Com os crescentes gastos em saúde, muitos países estão adotando a ferramenta de Avaliação de Tecnologias em Saúde (ATS) para auxiliar as suas decisões sobre introdução de novas tecnologias nos sistemas de saúde. No Brasil, a Comissão Nacional de Incorporação de Tecnologias no SUS (CONITEC) é responsável por assessorar o Ministério da Saúde em suas atribuições relacionadas à incorporação, à exclusão ou à alteração de tecnologias em saúde pelo SUS. Com o objetivo de aprimorar o trabalho da CONITEC, foi realizada uma pesquisa na base de dados MEDLINE, com a finalidade de identificar os critérios utilizados em outros países no processo de ATS para embasar suas decisões de adoção de medicamentos, dispositivos médicos, testes diagnósticos e equipamentos médicos. Foram selecionados 9 artigos e foram avaliadas as experiências de ATS de 16 países que, em conjunto, reportaram o uso de aproximadamente 21 critérios relacionados ao impacto da doença, à tecnologia, às questões econômicas, à qualidade de evidência e às questões de equidade, éticas e sociais e organizacionais. Em seguida, 12 relatórios elaborados pela CONITEC foram avaliados quanto à presença ou não de informações relacionadas a esses critérios. Da mesma forma que nos países avaliados, as informações sobre efetividade, segurança e custo-efetividade são consideradas extremamente relevantes no processo de incorporação de tecnologias pela CONITEC. A seleção apropriada de critérios e a sistematização da ATS podem conferir maior objetividade, transparência e legitimidade ao processo de incorporação de tecnologias, contribuindo para a tomada de decisões mais consistentes.

Introduction:Eculizumab is a monoclonal antibody indicated for the treatment of patients with paroxysmal nocturnal hemoglobinuria (PNH) or with atypical hemolytic uremic syndrome (aHUS). In Brazil in recent years eculizumab was the most expensive drug requested through court orders, obliging public health managers to import it from the USA. From 2012 to 2016, approximately BRL 424 million (USD 112 million) was spent on eculizumab. The purpose of this study was to assess the regulatory situation and the scientific evidence on the safety and efficacy of eculizumab.Methods:A literature search was conducted in PubMed, The Cochrane Library, and the Centre for Reviews and Dissemination databases on September 2017. The websites of regulatory agencies were also searched.Results:In 2007, the use of eculizumab was approved by the United States Food and Drug Administration and the European Medicines Agency. In Brazil, despite the provision of eculizumab through judicial proceedings since 2009, the manufacturer of eculizumab only requested a licence for it in 2017, after several meetings with the government when the company agreed to provide the drug at approximately half the price of the imported product. The efficacy of eculizumab in PNH patients was assessed in one randomized, placebo controlled study, one single arm study, and one long-term extension study. The drug reduced hemolysis and the need for transfusion, although the studies had methodological problems. The efficacy of eculizumab in the treatment of aHUS was assessed in four prospective, controlled open-label studies, two long-term extension studies, and one retrospective study. Eculizumab normalized platelet counts and reduced the need for plasmapheresis, although the studies had no control group. Eculizumab was well tolerated, with no meningococcal infections occurring after patients were immunized.Conclusions:Some companies have no interest in licensing their products in Brazil because their provision by judicial proceedings is more lucrative. This situation promotes litigation and irrational prescription of drugs, and also obligates the Brazilian government to import expensive health products.

Introduction:The National Committee for Health Technology Incorporation (CONITEC) has a structured process for the incorporation, disinvestment, or alteration of different health technologies in the Brazilian public health system and provides technical support for the decision-making process. Since its creation, CONITEC has received several submissions for the incorporation of medicines and the update of clinical practice guidelines for multiple sclerosis (MS). Nowadays, more than twelve different therapies are currently available to treat MS and the Brazilian clinical practice guideline, which was last updated in 2015, offers six medicines to treat MS that are divided into first, second, and third line treatments. The purpose of this study was to describe CONITEC's assessments of applications for incorporation, disinvestment, or alteration of medicines for MS.Methods:A case study method was used to evaluate information, retrieved from CONITEC's database, about the health technology reports developed by CONITEC's Executive Secretariat in response to applications received in the period from 2012 to 2017.Results:Ten technical reports on health technologies for MS were produced by CONITEC during the study period. This number represented four percent of the external submissions for incorporation of technologies for several clinical conditions in the public health system. Six medicines were evaluated. The highest number of submissions were for incorporation (n = 6), followed by alteration of treatment lines (n = 3), and disinvestment (n = 1); fifty percent of the submissions were not recommended. The main reasons for rejection were low or unproven efficacy, high budget impact, and inadequacy of the proposal based on the evidence presented. CONITEC's favorable recommendations caused a profound change in the current clinical practice guideline and had a significant impact on the health system.Conclusions:MS is considered a rare disease in Brazil, but there is significant pressure from society to provide better treatment options that will impact the MS scenario in the health system. The recent CONITEC assessments have led to a revolution in the treatment of MS in Brazil, which is now in the process of being updated.