O fim da extensão das patentes e as Parcerias para Desenvolvimento Produtivo: reflexos no acesso a medicamentos no Brasil / The end of patent extensions and the Productive Development Partnerships: effects on access to medicines in Brazil

Resumo A covid-19 jogou luz sobre o impacto negativo da propriedade intelectual na saúde e deu nova relevância à Ação Direta de Inconstitucionalidade 5529/DF, que, acatada pelo Supremo Tribunal Federal em 2021, culminou na extinção da extensão automática de patentes no Brasil. Este estudo busca analisar o efeito do julgamento histórico da ADI 5529/DF sobre pedidos de patente e as patentes de interesse das Parcerias para Desenvolvimento Produtivo (PDP). Trata-se de um estudo com base em uma pesquisa documental de análise do andamento, até 31 de dezembro de 2020, de 90 pedidos de patente relacionados a 15 medicamentos objetos de PDP. Nos sites do Instituto Nacional de Propriedade Industrial, do Ministério da Saúde, da Anvisa e da Câmara de Regulação do Mercado de Medicamentos, foram pesquisadas variáveis para comparar o cenário patentário dos medicamentos com o das PDP. De 88 pedidos válidos, 28 patentes foram concedidas, das quais dezessete foram estendidas para mais de vinte anos (média de 24 anos e nove meses). A decisão do STF resultou em mais de 68 anos de monopólio perdidos, potencialmente desanuviando alternativas para a produção de genéricos no país. Neste momento de retomada das PDP, estratégias para a superação de barreiras patentárias deveriam ser incorporadas à política.

Abstract The COVID-19 pandemic has shed light on the negative impact of intellectual property on health and has given new relevance to the Direct Action of Unconstitutionality 5529/DF, which was ruled by the Supreme Court in 2021, resulting in the extinction of automatic patent extensions in Brazil. This documentary case study analyzes the effects of the judicial decision on patent applications and patents of interest for Productive Development Partnerships (PDP), investigating the progress of 90 patent applications related to 15 PDPs drugs of interest until Decembre 31, 2020. Variables for comparing the drug patent scenario with that of the PDPs were researched on the websites of the National Institute of Industrial Property, the Ministry of Health, ANVISA, and the Brazilian Medicines Market Regulation Chamber. Of 88 valid applications, 28 patents were granted, 17 of which had been extended to more than 20 years (24 years and 09 months average). The court decision resulted in a loss of over 68 years of monopoly, potentially opening alternatives for generic production. This resumption of the PDP policy should incorporate strategies to overcome patent barriers.

Sedentary behavior, abdominal obesity and healthcare costs in Brazilian adults with cardiovascular diseases: a cross-sectional study

ABSTRACT BACKGROUND: Research on the economic burden of sedentary behavior and abdominal obesity on health expenses associated with cardiovascular diseases is scarce. OBJECTIVE: The objective of this study was to verify whether sedentary behavior, isolated and combined with abdominal obesity, influences the medication expenditure among adults with cardiovascular diseases. DESIGN AND SETTING: This cross-sectional study was conducted in the city of President Prudente, State of São Paulo, Brazil in 2018. METHODS: The study included adults with cardiovascular diseases, aged 30-65 years, who were treated by the Brazilian National Health Services. Sedentary behavior was assessed using a questionnaire. Abdominal obesity was defined by waist circumference. Medication expenditures were verified using the medical records of each patient. RESULTS: The study included a total of 307 adults. Individuals classified in the group with risk factor obesity combined (median [IQ] USD$ 29.39 [45.77]) or isolated (median [IQ] USD$ 27.17 [59.76]) to sedentary behavior had higher medication expenditures than those belonging to the non-obese with low sedentary behavior group (median [IQ] USD$ 13.51 [31.42]) (P = 0.01). The group with combined obesity and sedentary behavior was 2.4 (95%CI = 1.00; 5.79) times more likely to be hypertensive. CONCLUSION: Abdominal obesity was a determining factor for medication expenses, regardless of sedentary behavior, among adults with cardiovascular diseases.

Catadoras de materiais recicláveis: estratégias de resistência e medicamentos durante a pandemia de covid-19 / Women collectors of recyclables: resistance strategies and medicines during the COVID-19 pandemic

Resumo A catação de materiais recicláveis é uma ocupação com crescente número de trabalhadores, que encontram nela sustento diante das desigualdades socioeconômicas e do desemprego. Este artigo descreve as estratégias de resistência de mulheres catadoras que fazem parte de uma associação no Nordeste brasileiro, além da forma como elas lidaram com problemas de saúde durante a pandemia de covid-19, especialmente no que se refere aos medicamentos. Utilizamos uma abordagem quantitativa e uma etnográfica, coletando dados sociodemográficos de 13 participantes e dos medicamentos presentes em suas casas, e também realizando observação participante e entrevistas aprofundadas. Em meio à crise sanitária, foram mencionados problemas como o desemprego, as relações de gênero, a violência e os estigmas do trabalho. Se por um lado a pandemia vulnerabilizou ainda mais alguns segmentos populacionais, por outro potencializou estratégias coletivas de enfrentamento. As catadoras e a associação se organizaram para obter insumos e melhorias tanto na sede quanto no bairro. Identificamos 58 unidades de medicamentos, prescritos e obtidos principalmente na Unidade Básica de Saúde. Desses, 27,6% tinham ação sobre o sistema nervoso (analgésicos, psicolépticos e psicoanalépticos) e 17,2% sobre o sistema cardiovascular e sobre o trato alimentar e metabolismo. As catadoras desenvolveram práticas de autoatenção com os medicamentos, destacando o papel central deles no enfrentamento dos problemas de saúde.

