Política de Ciência, Tecnologia e Inovação em Saúde (CT& I/S): uma atualização para debate / Health Science, Technology and Innovation Policy (ST& I/H): an update for debate

Resumo O texto contém uma proposta atualizada de política de Ciência, Tecnologia e Inovação em Saúde no Brasil e a pertinência da atualização decorre do desastre nas atividades nesse terreno verificadas no país desde o início do atual governo federal em 2019, bem como dos desafios colocados pela emergência da pandemia COVID-19 desde 2020. Ele está organizado em cinco seções, a saber: Pesquisa em Saúde; Inovação Produtiva; Avaliação e Incorporação de Tecnologias em Saúde; Propriedade Intelectual em Saúde; Novos desafios colocados pela Pandemia. Os autores fazem parte do Comitê de Assessoramento em Ciência, Tecnologia e Inovação da Abrasco.

Abstract The text presents an updated proposal for a Health Science, Technology and Innovation Policy in Brazil, following the huge political turmoil in the country since 2019 and the COVID-19 pandemic since 2020. The proposal is presented in five sections: Scientific Research; Productive Innovation; Health Technology Assessment and Incorporation; Intellectual Property in Health; New challenges posed by the Pandemic. The authors take part in the Advisory Committee in Science, Technology and Innovation of the Brazilian Association of Collective Health.

Ranking the Criteria Used in the Appraisal of Drugs for Reimbursement: A Stated Preferences Elicitation With Health Technology Assessment Stakeholders Across Jurisdictional Contexts.

OBJECTIVES: Our goal was to estimate the relative importance assigned to health technology assessment (HTA) criteria by stakeholders involved in the HTA process. HTA is an increasingly common framework used in the appraisal of drugs for public reimbursement. It identifies clinical, economic, social, and organizational criteria to be considered. The criteria can vary across jurisdictions and are typically appraised by multidisciplinary expert committees. Guidance on the relative weighing of criteria is often absent. METHODS: We elicited stakeholders' preferences using a single-scenario discrete choice experiment and a best-worst scaling model with conviction scores to assess the weights assigned to selected criteria by HTA stakeholders. We recruited 111 HTA stakeholders across multiple jurisdictions, including members of expert committees, clinical and economic experts, patients, and public payer representatives. Each judged twelve hypothetical cancer drug profiles for suitability for public funding and identified which characteristics were best and worst. In addition to standard discrete choice experiment and best-worst scaling models, we estimated a hybrid model to obtain a ranking of criteria by importance they played in the appraisal. RESULTS: A strong clinical benefit proved the most important criterion, followed by cost considerations, presence of adverse events, and availability of other treatments. The importance of clinical benefit was moderated by unmet need, adverse events, and number of patients. CONCLUSION: Policymakers might want to consider providing an explicit weighing scheme, or moving to a 2-stage selection process with an assessment of the quality of clinical evidence as a gatekeeping step for a full HTA review.

Estado del Arte de la Evaluación de Tecnologías en Salud en América Latina / State of the Art of the Evaluation of Health Technologies in Latin America

Resumen El surgimiento y desarrollo de las tecnologías en salud ha hecho que se intensifique el papel de su evaluación en los últimos años, provocando un interés creciente en la Evaluación de Tecnologías en Salud (ETES) en América Latina, a través de intentos por proporcionar información útil al tomador de decisiones. Este artículo presenta una revisión del estado del arte de ETES en Latinoamérica, a partir del análisis de publicaciones en revistas y eventos especializados. Se abordaron tres aspectos: elementos de evaluación, métodos que se aplican y políticas resultantes. Se encontraron los siguientes aspectos de evaluación: seguridad, eficacia, efectividad y eficiencia, económico-financieros, clínicos y técnicos, estos últimos con mayor desarrollo. Con respecto a los métodos, se encontraron propuestas de estrategias empíricas para el análisis de la información y la toma de decisiones. La generación de políticas públicas relacionadas con la ETES en América Latina es incipiente, apenas se están identificando las problemáticas nacionales y las estrategias a seguir para su solución. Por lo tanto, se aprecia una necesidad de seguir trabajando en el desarrollo de políticas, estrategias y métodos de ETES en la región Latinoamericana que permitan responder a las problemáticas en salud de la población de cada país.

Abstract The emergence and development of health technologies have intensified the role of their evaluation in recent years, causing a growing interest in the Evaluation of Health Technologies (ETES) in Latin America, through attempts to provide useful information to the decision-maker. This article presents a review of the state of the art of ETES in Latin America, from the analysis of publications in journals and specific events. Three aspects were addressed: evaluation elements, methods that are applied, and resulting policies. The following evaluation aspects were found: safety, efficacy, effectiveness and efficiency, economic-financial, clinical and technical, the latter with more significant development. Concerning the methods, proposals for empirical strategies for the analysis of information and decision making were found. The generation of public policies related to the HTA in Latin America is under development, national problems are hardly being identified, and the strategies to be followed for their solution. Therefore, there is a need to continue working on the development of policies, and methods of HTA in the Latin American region that allow responding to the health problems of the population of each country.

Drug attributes associated with the selection of drugs for reimbursement: a pilot stated preferences experiment with Canadian stakeholders.

