La thèse examine les configurations d'acteurs et d'organisations impliquées dans la régulation de l'accès au marché des médicaments en France et en Angleterre, en charge du contrôle scientifique et administratif de la fixation du prix et du remboursement de ces produits. Cette activité s'autonomise de l'appréciation clinique de leur qualité, de leur efficacité et de leur innocuité (dominant la régulation de leur approbation sur le marché) au cours des années 1980. / This thesis examines the configurations of actors and organizations involved in the regulation of the market access for drugs in France and England. Since the mid-1980s, this process has entailed the development of administrative and scientific controls over the fixing of the price and the conditions of reimbursement of these products. These controls have become autonomus from clinical appreciation of their quality, efficacity and safety that dominate approval regulation as a whole.

Contexte : La pression croissante sur les soins primaires nécessite de développer des approches adaptées pour apprécier la valeur des innovations dans ce domaine et identifier les conditions favorables à leur diffusion, ce qui peut s'avérer difficile, puisque ces innovations sont souvent complexes et de nature organisationnelle. Objectifs : Explorer les arguments de valeur de 10 innovations identifiées comme prometteuses lors des symposiums sur les innovations du Collège québécois des médecins de famille et proposer des pistes pour leur bonification et diffusion. Méthodes : Approche délibérative multidimensionnelle avec un panel diversifié de 12 cliniciens, gestionnaires, patients et citoyens. À partir de données synthétisées par dimension, chaque participant a apprécié le potentiel de valeur des innovations par un score et des arguments justificatifs pour chaque dimension individuellement et suite à une délibération. Le potentiel de valeur moyen a été calculé par innovation, les arguments ont fait l’objet d’une synthèse qualitative. Ces résultats ont informé une seconde délibération visant à identifier des pistes d’amélioration et diffusion qui ont été organisées par analyse thématique. Résultats : Les innovations d’adaptation de l'organisation des soins aux populations vulnérables ont été considérées comme ayant le plus grand potentiel de valeur dans l’ensemble. Les innovations visant l’optimisation des processus cliniques et d’amélioration continue ont reçu des appréciations plus variables. 460 pistes ont été classées en six catégories : objectifs, modalités d’accès, fonctionnement, impacts, gouvernance et contexte externe. Conclusions : L’exploration du potentiel de valeur d’innovations complexes avec une approche délibérative multidimensionnelle permet d’identifier, pour certaines catégories d’innovations en soins primaires, des arguments de valeur communs, et facilite l’identification de pistes d’amélioration et diffusion. / Context: The increasing pressure on primary care calls for the development of appropriate approaches to assess the value of innovations in this field and identify the conditions for their diffusion, which can be difficult, as these innovations are often complex and organizational in nature. Objectives: To explore the value arguments of 10 innovations identified as promising during the Quebec College of Family Physicians' Innovation Symposia and to propose avenues for their improvement and diffusion. Methods: Multidimensional deliberative approach with a diverse panel of 12 clinicians, managers, patients and citizens. Using data synthesized by dimension, each participant assessed the value potential of the innovations by providing a score and supporting arguments for each dimension individually, and following group deliberation. The average value potential was calculated per innovation, and the arguments were qualitatively synthesized. These results informed a second deliberation aimed at identifying avenues for improvement and deployment that were organized by thematic analysis. Results: Innovations aimed at adapting the organization of care for vulnerable populations were considered to have the greatest value potential overall, while interventions aimed at optimizing clinical processes and continuous improvement received more variable appraisals. 460 avenues were categorized into six areas: objectives, access modalities, operations, impacts, governance, and external context. Conclusions: Exploring the value potential of complex innovations with a multidimensional deliberative approach allowed the identification of common value arguments for certain categories of primary care innovations and facilitated the identification of pathways for their improvement and dissemination.

