Brazil is a continental country with socioeconomic inequities, hampering access to essential goods and services, including health care. Brazil's Unified Health System (SUS) is a universal, publicly funded health system, warranting free access to health care, including medicines. Significant advances followed the creation of SUS, the implementation of the National Medicines Policy (PNM) and the National Pharmaceutical Services Policy (PNAF) between 1990 and 2000, such as country-wise organization and management of pharmaceutical services and provision of high-cost medicines, a segment including most medicines for rare diseases. The National Policy for Comprehensive Care for Individuals with Rare Diseases has fostered discussion and action on comprehensive care for individuals with rare conditions. However, insufficient funding, setbacks in provision, shortages and the pressures of innovation, patents and prices of medicines have burdened access initiatives. The paper presents the Brazilian scenario and discusses the main pathways for access to medicines for rare diseases in the country, beginning by a comprehensive description of administrative routes, as marketing authorization and incorporation into the health system, which constitute the more straightforward and norm-adherent paths. However, the alternative pathway, litigation for access, guaranteed by the Brazilian Constitution that upholds the right to health, has consolidated itself in the last 20 years, taking an enormous toll on expenditures and appropriate use of medicines. To overcome these challenges and expand access to drugs for rare diseases Brazil must ensure robust evidence-based use for adequate indications, monitoring and managed entry of new medicines and ethical allocation of resources.

Rare diseases are an important cause of infant mortality, yet their care presents many challenges. Legal claims have enabled access to medicines, but there are concerns about interference in health policy and patient safety. The present study analyzed 912 lawsuits, involving 23 medicines for 18 rare diseases between 2008 and 2022. Most plaintiffs were women and children, aged 0 to 5 years, residing in São Paulo and Minas Gerais. Legal aid was commonly granted, and 36 deaths were reported. The lawsuits originated from the Federal District, with limited use of alternative resolution methods. Private attorneys and prescriptions from private health services predominated. Court decisions were predominantly favorable to the plaintiffs, but only 4.2% of the actions indicated the inclusion of claimants in the Unified Health System (SUS). Despite Brazil's high rate of health litigation, some medicines involved in these lawsuits lacked strong evidence of efficacy and safety. Monitoring medicine use in this context is crucial to support future decisions. It is essential to develop innovative policies and coordination among different sectors to ensure effective and comprehensive care for individuals with rare diseases.

O desempenho dos sistemas de saúde está relacionado à garantia do direito à saúde, um importante componente dos direitos humanos, que inclui o direito à proteção da saúde. A ação regulatória do Estado visa diminuir riscos à saúde, contribuindo para melhorar o desempenho dos sistemas de saúde, especialmente no que tange aos princípios da integralidade e da equidade. Este artigo tem por objetivo discutir a resiliência na perspectiva das agências reguladoras nacionais, especialmente frente às Emergências de Saúde Pública (ESP) e sua contribuição para a resiliência dos sistemas de saúde, tomando como modelo a atuação da agência reguladora brasileira, a Agência Nacional de Vigilância Sanitária (Anvisa). A regulação sanitária, no contexto das ESP, vem aperfeiçoando suas práticas regulatórias e atuando em diversas frentes. As agências reguladoras se utilizaram de instrumentos regulatórios específicos para agilizar o acesso a produtos eficazes e seguros para o enfrentamento de pandemias, para a assistência de qualidade e para a comunicação com a sociedade, e, no caso da Anvisa, também atuou sobre novos objetos, como o acompanhamento do desenvolvimento de respiradores pulmonares. É crucial reconhecer a interdependência entre o aprimoramento da capacidade de resposta e o desempenho para enfrentar as ESP. São exemplos a rápida aprovação de vacinas contra a Covid-19, permitindo acelerar a vacinação, bem como a colaboração das agências reguladoras e, no caso brasileiro, a atuação da Anvisa para reforçar o papel do Sistema Único de Saúde (SUS) e do direito à saúde face à desinformação e ao negacionismo.