Abstract The collection of recyclable materials is an occupation with an increasing number of workers, who find in it a livelihood in a context of socioeconomic inequalities and unemployment. This article describes resistance strategies of women collectors who are part of an association in Northeast Brazil, in addition to describing how they managed health problems during the COVID-19 pandemic, especially regarding medicines. A quantitative and an ethnographic approach were used by collecting data on sociodemographic and medicines present in the homes of 13 participants and conducting participant observation and in-depth interviews. Amid the health crisis, the participants mentioned problems such as unemployment, gender relations, violence, and work stigmas. If, on the one hand, the pandemic has further increased vulnerability for certain population segments, on the other hand, it has enhanced collective resistance strategies. The collectors of the association organized to obtain various materials and benefits both for their headquarters and their neighborhood. We identified 58 units of medicines, prescribed and obtained mainly at the Basic Health Unit. Of these, 27.6% had action on the nervous system (analgesics, psycholeptics, and psychoanaleptics) and 17.2% on the cardiovascular system and on the alimentary tract and metabolism. The collectors developed self-attention practices with the medicines highlighting their central role in facing health problems.

Methodology for the Development of Clinical Practice Guidelines for Chinese Patent Medicine （Part 8）： Quality Evaluation with AGREE Ⅱ and RIGHT / 中医杂志

ObjectiveTo evaluate the methodological and reporting quality of clinical practice guidelines for Chinese patent medicine （CPM） with internationally recognized tools the appraisal of guidelines for research and evaluation （AGEREE） Ⅱ and reporting items for practice guidelines in healthcare （RIGHT）， thereby providing refe-rence for the clinical application and future development of CPM guidelines. MethodsDatabases including CNKI， VIP， Wanfang and Sinomed were searched for CPM guidelines， as well as medlive.cn， websites of China Association of Chinese Medicine and Chinese Medical Association， and reference lists of the included papers. The quality of the guidelines was evaluated using the AGREE Ⅱand RIGHT tools， and consistency tests were performed using Interclass Correlation Coefficient， and descriptive analysis and chi-square test were used to analyze the reporting rate for each domain and the average score for each item. ResultsFinally， 140 CPM guidelines were included， of which 51 were disease-oriented and 89 were drug-oriented， all of which were issued by China. For 51 disease-oriented CPM guidelines， the highest average score of all six AGREE Ⅱ domains was 73.32% for clarity， and the lowest was 26.80% for application； for 89 drug-oriented CPM guidelines， the highest average score was 55.62% for scope and purpose， and the lowest was 31.32% for rigour of development. In terms of the seven domains of the RIGHT checklist， the highest reporting rate was 68.26% for background， and lowest was 27.45% for other areas regarding the disease-oriented CPM guidelines； the highest reporting rate was 61.31% for background， and the lowest was 4.49% for other areas regarding drug-oriented CPM guidelines. The average reporting rate was higher for disease-oriented than drug-oriented CPM guidelines in three domains of AGREE Ⅱ （rigour of development， clarity of presentation， editorial independence）， as well as four domains of RIGHT checklist （basic information， evidence， funding and declaration and management of interests， and other areas）. ConclusionThe overall methodology and reporting quality of the current CPM guidelines still need to be improved. It is recommended that future guideline development teams should strictly refer to the AGREE Ⅱ and RIGHT checklist， and take into account of the characteristics of CPM guidelines and relevant methodo-logical suggestions in the development and reporting of CPM guidelines， thereby guiding the clinical use of CPM in a better way.

Role of Histone Methyltransferase in Fibrosis and Traditional Chinese Medicine Treatment： A Review / 中国实验方剂学杂志

Fibrosis， a tumor-like lesion between benign tissue and malignant tumor， mostly occurs in the liver， kidney， heart， lung， bone marrow and other organs and tissues. It can affect almost every organ and eventually induce multiple organ failure and cancers， seriously endangering human life. It will be of great importance to prevent cancer if the disease can be opportunely blocked in the fibrotic stage. The pathogenesis of fibrosis is still not completely clear. It is of great clinical significance to study the occurrence， development， and mechanism of fibrosis as well as to screen new therapeutic targets. Enhancer of zeste homolog 2 （EZH2） is mainly located in the nucleus and involved in the formation of the polycomb repressive complex 2. EZH2 is a methyltransferase which makes the lysine on position 27 of histone H3 （H3K27me3） undergo trimethyl modification induces gene silencing through classical or nonclassical actions， so as to inhibit or activate transcription. EZH2 plays a critical role in cell growth， proliferation， differentiation， and apoptosis， which is regulated by different targets and signaling pathways. EZH2 regulates the transformation of myofibroblasts and participates in the fibrosis of multiple organs. Recent studies have shown that EZH2 plays a role in fibrosis-related pathophysiological processes such as epithelial-mesenchymal transition， oxidative stress， and inflammation. EZH2 as the target of fibrosis， EZH2 inhibitors， and EZH2-related traditional Chinese medicine （TCM） formula and active compounds have gradually become hot research directions. EZH2 may be a powerful target for organ fibrosis. Exploring the structure， function， and distribution of EZH2， the role of EZH2 in fibrosis， the EZH2 inhibitors， and TCM formulas and active components targeting EZH2 has great meanings. This paper reviews the research progress in EZH2 and fibrosis， providing new ideas for the diagnosis， treatment， and drug development of fibrosis.

Política industrial farmacéutica, un requisito clave para la autonomía sanitaria de Colombia

Colombia depende de la importación de medicamentos, así como de gran parte de los materiales (principios activos y excipientes) requeridos para su elaboración; problemática que genera consecuencias sanitarias y macroeconómicas, las cuales se agudizan en el contexto de desindustrialización nacional y de disrupción tecnológica. De esta manera, se acepta que la disponibilidad y acceso a medicamentos y otras tecnologías sanitarias esenciales son un requisito fundamental para alcanzar la autonomía sanitaria de un país. Por lo tanto, resulta imprescindible coordinar esfuerzos entre diversos sectores sociales para desarrollar una agenda pública enfocada a la creación de condiciones que fortalezcan las capacidades científicas y tecnológicas de la industria farmacéutica local, y con ello, mejorar el suministro farmacéutico del país. En el presente documento se presentan conceptos teóricos y prácticos que deberían ser considerados en la definición y materialización de una política pública encaminada a fortalecer la industria farmacéutica y favorecer la autonomía sanitaria de Colombia.

