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.

Production components of agroindustrial interest of sugarcane varieties under subsurface drip irrigation in semiarid conditions

The identification of promising commercial varieties for irrigated crops in semiarid conditions based on characters of interest to sugarcane agro-industries is important, considering the search for high sugarcane yields to obtain sustainable crops. Thus, the objective of this work was to evaluate production components of agroindustrial interest of different sugarcane varieties grown under subsurface drip irrigation in the Semiarid region of Brazil, during two crop cycles. A randomized block experimental design was used, in an 8×2 factorial arrangement (varieties and cycles) with three replications. The sugarcane juice quality, sugar yield, and culm yield were evaluated in each cycle. Gross sugar percentage, sugarcane culm yield, juice apparent sucrose content, and juice purity were affected by the crop cycles. Soluble solids content presented strong significant correlation with most variables of agroindustrial interest. The path coefficient analysis showed that juice apparent sucrose content was the explanatory character with higher direct effect on soluble solids and that this variable presented high indirect effects on most explanatory variables. The sugarcane varieties RB72454 and Q124 are recommended for production managements with drip irrigation under semiarid conditions. Soluble solids content can be used as an alternative for direct selection of varieties with higher sugar yields in different cycles.(AU)

Evaluación de evidencia para drogas oncológicas de alto costo / Evaluating evidence for high-cost oncology drugs

INTRODUCCIÓN: El presente informe reporta la evaluación de tecnologías sanitarias realizada por el Departamento de Evaluación de Tecnologías Sanitarias de la División de Planificación Sanitaria, de la Subsecretaría de Salud Pública a solicitud del Departamento de Coordinación de Garantías y Prestaciones en Salud de la misma División, para tecnologías que actualmente son financiadas por Mecanismo de Financiamiento de Drogas Oncológicas de Alto Costo. METODOLOGÍA :Se solicitó la evaluación de 40 fármacos, entre los cuales se encuentran 8 corresponden a evaluaciones de alternativas terapéuticas (comparaciones head-to-head) y las restantes, a evaluaciones de un fármaco en comparación al tratamiento estándar o habitual. RESULTADOS: Cada una de las evaluaciones solicitadas se precisó en términos de una población, intervención y comparación específica, con un grupo de expertos clínicos. Este proceso estuvo compuesto por la evaluación de efectividad clínica y análisis económico. Por un lado, la evaluación de efectividad clínica se realizó a través de la revisión sistemática de la literatura y síntesis de la evidencia mediante metodología GRADE; considerando para la magnitud de los efectos la realización de un análisis de umbrales de beneficio, de acuerdo con los publicados por la European Society for Medical Oncology (ESMO), y en el caso de no contar con ellos, se realizó una estimación con las utilidades en salud para cada desenlace. Por otro lado, el análisis económico incluyó distintos escenarios de impacto presupuestario para cada una de las solicitudes. CONCLUSIÓN: De las 40 solicitudes de evaluación, se llevaron a cabo 37, ya que no se realizó la evaluación de carfilzomib en población infantil con leucemia linfoblástica aguda, debido a que el fármaco no cuenta con registro sanitario en el Instituto de Salud Pública de Chile (ISP) y, adicionalmente se fusionaron 2 preguntas correspondientes al uso de timoglobulina y linfoglobulina; y otras dos preguntas correspondientes al uso de pembrolizumab en comparación con nivolumab en personas con melanoma.

Pertinencia de la gestión de tecnología sanitaria para la seguridad y calidad en entornos hospitalarios / Pertinence of the health technology management for the safety and quality of hospital environments

Introducción: La gestión tecnológica en el sector de la salud, clave para la innovación y la calidad de la atención médica, precisa de criterios confiables de efectividad y seguridad para su aplicación en un entorno asistencial hospitalario. Objetivo: Valorar la pertinencia de la gestión de tecnología sanitaria para la seguridad y la calidad asistencial en entornos hospitalarios. Métodos: Se realizó una revisión bibliográfica en las bases de datos de ScienceDirect y SciELO y un estudio cualitativo exploratorio diseñado a partir de las opiniones recogidas en las entrevistas semiestructuradas que se aplicaron a una selección de profesionales de la salud con experiencia profesional y académica en dos organizaciones hospitalarias de Matanzas. Con la información obtenida se analizó la relevancia científica de los principales aspectos que se identificaron asociados a la relación gestión de tecnologías-seguridad y calidad. Resultados: La utilización, innovación y evaluación de las tecnologías, su influencia en la ocurrencia de errores humanos, el condicionamiento de las prácticas clínicas, terapéuticas y de restauración de la salud, y de entornos de cuidado de riesgo para los pacientes y el personal de trabajo fueron los criterios esbozados por los encuestados y delineados en correspondencia con la literatura científica. Conclusiones: El análisis temático y las opiniones de los encuestados coinciden en la importancia e impacto de la gestión de las tecnologías sanitarias en la seguridad y la calidad de la atención hospitalaria, la práctica asistencial y, la investigación(AU)

Introduction: Technological management in the health sector, that is a key for innovation and the quality of medical care, needs feasible effectiveness and safety criteria for its use in the care hospital environment. Objective: To evaluate the pertinence of the sanitary technology management for the care safety and quality in hospital environments. Methods: It was carried out a bibliographic review in ScienceDirect and SciELO databases, and a qualitative exploratory study that was designed from the opinions collected in the semi-structured interviews performed to a selection health workers with professional and scholar experience in two hospital organizations in Matanzas province. With the information collected, it was analyzed the scientific relevance of the main aspects that were identified as associated to the relation technologies management-safety and quality. Results: The use, innovation and assessment of technologies, their influence in the occurrence of human errors, the conditioning of clinical, therapeutic and health recovery practices, and of the environments for the risks care of patients and the health workers were the criteria stated by the respondents and those were in accordance with the scientific literature. Conclusions: The thematic analysis and the respondents' opinions coincide in the importance and impact of the heath technologies management in the safety an d quality of the hospital care, the care practice and research(AU)

Multiple Criteria Decision Analysis (MCDA) for evaluating cancer treatments in hospital-based health technology assessment: The Paraconsistent Value Framework.