Public Health Emergencies (PHE) have had repercussions on health systems on a global scale, and timely access to new health technologies is a challenge for health policy. The national regulatory authorities (NRA) play a key role in the evaluation and regulation of these technologies. The present study aims to analyze the main strategies and regulatory instruments used to deal with the challenges of regulating new technologies necessary for the health system's effective response during a PHE. This research, based on WHO and Brazilian NRA norms and documents, considered dimensions related to strategies for strengthening regulatory activities and regulatory instruments used to accelerate access to technologies, especially during PHEs. International cooperation between the NRA and the WHO were important strategies for strengthening the NRA, with emphasis on the use of reliance, regionalization, accelerated assessments, and work/information sharing, as well as the processes of regulatory harmonization and convergence. In addition to the use of existing regulatory instruments, efforts were also identified in order to implement new ones.

Abstract The article analyzes the cases of health system reprogramming in response to the Zika emergency in the states of Ceará (CE) and Rio de Janeiro (RJ), from 2015 to 2017. The research was anchored in the historical and institutionalist approach and the literature on regionalization and health care networks. It involved analyzing government documents and conducting interviews with key actors mobilized in the states’ response to the epidemic. For each state, the following aspects were primarily explored: context, political-financial situation and health agenda at the time of the epidemic; and the design and implementation of responses (meanings, actors, resources, strategies and repercussions). The reprogramming of the health system in the states of CE and RJ presented different conditions and different meanings of actions, strategies, mobilized actors and developments, with a focus on care initiatives for children with Congenital Zika Virus Syndrome (SCZV). The importance of the regionalized and coordinated network was highlighted, with deconcentration of the offer of specialized services and early stimulation procedures; the coordinating and investing role of the state government; the qualification initiatives of health professionals; of the performance of research institutions in the service, production of knowledge and in the dialogue with affected families.

The performance of health systems is related to ensuring the right to health, an important component of human rights that includes the right to health protection. Regulatory action by the state aims to reduce risks to health and thus help improve health system performance, particularly with respect to the principles of comprehensiveness and equity. The aim of this article is to discuss resilience from the perspective of national regulatory agencies - especially when they are faced with public health emergencies (PHEs) - and their contribution to health system resilience, taking as a model the work of the Brazilian Health Regulatory Agency (ANVISA). In the context of PHEs, health regulators have been improving their practices and working on several fronts. Regulatory agencies have used specific regulatory instruments to speed up access to effective and safe products for responding to pandemics, to ensure the quality of care, and to communicate with the public. ANVISA specifically has brought new areas of action under its purview, such as overseeing the development of mechanical ventilators. It is crucial to recognize the interdependence between improving response capacity and improving performance in dealing with PHEs. Examples include the fast-track approval of COVID-19 vaccines, which allowed more rapid vaccination; cooperation among regulatory agencies; and, in the case of Brazil, work by ANVISA to strengthen the role of the country's Unified Health System (SUS) and the right to health in the face of disinformation and denialism.

Resumo O artigo analisa a resposta federal dos Sistemas Nacionais de Vigilância em Saúde e de Vigilância Sanitária frente à epidemia do Zika vírus no Brasil, de 2015 a 2018, com foco nos contextos político-institucionais e no conteúdo das medidas governamentais desenvolvidas no período. O estudo ancorou-se na abordagem institucionalista histórica, compreendendo análise documental e entrevistas com atores-chave. A resposta se caracterizou pela priorização inicial da emergência sanitária na agenda política do governo federal, reduzindo-se ao longo do período, influenciada pelas crises financeira e política. Verificou-se multiplicidade de atores e instâncias voltadas à contingência, com certa articulação entre os dois sistemas, a partir de experiências pré-emergência próprias. O controle vetorial teve centralidade, valorizando ações intersetoriais e comunitárias, induzidas sobretudo pela Secretaria de Vigilância em Saúde do Ministério da Saúde e complementarmente pela Agência Nacional de Vigilância Sanitária. Observaram-se expressivas limitações alocativas de recursos financeiros novos e mudanças no aparato organizativo de resposta, com efeitos para a continuidade das políticas no pós-emergência, incluindo o desenvolvimento de medicamentos, vacinas e testes. A Emergência em Saúde Pública do Zika Vírus no Brasil foi marcada por limitada institucionalização de aprendizados e estratégias estruturantes, reduzindo oportunidades para a (re)organização das vigilâncias no Sistema Único de Saúde.