Colombia has a notorious dependency on the importation of medicines, as well as a large part of the materials (active ingredients and excipients) required for their manufacture. This problem generates health and macroeconomic consequences, which are exacerbated in the context of national deindustrialization and technological disruption. In this way, it is accepted that the availability and access to medicines and other essential health technologies are a fundamental requirement to achieve the health autonomy of a country. Therefore, it is crucial to coordinate efforts between several social sectors to develop a public agenda focused on creating conditions that allow strengthening the scientific and technological capabilities of the local pharmaceutical industry, thereby, improving the country's pharmaceutical supply. This document presents conceptual and practical topics that should be considered to defining and materializing a public policy aimed at strengthening the local pharmaceutical industry and favoring Colombia's sanitary autonomy.

A tecnologia digital como mecanismo auxiliador no envelhecimento ativo no século XXI / Digital technology as an auxiliary mechanism for active aging in the 21st century

Objetivo: Analisar os efeitos benéficos do uso da tecnologia digital no envelhecimento ativo, considerando o contexto pós-pandemia Sars-Cov-2-covid-19. Além disso, pretende-se identificar as vantagens e desvantagens do uso dessas tecnologias pelas pessoas idosas no mundo contemporâneo. Método: estudo descritivo, tipo análise teórico reflexiva, desenvolvido a partir de duas questões norteadoras relacionados à temática, subsidiado por levantamento bibliográfico, considerando publicações pertinentes à temática, disponíveis nas bases de dados do Portal Regional da BVS, Portal de Periódicos Capes, SciELO e Pubmeb. por meio dos descritores controlados DECS /MeSH. Resultados: as TIC's estão desempenhando um papel crucial na vida das pessoas idosas, melhorando a comunicação, promovendo a saúde, facilitando o aprendizado e proporcionando um acesso mais fácil à informação e aos cuidados médicos. Conclusão: a tecnologia digital é um mecanismo auxiliador no envelhecimento ativo; quando bem implementado pode trazer mais vantagens do que desvantagens.(AU)

Objective: To analyze the beneficial effects of digital technology usage in active aging, considering the post-Sars-Cov-2-covid-19 pandemic context. Additionally, the aim is to identify the advantages and disadvantages of older individuals using these technologies in the contemporary world. Method: A descriptive study, specifically a theoretical reflective analysis, developed based on two guiding questions related to the theme. The study was supported by a literature review, considering publications relevant to the topic available in the databases of the Regional Portal of BVS, Capes Periodicals Portal, SciELO, and Pubmeb, using controlled descriptors such as DECS/MeSH. Results: Information and communication technologies (ICTs) are playing a crucial role in the lives of older individuals, enhancing communication, promoting health, facilitating learning, and providing easier access to information and medical care. Conclusion: Digital technology serves as an auxiliary mechanism in active aging; when well implemented, it can bring more advantages than disadvantages.(AU)

Objetivo: Analizar los efectos beneficiosos del uso de la tecnología digital en el envejecimiento activo, considerando el contexto post-pandemia de Sars-Cov-2-covid-19. Además, se pretende identificar las ventajas y desventajas del uso de estas tecnologías por parte de las personas mayores em mundo contemporáneo. Método: Estudio descriptivo, tipo análisis teórico reflexivo, desarrollado a partir de dos preguntas orientadoras relacionadas con el tema, respaldado por revisión bibliográfica que considera publicaciones pertinentes disponibles en las bases de datos del Portal Regional de BVS, Portal de Periódicos Capes, SciELO y Pubmeb, mediante descriptores controlados DECS/MeSH. Resultados: tecnologías de la información y la comunicación (TIC) desempeñan un papel crucial en la vida de las personas mayores, mejorando la comunicación, promoviendo la salud, facilitando aprendizaje y proporcionando acceso fácil a información y atención médica. Conclusión: La tecnología digital es un mecanismo auxiliar en el envejecimiento activo cuando se implementa adecuadamente puede aportar más ventajas que desventajas.(AU)

Estudo comparativo entre empresa farmacêutica global e instituição pública de produção e inovação em saúde / Comparative study between global pharmaceutical company and public institution of production and innovation in health

RESUMO O cenário tecnológico no campo da saúde é um fato alarmante, mormente no contexto provocado pela Covid-19. Nessa conjuntura, a Fundação Oswaldo Cruz, por meio da Unidade de Bio-Manguinhos, e o Butantan foram protagonistas para o acesso universal, dialogando com estratégicas internacionais. No adensamento da discussão estratégica para Instituições Públicas de Produção e Inovação em Saúde (Ippis), o uso de diretrizes da Avaliação de Tecnologias em Saúde destaca-se como via de mudança paradigmática para a introdução de tecnologias no Sistema Único de Saúde (SUS) alinhada à visão de inovação de futuro em saúde consonante às demandas nacionais. Este artigo, desenvolvido metodologicamente mediante pesquisas descritiva qualitativa, bibliográfica, documental e trabalho de campo, buscou traçar simetrias e assimetrias baseado nas experiências coletadas em empresa farmacêutica global e instituição de referência nacional pública do campo de incorporação tecnológica em saúde. Como resultados, são explicitados pontos-chave para o fortalecimento técnico e político do Complexo Econômico-Industrial da Saúde, por meio da revisão organizacional das Ippis quanto a aspectos de inovação e de gestão, culminado na promoção de melhorias na Política de ciência, tecnologia e inovação em resposta ao desafio da sustentabilidade, efetividade e acesso no SUS.

ABSTRACT The technological scenario in the field of health is an alarming fact, especially in the context caused by COVID-19. In this context, the Oswaldo Cruz Foundation, through the Bio-Manguinhos Unit, and the Butantan Institute were protagonists for universal access, dialoguing with international strategies. In the strategic discussion for Public Institutions of Production and Innovation in Health (IPPIS), the use of guidelines of the Technological Assessment in Health stands out as a way of paradigmatic change for the introduction of technologies in the Unified Health System (SUS) in line with the vision of future innovation in health in accordance with national demands. This article, methodologically developed through qualitative descriptive research, bibliographical, documentary and field work, sought to trace symmetries and asymmetries based on the experiences collected in a global pharmaceutical company and a public national reference institution in the field of technological incorporation in health. As a result, key points are explained for the technical and political strengthening of the Health Economic-Industrial Complex, through the organizational review of the IPPIS regarding innovation and management aspects, culminating in the promotion of improvements in the science, technology and innovation policy in response to the challenge of sustainability, effectiveness and access in the SUS.