BACKGROUND: In recent years, the potential of multi-criteria decision analysis (MCDA) in the health field has been discussed widely. However, most MCDA methodologies have given little attention to the aggregation of different stakeholder individual perspectives. OBJECTIVE: To illustrate how a paraconsistent theory-based MCDA reusable framework, designed to aid hospital-based Health Technology Assessment (HTA), could be used to aggregate individual expert perspectives when valuing cancer treatments. METHODS: An MCDA methodological process was adopted based on paraconsistent theory and following ISPOR recommended steps in conducting an MCDA study. A proof-of-concept exercise focusing on identifying and assessing the global value of first-line treatments for metastatic colorectal cancer (mCRC) was conducted to foster the development of the MCDA framework. RESULTS: On consultation with hospital-based HTA committee members, 11 perspectives were considered in an expert panel: medical oncology, oncologic surgery, radiotherapy, palliative care, pharmacist, health economist, epidemiologist, public health expert, health media expert, pharmaceutical industry, and patient advocate. The highest weights were assigned to the criteria "overall survival" (mean 0.22), "burden of disease" (mean 0.21) and "adverse events" (mean 0.20), and the lowest weights were given to "progression-free survival" and "cost of treatment" (mean 0.18 for both). FOLFIRI and mFlox scored the highest global value score of 0.75, followed by mFOLFOX6 with a global value score of 0.71. mIFL was ranked last with a global value score of 0.62. The paraconsistent analysis (para-analysis) of 6 first-line treatments for mCRC indicated that FOLFIRI and mFlox were the appropriate options for reimbursement in the context of this study. CONCLUSION: The Paraconsistent Value Framework is proposed as a step beyond the current MCDA practices, in order to improve means of dealing with individual expert perspectives in hospital-based HTA of cancer treatments.

Multicriteria decision analysis in health care decision in oncology: a systematic review.

INTRODUCTION: Multicriteria decision analysis (MCDA) has been used to inform health decisions in health technology assessments (HTA) processes. This is particularly important to complex treatment decisions in oncology. AREAS COVERED: Five databases (PubMed, EMBASE, LILACS, Web of Science and CRD's NHS Economic Evaluation Database) were searched for studies comparing health technologies in oncology, involving the concept MCDA. The ISPOR MCDA Good Practices Guidelines were used to assess the reporting quality. Study selection, appraisal, and data extraction were performed by two reviewers. Fifteen studies were included. The main decision problem was related to health technology assessment of cancer treatments. Clinicians and public health experts were the most frequent stakeholders. The most frequently included criteria comprised therapeutic benefit, and socio-economic impact. Value measurement approach, direct rating techniques, and additive model for aggregation were used in most studies. Uncertainty analysis revealed the impact of posology and costs on the studies' results. All studies showed some level of overlapping decision criteria. EXPERT OPINION: There is considerable diversity of methods in MCDA for healthcare decision-making in oncology. The evidence presented can serve as a resource when considering which stakeholders, criteria, and techniques to include in future MCDA studies in oncology.

A framework for action to improve patient and public involvement in health technology assessment.

BACKGROUND: Patient and public involvement (PPI) in the Brazilian Health Technology Assessment (HTA) process occurs in response to a legislative mandate for "social participation." This resulted in some limited patient participation activities, and, therefore, a more systematic approach was needed. The study describes the development of a suggested framework for action to improve PPI in HTA. METHODS: This work used formal methodology to develop a PPI framework based on three-phase mixed-methods research with desktop review of Brazilian PPI activities in HTA; workshop, survey, and interviews with Brazilian stakeholders; and a rapid review of international practices to enact effective patient involvement. Patient partners reviewed the draft framework. RESULTS: According to patient group representatives, their involvement in the Brazilian HTA process is important but could be improved. Different stakeholders perceived barriers, identified values, and made suggestions for improvement, such as expansion of communication, capacity building, and transparency, to support more meaningful patient involvement. The international practices identified opportunities for earlier, more active, and collaborative PPI during all HTA stages, based on values and principles that are relevant for Brazilian patients and the public. These findings were synthesized to design a framework that defines and systematizes actions to support PPI in Brazil, highlighting the importance of evaluating these strategies. CONCLUSIONS: Since the publication of this framework, some of its suggestions are being implemented in the Brazilian HTA process to improve PPI. We encourage other HTA organizations to consider a systematic and planned approach with regular evaluation when pursuing or strengthening involvement practices.

