Pharmaceutical innovativeness index: methodological approach for assessing the value of medicines - a case study of oncology drugs.

BACKGROUND: We propose a framework to assess the value of pharmaceutical innovations, with explicit clinical and methodological parameters, based on the therapeutic value and health needs. RESEARCH DESIGN AND METHODS: The study was based on the adaptation of health technology assessment methods documented in the literature, which was applied to a sample of oncological drugs. Difficulties and issues during the application of those tools were identified and addressed to develop a new framework with new and revised domains and clear classification criterion for each domain. Scores were assigned to each level and domain according to their relevance to generate the final score of innovativeness. RESULTS: The Pharmaceutical Innovation Index (PII) includes four domains, two related to clinical and social dimensions - Therapeutic Need and Added Therapeutic Value - and other two about methodological features - Study Design and Quality (risk of bias). The scores combined after assigned to each domain results Index of the Innovativeness of the medicines represents the degree of pharmaceutical innovation. CONCLUSION: This work proposes a transparent methodology with well-defined criteria and script; the algorithm developed with authors' weightings and criteria may be switched to best adjust to other applications, perspective or clinical indications, while keeping the transparency and objectiveness.

Methodological guidelines and publications of benefit-risk assessment for health technology assessment: a scoping review.

OBJECTIVES: To map the available methodological guidelines and documents for conducting and reporting benefit-risk assessment (BRA) during health technologies' life cycle; and to identify methodological guidelines for BRA that could serve as the basis for the development of a BRA guideline for the context of health technology assessment (HTA) in Brazil. DESIGN: Scoping review. METHODS: Searches were conducted in three main sources up to March 2023: (1) electronic databases; (2) grey literature (48 HTA and regulatory organisations) and (3) manual search and contacting experts. We included methodological guidelines or publications presenting methods for conducting or reporting BRA of any type of health technologies in any context of the technology's life cycle. Selection process and data charting were conducted by independent reviewers. We provided a structured narrative synthesis of the findings. RESULTS: From the 83 eligible documents, six were produced in the HTA context, 30 in the regulatory and 35 involved guidance for BRA throughout the technology's life cycle. We identified 129 methodological approaches for BRA in the documents. The most commonly referred to descriptive frameworks were the Problem, Objectives, Alternatives, Consequences, Trade-offs, Uncertainty, Risk and Linked decisions and the Benefit-Risk Action Team. Multicriteria decision analysis was the most commonly cited quantitative framework. We also identified the most cited metric indices, estimation and utility survey techniques that could be used for BRA. CONCLUSIONS: Methods for BRA in HTA are less established. The findings of this review, however, will support and inform the elaboration of the Brazilian methodological guideline on BRA for HTA. TRIAL REGISTRATION NUMBER: https://doi.org/10.17605/OSF.IO/69T3V.

Política Andina de Evaluación de Tecnologías Sanitarias / Andean Health Technology Assessment Policy

La actualización de la presente política reafirma el compromiso del Organismo Andino de Salud ­ Convenio Hipólito Unanue con la toma de decisiones en salud, basadas en la mejor evidencia científica mediante la evaluación de tecnologías sanitarias. Para lograr este objetivo general en nuestra región, se han trazado 4 ejes estratégicos para su implementación: políticas públicas basadas en evidencia, fortalecimiento de las evaluaciones de tecnologías sanitarias en la región andina, establecer un marco de calidad y fomentar el uso racional de las tecnologías. Los indicadores propuestos en esta política monitorearán la implementación de las evaluaciones de tecnologías sanitarias en la región Andina y ­en un futuro próximo­, pueden ser el punto de partida de una lista de chequeo para acreditar agencias de evaluación de tecnologías sanitarias en la región

HTA criteria adopted in different models of public healthcare systems for orphan drugs: A scoping review.

Access to drugs for rare diseases constitutes a challenge to healthcare systems, especially those with public funding. This study aimed to map and summarize the criteria used by HTA agencies in different healthcare systems to evaluate reimbursement recommendations for orphan drugs. A comprehensive literature search was performed on the databases PubMed, LILACS, Scopus, and Embase and the gray literature (Google Scholar and websites of HTA agencies). Publications addressing the criteria used by HTA agencies in countries with public healthcare systems when evaluating reimbursement recommendations for orphan drugs were included. This scoping review included 23 studies published between 2014 and 2023, mostly consisting of reviews of HTA reports, guidance documents, and original articles. The criteria were mapped from 19 countries and ranked within three models of healthcare systems (National Health System, National Health Insurance, and Social Health Insurance). All models shared concerns about unmet needs and disease nature. In addition, NHS countries (e.g., United Kingdom, Sweden, and Italy) prioritized innovation and system-level impact, while SHI countries (e.g., Germany, France, the Netherlands) usually valued budget impact and employed expedited evaluation processes. This review provides a comprehensive understanding of the general tendencies of each healthcare system model in establishing differentiated criteria to address the challenges posed by the limited evidence and investment in the field of rare diseases.

Application of natural language processing to predict final recommendation of Brazilian health technology assessment reports.