Caracterización de la oferta de medicamentos en la República Argentina / Characterization of the drug supply in Argentina

Resumen Introducción : Conocer las características del mercado farmacéutico permite obtener información sensible para entender la oferta, la demanda y el acceso de la pobla ción a los medicamentos. Con el objetivo de aportar da tos primarios respecto a la comercialización de fármacos en Argentina, se desarrolló la siguiente investigación. Métodos : Se trata de un estudio descriptivo cuanti tativo-cualitativo transversal del mercado farmacéutico argentino, tomando en cuenta 30 años de información oficial aportada por la Agencia Reguladora Nacional (ANMAT). Resultados : Se identificaron 216 laboratorios (182 nacionales) productores/importadores de medicamentos, 53 distribuidores y 479 droguerías (establecimientos de distribución de medicamentos al por mayor). Se detectó una alta concentración de la comercialización, agrupán dose el 90% de la misma, en solo 5 intermediarios. En el país existen actualmente 6670 productos/certificados, cantidad que fluctuó a lo largo de los últimos 30 años. Seis laboratorios son dueños de entre 116 y 208 certifica dos. El 84% de estos productos provienen de laboratorios nacionales, 5002 son monofármacos, mientras que el 83% se comercializa bajo un nombre de fantasía. Las tres principales indicaciones a las que se destina el registro de medicamentos en la Argentina son enfermedades del aparato digestivo, sistema nervioso, e infecciosas; el 58% es comercializado como formulaciones orales. Discusión : el presente trabajo muestra que el mer cado farmacéutico argentino tiene una participación mayoritaria de capitales nacionales, existiendo gran concentración en pocas empresas productoras y distri buidoras. Los productos son mayormente monodrogas comercializadas en forma oral y ofrecidas por su nombre de fantasía.

Abstract Introduction : Knowing the characteristics of the phar maceutical market allows obtaining sensitive informa tion to understand the supply, demand and access of the population to medicines. In order to provide primary data regarding the marketing of drugs in Argentina, the following research was performed. Method : This is a cross-sectional quantitative-quali tative descriptive study of the Argentine pharmaceutical market, taking into account 30 years of official information provided by the National Regulatory Agency (ANMAT). Results : Two hundred and sixteen laboratories (182 national) drug producers/importers, 53 distributors and 479 drugstores (wholesale drug distribution establishments) were identified. A high concentration of market ing was detected, grouping 90% in only 5 intermediaries. There are currently 6670 products/certificates in the country, an amount that fluctuated over the last 30 years. Six laboratories are owners of between 116 and 208 certificates; 84% of these products come from na tional laboratories, 5002 are mono-drugs, while 83% are marketed under a fancy name. The three main indica tions for which the registration of drugs in Argentina is intended are diseases of digestive system, nervous system and infectious diseases; 58% of the marketed products consist of oral formulations. Discussion : Based on data provided by this study, it is possible to assert that the Argentinian pharmaceutical market has a majority share of national capital, with a great concentration in a few pharmaceutical companies and distributors. The products are mostly available as non-combined drugs, in their oral form, and available by their brand names.

Acesso a medicamentos anti-hipertensivos pelos usuários de uma unidade ambulatorial / Antihypertensive drugs access by users of ambulatory unit

Objetivo: caracterizar o acesso aos medicamentos anti-hipertensivos pelas pessoas com hipertensão arterial atendidas em uma unidade ambulatorial. Metodologia: estudo descritivo, quantitativo, desenvolvido com 103 pessoas com hipertensão arterial em uso de anti-hipertensivos. Os dados foram coletados por meio de questionário com perguntas sociodemográficas, sobre tratamento e acesso aos medicamentos anti-hipertensivos. Utilizou-se a estatística descritiva e teste qui-quadrado de Pearson ou exato de Fisher para análise dos dados. Resultados: Predominou a faixa etária de 50-69 (68,9%), sexo feminino (85,4%) e raça/cor autodeclarada preta (46,6%). Quanto ao acesso aos anti-hipertensivos, 70,9% relataram ter acesso gratuito, 60,2% os obtêm nas unidades de saúde, 65,7% não referiram dificuldades na aquisição e 86,4% que tinham acesso total. Todos os participantes que tinham dificuldade econômica também tinham dificuldade de acesso aos anti-hipertensivos. Verificou-se associação significativa entre a dificuldade de acesso aos anti-hipertensivos com forma de acesso (total ou parcial), quantidade de drogas e disponibilidade do medicamento nas farmácias (p<0,005). Conclusão: observou-se que, embora a maioria dos participantes do estudo não encontre dificuldades para obtenção dos anti-hipertensivos nas farmácias das unidades básicas de saúde, ainda assim, existe uma parcela da população sem acesso total aos anti-hipertensivos de forma gratuita, sendo essencial melhorias dos programas de fornecimento de medicamentos.

Objective: to characterize access to antihypertensive drugs by patients with arterial hypertension treated at an ambulatory unit. Methodology: descriptive, quantitative study, developed with a group of 103 people with arterial hypertension currently using antihypertensive drugs. The data were collected through a questionnaire with sociodemographic questions, with respect to treatment and access to antihypertensive drugs. Descriptive statistics and Pearson's chi-square test or Fisher's exact test were used for data analysis. Results: the age group 50-69 (68.9%), female (85.4%) and black self-declared race (46.6%) predominated. Regarding access to antihypertensive drugs, 70.9% reported having free access, 60.2% obtained them at health units, 65.7% did not mention difficulties in acquiring them and 86.4% that had full access. All participants who had economic difficulties also had difficulty accessing antihypertensive drugs. It was identified a significant association between difficulty in accessing antihypertensive drugs and the means of access (total or partial), quantity of drugs and availability of the drug in pharmacies (p<0.005). Conclusion: it was observed that, that most of the study participants did not find it difficult to obtain antihypertensive drugs in the pharmacies of basic health units, notwithstanding, there is a portion of the population without full access to antihypertensive drugs free of charge, improvements in drug supply programs are essential.