Resumo As Emergências em Saúde Pública (ESP) têm repercutido nos sistemas de saúde em escala global. O acesso às novas tecnologias em saúde em tempo oportuno é um desafio para a política de saúde. As autoridades reguladoras nacionais (ARN) têm papel fundamental na avaliação e regulação dessas tecnologias. O estudo objetiva analisar as principais estratégias e instrumentos regulatórios utilizados para lidar com os desafios da regulação de novas tecnologias necessárias à resposta do sistema de saúde durante as ESP. Trata-se de uma pesquisa normativa e documental, tendo como fonte a OMS e a ARN brasileira. Foram consideradas as dimensões relacionadas às estratégias para o fortalecimento das atividades regulatórias e os instrumentos regulatórios utilizados para acelerar o acesso às tecnologias, especialmente durante as ESP. A cooperação e a colaboração internacional entre as ARN e com a OMS foram importantes estratégias para o fortalecimento das ARN, com destaque para o uso de confiança, regionalização, avaliações aceleradas e compartilhamento de trabalho/informações, bem como os processos de harmonização e convergência regulatória. Identificou-se, além da utilização de instrumentos regulatórios já existentes, esforços na implementação de novos, com destaque para Autorização de Uso Emergencial.

Abstract The article analyzes the federal response of the National Public Health Surveillance and Brazilian Health Regulatory Systems to the Zika virus epidemic in Brazil, from 2015 to 2018, focusing on the political-institutional contexts and the content of government measures developed during the period. The study was anchored in the historical institutionalist approach, comprising documentary analysis and interviews with key actors. The response was characterized by the initial prioritization of the health emergency on the federal government's political agenda, which was reduced over the period, influenced by the financial and political crises. There was a multiplicity of actors and instances focused on contingency, with a certain articulation between the two systems, based on their own pre-emergency experiences. Vector control had centrality, valuing intersectoral and community actions, induced mainly by the Health Surveillance Secretariat of the Ministry of Health and additionally by the National Health Surveillance Agency. Significant allocation limitations of new financial resources and changes in the organizational response apparatus were observed, with effects on the continuity of post-emergency policies, including the development of medicines, vaccines and tests. The Zika Virus Public Health Emergency in Brazil was marked by limited institutionalization of learning and structuring strategies, reducing opportunities for the (re)organization of surveillance in the Unified Health System.

The performance of health systems is related to ensuring the right to health, an important component of human rights that includes the right to health protection. Regulatory action by the state aims to reduce risks to health and thus help improve health system performance, particularly with respect to the principles of comprehensiveness and equity. The aim of this article is to discuss resilience from the perspective of national regulatory agencies - especially when they are faced with public health emergencies (PHEs) - and their contribution to health system resilience, taking as a model the work of the Brazilian Health Regulatory Agency (ANVISA). In the context of PHEs, health regulators have been improving their practices and working on several fronts. Regulatory agencies have used specific regulatory instruments to speed up access to effective and safe products for responding to pandemics, to ensure the quality of care, and to communicate with the public. ANVISA specifically has brought new areas of action under its purview, such as overseeing the development of mechanical ventilators. It is crucial to recognize the interdependence between improving response capacity and improving performance in dealing with PHEs. Examples include the fast-track approval of COVID-19 vaccines, which allowed more rapid vaccination; cooperation among regulatory agencies; and, in the case of Brazil, work by ANVISA to strengthen the role of the country's Unified Health System (SUS) and the right to health in the face of disinformation and denialism.