INTRODUCTION: Health technology assessment (HTA) plays a vital role in healthcare decision-making globally, necessitating the identification of key factors impacting evaluation outcomes due to the significant workload faced by HTA agencies. OBJECTIVES: The aim of this study was to predict the approval status of evaluations conducted by the Brazilian Committee for Health Technology Incorporation (CONITEC) using natural language processing (NLP). METHODS: Data encompassing CONITEC's official report summaries from 2012 to 2022. Textual data was tokenized for NLP analysis. Least Absolute Shrinkage and Selection Operator, logistic regression, support vector machine, random forest, neural network, and extreme gradient boosting (XGBoost), were evaluated for accuracy, area under the receiver operating characteristic curve (ROC AUC) score, precision, and recall. Cluster analysis using the k-modes algorithm categorized entries into two clusters (approved, rejected). RESULTS: The neural network model exhibited the highest accuracy metrics (precision at 0.815, accuracy at 0.769, ROC AUC at 0.871, and recall at 0.746), followed by XGBoost model. The lexical analysis uncovered linguistic markers, like references to international HTA agencies' experiences and government as demandant, potentially influencing CONITEC's decisions. Cluster and XGBoost analyses emphasized that approved evaluations mainly concerned drug assessments, often government-initiated, while non-approved ones frequently evaluated drugs, with the industry as the requester. CONCLUSIONS: NLP model can predict health technology incorporation outcomes, opening avenues for future research using HTA reports from other agencies. This model has the potential to enhance HTA system efficiency by offering initial insights and decision-making criteria, thereby benefiting healthcare experts.

Real-world evidence: experiences and challenges for decision making in Latin America.

OBJECTIVE: The Health Technology Assessment (HTA) process aims to optimize health system funding of technologies. In recent years there has been an increase in what is known as Real-World Evidence (RWE) as a complement to clinical trials. The objective of Health Technology Assessment International's Latin American Policy Forum 2022 was to explore the utility of incorporating RWE into HTA and decision-making processes in the region. METHODS: This article is based on a background document, survey, and the deliberative work of the country representatives who participated in the Forum. RESULTS: There is a growing interest in the use of Real-World Data / Real-World Evidence in HTA processes in Latin America, although currently there are no specific local guidelines for RWE use by HTA agencies. At present, its use is limited to certain areas such as adding context to HTA reports, the evaluation of adverse events, or cost estimation.Potential future uses of RWE were identified, including the creation of risk-sharing agreements, the assessment of technology performance in routine practice, providing information on outcomes that are not so easily evaluated in clinical trials (e.g., the identification of specific subpopulations or quality of life), and the estimation of input parameters for economic evaluations. CONCLUSIONS: The participants agreed that there are several areas presenting significant potential to expand the application of RWD/RWE and that the development of normative frameworks for its use could be helpful.

Challenges and opportunities in building a health economic framework for personalized medicine in oncology.

Personalized medicine has allowed for knowledge at an individual level for several diseases and this has led to improvements in prevention and treatment of various types of neoplasms. Despite the greater availability of tests, the costs of genomic testing and targeted therapies are still high for most patients, especially in low- and middle-income countries. Although value frameworks and health technology assessment are fundamental to allow decision-making by policymakers, there are several concerns in terms of personalized medicine pharmacoeconomics. A global effort may improve these tools in order to allow access to personalized medicine for an increasing number of patients with cancer.

Estratégias de Avaliação de Tecnologias em Saúde (ATS) para medicamentos para doenças raras: uma revisão rápida de escopo / Health Technology Assessment (HTA) strategies for drugs in rare diseases: a rapid scope review

A Avaliação de Tecnologias em Saúde (ATS) considera os domínios de benefícios clínicos, perfil epidemiológico, inovação, custo-efetividade, ética e de equidade no processo de decisão dos gestores em saúde. No contexto dos medicamentos para doenças raras, é desafiador o trabalho da ATS, dada a baixa disponibilidade de evidências robustas e o alto custo unitário das tecnologias. O objetivo da revisão foi analisar as estratégias disponíveis de avaliação das demandas de incorporação de medicamentos para o tratamento de doenças raras em sistemas de saúde. Foi realizada uma revisão rápida com busca estruturada na base de dados MEDLINE (via PubMed), Cochrane Library e Health Systems Evidence. Incluíram-se estudos sobre estratégias de avaliação de medicamentos utilizados para tratamento de doenças raras. Adicionalmente, foram realizadas buscas nas Agências de ATS do Brasil, Austrália, Nova Zelândia, Canadá, Reino Unido, França, Estados Unidos e Alemanha. A síntese dos resultados foi qualitativa com o agrupamento dos achados nos seguintes eixos temáticos: Segurança e efetividade, Custo-efetividade, Impacto orçamentário e Perspectiva da sociedade. Foram identificadas 267 publicações, sendo selecionadas 16 das bases de dados indexadas e 7 da literatura cinzenta. Com a análise dos documentos, pode-se concluir que a adoção de critérios específicos harmonizada com o atual modelo de ATS é um possível caminho a ser seguido no contexto dos medicamentos para doenças raras. Concomitante a isso, abordagens no sentido de incentivo a pesquisa e produção de dados de mundo real e a criação de comitês específicos para tratativa do tema nas agências de ATS apresentam-se como alternativa para lidar com as fragilidades no contexto de doenças raras.

