (79.6%). The population assessed was heterogeneous varying from patients with melanoma in general to specific types of melanoma, or even more restricted to patients with specific conditions. The evidence found was mostly concerning clinical outcomes (n=46), patients' clinical profile (n=44) and demographic characterization (n=48). Treatment information was described in 30 studies whereas only 18 reported epidemiological parameters. Studies were mainly performed by the major oncology centers in Lisbon, Oporto and Coimbra, and only two evaluated the entire Portuguese population. To allow comparability, only studies including patients with cutaneous malignant melanoma were considered (13 of the 54) for outcomes evaluation analysis. Median OS varied from 18 to 36 months, assessed after melanoma treatment. Incidence was the most reported epidemiological parameter, confirming the increasing number of cutaneous malignant melanoma patients over the years. Only one study reported prevalence and four reported mortality rates. CONCLUSIONS: The evidence found confirms the lack of information about malignant melanoma in Portugal, highlighting the need of real-world studies to assess melanoma prevalence and incidence rates, current treatment approaches, and clinical characterization of these patients.

[The Glivec® case: the first example of a global debate on the drug patent system]. / El caso Glivec®: primer ejemplo de debate global en torno al sistema de patentes de medicamentos.

OBJECTIVE: To describe the sequence of events involving the Glivec® case in India and to analyze the opinions generated in distinct settings. METHOD: We performed a systematic search for articles concerning the imatinib (Glivec®) patent in India. We selected those sources that described the events, decisions of the authorities involved, and press and scientific opinions. Dates and arguments presented by the involved parties were clearly identified. RESULTS: Of 886 documents initially obtained, we selected 40 documents published between 2003 and 2013. Most of them were press news and commentaries. The process lasted 7 years, starting in 2006 when the Indian Patent Office rejected the patent application filed by Novartis. It ended in 2013 when the Indian Supreme Court upheld this decision. It was argued that the Indian Patent Law would facilitate access to medicines in the Third World and the final decision has received support by the general population. Although the court's final decision has been supported by several institutions, an objective analysis should also take into account the arguments of the pharmaceutical companies and other entities. CONCLUSION: The Glivec® case gave rise to an intense debate on the appropriateness of international standards on patents, their applicability and how they should be adopted in each country. This case, as well as other cases, should serve to stimulate reflection on the international patent system and to achieve scenarios in which the health of the poorest populations is protected but also balanced against intellectual property protection and innovation.

[Incremental cost evaluation study of INR self-monitoring versus laboratorial control or monitoring in health care centres, in Portugal]. / Estudo de avaliação do custo incremental da auto-monitorização de INR versus controlo laboratorial ou monitorização no centro de saúde, em Portugal.

UNLABELLED: Oral anticoagulant therapy with vitamin K blockers has been increasing in the last few years and its effectiveness is directly related to maintaining INR values within the target range. The objective of this study is to evaluate the cost-effectiveness of INR auto-monitoring versus monitoring in a laboratory or a health care centre, in Portugal. METHODOLOGY: The study was conducted from a social perspective and considered only the direct costs. A Markov model was developed that considered the evolution of a hypothetical patient cohort, in annual cycles, with a time horizon of 5 years. For each patient, prothrombin time monitoring was predicted in three different ways: auto-monitoring, laboratory monitoring, and monitoring in a health care centre. The parameters analyzed were life-years gained and QALYs. RESULTS: The auto-monitoring option had a cost-effectiveness per QALY of 542euro/QALY (4.71 QALYs and 4.74 life-years). Health centre monitoring showed a costeffectiveness of 526euro/QALY (4.66 QALYs and 4.70 life-years) and the laboratory option had a cost-effectiveness of 704euro/QALY (4.64 QALYs and 4.68 life-years). CONCLUSIONS: Results showed that auto-monitoring is the option that presents more benefits. Results for both auto-monitoring and monitoring in a health care centre are better than monitoring in a laboratory.