Resumo O artigo analisa os casos de reprogramação do sistema de saúde em resposta à emergência de zika dos estados do Ceará (CE) e do Rio de Janeiro (RJ), no período de 2015 a 2017. A pesquisa ancorou-se na abordagem institucionalista histórica e na literatura sobre regionalização e redes de atenção à saúde. Envolveu a análise de documentos governamentais e a realização de entrevistas com atores-chave mobilizados na resposta dos estados à epidemia. Para cada estado, foram explorados, prioritariamente, os seguintes aspectos: contexto, conjuntura político-financeira e agenda da saúde no momento da epidemia; e o desenho e implementação das respostas (sentidos, atores, recursos, estratégias e repercussões). A reprogramação do sistema de saúde nos estados do CE e RJ apresentou distintas condições e diferentes sentidos das ações, estratégias, atores mobilizados e desdobramentos, com foco nas iniciativas de atenção às crianças com síndrome congênita do Zika vírus (SCZV). Evidenciou-se a importância: da rede regionalizada e coordenada, com desconcentração da oferta de serviços especializados e dos procedimentos de estimulação precoce; do papel coordenador e investidor do governo estadual; das iniciativas de qualificação dos profissionais de saúde; da atuação de instituições de pesquisa no atendimento, produção de conhecimento e no diálogo com famílias afetadas.

Context: Breast cancer is the most common cancer, except for non-melanoma skin cancer, among women in Brazil and worldwide. Breast cancer treatment involves surgery, radiotherapy and chemotherapy, which is used in 70% of patients. This study analyzes the utilization of antineoplastic agents among women undergoing their first round of chemotherapy in Brazil's public health system (SUS) in the state of Rio de Janeiro. Methods: Data from the SUS Outpatient Information System's authorizations for high-complexity outpatient procedures (APACs) billed between January 2013 and December 2019 were extracted, and three datasets were created: all type 1 and type 2 APACs (including all chemotherapy procedures performed); all type 1 APACs; and first type 1 APACs (containing data only for the first round of breast cancer chemotherapy). Names of antineoplastic agents were standardized to enable the subsequent classification of therapy regimens, mitigating limitations related to data quality. Absolute and relative frequencies were used to describe sociodemographic, clinical and treatment characteristics, therapy regimen and supportive drugs. Results: We analyzed 23,232 records of women undergoing their first round of chemotherapy. There was a progressive increase in the number of procedures over time. Women were predominantly white, lived in the capital and close to the treatment center. Most had stage 3 cancer at diagnosis (50.51%) and a significant proportion had regional lymph node invasion (37.9%). The most commonly used chemotherapy regimens were TAC (docetaxel, doxorubicine, cyclophosphamide) (21.05%) and and cyclophosphamide (17.71%), followed by tamoxifen (15.65%) and anastrozole (12.94%). Supportive drugs were prescribed to 386 women and zoledronic acid was predominant (59.58%). Conclusion: The findings point to important bottlenecks and possible inequities in access to treatment and medicine utilization for breast cancer patients in Brazil. Efforts to improve breast cancer treatment and prevention should not only focus on interventions at the individual level but address the disease as a public health problem. The study focused on women undergoing their first round of treatment, providing valuable insight into patient and treatment characteristics to inform policy decisions.

Background Breast cancer is the most common cancer among women in Brazil and the country’s public health care system is the main care provider. Timely treatment can increase the chance of cure, prevent metastasis and improve quality of life. Effective public procurement of antineoplastic agents can therefore improve access to drug therapy. This study investigates patterns in the procurement of selected antineoplastic agents used for treating breast cancer by public bodies and avoidable expenditure on these drugs between January 2013 and December 2019. Methods We selected antineoplastic agents used for adjuvant or preoperative chemotherapy listed in the 2018 Breast Cancer Diagnosis and Treatment Guidelines and included in category L of the WHO Anatomical Therapeutic Chemical classification system. We analyzed regular purchases of antineoplastic agents registered in the Integrated General Services Administration System (SIASG), considering purchased quantity, unit price, date of purchase and procuring entity. Prices were inflation-adjusted to July 2019 based on the National Consumer Price Index. Results A total of 10 antineoplastic agents were selected. Trastuzumab and tamoxifen accounted for the largest share of total spending and largest volume of purchases, respectively. The Ministry of Education was the largest purchaser in volume terms of all the drugs studied, except trastuzumab 440 mg, where the category “Other Institutions” accounted for most purchases, and vinorelbine 20 mg, where the Ministry of Health made most purchases. The category “Other Institutions” accounted for the largest share of total spending. Total avoidable expenditure was R$99,130,645. Prices paid for medicines and avoidable expenditure were highest in the Ministry of Defense. Conclusions The differences observed in the performance of different categories of buyers as to amounts purchased and prices practiced for antineoplastic agents could be reduced by employing strategies to expand the centralization of purchases, resulting in expanded access to breast cancer medicines in the public sector.