The Health Technology Assessment (HTA) considers evidence regarding clinical benefits, epidemiological profile, innovation, cost-effectiveness, ethics and equity in its assessment process to support managers' decisions. In the context of drugs in rare diseases, the work of the ATS is challenging given the low availability of evidence and the high cost of technologies. The objective of the review was to analyze the available strategies for evaluating the demands for incorporating drugs for the treatment of rare diseases in health systems. A rapid review was performed with a structured search in the MEDLINE database (via PubMed), the Cochrane Library and Health Systems Evidence. Studies on strategies for evaluating drugs used to treat rare diseases were included and, additionally, searches were carried out in ATS Agencies in Brazil, Australia, New Zealand, Canada, United Kingdom, France, United States and Germany. The synthesis of the results was qualitative, grouping the major ones into thematic axes: Safety and effectiveness, Cost-effectiveness, Budgetary impact and Society's perspective. 267 publications were identified, 16 selected from indexed databases and 7 from gray literature. With the analysis of the documents, it can be concluded that the adoption of specific criteria harmonized with the current ATS model is a possible path to be followed in the context of drugs for rare diseases. At the same time, approaches to encourage research and the creation of specific committees to deal with the issue in HTA agencies would complement actions towards the consolidation of this work.

New challenges in health technology assessment (HTA): the case of Zolgensma. / Novos desafios na avaliação de tecnologias em saúde (ATS): o caso Zolgensma.

Rare or orphan diseases have played an important role in the pharmaceutical industry. On the other hand, the impact of new technologies derived from genomic research has been growing in this industry, with new drugs being launched on the market at unsustainable prices for health systems and patients. This double tendency poses important and growing challenges to public policies on Health Technology Assessment, whose hegemonic rationale is based on cost-benefit analysis between therapies. The very high prices of these drugs require revisiting this rationale and the recent negotiations between the Brazilian Ministry of Health and Novartis regarding a possible risk-sharing agreement for the incorporation of the drug Zolgensma is an opportunity for this revisitation.

As doenças raras ou órfãs entraram de modo importante nos negócios da indústria farmacêutica. Por outro lado, o impacto das novas tecnologias derivadas da pesquisa genômica vêm tendo presença crescente nessa indústria, com novos medicamentos sendo lançados no mercado a preços não sustentáveis para sistemas de saúde e pacientes. Essa dupla tendência coloca desafios importantes e crescentes para as políticas públicas de avaliação e incorporação de tecnologias, cujo racional hegemônico está lastreado em análise de custo-benefício entre terapias. Os preços muito altos desses medicamentos exigem revisitar esse racional e a recente tratativa entre o Ministério da Saúde e a Novartis relativa a um possível acordo de compartilhamento de risco para a incorporação do medicamento Zolgensma é uma oportunidade para essa revisita.

Effectiveness and safety of caplacizumab in acquired thrombotic thrombocytopenic purpura: health technology assessment and classification according to the methodology established in Colombia.

OBJECTIVES: Acquired thrombotic thrombocytopenic purpura (aTTP) is a rare hematological disease whose clinical management includes caplacizumab along with plasma exchange and immunosuppression, according to international guidelines. Caplacizumab has been available in Colombia since 2022. This study seeks to determine the therapeutic classification of caplacizumab according to the methodology of the Instituto de Evaluación Tecnológica en Salud. METHODS: The classification was carried out through a deliberative process following the modified Delphi technique, with a panel of experts, made up of four hemato-oncologists, a pharmaceutical chemist, and a patient. The results of effectiveness and safety obtained through a systematic review, therapeutic thresholds (clinical significance), and degree of acceptability (willingness to use the technology) were used for the classification. RESULTS: Fourteen effectiveness and safety outcomes were submitted for the classification process. Caplacizumab showed clinical significance for some effectiveness outcomes, was not considered inferior in terms of safety, and displayed acceptability of use. Through consensus, the panel determined that caplacizumab plus the standard regimen is superior to the standard regimen in terms of treatment response and composite outcome, and no different for the other effectiveness and safety outcomes. Likewise, in overall terms, the panel determined that caplacizumab together with the standard regimen is superior to the standard regimen. CONCLUSION: Treatment with caplacizumab together with the standard regimen was considered superior to the standard regimen for the treatment of patients with aTTP, as it showed clinically significant benefits in critical outcomes for decision making, and a safety profile no different to its comparator.

Exploring the COVID-19 Pandemic's Impact on the Health Technology Assessment Process of the National Commission for the Incorporation of Technologies Into the Brazilian Health System.