Avanços e desafios das políticas públicas de gestão das tecnologias em saúde nas américas: scoping review / Avances y desafíos de las políticas públicas de gestión de las tecnologías en salud en las américas: scoping review / Advances and challenges of public policies for the management of health technologies in the americas: scoping review

RESUMO Objetivo: mapear evidências científicas sobre os avanços e desafios das políticas públicas de gestão das tecnologias em saúde nas Américas. Método: trata-se de uma scoping reviewrealizada em junho de 2021, nos portais e base de dados: National Library of Medicine (PubMed), Biblioteca Virtual da Saúde (BVS) e ScientificElectronic Library Online (SciELO). Resultados: ao final, 16 estudos foram selecionados. A discussão sobre as políticas públicas de gestão das tecnologias em saúde estava pautada principalmente na Avaliação de Tecnologias em Saúde (ATS). Como desafios apresentados destacaram-se: falta de investimentos e de recursos humanos; alinhamento da inovação em saúde com demandas de saúde pública; baixa contextualização do papel das tecnologias em saúde; metodologias que permitem avaliação de tecnologias leves. Em relação aos avanços, ressalta-se aumento de pesquisas com ênfase no componente tecnológico e busca de inovação, bem como avanços relacionados à ATS nos países identificados. Conclusão: a maioria dos avanços revelados são referentes ao processo de avaliação dessas tecnologias em saúde, mas existem oportunidades de melhorias, principalmente no investimento, expansão das áreas de conhecimento e no reconhecimento das tecnologias leves e leve-duras.

RESUMEN Objetivo: mapear evidencias científicas sobre los avances y desafíos de las políticas públicas de gestión de las tecnologías en saluden las Américas. Método: se trata de una scopingreviewrealizada en junio de 2021, en los enlaces y base de datos: National Library of Medicine (PubMed), Biblioteca Virtual da Saúde (BVS) yScientific Electronic Library Online (SciELO). Resultados: al final, 16 estudios fueron seleccionados. La discusión sobre las políticas públicas de gestión de las tecnologías en salud estaba basada principalmente en la Evaluación de Tecnologías en Salud (ATS). Como desafíos presentados se destacaron: falta de inversiones y de recursos humanos; organización de la innovación en salud con demandas de salud pública; baja contextualización delrol de las tecnologías en salud; metodologías que permiten evaluación de tecnologías blandas. Respecto a los avances, se destaca el aumento de investigaciones con énfasis en el componente tecnológico y búsqueda de innovación, así como avances relacionados a la ATS en los países identificados. Conclusión: la mayoría de los avances revelados son referentes al proceso de evaluación de estas tecnologías en salud, pero existen oportunidades de mejorías, principalmente en la inversión, expansión de las áreas de conocimiento yen el reconocimiento de las tecnologías blandas y blandas-duras.

ABSTRACT Objective: to outline scientific evidence on the advances and challenges of public policies for the management of health technologies in the Americas. Method: this is a scoping review conducted in June 2021, in portals and databases: National Library of Medicine (PubMed), Virtual Health Library (VHL), and Scientific Electronic Library Online (SciELO). Results: in the end, 16 studies were selected. The discussion on public policies for the management of health technologies was mostly based on the Health Technology Assessment (HTA). As challenges, the following were highlighted: a shortage of investments and human resources; matching health innovation with public health demands; poor contextualization of the health technologies' roles; methodologies that allow evaluation of light technologies. As for advances, there was an increase in research with an emphasis on technology and the search for innovation, as well as advances related to HTA in the identified countries. Conclusion: most of the advances are related to the evaluation process of these health technologies, but there are opportunities for improvement, especially regarding investment, expansion of areas of knowledge, and acknowledgment of light and light-hard technologies.

Utilização de sistema de informação e suas influências sobre a segurança de pacientes / Uso del sistema de información y sus influencias en la seguridad del paciente / Use of the information system and its influences on patient safety

Buscamos analisar a utilização de um sistema de informação e suas influências sobre a segurança de pacientes. Estudo de caso, de abordagem qualitativa, onde realizaram-se entrevistas com 21 profissionais e observações relacionadas à utilização de um sistema de informação em uma Unidade de Pronto Atendimento regional em Minas Gerais, Brasil. Analisou-se os dados a partir da Análise de Conteúdo, modalidade Temático-Categorial. Verificamos que há deficiências na conectividade; incompatibilidades da tecnologia com o trabalho na urgência; baixa qualidade das informações; sobrecarga de trabalho; pressões externas e internas; fragilidades na capacitação, no suporte técnico e na avaliação da tecnologia. Tais situações fragilizaram a utilização da tecnologia, potencializando riscos à segurança de pacientes. É imperativo desenvolver tecnologias seguras, incentivar a utilização segura e aprimorá-las, considerando os profissionais como participantes destes processos(AU)

En este estudio nos propusimos analizar el uso de un sistema de información y sus influencias en la seguridad del paciente. Se realizó un estudio con enfoque cualitativo, donde se realizaron entrevistas a 21 profesionales y observaciones relacionadas con el uso de un sistema de información en un Servicio Regional de Emergencias en Minas Gerais, Brasil. Los datos fueron procesados mediante el análisis de contenido y la modalidad temático-categorial. Se encontró que existen deficiencias en la conectividad; incompatibilidades entre tecnología y trabajo de emergencia; baja calidad de la información; sobrecarga de trabajo; presiones externas e internas; debilidades en la capacitación, en el soporte técnico y en la evaluación tecnológica. Estas situaciones han debilitado el uso de la tecnología, lo que ha aumentado los riesgos para la seguridad del paciente. Es preciso desarrollar tecnologías seguras, fomentar su uso y mejorarlas, considerando a los profesionales como partícipes de estos procesos(AU)

In this study we analyzed the use of an information system and its influences on patient safety. A study with a qualitative approach was conducted, where interviews were carried out with 21 professionals and observations related to the use of an information system in a Regional Emergency Service in Minas Gerais, Brazil. The data were processed through content analysis and thematic-categorical modality. It was found that there are deficiencies in connectivity; incompatibilities between technology and emergency work; low quality of information; work overload; external and internal pressures; weaknesses in training, technical support and technological evaluation. These situations have weakened the use of technology, which has increased risks to patient safety. It is necessary to develop safe technologies, promote their use and improve them, considering professionals as participants in these processes(AU)

Multi-criteria Decision Analysis Software in Healthcare Priority Setting: A Systematic Review.