Innovative mechanisms of pre-marketing authorization access for rare diseases in Brazil: a case study of pabinafusp-alfa for mucopolysaccharidosis type II / Mecanismos inovadores de acesso pré-comercialização a tecnologias para doenças raras no Brasil: um estudo de caso de alfapabinafuspe para mucopolissacaridose tipo II

Patients with rare diseases frequently face unmet medical needs due to the high costs, lengthy development times, and slow approval processes for new treatments. This case study discusses innovative access alternatives for rare diseases in Brazil, focusing on early access to pabinafusp-alfa for mucopolysaccharidosis type II (MPS-II), a rare genetic lysosomal storage disease characterized by a deficiency of the enzyme iduronate-2-sulfatase. From September 2018 to March 2023, 20 Brazilian MPS-II patients received pabinafusp-alfa through a clinical research protocol. This enzyme replacement therapy (ERT) crosses the blood-brain barrier to address central nervous system manifestations unmet by existing treatments. Patients' participation in the clinical study resulted in an estimated BRL 65 million in cost savings for the public healthcare system compared to conventional ERT with idursulfase-alfa and potentially better clinical outcomes. The case study underscores the importance of innovative mechanisms in addressing patients' medical needs. Early access alternatives include: a) clinical study access, with execution/development aligned with healthcare managers and linked to future access strategies; b) regulatory-level risk-sharing, considering effectiveness uncertainties and the possibility of market withdrawal and/or reimbursement in case of negative results; and c) drug pre-delivery, with payment contingent on positive phase III clinical study outcomes. Although public-private partnerships in clinical research are underused, they could benefit all stakeholders by accelerating drug development, facilitating early patient access to innovative medicines, and generating healthcare system savings, particularly for rare diseases.

Pacientes com doenças raras frequentemente enfrentam necessidades médicas não atendidas devido aos altos custos, longos tempos de desenvolvimento e processos de aprovação lentos para novos tratamentos. Este estudo de caso discute alternativas inovadoras de acesso para doenças raras no Brasil, com foco no acesso precoce ao alfapabinafuspe para mucopolissacaridose tipo II (MPS-II), uma doença lisossômica de armazenamento genético rara, caracterizada por uma deficiência da enzima iduronato-2-sulfatase. De setembro de 2018 a março de 2023, 20 pacientes brasileiros com MPS-II receberam alfapabinafuspe por meio de pesquisa clínica. Essa terapia de reposição enzimática (TRE) atravessa a barreira hematoencefálica para tratar manifestações do sistema nervoso central não atendidas pelos tratamentos existentes. A participação dos pacientes no estudo clínico resultou em uma economia estimada de 65 milhões de reais para o sistema público de saúde, em comparação com a TRE convencional com idursulfase alfa, além de potencialmente melhores resultados clínicos. O estudo de caso destaca a importância de mecanismos inovadores no atendimento das necessidades médicas dos pacientes. As alternativas de acesso precoce incluem: a) acesso por meio de estudos clínicos, com execução/desenvolvimento alinhada aos gestores de saúde e vinculada a estratégias futuras de acesso; b) compartilhamento de risco em nível regulatório, considerando as incertezas de eficácia e a possibilidade de retirada do mercado e reembolso em caso de resultados negativos; e c) pré-entrega do medicamento, com pagamento condicionado aos resultados positivos do estudo clínico de fase III. Embora as parcerias público-privadas em pesquisa clínica sejam subutilizadas, elas poderiam beneficiar todas as partes interessadas ao acelerar o desenvolvimento de medicamentos, facilitar o acesso precoce dos pacientes a medicamentos inovadores e gerar economias para o sistema de saúde, especialmente para doenças raras.

Use of complementary and alternative medicines in ENT outpatient department of tertiary care hospital in Southern Rajasthan

Background: Complementary and alternative medicine (CAM) use is highly gaining popularity along with main treatment streams these days. Physicians cannot ignore this fact and should question and counsel patients about pros and cons of CAM for cure of different ailments. Aim and Objectives: The objective of the study was to know about use of CAM among patients visiting Ear, Nose, and Throat (ENT) outpatient department (OPD) in tertiary care hospital of Southern Rajasthan. Materials and Methods: An observational cross-sectional study was conducted in ENT OPD for 2 months. Two hundred and fifty patients were considered for the study. Semi-structured questionnaire was prepared and made to be filled by the patients during their visit to ENT OPD after taking informed consent. Various questions were asked like extent of use of alternative therapies, type of CAM used by the patients, patient’s knowledge about ADRs and drug interactions, reasons for which alternative medicine might have been used. Results: Among 250 patients, CAMs were used by 36 patients. The most common CAMs were either Ayurveda or Homoeopathy medicines or both. The majority of patients obtained their information from family and friends. Maximum patients used these alternative medicines for relief of cough and hoarseness of voice. Conclusion: The study throws light on use of alternative drug therapy and streams of medicine along with routine hospital treatment among patients visiting ENT OPD. This knowledge helps to understand about patients’ alternative approach to diseases, about self-medication, patient’s belief about different types of therapies and also ignorance of possible health hazards, side effects, and complications due to use of concomitant main and alternative medicines for treatment purposes.

Política de medicamentos de alto costo en un hospital público pediátrico: Análisis de utilización y costos / High-cost medication policy at a public pediatric hospital: utilization and cost analysis