El caso Glivec(R): primer ejemplo de debate global en torno al sistema de patentes de medicamentos / The Glivec(R) case: the first example of a global debate on the drug patent system

Objetivo. Describir la secuencia de hechos acontecidos respecto al caso Glivec® en la India y analizar las opiniones generadas en diferentes ámbitos. Método. Búsqueda sistemática de artículos sobre la patente de imatinib (Glivec®) en la India. Se seleccionaron los que describían los acontecimientos, las decisiones de las instancias implicadas y las opiniones en la prensa general y científica, con identificación de las fechas y de los argumentos de las partes implicadas. Resultados. De los 886 documentos obtenidos se seleccionaron 40, publicados entre 2003 y 2013, mayoritariamente de tipo periodístico y comentarios. El caso duró 7 años, desde el rechazo de la patente presentada por Novartis ante la oficina de patentes de la India en 2006 hasta la ratificación de esta decisión por el tribunal supremo en 2013. Entre las controversias surgidas destacaron los argumentos en defensa de la ley de patentes India, promulgada para favorecer el acceso a los medicamentos en los países del Tercer Mundo. El fallo judicial ha recibido el apoyo de diversas instituciones, si bien un análisis objetivo implica considerar también los argumentos presentados por las empresas farmacéuticas y otras instituciones. Conclusión. El caso Glivec® ha planteado el debate sobre la adecuación de las normas internacionales, su aplicabilidad en el entorno económico y su adaptación en cada país. Este caso, y otros, deberían contribuir a la reflexión sobre el sistema internacional de patentes a fin de preservar la salud de la población más necesitada de una atención sanitaria adecuada en equilibrio con la protección de la propiedad intelectual y la innovación (AU)

Objective. To describe the sequence of events involving the Glivec® case in India and to analyze the opinions generated in distinct settings. Method. We performed a systematic search for articles concerning the imatinib (Glivec®) patent in India. We selected those sources that described the events, decisions of the authorities involved, and press and scientific opinions. Dates and arguments presented by the involved parties were clearly identified. Results. Of 886 documents initially obtained, we selected 40 documents published between 2003 and 2013. Most of them were press news and commentaries. The process lasted 7 years, starting in 2006 when the Indian Patent Office rejected the patent application filed by Novartis. It ended in 2013 when the Indian Supreme Court upheld this decision. It was argued that the Indian Patent Law would facilitate access to medicines in the Third World and the final decision has received support by the general population. Although the court's final decision has been supported by several institutions, an objective analysis should also take into account the arguments of the pharmaceutical companies and other entities. Conclusion. The Glivec® case gave rise to an intense debate on the appropriateness of international standards on patents, their applicability and how they should be adopted in each country. This case, as well as other cases, should serve to stimulate reflection on the international patent system and to achieve scenarios in which the health of the poorest populations is protected but also balanced against intellectual property protection and innovation (AU)

[Characterization of oncological disease: racional, objectives and methodology - perfil study]. / Perfil da doença oncológica em Portugal racional, objectivos e metodologia - estudo perfil.

Oncologic disease is one of the main causes of death in Portugal, as well as a high morbidity rate. Over the past few years, these diseases have been targeted with several strategies which aims at optimizing the use of available therapeutic and diagnostic options. Nevertheless, the amount of quantitative information available regarding the disease, patient profile, and treatment and monitoring practices is very low. In order to optimise the implementation of health policies specifically directed at oncologic diseases reliable and up-to-date information is needed, permitting optimisation and balancing of costs and benefits. The PERFIL national epidemiologic study is a multi-centre, retrospective cohort study which aims at evaluating the clinical practice regarding treatment of 6 neoplasias -colorectal, gastric, breast, prostate, lung, and lymphoma, in Portugal, between 2003 and 2007. This study is based upon data collection from the clinical files of patients being followed in the participating centres from 2003 to 2007. Qualitative evaluation (diagnosis and stage) was collected from the clinical records of all patients seen in each participating centre on a given month in each year from 2003 to 2007. The characterization of both patient profile and treatment and monitoring patterns will be based on a far-reaching analysis of 10,000 of those files, selected in a random and stratified sampling. This article describes the PERFIL study rationale, its objectives, and methodology.

[Guidelines for prevention of febrile neutropenia]. / Orientações para a prevenção da neutropenia febril em doentes submetidos a quimioterapia.

Neutropenia and febrile neutropenia are common consequences of some cytotoxic chemotherapy regimens. This situation leads to modifications of the therapeutic regimen, conducting to either dose reduction or cycle delays. Granulocyte colony stimulating factors are commonly used to minimize chemotherapy cytotoxic effect on the granulocytic series. The objective of this study is to assess the available evidence in what concerns the efficacy and safety of granulocyte colony stimulating factors, in several settings of their use. An extensive bibliographic review was performed, including clinical trials, observational studies, systematic reviews, and international guidelines for neutropenia prophylaxis, which aims to establish recommendations on their use, in adequacy to the National reality.