OBJECTIVE: The objectives of this systematic review were to identify studies using Multi-Criteria Decision Analysis (MCDA) software tools to support health prioritisation processes and describe the technical capabilities of the MCDA software tools identified. METHODS: First, a systematic literature review was conducted in the MEDLINE, EMBASE, Web of Science, EconLit and Cochrane databases in July 2019 to identify studies that have used MCDA software for priority setting in health-related problems. Second, the MCDA software tools found in the review were downloaded (full versions, where freely available, and trial versions otherwise) and tested to extract their key technical characteristics. RESULTS: Nine studies were included, from which seven different software tools, 1000minds®, M-MACBETH, Socio Technical Allocation of Resources (STAR), Strategic Multi-Attribute Ranking Tool (SMART), Visual PROMETHEE, EVIDEM and the Prioritisation Framework, were identified. These software tools differed in terms of the operating systems (including web interface), MCDA technique(s) available for use, visualisation features, and the capability to perform Value for Money (VfM) and sensitivity analyses. CONCLUSIONS: The use of MCDA software in prioritisation processes has a number of advantages such as inclusion of several types of stakeholders and the ability to analyse a greater number of alternatives and criteria and perform real-time sensitivity analyses. Proprietary software (i.e. software with licensing fees) seemed to have more features than freely available software. However, this field is still developing, with only a few studies where MCDA software was used to support health priority setting and opportunity costs not explicitly captured in many software tools.

Análise das demandas judiciais por medicamentos no Estado do Rio Grande do Norte / Analysis of lawsuits for medicines in the State of Rio Grande do Norte

A judicialização da saúde, especialmente no acesso a medicamentos, é um fenômeno complexo, multifatorial que envolve aspectos técnico-científicos, legais, econômicos e sociais, podendo trazer implicações diversas para a saúde pública. Com o reconhecimento constitucional da saúde como um direito e a implantação do Sistema Único de Saúde (SUS), um número cada vez maior de cidadãos tem buscado o Poder Judiciário para garantir a promessa constitucional, e o número de processos tem crescido ao longo dos anos. Dada sua relevância, o tema da judicialização vêm sendo debatido em diversos espaços: na sociedade, no meio acadêmico, por instituições jurídicas e por gestores da saúde. Entretanto ainda não foi possível traçar um panorama nacional da judicialização de medicamentos no Brasil, havendo uma marcante concentração de pesquisas nas regiões Sudeste e Sul do país. Nesse contexto, o objetivo desse estudo foi identificar e analisar as demandas judicias por medicamentos no Estado do Rio Grande do Norte, buscando descrever as características sociodemográficas, médico-sanitárias e judiciais das ações, fazendo uma análise à luz das políticas de medicamentos vigentes e analisando suas possíveis interfaces com o processo de incorporação de tecnologias no SUS. Para tanto, foi conduzido um estudo descritivo, exploratório e retrospectivo, cuja unidade de análise foram os processos individuais solicitando medicamentos ao Estado do Rio Grande do Norte, entre os anos de 2013 a 2017. Os dados foram obtidos junto a Secretaria Estadual de Saúde Pública (SESAP/RN), e as informações dos processos foram coletadas através de consulta ao sítio eletrônico do Tribunal de Justiça do RN. Foram analisados 987 processos, em que foram solicitados 1517 medicamentos. A maioria dos demandantes foi do sexo feminino (58,8%), residentes no interior do Estado, com idade média de 48,3 anos, representadas predominantemente por assistência jurídica pública (52,8%) e com prescrição oriunda de serviços médicos privados (38,1%). A maioria dos medicamentos pleiteados (61,7%) não estavam incorporados ao SUS, porém, em 75% dos casos, havia alternativa terapêutica. Em 13,6% das ações, ao menos 1 medicamento foi prescrito para uso off-label. Mesmo os medicamentos judicializados que faziam parte da Relação Nacional de Medicamentos Essenciais (Rename), foram constantemente solicitados para indicações não recomendadas nos protocolos oficiais. Em 68% dos casos, houve acesso ao medicamento, sendo o autor responsável pela compra em 56,1% deles, via bloqueio de verbas públicas. O medicamento mais solicitado foi a insulina glargina (74 pedidos). Medicamentos não disponíveis no SUS e frequentemente solicitados nas demandas judiciais tendem a ser incorporados posteriormente, passando a integrar a política de saúde. Dos 10 medicamentos com maior número de ações judiciais, 4 foram posteriormente incorporados ao SUS, com destaque para as insulinas análogas. Em relação ao processo de incorporação desses medicamentos, observou-se que predominaram demandas internas (60%), e a minoria contou com avaliações econômicas (40%), sendo a principal justificativa utilizada no embasamento das decisões, a presença de evidência científica. Porém, foi observada mudança de posicionamento após a expressiva participação em consultas públicas e a judicialização dos medicamentos é mencionada em alguns relatórios. Os resultados mostraram que a via judicial tem se consolidado como forma de acesso a medicamentos no Rio Grande do Norte, inclusive por cidadãos residentes no interior do Estado, que conseguem assistência jurídica gratuita. Foram solicitados principalmente medicamentos ainda não incorporados ao SUS, e essas solicitações frequentemente violaram regras sanitárias e de gestão da Assistência Farmacêutica. O bloqueio de verbas públicas para o cumprimento dessas decisões é outro fator preocupante para o gestor do SUS, pois compromete a execução das políticas de medicamentos programadas, podendo enfraquecer a execução dessas. No processo de incorporação de medicamentos alvo de demandas judiciais, observou-se que apesar da preponderância do embasamento técnico-científico nas decisões de incorporação de medicamentos no SUS, houve influência indireta da judicialização no processo de tomada de decisão (AU).