Objetivos. Analizar el circuito de utilización de los medicamentos de alto costo (MAC) y los resultados clínicos obtenidos en un hospital de pediatría público de alta complejidad de Argentina y presentar una estrategia de selección replicable para otras instituciones de similares características de la región. Métodos: Estudio prospectivo, descriptivo, aleatorizado, conducido en el Hospital de Pediatría Juan P. Garrahan de la Ciudad Autónoma de Buenos Aires en el período entre el 1 de setiembre de 2018 y el 31 de marzo de 2019. Se evaluaron dos unidades de estudio, la unidad paciente y la unidad MAC. Resultados: Los MAC consumen 7.921.200 dólares estadounidenses (USD) anuales y representan el 41% del costo de los medicamentos del hospital de alta complejidad. El 50% del costo de los MAC estuvo representado por la gammaglobulina (medicamento utilizado en diferentes enfermedades). Los pacientes proceden de toda la Argentina y otros países y un 44% tiene cobertura de salud. Los diagnósticos para los que se prescribieron MAC con mayor frecuencia fueron los relacionados con patología oncológica (leucemia linfoide aguda, leucemia mieloblástica aguda). El 54% de los pacientes presentó mejoría atribuible directamente a la administración de los MAC, 39% no presentó cambios y el 7% empeoró. Conclusiones: La efectividad en los resultados clínicos y el análisis de los circuitos de aprobación indican que, además de la aprobación por las entidades nacional e internacionales, la evaluación responsable por parte de las instituciones efectoras, mediante la discusión interdisciplinaria basada en la mejor evidencia, contribuye a optimizar la utilización de los MAC y la seguridad de los pacientes (AU)

Objectives. To analyze the utilization circuit of high-cost medications (HCM) and the clinical results obtained in a tertiarycare public pediatric hospital in Argentina and to present a selection strategy that may be disseminated to other institutions of similar characteristics in the region. Methods: A prospective, descriptive, randomized study was conducted at Hospital de Pediatría Juan P. Garrahan in Buenos Aires between September 1, 2018 and March 31, 2019. Two study units were evaluated, the patient and the HCM. Results: HCMs account for 7,921,200 US dollars (USD) per year and represent 41% of the cost of drugs in this tertiary-care hospital. Gamma globulin (a drug used for different diseases) accounted for 50% of the cost of HCMs. Patients came from Argentina and other countries and 44% had a health insurance. Cancer (acute lymphoid leukemia, acute myeloblastic leukemia) was the diagnosis for which HCMs were most frequently prescribed. Fifty-four percent of patients showed improvement directly attributable to the administration of HCMs, 39% showed no change, and 7% worsened. Conclusions: The effectiveness in clinical outcomes and the analysis of approval circuits show that, in addition to approval by national and international entities, responsible evaluation by the effector institutions through interdisciplinary discussion based on the best evidence contributes to optimizing the use of HCMs and patient safety (AU)

Creación e implementación del Sistema de Entrega de Medicamentos a Pacientes Crónicos SIENMECRO: estudio cualitativo

Introducción: Frente a pandemia producida por el SARS-CoV-2, el gobierno peruano implementó diversas medidas como el cierre temporal de los establecimientos del primer nivel de atención y el sistema sanitario implementó diversas políticas y estrategias; una de ellas, fue la creación e implementación del proyecto del Sistema de Entrega de Medicamentos a Pacientes Crónicos (SIENMECRO). Objetivo: Describir el proceso de creación e implementación del proyecto SIENMECRO. Métodos: Investigación cualitativa de tipo estudio de caso que implicó la revisión de la documentación existente como textos y videos y, realización de entrevistas en profundidad a funcionarios y colaboradores involucrados desde la elaboración de la propuesta del SIENMECRO. Resultados: El proyecto SIENMECRO surge como una iniciativa positiva con la aspiración de impulsar un sistema integrado de salud que aborde la realidad sanitaria desde los determinantes sociales de la salud, debido a las dificultades de los pacientes para acudir a sus controles y recibir su tratamiento. A pesar de que no se contó con un plan integral de implementación y control del proyecto, fue fundamental para cubrir la necesidad de atención sanitaria a un segmento vulnerable de la población, evitando su traslado y preservando la salud de las personas más susceptibles de padecer complicaciones por la infección por COVID-19. Conclusiones: Es una iniciativa donde el sistema sanitario toma un rol más proactivo en beneficio de la población, implementando el uso de las tecnologías de información y comunicación en la atención de salud, articulando con los diversos prestadores y acercando los servicios a los ciudadanos más vulnerables.

Introduction: Faced with the pandemic produced by SARS-CoV-2, the Peruvian government implemented several measures such as the temporary closure of primary care facilities and the health system implemented various policies and strategies; one of them was to create and implement the Drug Delivery System for Chronic Patients (SIENMECRO) project. Objective: To describe the creation and implementation process of SIENMECRO project. Methods: Qualitative case study that involved reviewing existing documentation such as texts and videos and conducting in-depth interviews with officials and collaborators involved since the preparation of SIENMECRO proposal. Results: SIENMECRO project emerges as a positive initiative with the aspiration of promoting an integrated health system that addresses the health reality from the social determinants of health, due to patients' difficulties to go to their controls and to receive their treatment. Although there was no comprehensive plan for the implementation and control of the project, it was essential to cover the need for health care for a vulnerable segment of the population, avoiding their transfer and preserving the health of the people most likely to suffer complications from COVID-19 infection. Conclusions: It is an initiative where the health system takes a more proactive role for the benefit of the population, implementing the use of technology of the information and communication in health care, articulating with the various providers and bringing services closer to the most vulnerable citizens.

Judicialização no acesso a medicamentos: análise das demandas judiciais no estado do Piauí, Brasil / Judicialization in access to medicines: analysis of lawsuits in the State of Piauí, Brazil

Resumo: Este trabalho objetivou analisar acórdãos de fornecimento de medicamentos pelo Estado no Tribunal de Justiça do Estado do Piauí (TJ-PI) entre 2018 e 2021. Foram analisados 209 acórdãos, tendo sido predominantes mandados de segurança (49%), concessão de liminar (95%), decisões favoráveis (93%), medicamentos do sistema nervoso (24,6%) e antineoplásicos (23,7%), transtornos mentais (13,4%) e neoplasias (9,8%). Os resultados e análises demonstram o dever do Estado em prover os medicamentos solicitados.

Abstract: This work aimed to analyze judgments on the supply of medicines by the State in the Court of Justice of the State of Piauí (TJ-PI) between 2018 and 2021. 209 judgments were analyzed, predominantly injunctions (49%); granting of an injunction (95%); favorable decisions (93%); nervous system drugs (24.6%) and antineoplastics (23.7%); mental disorders (13.4%) and neoplasms (9.8%). The results and analyses demonstrate the State's duty to provide the requested medication.