OBJECTIVES: This study aimed to evaluate the impact of the COVID-19 pandemic on Brazilian health technology assessment processes based on public reports from the National Committee for Health Technology Incorporation (CONITEC). METHODS: This descriptive study analyzed CONITEC's official reports on Brazil available on its website between 2018 and 2021 that aimed to propose recommendations for technologies to be incorporated into its public healthcare system. We used descriptive statistics covering the number of technologies and number of reports about drugs per year, objective, type of technology, demanding sector, and outcome before 2018 to 2019 and during the COVID-19 pandemic (2020-2021). Furthermore, we used logistic regression to explore any association between the final decision labeled as "incorporated" and the emergence of the COVID-19 pandemic. RESULTS: A total of 278 reports were analyzed. Approximately 85% (136 of 278), 79% (220 of 278), and 45% of the reports (125 of 278) were about drugs, for incorporation, and requested by the government, respectively. Moreover, 74 of 130 (57%) and 56 of 148 decisions (38%) were "incorporated" before and during the pandemic, respectively. No significant association was noted between incorporated decisions and the arrival of the COVID-19 pandemic for all technologies (odds ratio 1.43; 95% CI 0.84-2.46; P = .192) and for drugs (odds ratio 1.43; 95% confidence interval 0.81-2.53; P = .223) while adjusting for the type of technology and demandant. CONCLUSIONS: The COVID-19 pandemic has brought many challenges, but it does not seem to have had a significant impact on the health technology assessment approval decisions of CONITEC in Brazil.

High cost drugs in Latin America: access and barriers.

INTRODUCTION: Latin America comprises a large set of culturally diverse middle-income countries sharing an inequality gap and a rapidly aging population. A better informed growing middle class adds to the pressure on fragmented health systems that strive to attain universal coverage. Cost containment becomes crucial for sustainability. AREAS COVERED: Using 'high cost' as free term, together with individual country names, a search was performed in Pubmed and Scopus databases for relevant documents centered on pharmaceutical products. References of selected articles were also reviewed. EXPERT OPINION: In the region as elsewhere improving health information systems has been the starting point. Official health technology assessment agencies have been established in several countries, supporting decisions on best available evidence. A few centralized procurement and price regulation schemes using international reference pricing have been successful. Fast-track approval of generics and biosimilars, or establishing a separate funding source for high cost technologies are other options that, with varying degrees of success, have been. Since Latin America is characterized by its social, geographical and political diversity, each health system needs to recognize its individual priorities, learn from successful experiences elsewhere, and adapt possible alternative interventions to the different local contexts.

La importancia de la evaluación de tecnologías sanitarias: estado del arte / The importance of health technology assessment: state of the art / A importância da avaliação da tecnologia em saúde: o estado da arte

En un contexto de atención médica en constante evolución, la evaluación de tecnologías sanitarias se ha vuelto esencial. Este proceso evalúa la efectividad, seguridad y costo-efectividad de las tecnologías médicas, desde medicamentos hasta dispositivos. La evaluación respalda decisiones clínicas informadas, reduciendo la variabilidad en la práctica y mejorando la atención al paciente. Además, desempeña un papel crucial en las políticas de salud al guiar la asignación eficiente de recursos y promover el acceso equitativo a las tecnologías sanitarias. Como conclusión se puede decir que la evaluación de tecnologías sanitarias es esencial para garantizar sistemas de salud sostenibles y una atención de calidad.

In an ever-evolving healthcare environment, health technology assessment has become essential. This process evaluates the effectiveness, safety and cost-effectiveness of medical technologies, from drugs to devices. The assessment supports informed clinical decisions, reducing practice variability and improving patient care. It also plays a crucial role in health policy by guiding the efficient allocation of resources and promoting equitable access to health technologies. In conclusion, health technology assessment is essential to ensure sustainable health systems and quality care.

Em um contexto de saúde em constante evolução, a avaliação da tecnologia da saúde tornou-se essencial. Esse processo avalia a eficácia, a segurança e a relação custo-benefício das tecnologias médicas, de medicamentos a dispositivos. A avaliação apóia decisões clínicas fundamentadas, reduzindo a variabilidade na prática e melhorando o atendimento ao paciente. Ela também desempenha um papel fundamental na política de saúde, orientando a alocação eficiente de recursos e promovendo o acesso equitativo às tecnologias de saúde. Em conclusão, a avaliação da tecnologia em saúde é essencial para garantir sistemas de saúde sustentáveis e atendimento de qualidade.

Avaliação da eficácia e segurança de pegaspargase, em comparação à L-asparaginase, e sua viabilidade econômica no tratamento de leucemia linfoblástica aguda / Evaluation of the efficacy and safety of pegaspargase, compared to L-asparaginase, and its economic viability in the treatment of acute lymphoblastic leucemia