Population aging brings challenges to the health system. Increased degenerative joint diseases and occurrence of falls may require hip arthroplasties. The objective of this study was to evaluate factors associated with hospital deaths due to hip arthroplasties in the Brazilian Unified National Health System (SUS), in the State of Rio de Janeiro, Brazil. A cross-sectional study was carried out using the Brazilian Hospital Information System of the SUS, considering hospitalizations and deaths from hip arthroplasties that occurred between 2016 to 2018. The analyses considered hospitalizations by type of care (elective, urgency and accidents or injury and poisoning). Binary logistic regression was performed to obtain a ratio for the chance of death. Mortality increased according to the severity of hospitalization. Most of the hospitalizations with death were due to femoral fracture, demanded intensive care, had mean length of stay of 21.5 days, and involved women aged 80 years and over, of the white race/color, who had their procedure performed in municipal units qualified in orthopedics and traumatology of high complexity and classified as general II, located in the patients' region of residence. The chance of death was significant for the age of the patient, use of intensive care unit (ICU), and length of hospital stay. We expect an increase in the demand for hip arthroplasties and physical rehabilitation due to population aging. Health teams must be prepared to deal with an ageing population. Knowing the factors that increase the risk of death favors care planning and management by hospital staff, including in the reduction of hospital stay, which is so impactful on the functional condition of the elderly.

This study analyzed the court proceedings, health characteristics of the patients, and plaintiffs’ characteristics in lawsuits for hip replacement surgery from public health services in the state of Rio de Janeiro, Brazil. The data employed was from the Technical Support Center for the Judiciary of the Rio de Janeiro Court of Justice (NAT-JUS) database, from January 2016 to December 2018. Data were analyzed according to tiers in the court system. There were 89 lawsuits filed for hip replacement surgery and most of them were tried in the Court of Justice of the State of Rio de Janeiro (TJRJ), with joint plaintiffs, represented by the Public Defender’s Office. They had advance relief granted, and were ruled in favor of the plaintiffs, with no appeals filed. More than half of the cases were referred to NAT-JUS. The osteoarthritis group waited approximately seven months, while the emergency conditions group waited nearly two months from the date of filing to obtain the procedure. The lawsuits for hip replacement surgery showed similarities and differences in relation to other claims, object of others studies, which means that the strategies for their qualification. The study found that access to hip replacement surgery related to lawsuits in the Brazilian Unified National Health System (SUS) in the State of Rio de Janeiro is jeopardized, with the claims considered as an indicator of unmet needs

Executive Secretariat of the Drug Market Regulation Chamber, Brazilian Health Regulatory Agency, Brasília, Distrito Federal, Brazil Department of Health Policy, The London School of Economics and Political Science, London, UK Population Medicine, Harvard Medical School, Boston, Massachusetts, USA National School of Public Health Sergio Arouca, Department of Health Administration and Planning, Oswaldo Cruz Foundation, Rio de Janeiro, Rio de Janeiro, Brazil