The judicialization of health, especially in access to medicines, is a complex and multifactorial phenomenon that involves technical-scientific, legal, economic and social aspects, and may have different implications for public health. With the constitutional recognition of health as a right and the implementation of the Unified Health System (Sistema Único de Saúde - SUS), an increasing number of citizens have sought the Judiciary to guarantee the constitutional promise, and the number of lawsuits has grown over the years. As a relevant question, the theme of judicialization has been debated in several spaces: in society, in academia, by legal institutions and by health managers. However, it has not yet been possible to draw a national panorama of the judicialization of medicines in Brazil, with a marked concentration of research in the Southeast and South regions of the country. In this context, the aim of this study was to identify and analyze the lawsuits for medicines in the State of Rio Grande do Norte, describing the sociodemographic, medical-sanitary and judicial characteristics of the processes, making an analysis in the light of current drug policies and evaluating its possible interfaces with the process of incorporating technologies into SUS. It was conducted a descriptive, exploratory and retrospective, whose unit of analysis was the individual processes requesting medicines from the State of Rio Grande do Norte, between the years 2013 to 2017. The data were collected from the State Department of Public Health (SESAP/RN), and the information on the cases was collected through consultation with the website of the Court of Justice of RN. Nine hundred eighty-seven (987) processes were analyzed, in which 1517 medicines were requested. Most of the claimants were female (58.8%), residing in the interior of the State, with an average age of 48.3 years, represented predominantly by public legal assistance (52.8%) and with prescription from private medical services (38.1%). Most of the requested drugs (61.7%) were not incorporated into SUS, however, in 75% of the cases, there was a therapeutic alternative. In 13.6% of the actions, at least 1 medicine was prescribed for off-label use. Even the judicialized drugs that were part of the National List of Essential Medicines (Rename), were constantly requested for indications not recommended in official protocols. In 68% of the cases, access to the drug was available, with the author responsible for the purchase in 56.1% of them, through blocking public funds. The most requested medicine was insulin glargine (74 lawsuits). Drugs not available in SUS and frequently requested in court demands tend to be incorporated later, becoming part of health policy. Of the 10 drugs with the highest number of lawsuits, 4 were subsequently incorporated into SUS, mainly insulin analogues. Regarding the process of incorporating these drugs, it was observed that internal demands predominated (60%), and the minority had economic assessments (40%), the main justification used to support decisions being the presence of scientific evidence. However, a change of position was observed after the expressive participation in public consultations and the judicialization of medicines is mentioned in some reports. The results showed that the judicial system has been consolidated as a way of accessing medicines in Rio Grande do Norte, including by citizens residing in the interior of the State, who obtain free legal assistance. The most of the drugs requested were not yet incorporated into SUS, and these requests frequently violated health and Pharmaceutical Assistance management rules. The blocking of public funds for the fulfillment of these decisions is another worrying factor for the SUS manager, as it compromises the execution of the programmed medicines policies, which impair their execution. In the process of incorporating drugs targeted by lawsuits, it was observed that despite the preponderance of the technical-scientific basis in decisions to incorporate drugs into SUS, there was an indirect influence of judicialization in the decision-making process (AU).

Public engagement in health technology assessment in Brazil: the case of the Trastuzumab public consultation.

BACKGROUND: Public engagement in health technology assessment (HTA) is increasing worldwide. There are several forms of public engagement and it is not always possible to determine which stakeholders participate in the HTA process and how they contribute. Our objective was to investigate which types of social representatives contributed to the public consultation on the incorporation of Trastuzumab for early-stage breast cancer treatment within the public health system in Brazil, held in 2012 by the National Committee for Health Technology Incorporation (CONITEC). METHODS: A mixed methods approach was used to analyze social representativeness and the composition of the corpus from the public consultation, which consisted of 127 contributions. Three types of analysis were performed using IRaMuTeQ software: classic lexical analysis, descending hierarchical classification and specificities analysis. The contributions were clustered according to the main categories of discourse observed, into four social representation categories: 1) patient representation/advocacy; 2) pharmaceutical industry/advocacy; 3) healthcare professionals; and 4) individual contributions. RESULTS: Category 1 contained words related to increased survival due to use of the drug and a low score for words pertaining to studies on Trastuzumab. The word "safety" obtained a positive score only in category 2, which was also the only category that exhibited a negative score for the word "risk". Category 3 displayed the lowest scores for "diagnosis" and "safety". The word "efficacy" had a negative score only in category 4. CONCLUSIONS: Each category exhibited different results for words related to health systems and to key concepts linked to HTA. Our analysis enabled the identification of the most prominent contributions for each category. Despite the promising results obtained, further research is needed to validate this software for use in analyzing public contributions.