Introdução: O tratamento da leucemia linfoblástica aguda (LLA) é relacionado a eventos adversos (EA) e mortalidade por toxicidade dos medicamentos utilizados. Protocolos com L-asparaginase (L-Asp) têm demonstrado melhor prognóstico, porém podem causar hipersensibilidade e desenvolvimento de anticorpos neutralizantes por ser produzida a partir da Escherichia coli. A conjugação de E. coli L-Asp com monometoxipolietilenoglicol resulta na PEG-Asp, com menor imunogenicidade e maior meia-vida. Objetivo: Avaliar eficácia e segurança da PEG-Asp, em comparação à L-Asp, no tratamento de LLA, e sua viabilidade econômica para subsidiar a tomada de decisão quanto à sua incorporação. Material e Métodos: Estudo descritivo de síntese de evidências e avaliação econômica. Busca de evidências foi realizada no MEDLINE, Cochrane Library, Embase, Epistemonikos, e agências de avaliação de tecnologias em saúde. Foram considerados elegíveis revisões sistemáticas, ensaios clínicos e estudos observacionais, publicados em inglês, espanhol e português, independente da data. Viabilidade econômica foi calculada a partir do custo do uso da PEG-Asp no protocolo GRAALL ­ 2003 frente ao valor faturado via Autorização de Procedimentos de Alta Complexidade. Resultados: As evidências demonstraram eficácia semelhante entre PEG-Asp e L-Asp para maioria dos desfechos de interesse, com superioridade na prevenção de leucemia no sistema nervoso central em adultos e comodidade posológica. PEG-Asp demonstrou maior frequência de EA em adultos recém diagnosticados, e ausência de diferença na toxicidade e mortalidade nos recidivados. A incorporação da PEG-Asp se mostrou economicamente viável para pacientes adultos, e desvantajosa naqueles com 18 e 19 anos incompletos, considerando superfície corpórea média de 1,7m². Conclusão: Recomendou-se a incorporação de PEG-Asp para tratamento de LLA em pacientes acima de 18 anos e naqueles com 18 a 19 anos incompletos, deve-se avaliar a viabilidade econômica em função da superfície corpórea. Além do perfil de eficácia e segurança da PEG-Asp, não há medicamentos dessa classe terapêutica com registro para adultos na Agência Nacional de Vigilância Sanitária.

Introduction: Treatment of acute lymphoblastic leukemia (ALL) is associated with adverse events (AEs) and mortality due to toxicity of drugs used. Protocols with L-asparaginase (L-Asp) have shown improved prognosis, but can cause hypersensitivity and development of neutralizing antibodies, as L-Asp is produced from Escherichia coli. The conjugation of E. coli L-Asp with monomethoxypolyethylene glycol results in PEG-Asp, with lower immunogenicity and longer half-life. Objective: To evaluate the efficacy and safety of PEG-Asp, compared to L-Asp, in ALL treatment, and its economic viability in order to subsidize decision making regarding its incorporation. Material and Methods: Descriptive study of evidence synthesis and economic evaluation. Evidence was searched in MEDLINE, Cochrane Library, Embase, Epistemonikos, and Health technology assessment agencies. Systematic reviews, clinical trials and observational studies published in English, Spanish and Portuguese, regardless of date, were considered eligible. Economic viability was calculated based on the cost of using PEG-Asp in GRAALL - 2003 protocol compared to the amount billed via High-complexity Procedures Authorization. Results: Evidence showed similar efficacy between PEG-Asp and L-Asp for most of the outcomes of interest, with superiority in prevention of Central Nervous System leukemia in adults and in dosage convenience. PEG-Asp showed a higher frequency of AEs in newly diagnosed adults, and no difference in toxicity and mortality in relapsed adults. The incorporation of PEG-Asp proved to be economically viable for adult patients, and disadvantageous for patients between 18 and 19 years of age, considering a mean body surface area of 1.7m². Conclusion: Incorporation of PEG-Asp for the treatment of ALL in patients over 18 years was recommended, and in those aged 18 to 19 years incomplete, the economic viability should be assessed according to body surface area. In addition to the efficacy and safety profile of PEG-Asp, there are no drugs in this therapeutic class for adults registered within ANVISA.

Perfil de notas técnicas para suporte técnico-científico à decisão judicial do medicamento Zolgensma® (onasemnogeno abeparvoveque) / Profile of technical notes for scientific and technical support to judicial decision on the Zolgensma® (onasemnogene abeparvovec)

Introdução: O aumento da judicialização da saúde tem destacado a importância dos centros de avaliação judicial em decisões baseadas em evidências. A atrofia muscular espinhal (AME) é uma doença neurodegenerativa caracterizada principalmente por hipotonia muscular progressiva e alta mortalidade nos primeiros dois anos de vida. Embora o medicamento Zolgensma® (onasemnogeno abeparvoveque) seja indicado para o tratamento da AME, seu custo elevado e a indisponibilidade pelo sistema público de saúde brasileiro tornam o tratamento inviável, resultando em processos judiciais. Objetivo: Descrever o perfil das informações contidas nas notas técnicas (NT) disponíveis no Sistema Nacional de Pareceres e Notas Técnicas (e-NatJus), utilizadas como apoio nas decisões judiciais relacionadas ao Zolgensma®. Material e Métodos: Foi realizado um estudo descritivo com dados de notas técnicas disponíveis no e-NatJus no período entre setembro de 2021 a setembro de 2023. Resultados: Foram identificadas 63 notas técnicas, sendo que uma foi excluída por se tratar de nota complementar. Das 62 NTs consideradas elegíveis para análise neste estudo, a idade média dos participantes foi de 2,03 (± 1,54) anos, sendo a maioria do sexo masculino. A maioria das solicitações foi para tratamento de AME tipo 1 (N= 52; 82%). Destas, somente 23 atendiam a faixa etária inferior a 2 anos. Quanto à recomendação final, 14 NTs foram favoráveis ao fornecimento do Zolgensma®. Conclusão: Os resultados revelaram o perfil das NTs baseadas em evidências científicas, que subsidiam decisões judiciais visando minimizar impactos no orçamento da saúde. Iniciativas como o Sistema e-NatJus são fundamentais para acesso a esses subsídios, embora a disseminação de informações e capacitações continuadas sejam necessárias. Pesquisas adicionais sobre o impacto dos Núcleos de Avaliação de Tecnologias em Saúde (NATS) e Núcleos de Apoio Técnico ao Judiciário (NATJUS) na judicialização da saúde são importantes.