INTRODUCTION: Health Technologies Assessment requires that evidence about clinical, economic, social, and organizational aspects be considered and weighted in the selection of drugs for reimbursement. We investigate how evidence is balanced by committee members in Canada, where neither explicit weighing schemes nor thresholds are provided. METHODS: Thirty-six past and present members of cancer drug appraisal committees participated in an online stated preferences experiment. The experiment included a ranking of drug attributes, a discrete choice experiment asking to vote in favor or against the funding of drugs described using five attributes, and a best-worst scaling experiment using the same drug descriptions. RESULTS: Respondents focused on the clinical attributes of drugs, particularly on the survival benefit relative to a comparator. As a second criterion, respondents either consider economic attributes or they consider patient relevant attributes, depending on how questions are framed. The small sample size is a limitation to generalizability. CONCLUSION: Understanding how individuals involved in HTA weigh evidence is important to the development of policy guidelines for the drug selection process. Our pilot results suggest that non-clinical criteria can become marginalized in the appraisal process in the absence of clear guidelines to their use. Avenues for further research are discussed.

Requests for post-registration studies (PRS), patients follow-up in actual practice: Changes in the role of databases.

Early market access of health products is associated with a larger number of requests for information by the health authorities. Compared with these expectations, the growing expansion of health databases represents an opportunity for responding to questions raised by the authorities. The computerised nature of the health system provides numerous sources of data, and first and foremost medical/administrative databases such as the French National Inter-Scheme Health Insurance Information System (SNIIRAM) database. These databases, although developed for other purposes, have already been used for many years with regard to post-registration studies (PRS). The use thereof will continue to increase with the recent creation of the French National Health Data System (SNDS [2016 health system reform law]). At the same time, other databases are available in France, offering an illustration of "product use under actual practice conditions" by patients and health professionals (cohorts, specific registries, data warehouses, etc.). Based on a preliminary analysis of requests for PRS, approximately two-thirds appeared to have found at least a partial response in existing databases. Using these databases has a number of disadvantages, but also numerous advantages, which are listed. In order to facilitate access and optimise their use, it seemed important to draw up recommendations aiming to facilitate these developments and guarantee the conditions for their technical validity. The recommendations drawn up notably include the need for measures aiming to promote the visibility of research conducted on databases in the field of PRS. Moreover, it seemed worthwhile to promote the interoperability of health data warehouses, to make it possible to match information originating from field studies with information originating from databases, and to develop and share algorithms aiming to identify criteria of interest (proxies). Methodological documents, such as the French National Authority for Health (HAS) recommendations on "Les études post-inscription sur les technologies de santé (médicaments, dispositifs médicaux et actes). Principes et méthodes" [Post-registration studies on health technologies (medicinal products, medical devices and procedures). Principles and methods] should be updated to incorporate these developments.

Herramienta de evaluación de tecnologías para la adquisición de equipos biomédicos / Health technology assessment tools for biomedical equipment acquisition / Ferramenta de avaliação de tecnologias para a aquisição de equipamentos biomédicos

La Evaluación de Tecnologías en Salud es una práctica que permite a las Instituciones de Salud valorar de forma integral las consecuencias del uso de una tecnología, sin embargo, la falta de estandarización y de metodologías que guíen el proceso sigue siendo un reto por superar. Este trabajo presenta el desarrollo y la validación de una herramienta de Evaluación de Tecnologías en Salud la cual se realizó en tres fases: primero se desarrolló la herramienta que permite valorar el proceso de adquisición de equipos biomédicos, después se conformó un grupo de discusión para obtener diferentes percepciones frente a la metodología empleada, el diseño y desarrollo de la herramienta para finalmente implementarla mediante casos de estudio evaluando su uso real y aplicando una encuesta que permitió valorar su utilidad en los procesos de adquisición de equipos biomédicos. Se ponderó la importancia de cada tipo de evaluación de tecnologías seleccionada y se desarrollaron los módulos para las evaluaciones técnica, clínica, económica y ética, social y organizacional. Por su parte, la validación con expertos demostró la utilidad de la herramienta en los procesos de selección y adquisición de equipos biomédicos de manera que se disminuyan costos y se realicen mejores inversiones.

Health Technologies Assessment is a practice that enablesthe evaluation of the consequences of the use of a technology. However, the lack of standardization and methodologies that guide the process remains a challenge. This paper presents the development and validation of a Health Technology Assessment tool which process was carried out in three phases. First, the tool was developed to assess the acquisition process of medical equipment, after which a discussion group was formed to obtain different perceptions regarding the tool. Finally,was the implementation through case studies, evaluating its real use and applying an evaluation survey to measure its usefulness in the processes of acquisition of medical equipment. The importance of each type of technology assessment was weighed and modules for technical, clinical, economic, ethical, social and organizational evaluation were developed. In addition, validation with experts demonstrated the usefulness of the tool in the selection and acquisition processes of medical equipment in a way that will reduce costs and enable better investments.

A Avaliação de Tecnologias em Saúde é uma prática que permite às Instituições de Saúde valorizar de forma integral as consequências do uso de uma tecnologia, no entanto, a falta de estandardização e de metodologias que guiem o processo segue sendo um desafio por superar. Este trabalho apresenta o desenvolvimento e a validação de uma ferramenta de Avaliação de Tecnologias em Saúde a qual se realizou em três fases: primeiro desenvolveu-se a ferramenta que permite valorizar o processo de aquisição de equipamentos biomédicos, depois se conformou um grupo de discussão para obter diferentes percepções em frente à metodologia empregada, o desenho e desenvolvimento da ferramenta para finalmente implementá-la mediante casos de estudo avaliando seu uso real e aplicando uma pesquisa que permitiu valorizar sua utilidade nos processos de aquisição de equipamentos biomédicos. Ponderou-se a importância de cada tipo de avaliação de tecnologias selecionadas e desenvolveram-se os módulos para as avaliações técnica, clínica, económica, ética, social e organizacional. Por sua vez, a validação com experientes demonstrou a utilidade da ferramenta nos processos de seleção e aquisição de equipamentos biomédicos de maneira que se diminuam custos e se realizem melhores investimentos.