Acesso equitativo a vacinas: lições aprendidas e perspectivas futuras / Equal access to vaccines: lessons learned and future perspectives

Resumo A vacinação é um componente essencial da atenção primária à saúde e do enfrentamento de emergências em saúde. No entanto, apesar do progresso ocorrido nas últimas décadas, persistem importantes barreiras que resultam na queda de coberturas e disparidades entre os países no acesso a novas vacinas. Neste cenário, a Organização Mundial da Saúde (OMS) lançou, em 2020, a Agenda de Imunização para o decênio 2021-2030 (AI2030). Este artigo tem o objetivo de debater os principais fatores que afetam o acesso às vacinas e as estratégias para promoção da equidade no acesso a elas a nível global e nacional. Tais fatores são multisetoriais e precisam ser considerados em ambos os níveis, destacando-se as barreiras financeiras e geográficas, os desafios de infraestrutura, fatores socioeconômicos e culturais, políticas públicas e governança. O texto aponta a necessidade de remodelação da arquitetura global das cadeias produtivas e dos centros de pesquisa e inovação, criando e/ou fortalecendo as existentes em países de baixa e média renda. Além disso, é necessário estabelecer novos mecanismos e modelos de produção e comercialização de vacinas. As estratégias adotadas para acesso a vacinas e outras tecnologias em saúde estão no centro do debate da agenda de saúde global.

Abstract Vaccination is an essential component of primary health care and coping with health emergencies. However, despite the progress from the last decades, important barriers persist resulting in lower access and disparities between the countries in the access to new vaccines. In this scenario, the World Health Organization (WHO) launched, in 2020, the Immunization Agenda for the 2021-2030 decade (AI2030). This article aims to discuss the main factors that affect access to vaccines and strategies to promote equity in access to them at global and national levels. These factors are multi-sectoral and need to be considered in both levels, with emphasis on financial and geographic barriers, infrastructure challenges, socioeconomic and cultural factors, public policies, and governance. The text points the need to remodel the global architecture of production chains and research and innovation centers, creating and/or strengthening existing ones in low- and middle-income countries. In addition, establishing new mechanisms and models for the production and commercialization of vaccines is necessary. The strategies adopted for accessing vaccines and other health technologies are at the center of the global health agenda debate.

Acesso a Medicamentos do Componente Especializado da Assistência Farmacêutica: uma Revisão Integrativa / Access to drugs from the specialized component of pharmaceutical assistance: an integrative review / Acceso a medicamentos del componente especializado de asistencia farmacéutica: una revisión integradora

Objetivo:Descrever as dificuldades e/ ou facilidades encontradas no acesso a medicamentos do CEAF. Método: Revisão integrativa de literatura, com levantamento online no Portal de Periódicos Eletrônicos disponibilizado pela CAPES, nas Bases de Dados DOAJ, Latindex, SciELO Brazil e Medline Complete, com os Descritores em Ciências da Saúde: Assistência farmacêutica, Medicamentos do Componente Especializado da Assistência Farmacêutica e Acesso aos serviços de saúde. Foram selecionados nove artigos, sendo realizada análise descritiva, com a criação das categorias: Facilidades no acesso aos medicamentos do CEAF; Dificuldades no acesso aos medicamentos do CEAF. Resultados:As facilidades encontradas foram: descentralização; aumento de usuários; qualificação da equipe; encaminhamento dos documentos; preenchimento da prescrição médica com Denominação Comum Brasileira; infraestrutura; participação do farmacêutico; oferta de medicamentos em casa. Já as dificuldades foram: insuficiência de recursos; acesso a consultas; tempo para avaliação das solicitações; necessidade de incorporação de medicamentos; insuficiência de farmacêutico; falta de estrutura física; déficit de conhecimento dos médicos e farmacêuticos sobre os PCDT; ações judiciais. Conclusão: É necessário superar os desafios encontrados para que o acesso aos medicamentos do CEAF seja efetivado na prática enquanto direito de cidadania

Objective:To describe the difficulties and/or facilities encountered in accessing CEAF medication. Method:Integrative literature review, with online survey in the Electronic Journals Portal made available by CAPES, in the DOAJ, Latindex, SciELO Brazil and Medline Complete Databases, with the Health Sciences Descriptors: Pharmaceutical assistance, Medicines ofthe Specialized Component of Assistance Pharmaceuticals and Access to Health Services. Nine articles were selected, and a descriptive analysis was carried out, with the creation of the categories: Ease of access to CEAF medicines; Difficulties in accessing CEAF medicines. Results:The facilities found were: decentralization; increase in users; team qualification; routing of documents; filling out the medical prescription with Brazilian Common Denomination; infrastructure; pharmacist participation; supply of medicines at home. The difficulties were: insufficient resources; access to queries; time for evaluating requests; need for medication incorporation; pharmacist insufficiency; lack of physical structure; lack of knowledge of physicians and pharmacists about PCDT; judicial actions. Conclusion:It is necessary to overcome the challenges encountered so that access to CEAF medicines is implemented in practice as a right of citizenship.

Objetivo: Describir las dificultades y/o facilidades encontradas en el acceso a la medicación CEAF. Método:Revisión integrativa de la literatura, con encuesta en línea en el Portal de Revistas Electrónicas disponible por la CAPES, en las Bases de Datos DOAJ, Latindex, SciELO Brasil y Medline Complete, con los Descriptores de Ciencias de la Salud: Asistencia Farmacéutica, Medicamentos del Componente Especializado de Asistencia Farmacéutica y Acceso a los Servicios de Salud. Se seleccionaron nueve artículos y se realizó un análisis descriptivo, con la creación de las categorías: Facilidad de acceso a los medicamentos del CEAF; Dificultades en el acceso a los medicamentos del CEAF. Resultados:Las facilidades encontradas fueron: descentralización; aumento de usuarios; calificación del equipo; enrutamiento de documentos; diligenciamiento de la prescripción médica con Denominación Común Brasileña; infraestructura; participación farmacéutica; suministro de medicamentos a domicilio. Las dificultades fueron: recursos insuficientes; acceso a consultas; tiempo para evaluar las solicitudes; necesidad de incorporación de medicamentos; insuficiencia farmacéutica; falta de estructura física; falta de conocimiento de médicos y farmacéuticos sobre PCDT; acciones judiciales. Conclusión:Es necesario superar los desafíos encontrados para que el acceso a los medicamentos del CEAF sea implementado en la práctica como un derecho de ciudadanía.