Contribuições das tecnologias em saúde para a segurança do paciente / Contribuciones de las tecnologías en salud para la seguridad del paciente / Contributions of Health Technologies to Patient Safety

RESUMO Introdução: O uso das tecnologias no campo da saúde é uma realidade e colabora para a promoção da segurança do paciente. Objetivo: Identificar na literatura científica as relações entre os tipos de tecnologias e a segurança do paciente. Métodos: Trata-se de uma revisão integrativa, realizada entre novembro e dezembro de 2016, a partir de buscas nas bases Cumulative Index to Nursing & Allied Health Literature, Web of science, SCOPUS e Literatura Latino-americana e do Caribe em Ciências da Saúde. O levantamento dos artigos foi feito com o uso dos descritores controlados em ciências da saúde: segurança do paciente; tecnologias em saúde; serviços de saúde e os MeSH: patient safety; technology; health services. Incluíram-se as publicações disponíveis gratuitamente em texto completo e em meio eletrônico; artigos que abordam a temática de interesse nos idiomas português, inglês e espanhol e sem delimitação temporal. Foram excluídas do estudo as publicações em formato de editoriais, carta ao editor, artigos de revisão e de opinião e os artigos duplicados. Os artigos selecionados foram analisados por estatística descritiva e apresentados em quadros. Conclusão: Apesar de ter sido evidenciado que o uso das tecnologias melhora a segurança e a qualidade no cuidado, é necessária maior abordagem em relação às tecnologias leves e leve-duras, uma vez que as mesmas são indispensáveis para o cuidado(AU)

RESUMEN Introducción: El uso de tecnologías en el campo de la salud es una realidad y contribuye a la promoción de la seguridad del paciente. Objetivo: Identificar, en la literatura científica, la relación entre los tipos de tecnologías y la seguridad del paciente. Métodos: Se trata de una revisión integrativa realizada entre noviembre y diciembre de 2016, a partir de la búsqueda en las bases Cumulative Index to Nursing & Allied Health Literature, Web of science, SCOPUS y Literatura Latinoamericana y del Caribe en Ciencias de la Salud. La acumulación de los artículos se hizo con el uso de los descriptores controlados en ciencias de la salud: seguridad del paciente; tecnologías en salud; servicios de salud y MeSH: patient safety; tecnología; servicios de salud. Se incluyeron las publicaciones disponibles gratuitamente en texto completo y en medio electrónico; artículos que abordan la temática de interés en los idiomas portugués, inglés y español, sin delimitación temporal. Se excluyeron del estudio las publicaciones en formato de editoriales, carta al editor, artículos de revisión y de opinión y los artículos duplicados. Los artículos seleccionados fueron analizados por estadística descriptiva y presentados en cuadros. Conclusión: A pesar de que se ha evidenciado que el uso de las tecnologías mejora la seguridad y la calidad en el cuidado, es necesario un mayor enfoque en las tecnologías leves y leve-dura, una vez que las mismas son indispensables para el cuidado(AU)

ABSTRACT Introduction: The use of technologies in the field of health is a reality and contributes to the promotion of patient safety. Objective: To identify, in the scientific literature, the relationship between the types of technologies and patient safety. Methods: This is an integrative review carried out between November and December 2016, based on the search in the databases Cumulative Index to Nursing & Allied Health Literature, Web of science, SCOPUS, and Latin American and Caribbean Literature in Health Sciences. The gathering of articles was carried out with the use of descriptors controlled within health sciences: seguridad del paciente [patient safety], tecnologías en salud [health technologies], servicios de salud [health services], and MeSH. Publications available for free in full text and electronic media were included, together with articles that address the topic of interest in the Portuguese, English and Spanish languages, without temporal delimitation. Publications in editorial format, letter to the editor, review and opinion articles and duplicate articles were excluded from the study. The selected articles were analyzed by descriptive statistics and presented in tables. Conclusion: Although it has been shown that the use of technologies improves safety and quality in care, a greater focus on mild and mild-hard technologies is necessary, once they are essential for care(AU)

[Incorporation of technologies by the Canadian and Brazilian health systems: prospects for progress in assessment processes]. / Incorporação de tecnologias nos sistemas de saúde do Canadá e do Brasil: perspectivas para avanços nos processos de avaliação.

One of the main challenges for modern health systems is to guarantee equitable access to technologies with proven quality, safety, efficacy, and cost-effectiveness, as well as to ensure that their use is based on high-quality scientific evidence. Health technology assessment (HTA) is one of the most widely used strategies in the world to support decisions on health technologies. The article analyzes how HTA systems are organized in Brazil and Canada and discusses the implications for planning the incorporation of technologies in Brazil, considering the challenges posed by the regionalization process and the establishment of healthcare networks. This is an exploratory comparative study based on secondary data. The results show that both countries have fragmented HTA systems with different levels of maturity. The systems are characterized by multiple organizations working in the field of HTA, the scope of activities, and the concentration of activities in national agencies/bodies. Both systems have weaknesses, but the Brazilian case presents a series of factors (insufficient resources, impact of court rulings, heavy dependence on foreign technologies, and incipient regional HTA processes and planning) that make the scenario more complex. The article argues that the regionalized structure for planning the incorporation of technologies in Canada can serve as an interesting experience for the Brazilian system, despite the different contexts in the two countries.

Garantir o acesso equitativo a tecnologias que têm qualidade, segurança, eficácia e custo-efetividade comprovados, bem como assegurar que sua utilização seja baseada em evidências científicas de qualidade, constitui um dos principais desafios dos modernos sistemas de saúde. A avaliação de tecnologias em saúde (ATS) é uma das estratégias mais usadas em todo o mundo para apoiar a tomada de decisão relativa às tecnologias em saúde. O objetivo do artigo é examinar como os sistemas de ATS estão organizados no Brasil e no Canadá e discutir suas implicações para o planejamento da incorporação de tecnologias no Brasil, considerando os desafios impostos pelo processo de regionalização e da constituição das redes de atenção à saúde. Trata-se de um estudo exploratório, em perspectiva comparada, com base em dados secundários. Os resultados mostram que os dois países contam com sistemas de ATS fragmentados, com níveis diferenciados de maturidade. Caracterizam-se pela multiplicidade de organizações que atuam no campo da ATS, pela abrangência do escopo das atividades desenvolvidas e pela concentração das atividades em agências/órgãos nacionais. Os dois sistemas têm fragilidades, mas o caso brasileiro apresenta um conjunto de fatores (recursos insuficientes, impacto das decisões judiciais, forte dependência de tecnologias provenientes do exterior, e processos e planejamentos regionais incipientes no campo da ATS) que torna o cenário mais complexo. Argumenta-se que a estrutura regionalizada para o planejamento da incorporação de tecnologias no Canadá pode ser uma experiência interessante para o sistema brasileiro, a despeito das diferenças de contexto entre os dois países.