Introduction: The increasing judicialization of health has highlighted the importance of judicial evaluation centers in evidence-based decisions. Spinal Muscular Atrophy (SMA) is a neurodegenerative disease characterized mainly by progressive muscle hypotonia and high mortality in the first two years of life. Although the medication Zolgensma® (onasemnogene abeparvovec) is indicated for the treatment of SMA, its high cost and unavailability through the Brazilian public health system make the treatment unfeasible, resulting in legal proceedings. Objective: To describe the profile of the information contained in the Technical Notes (TN) available in the e-NatJus System used as support in Zolgensma® judicial decisions. Material and Methods: A descriptive study was conducted with data from technical notes available in the National System of Opinions and Technical Notes (e-NatJus) from September 2021 to September 2023. Results: 63 Technical Notes were identified, with one excluded as a Complementary Note. Of the 62 TNs considered eligible for analysis in this study, the mean age of the participants was 2.03 (± 1.54) years, with the majority being male. Most requests were for the treatment of SMA type 1 (N= 52; 82%). Of these, only 23 met the age criteria of less than 2 years. Regarding the final recommendation, 14 TNs were favorable to the provision of Zolgensma®. Conclusion: The results revealed the profile of technical notes based on scientific evidence, which support judicial decisions aimed at minimizing impacts on healthcare budgets. Initiatives like the e-NatJus System are essential for accessing these subsidies, although the dissemination of information and continuous training are necessary. Further research on the impact of NATS and NATJUS on healthcare judicialization is important.

Elaboração de guia para Núcleos de Avaliação de Tecnologias em Saúde da Rede Ebserh: relato de experiência / Elaboration of a guide for Health Technology Assessment Centers in the Ebserh network: an experience report

Introdução: A Empresa Brasileira de Serviços Hospitalares (Ebserh) gere 41 hospitais universitários federais (HUFs), atualmente com 36 Núcleos de Avaliação de Tecnologias em Saúde (NATS). No contexto hospitalar, o NATS desenvolve produtos de avaliação de tecnologias em saúde (ATS) para subsidiar o gestor na tomada de decisões, sendo essencial para a sustentabilidade do Sistema Único de Saúde. A implantação efetiva dos NATS ainda é desafiadora, devido à insuficiência de diretrizes específicas para ATS hospitalar. Objetivo: Relatar atuação do Grupo de Trabalho em ATS (GT-ATS) da Ebserh na elaboração de guia para orientar a estruturação e fortalecimento dos NATS dos HUFs.Relato de Experiência: O GT-ATS foi instituído por portaria, composto por representantes de NATS dos HUFs e da administração central da Ebserh. Inicialmente, fez-se levantamento dos processos envolvendo os NATS, da prática de ATS na Ebserh e foi realizado diagnóstico situacional dos NATS. Em seguida, elaborou-se o guia a partir da expertise dos membros do GT-ATS e das melhores práticas e recomendações sobre ATS hospitalar, identificadas em revisão da literatura. O "Guia para organização e funcionamento dos NATS na Rede Ebserh" foi publicado em março de 2023 no portal eletrônico da Ebserh e inclui contextualização da ATS hospitalar no Brasil, objetivos e escopo de atuação dos NATS da Ebserh, orientações para organização e funcionamento dos núcleos e modelo de regulamento interno. Também foram elaborados modelos de nota técnica, declaração de conflito de interesses, formulário de solicitação e termo de compromisso de confidencialidade. Conclusão: O GT-ATS, por meio do Guia e dos documentos elaborados, contribuiu para institucionalização, harmonização e implementação dos processos de ATS nos hospitais da Ebserh, respeitando a heterogeneidade dos contextos e níveis de maturidade dos NATS. O trabalho colaborativo dentro do GT-ATS permitiu articulação de saberes interdisciplinares e práticas interinstitucionais, ampliando a dimensão, alcance e efetividade das ações de ATS.

Introduction: The Brazilian Hospital Services Company (Ebserh) manages 41 federal university hospitals (HUFs), currently with 36 Health Technology Assessment Centers (NATS). In the hospital context, NATS develop health technology assessment (HTA) products to help managers make decisions and are essential for the sustainability of the Unified Health System. The effective implementation of NATS is still a challenge, due to the lack of specific guidelines for hospital HTA. Objective: To report on the work of Ebserh's HTA Working Group (GT-ATS) in drawing up a guide for structuring and strengthening NATS HUFs. Experience Report: The GT-ATS was set up by ordinance, made up of representatives from NATS of HUFs and from Ebserh's central administration. Initially, a survey about the processes involving the NATS and the practice of HTA at Ebserh was carried out to make a situational diagnosis. The guide was then drawn up based on the expertise of the GT-ATS members and the best practices and recommendations on hospital HTA identified in literature review. The "Guide for the organization and operation of NATS in the Ebserh network" was published in March 2023 on Ebserh's electronic portal and includes a contextualization of hospital HTA in Brazil, the objectives and scope of action of Ebserh's NATS, guidelines for the organization and operation of the centers and a model of internal regulations. Templates have also been drawn up for technical note, declaration of conflict of interests, application form and confidentiality agreement. Conclusion: Through the Guide and the documents produced, the GT-ATS contributed to the institutionalization, harmonization and implementation of HTA processes in Ebserh hospitals, while respecting the heterogeneous contexts and maturity levels of the NATS. The collaborative work within the GT-ATS has allowed interdisciplinary knowledge and inter-institutional practices to be brought together, expanding the dimension, scope and effectiveness of HTA actions.