Judicialization and right to health in Brazil: a trajectory of matches and mismatches / Judicialização e direito à saúde no Brasil: uma trajetória de encontros e desencontros

ABSTRACT This study discusses the impacts of judicialization on the guarantee of the right to health in Brazil and the need to reassess the role of the Judicial system in its protection. We used evidence from the technical-scientific literature and information on the budgetary-financial execution and the acquisition of medicines from the Brazilian Ministry of Health to substantiate the arguments. In 2019, lawsuits consumed 25.2% of the resources of the Specialized Component of Pharmaceutical Care, 21% for 10 medicines. Although the Judicial promotes this right when the State fails to ensure access to medicines incorporated into the Brazilian Unified Health System (SUS), this system compromises access to medicines of the population with the determinations of acquisition of non-incorporated products. The Judicial needs to guide its control over compliance with constitutional and legal precepts in public policies, especially in fiscal policy, given its impact on the financing of the SUS.

RESUMO Neste texto, discutem-se os impactos da judicialização na garantia do direito à saúde no Brasil e a necessidade de reavaliação do papel do Judiciário na sua proteção. Evidências da literatura técnico-científica e informações sobre a execução orçamentário-financeira e a aquisição de medicamentos do Ministério da Saúde foram utilizadas para fundamentar os argumentos. Mostra-se que, em 2019, as ações judiciais consumiram 25,2% dos recursos do Componente Especializado da Assistência Farmacêutica, sendo 21% para 10 medicamentos. Argumenta-se que, embora o Judiciário promova esse direito quando o Estado falha em assegurar o acesso a medicamentos incorporados ao Sistema Único de Saúde (SUS), ele compromete o acesso a medicamentos da população com as determinações de aquisição de produtos não incorporados. Defende-se a necessidade de o Judiciário pautar seu controle sobre a observância dos preceitos constitucionais e legais nas políticas públicas, especialmente na política fiscal, dado seu impacto sobre o financiamento do SUS.

A prospective descriptive study on emergency medicines used most frequently from the emergency medicine packs at Universitas Academic Hospital and the associated financial loss of expired emergency medicines

Resuscitation trolleys with equipment and medicines for emergencies are required in all clinical areas in hospitals. Emergency medicines kept separately are more likely to expire than non-emergency medicines, as these are generally used less frequently. An updated list of essential emergency medicines was implemented in 2016 at an academic hospital in central South Africa. The aim of this study was to determine the frequency of both the use and the expiry, with associated financial losses, of the emergency medicines on this list. Methods: A prospective, descriptive study was conducted over a six-month period in 2019. Forms accompanying the emergency packs were returned to the pharmacy whenever packs expired or were opened. These forms were collected and the type and quantity of medicines either used or expired were recorded. Results: In total, 168 of the returned forms were included. The most frequently used emergency medicines were adrenaline, sodium bicarbonate and Ringer's lactate. Adrenaline use was recorded on 52.8% and 25% of the forms from the adult/paediatric and neonatal emergency packs, respectively. Medicines that were never used included: betamethasone, Darrow's half-strength solution and most of the neonatal pack medications. Neonatal emergency medicine packs were rarely used and the neonatal fluid packs were never used. The total cost of the expired medicines was R7 960.29 (US$569.41 at the time of the study). Adrenaline was the medication that expired most frequently but the greatest financial losses occurred from expired sodium bicarbonate. Conclusion: The total financial losses were low but can be reduced further. The contents of the emergency packs should be reviewed to possibly reduce the amount of items used infrequently. Also, the removal of those items not recommended in emergency care guidelines could be considered. Ensuring the policy was followed of returning unused medicines close to expiry and redistributing these to high-use areas would further reduce wastage

Acute and subacute oral toxicity characterization and safety assessment of COVID organics® (Madagascar's Anti­COVID Herbal Tea) in animal models

Introduction: Coronavirus disease 2019 (COVID 19) is an infectious disease caused by severe acute respiratory syndrome coronavirus 2. No drug has been generally approved as safe and effective for the treatment of COVID 19. Several therapeutic agents such as COVID Organics® (CVO) have been explored as treatment options. CVO is an herbal tea composed of 62% of Artemisia annua and 38% of other plants. There is presently no existing scientific report and data on the safety and efficacy of CVO herbal drug. Thus, acute and subacute toxicity studies were undertaken to evaluate the safety and toxicity of CVO on short and long term usage in animal models. Materials and Methods: Phytochemical and nutritional compositions of CVO were determined using standard methods. Acute oral toxicity was investigated using female Swiss albino mice (three per group). While subacute oral toxicity was done using female and male Swiss albino rats (five per group). The animals were administered 2000 mg/kg, 5000 mg/kg, therapeutic dose; 5500 mg/kg and supratherapeutic dose; 11,000 mg/kg of CVO herbal product. The control group received water ad libitum. The oral toxicity studies were done in accordance with Organization for Economic Corporation and Development guidelines. The experimental protocol was approved by the Institutional Animal Care and Use Committee, Nigerian Institute of Medical Research (Ethics No. IRB/17/043). Results: CVO is rich in antioxidants: flavonoids(10.3%), tannins(29.1%), and phenolics(434.4 mg). It contains proteins (33.8%), carbohydrates (34.5%), fat (6.8%), and fiber (0.5%). In the acute toxicity study, no mortality was recorded in all the treated and untreated groups. The lethal dose of CVO is >5000 mg/kg body weight. The hematological, biochemical, lipid profile, and histologic parameters were all normal at therapeutic doses when compared to the control group. Conclusion: The acute and subacute oral toxicity studies revealed that CVO is not toxic. The specific organ toxicity evaluations also indicated that CVO has no toxic effects on blood parameters and vital organs structure and function at therapeutic dose. Thus, CVO is safe for short and long term usage. We recommend that CVO should be subjected to efficacy studies to investigate whether it is effective for COVID 19 treatment as claimed by the manufacturer.