Garantizar el acceso equitativo a tecnologías que poseen calidad, seguridad, eficacia y costo-efectividad comprobados, así como asegurar que su utilización esté basada en evidencias científicas de calidad, constituye uno de los principales desafíos de los sistemas de salud modernos. La evaluación de tecnologías en salud (ETS) es una de las estrategias más usadas en todo el mundo para apoyar en la toma de decisiones relativa a las tecnologías en salud. El objetivo del artículo es examinar cómo los sistemas de ETS están organizados en Brasil y en Canadá y discutir sus implicaciones para la planificación de la incorporación de tecnologías en Brasil, considerando los desafíos impuestos por el proceso de regionalización y la constitución de las redes de atención a la salud. Se trata de un estudio exploratorio, desde una perspectiva comparada, basado en datos secundarios. Los resultados muestran que los dos países cuentan con sistemas de ETS fragmentados, con niveles diferenciados de madurez. Se caracterizan por la multiplicidad de organizaciones que actúan en el campo de la ETS, por la amplitud del alcance de las actividades desarrolladas y por la concentración de las actividades en agencias/órganos nacionales. Los dos sistemas tienen fragilidades, pero el caso brasileño presenta un conjunto de factores (recursos insuficientes, impacto de decisiones judiciales, fuerte dependencia de tecnologías provenientes del exterior, y procesos y planificaciones regionales incipientes en el campo de la ETS) que hacen que el escenario sea más complejo. Se argumenta que la estructura regionalizada para la planificación de la incorporación de tecnologías en Canadá puede ser una experiencia interesante para el sistema brasileño, a pesar de las diferencias de contexto entre ambos países.

Health technology reassessment in the Brazilian public health system: Analysis of the current status.

BACKGROUND: The reassessment of technologies and services offered by healthcare systems is recent initiative and still without a widely adopted and evaluated method. To a better understanding of this process in Brazil, we have described the health technology reassessment (HTR) performed by the National Committee for Health Technology Incorporation (Conitec) into Brazilian public health system (SUS). METHODS: A documental, exploratory, descriptive, retrospective study with qualitative-quantitative approach regarding the HTR performed by Conitec from January 2012 to November 2017. RESULTS: After applying the criteria of inclusion and exclusion, we selected 47 technologies for this study. The vast majority of the demands (41 demands) came from the public sector, and only six from the private sector. Most of the requests referred to the exclusion of specific indication; followed by extension of use, withdraw of the technology from SUS, maintenance, and restriction of use. The dimensions of analysis found in the recommendation reports were scientific evidence on efficacy, effectiveness and safety, disease-related issues, issues related to the use of technology, costs, and social participation. However, these dimensions were not included in all analysis, and a standardized structure of the reports has not been observed. The most relevant decision factors considered for decision-making were efficacy, safety and use of the technology. CONCLUSION: During a six-year period of Conitec actuation, we could find some reassessments of technologies that are available in SUS. We observed that these activities had enabled progress, however, they are still not yet structured, with gaps in the selection process, and the assessment since no methodology and criteria for proper conduct were established.

Cost-effectiveness of infusion pumps to reduce errors in a Pediatric ICU.

OBJECTIVE: To analyze cost-effectiveness and to calculate incremental cost-effectiveness ratio of the use of infusion pumps with drug library to reduce errors in intravenous drug administration in pediatric and neonatal patients in Intensive Care Units. METHODS: Mathematical modeling for economic analysis of the decision tree type. The base case was composed of reference and alternative settings. The target population was neonates and pediatric patients hospitalized in Pediatric and Neonatal Intensive Care Units, comprising a cohort of 15,034 patients. The cost estimate was based on the bottom-up and top-down approaches. RESULTS: The decision tree, after RollBack, showed that the infusion pump with drug library may be the best strategy to avoid errors in intravenous drugs administration. CONCLUSION: The analysis revealed that the conventional pump, although it has the lowest cost, also has lower effectiveness.

International experiences in multicriteria decision analysis (MCDA) for evaluating orphan drugs: a scoping review.

Introduction: Orphan diseases are low-prevalence conditions with chronically debilitating or life-threatening consequences. Their treatments are generally called orphan drugs (OD). Health-technology assessment processes have traditionally considered cost-effectiveness analysis (CEA), when making reimbursement and pricing decisions for health-care plans. Valuing OD with standard CEA raises important issues due to uncertain evidence, inability to meet cost-effectiveness thresholds for reimbursement and high budget impact, among others. Multi-criteria decision analysis (MCDA) allows to overcome these issues and improve the technical and ethical quality of decisions regarding prioritization, coverage, and reimbursement of OD. Areas covered: A scoping review was conducted in order to characterize MCDA frameworks for assessing OD and implementation experiences. We reviewed electronic databases (Medline, Embase, Cochrane Library, EBSCO, CINAHL, EconLit, Web of Science, LILACS, Google Scholar) key journals (Orphanet Journal of Rare Diseases and Value in Health) and organization repositories. Expert opinion: The theoretical framework for MCDA considers areas related to characteristics of orphan diseases and their technologies' clinical and economic impact. Participation processes are critical in incorporating societal values in weighting different dimensions and constructing decision rules. Local implementation pilots considering different stakeholders are necessary in order to pinpoint specific barriers and opportunities.