Parches transdérmicos anticonceptivos: evaluación de tecnología sanitaria / Contraceptive transdermal patches: health technology assessment

INTRODUCCIÓN: La anticoncepción es un derecho, y es obligación del Estado garantizar el acceso a métodos anticonceptivos efectivos, seguros y de calidad. Se realizó una evaluación de tecnología sanitaria sobre los parches anticonceptivos transdérmicos. MÉTODOS: Un equipo multidisciplinario e independiente designado por el Comité Provincial de Biotecnologías de Neuquén buscó información epidemiológica, regulatoria y evidencias científicas sobre eficacia, seguridad y adherencia. Se analizó y sistematizó siguiendo metodología GRADE (Grading of Recommendations Assessment, Development and Evaluation) y CASPe (Critical Appraisal Skills Programme Español). RESULTADOS: El único parche autorizado en Argentina para su comercialización libera 33,9 µg/día de etinilestradiol y 203 µg/día de norelgestromina. Su prospecto en Argentina, EE.UU. y Europa lo asocia al doble de riesgo de enfermedad tromboembólica venosa si se compara con las píldoras anticonceptivas que provee el Estado. Esto coincide con resultados de estudios de cohortes de alta calidad. Los parches proveen similar eficacia anticonceptiva a corto plazo, pero con altas tasas de abandono en el seguimiento. La Organización Mundial de la Salud no los ha incluido en su listado de medicamentos esenciales. Los parches son más costosos que otros métodos disponibles. DISCUSIÓN: Sobre la base de los principios de beneficencia, no maleficencia, de precaución y de proporcionalidad, no se recomienda la incorporación de parches.(AU)

INTRODUCTION: Contraception is a right, being an obligation of the State to guarantee access to effective, safe and quality contraceptive methods. A health technology assessment was carried out on transdermal contraceptive patches. METHODS: A multidisciplinary and independent team appointed by the Provincial Biotechnology Committee of Neuquén searched for epidemiological and regulatory information and scientific evidence on efficacy, safety and adherence. It was analyzed and systematized following the GRADE (Grading of Recommendations Assessment, Development and Evaluation) and CASPe (Critical Appraisal Skills Programme Español) methodology. RESULTS: The only patch authorized for commercialization in Argentina releases 33.9 µg/day of ethinylestradiol and 203 µg/day of norelgestromin. Its package insert in Argentina, the US and Europe highlights that the risk of venous thromboembolic disease is twice as high compared to the contraceptive pills provided by the State. This is consistent with results from high-quality cohort studies. Patches provide similar short-term contraceptive efficacy, but with high dropout rates at follow-up. The World Health Organization has not included them in its list of essential medicines. Patches are more expensive than other available methods. DISCUSSION: Based on the principles of beneficence, non-maleficence, precaution and proportionality, the incorporation of patches is not recommended.(AU)

Síntesis de evidencia para intervenciones en COVID-19: informes de actualización de la Comisión Nacional de Evaluación de Tecnologías de Salud de Argentina

[RESUMEN]. Este informe tiene por objetivo exponer el trabajo realizado sobre el diseño, la publicación y el impacto de las actualizaciones para intervenciones en COVID-19 basadas en la evidencia, para brindar síntesis de evi- dencia actualizadas a partir de revisiones sistemáticas vivas sobre intervenciones terapéuticas para dar soporte en la toma de decisiones. Para ello, se generó un grupo específico de trabajo en el ámbito de la Comisión Nacional de Evaluación de Tecnologías de Salud (CONETEC) en el Ministerio de Salud de Argentina en colaboración con la Organización Panamericana de la Salud (OPS). Se diseñó una metodología para ela- borar y publicar documentos de evaluación orientados a dar apoyo en la toma de decisiones. Estos informes de actualización se respaldaron en la exploración de tres dominios: los efectos en la salud, la factibilidad de la implementación y las recomendaciones basadas en evidencia. Las conclusiones se adaptaron en un esquema semaforizado según el balance entre los beneficios y los aspectos negativos para los distintos esce- narios clínicos, a fin de mejorar su interpretación y aplicación. Durante el año 2021 se publicaron 16 informes de síntesis de evidencia (13 originales y 3 actualizaciones completas) que recibieron un destacado número consultas desde la página web de CONETEC y la Base Regional de Informes de Evaluación de Tecnologías en Salud de las Américas (BRISA). Así se hizo visible la necesidad de disponer de resúmenes de evidencia robustos, actualizados y confiables con adaptación al contexto de aplicación en el sistema sanitario del país. El desafío se presenta, además, en la actualización constante de la información, la diseminación apropiada y el sostenimiento en la rigurosidad en el desarrollo de los informes.