Em momentos de Emergência em Saúde Pública, o uso de tecnologias diagnósticas se dá sob condições especiais, visto a necessidade do rápido acesso. Na Emergência em Saúde Pública do vírus zika, os testes comerciais foram importantes para identificar casos e responder seus desafios. No Brasil, a comercialização de testes diagnósticos ocorre após a concessão do registro pela Agência Nacional de Vigilância Sanitária. O objetivo deste artigo foi analisar o registro dos testes comerciais para o diagnóstico da infecção pelo vírus zika no período de 2016 a 2019 no Brasil. O estudo exploratório-descritivo utilizou-se do banco de dados e de documentos do site da Agência Nacional de Vigilância Sanitária, bem como de documentos disponíveis nas páginas eletrônicas dos fabricantes detentores de registro. Quatro dimensões foram consideradas para a análise dos dados coletados: registro, testes, fabricantes e detentores de registro. Foram registrados 68 testes, sendo 91% de empresas privadas; 82% específicos para o vírus zika; 54% importados; 78% sorológicos; e 37% testes rápidos. Durante a Emergência em Saúde Pública do vírus zika, a Agência Nacional de Vigilância Sanitária priorizou o registro dos testes, sem fazer uso da Autorização de Uso Emergencial, dispositivo preconizado internacionalmente. Em 2016, o tempo médio para análise de registro foi menor e o número de exigências, maior. Não se identificou informação sobre o desempenho em 50% dos testes. A recuperação e a geração de novas evidências, durante a utilização, são os grandes desafios e mostra-se importante disponibilizar informações sobre o desempenho dos testes.

O envelhecimento populacional traz desafios ao sistema de saúde. O aumento das doenças degenerativas articulares e a ocorrência de quedas podem demandar a realização de artroplastia de quadril. Objetivou-se avaliar os fatores associados a óbitos hospitalares por artroplastia de quadril no Sistema Único de Saúde (SUS), no Estado do Rio de Janeiro, Brasil. Realizou-se um estudo transversal utilizando o Sistema de Informações Hospitalares do SUS, considerando internações e óbitos por artroplastias de quadril ocorridos entre 2016-2018. As análises consideraram as internações por caráter de atendimento (eletivo, urgência e acidentes ou lesão e envenenamento). Realizou-se regressão logística binária para obter a razão de chance de óbito. A mortalidade aumentou conforme a gravidade da internação. A maioria das internações com óbito foi por fratura de fêmur, demandando cuidados intensivos, tempo médio de permanência de 21,5 dias, envolveu mulheres com 80 anos e mais, da raça/cor branca, que realizaram procedimento em unidades habilitadas em alta complexidade em ortopedia e traumatologia, municipais e classificadas como geral II, localizadas na região de moradia do usuário. A chance de óbito se mostrou significativa para a idade do usuário, uso de unidade de terapia intensiva (UTI) e permanência hospitalar. Espera-se aumento da demanda por artroplastias de quadril e reabilitação física com o envelhecimento populacional. As equipes de saúde devem estar preparadas para lidar com uma população envelhecida. Conhecer os fatores que incrementem o risco de óbito favorece o planejamento e o manejo do cuidado pela equipe hospitalar, inclusive no sentido de diminuir a permanência hospitalar, tão impactante na condição funcional de pessoas idosas.

In times of Public Health Emergencies, the use of diagnostic technologies occurs under special conditions, given the need for quick access. In the Public Health Emergencies of Zika virus, commercial tests were important to identify cases and respond to their challenges. In Brazil, commercialization occurs after registration is granted by the Brazilian Health Regulatory Agency. The objective was to analyze the registration of commercial tests for the diagnosis of Zika virus infection in Brazil from 2016 to 2019. This exploratory-descriptive study used the database of the Brazilian Health Regulatory Agency, and documents from the pages of the manufacturers, registration holders and the agency. Four dimensions were considered: registry; tests; manufacturers; and registration holders. Sixty-eight tests were registered, 91% of which were from private companies, 82% specific to Zika virus, 54% imported, 78% serological and 37% rapid tests. During the Public Health Emergencies of Zika virus, the Brazilian Health Regulatory Agency prioritized the registration of tests, not using the Emergency Use Authorization, an internationally recommended instrument. The average time for analyzing the registration process was shorter and the number of requirements was higher in 2016. No information on performance was identified in 50% of the tests. The recovery and generation of new evidence, during use, is a major challenge, and it is important to provide information on tests performance.