Health technology assessment in Brazil - an international perspective. / O processo de incorporação de tecnologias em saúde no Brasil em uma perspectiva internacional.

Given the financial impact of the adoption of new health technologies in health systems, choosing what technology should be introduced and when poses a major challenge for health managers. The health technology assessment (HTA) process should therefore be underpinned by transparent and objective criteria. The objective of this study was to analyze HTA processes in Brazil, overseen by the National Commission for the Incorporation of Health Technology (CONITEC), and to compare these processes with those in countries considered to be at the forefront of this field: Australia, Canada, and the United Kingdom. The following categories were used for the comparative analysis: program structure, definition and selection of topics, evidence review, use of HTA in decision making, program products and dissemination, and transparency. The findings show that there are more similarities than differences between these countries' processes and the CONITEC processes. The main differences identified were: composition of committees, entitlement to appeal, program evaluation, and timeframes for the implementation of recommendations/decisions. Despite making major strides in recent years, Brazil should continue to promote continuous improvement of its HTA process.

Dado o impacto financeiro da incorporação de novas tecnologias em saúde, é um desafio para os gestores escolher qual delas deve ser incorporada e quando isto deve ocorrer. Assim, é necessário contar com um processo de avaliação e de incorporação de tecnologias baseado em critérios transparentes e objetivos. Neste trabalho objetivou-se analisar o processo nacional da Comissão Nacional de Incorporação de Tecnologias em Saúde do Ministério da Saúde (Conitec) e compará-lo com o de agências de países de referência: Austrália, Canadá e Reino Unido. Utilizaram-se as seguintes categorias para a comparação: estrutura, indicação e seleção de temas, condução da revisão de evidências, uso de Avaliação Tecnológica em Saúde (ATS) na tomada de decisão, produtos do programa de ATS, divulgação e transparência. O processo da Conitec legalmente previsto apresentou mais similaridades do que distinções em comparação com os das agências estudadas. As principais diferenças foram em relação a: composição dos comitês, apresentação de recursos, avaliação do programa, seleção e prazos para oferta da tecnologia incorporada. Apesar dos avanços, a incorporação de tecnologias em saúde no Brasil deve buscar a melhoria contínua.

Cost-effectiveness of infusion pumps to reduce errors in a Pediatric ICU / El costo-efectividad de bombas de infusión para la reducción de errores en una UTI Pediátrica / Custo-efetividade de bombas de infusão para a redução de erros em uma UTI Pediátrica

ABSTRACT Objective: To analyze cost-effectiveness and to calculate incremental cost-effectiveness ratio of the use of infusion pumps with drug library to reduce errors in intravenous drug administration in pediatric and neonatal patients in Intensive Care Units. Methods: Mathematical modeling for economic analysis of the decision tree type. The base case was composed of reference and alternative settings. The target population was neonates and pediatric patients hospitalized in Pediatric and Neonatal Intensive Care Units, comprising a cohort of 15,034 patients. The cost estimate was based on the bottom-up and top-down approaches. Results: The decision tree, after RollBack, showed that the infusion pump with drug library may be the best strategy to avoid errors in intravenous drugs administration. Conclusion: The analysis revealed that the conventional pump, although it has the lowest cost, also has lower effectiveness.

RESUMEN Objetivo: Analizar el costo-efectividad y calcular la razón de costo-efectividad incremental del uso de bombas de infusión con una biblioteca de fármacos para reducir errores en la administración de medicamentos por vía intravenosa, en pacientes pediátricos y neonatales en unidades de terapia intensiva. Método: Modelaje matemático para el análisis económico, del tipo árbol de decisión. El caso base se compone de escenarios de referencia y alternativo. La población objetivo fueron pacientes neonatos y pediátricos internados en unidades de terapia intensiva pediátrica y neonatal, componiendo una cohorte de 15.034 pacientes. La estimación de costos se basó en los enfoques bottom-up y top-down. Resultados: El árbol de decisión, después de Roll Back, mostró que la bomba de infusión con biblioteca de fármacos puede ser la mejor estrategia para evitar errores en la administración de medicamentos intravenosos. Conclusión: El análisis reveló que la bomba convencional, aunque tiene el menor costo, tiene también menor efectividad.

RESUMO Objetivo: Analisar o custo-efetividade e calcular a razão de custo-efetividade incremental do uso de bombas de infusão com biblioteca de fármacos para reduzir erros na administração de medicamento pela via intravenosa, em pacientes pediátricos e neonatais em Unidades de Terapia Intensiva. Método: Modelagem matemática para análise econômica, do tipo árvore de decisão. O caso-base foi composto pelos cenários de referência e alternativo. A população alvo foram pacientes neonatos e pediátricos internados em Unidades de Terapia Intensiva pediátrica e neonatal, compondo uma coorte de 15.034 pacientes. A estimativa de custos foi baseada nas abordagens bottom-up e top-down. Resultados: A árvore de decisão, após RollBack, mostrou que a bomba de infusão com biblioteca de fármacos pode ser a melhor estratégia para evitar erros na administração de medicamentos intravenosos. Conclusão: A análise revelou que a bomba convencional, embora tenha o menor custo, tem também menor efetividade.