[ABSTRACT]. This report presents the work done on the design, publication, and impact of updates on evidence-based COVID-19 interventions, in order to support decision-making with updated evidence syntheses based on living systematic reviews of therapeutic interventions. To this end, a specific working group was created within the National Commission for Health Technology Assessment (CONETEC) of the Ministry of Health of Argentina, in collaboration with the Pan American Health Organization (PAHO). A methodology was designed to develop and publish assessment documents aimed at supporting decision-making. These update reports were based on the exploration of three areas: health effects, feasibility of implementation, and evidence-based recom- mendations. A color-coded system was used to present the conclusions according to the balance between their positive and negative effects in different clinical scenarios, in order to improve their interpretation and implementation. In 2021, 16 evidence synthesis reports were published (13 original reports and three full updates). These were downloaded many times from the CONETEC website and the Regional Database of Health Technology Assessment Reports of the Americas (BRISA), highlighting the need for robust, up-to-date, reliable evidence summaries adapted for implementation in the country's health system. Other challenges include constant updating of information, appropriate dissemination, and sustained rigorous preparation of the reports.

[RESUMO]. Este relatório tem como objetivo apresentar o trabalho realizado sobre o delineamento, publicação e impacto das atualizações para intervenções em COVID-19 baseadas em evidências, que visam fornecer sínteses de evidências atualizadas provenientes de revisões sistemáticas vivas sobre intervenções terapêuticas para apoiar a tomada de decisões. Para isso, foi criado um grupo de trabalho específico no âmbito da Comis- são Nacional de Avaliação de Tecnologias de Saúde (CONETEC, na sigla em espanhol), do Ministério da Saúde da Argentina, em parceria com a Organização Pan-Americana da Saúde (OPAS). Foi desenhada uma metodologia para a elaboração e publicação de documentos de avaliação destinados a apoiar a tomada de decisões. Esses relatórios de atualização se basearam na exploração de três domínios: efeitos na saúde, viabilidade da implementação e recomendações baseadas em evidências. As conclusões foram adaptadas a uma escala semafórica de acordo com o equilíbrio entre os benefícios e os aspectos negativos para os dife- rentes cenários clínicos, de forma a melhorar a sua interpretação e aplicação. Durante o ano de 2021, foram publicados 16 relatórios de síntese de evidências (13 originais e 3 atualizações completas), que receberam um número significativo de consultas no site da CONETEC e da Base Regional de Informes de Avaliação de Tecnologias em Saúde das Américas (BRISA). Assim, tornou-se visível a necessidade de contar com resumos de evidências robustos, atualizados e confiáveis adaptados ao contexto de aplicação no sistema de saúde do país. A atualização constante das informações, sua divulgação adequada e a manutenção do rigor na elaboração dos relatórios também apresentam desafios.

Reversal of the recommendations issued by the National Commission for the Incorporation of Technologies in SUS after Public Consultations. / Reversão das recomendações emitidas pela Comissão Nacional de Incorporação de Tecnologias no SUS após Consultas Públicas.

The work analyzed the effect of Public Consultations (PC) and their contributions to the recommendations of the National Commission for the Incorporation of Technologies (CONITEC). This is a descriptive and retrospective study with a qualitative-quantitative approach using a secondary data source of public access, between 2012 and 2017. A database was developed to characterize the PC of medications and their contributions, which allowed the identification of cases of reversals between the preliminary and final recommendation of CONITEC. We analyzed the contributions in cases of reversal for characterization of argumentative axes and type of basis. Of the 307 demands for incorporation of medications, 205 went through PC, with 23,894 contributions. The reversal of the recommendations occurred in 9% of the open PC (15 medications), all in the sense of non-incorporation for incorporation. Main argumentative axes dealt with clinical benefits and minor adverse events, with prevalence of the submission of clinical experiences and opinions. Advances in the processes of incorporation of technologies in the SUS by performing PC were found and the challenge that decision makers face in institutional spaces for the improvement of social participation to strengthen the public benefit was clear.

O trabalho analisou o efeito das Consultas Públicas (CP) e suas contribuições nas recomendações da Comissão Nacional de Incorporação de Tecnologias (CONITEC). Trata-se de estudo descritivo e retrospectivo, com abordagem qualiquantitativa, com fonte de dados secundárias de acesso público, entre 2012 e 2017. Elaborou-se banco de dados para caracterizar as CP de medicamentos e suas contribuições, o que permitiu identificar casos de reversões entre a recomendação preliminar e final da CONITEC. Analisou-se as contribuições nos casos de reversão para caracterização de eixos argumentativos e tipo de embasamento. Das 307 demandas de incorporação, 205 destes passaram por CP, com 23.894 contribuições. A reversão das recomendações ocorreu em 9% das CP abertas (15 medicamentos), todas no sentido da não incorporação para incorporação. Principais eixos argumentativos trataram de benefícios clínicos e menores eventos adversos, prevalecendo o envio de experiências clínicas e opiniões. Evidencia-se avanços nos processos de incorporação de tecnologias no SUS pela realização da CP e ficou claro o desafio que os tomadores de decisão enfrentam nos espaços institucionais para o aprimoramento da participação social no sentido de fortalecer o benefício público.