Objectif principal: Il n’est pas démontré que les interventions visant à maîtriser voire modérer la médicamentation de patients atteints d’hypertension peuvent améliorer leur gestion de la maladie. Cette revue systématique propose d’évaluer les programmes de gestion contrôlée de la médicamentation pour l’hypertension, en s’appuyant sur la mesure de l’observance des traitements par les patients (CMGM). Design: Revue systématique. Sources de données: MEDLINE, EMBASE, CENTRAL, résumés de conférences internationales sur l’hypertension et bibliographies des articles pertinents. Méthodes: Des essais contrôlés randomisés (ECR) et des études observationnelles (EO) ont été évalués par 2 réviseurs indépendants. L’évaluation de la qualité (de ce matériel) a été réalisée avec l’aide de l’outil de Cochrane de mesure du risque de biais, et a été estimée selon une échelle à quatre niveaux de qualité Une synthèse narrative des données a été effectuée en raison de l'hétérogénéité importante des études. Résultats: 13 études (8 ECR, 5 EO) de 2150 patients hypertendus ont été prises en compte. Parmi elles, 5 études de CMGM avec l’utilisation de dispositifs électroniques comme seule intervention ont relevé une diminution de la tension artérielle (TA), qui pourrait cependant être expliquée par les biais de mesure. L’amélioration à court terme de la TA sous CMGM dans les interventions complexes a été révélée dans 4 études à qualité faible ou modérée. Dans 4 autres études sur les soins intégrés de qualité supérieure, il n'a pas été possible de distinguer l'impact de la composante CMGM, celle-ci pouvant être compromise par des traitements médicamenteux. L’ensemble des études semble par ailleurs montrer qu’un feed-back régulier au médecin traitant peut être un élément essentiel d’efficacité des traitements CMGM, et peut être facilement assuré par une infirmière ou un pharmacien, grâce à des outils de communication appropriés. Conclusions: Aucune preuve convaincante de l'efficacité des traitements CMGM comme technologie de la santé n’a été établie en raison de designs non-optimaux des études identifiées et des ualités méthodologiques insatisfaisantes de celles-ci. Les recherches futures devraient : suivre les normes de qualité approuvées et les recommandations cliniques actuelles pour le traitement de l'hypertension, inclure des groupes spécifiques de patients avec des problèmes d’attachement aux traitements, et considérer les résultats cliniques et économiques de l'organisation de soins ainsi que les observations rapportées par les patients. / Objective: Whether interventions including measurement and correction of patients’ attitude to antihypertensive medication can improve hypertension management is unclear. The review aims to determine the effectiveness of patient compliance measurement-guided medication management (CMGM) programs in essential hypertension. Design: Systematic review. Data sources: MEDLINE, EMBASE, CENTRAL, hypertension meetings abstracts, and bibliographies of identified articles. Methods: Randomized controlled trials (RCT) and observational studies (OS) were assessed by 2 reviewers independently. Quality assessment was performed with the Cochrane risk of bias tool and evaluated in a four-point continuum. A narrative data synthesis was performed due to significant heterogeneity among studies. Results: 13 studies (8 RCT, 5 OS) involving 2150 hypertensives were included. Five trials of CMGM with electronic devices as a sole intervention suggested decrease in blood pressure (BP) but the result may have been due to bias. Short-term BP improvement under CMGM in complex interventions was revealed in 4 studies of low-to-moderate quality. In 4 integrated care studies of higher quality the impact of CMGM component was not possible to distil and may be compromised by medication regimens. Regular feedback to the treating physician seems to be an essential component of CMGM and may be effectively mediated by a nurse or a pharmacist and via telecommunication. Conclusions: No convincing evidence for the effectiveness of CMGM as a health technology was found due to non-optimal study designs and methodological quality. Future research should follow accepted quality standards and current guidelines for the treatment of hypertension, include specific groups of patients with compliance problems and consider clinical, economic, patient-reported and organizational outcomes.

La littérature abordant les enjeux socio-éthiques et réglementaires associés aux médicaments est relativement abondante, ce qui n’est pas le cas des dispositifs médicaux (DM). Ce dernier secteur couvre une très large diversité de produits qui servent à de multiples applications: diagnostic, traitement, gestion des symptômes de certaines conditions physiques ou psychiatriques, restauration d’une fonction débilitante, chirurgie, etc. À tort, on a tendance à croire que les DM sont réglementés de la même manière que les médicaments, que ce soit pour les exigences concernant leur mise en marché ou des pratiques de surveillance après mise en marché. Or, au cours des dernières années, leur usage élargi, leur impact sur les coûts des soins de santé, et les rappels majeurs dont certains ont fait l’objet ont commencé à inquiéter la communauté médicale et de nombreux chercheurs. Ils interpellent les autorités réglementaires à exercer une plus grande vigilance tant au niveau de l’évaluation des nouveaux DM à risque élevé avant leur mise en marché, que dans les pratiques de surveillance après mise en marché. Une stratégie plus rigoureuse d’évaluation des nouveaux DM permettrait d’assurer un meilleur suivi des risques associés à leur utilisation, de saisir la portée des divers enjeux socio-éthiques découlant de l’utilisation de certains DM, et de préserver la confiance du public. D’emblée, il faut savoir que les autorités nationales n’ont pas pour mandat d’évaluer la portée des enjeux socio-éthiques, ou encore les coûts des DM qui font l’objet d’une demande de mise en marché. Cette évaluation est essentiellement basée sur une analyse des rapports risques-bénéfices générés par l’usage du DM pour une indication donnée. L’évaluation des impacts socio-éthiques et l’analyse coûts-bénéfices relèvent des agences d’Évaluation des technologies de santé (ÉTS). Notre recherche montre que les DM sont non seulement peu fréquemment évalués par les agences d’ÉTS, mais l’examen des enjeux socio-éthiques est trop souvent encore incomplet. En fait, les recommandations des rapports d’ÉTS sont surtout fondées sur une analyse coûts-bénéfices. Or, le secteur des DM à risque élevé est particulièrement problématique. Plusieurs sont non seulement porteurs de risques pour les patients, mais leur utilisation élargie comporte des impacts importants pour les systèmes de santé. Nous croyons que le Principisme, au cœur de l’éthique biomédicale, que ce soit au plan de l’éthique de la recherche que de l’éthique clinique, constitue un outil pour faciliter la reconnaissance et l’examen, particulièrement par les agences d’ÉTS, des enjeux socio-éthiques en jeu au niveau des DM à risque élevé. Également, le Principe de Précaution pourrait aussi servir d’outil, particulièrement au sein des agences nationales de réglementation, pour mieux cerner, reconnaître, analyser et gérer les risques associés à l’évaluation et l’utilisation de ce type de DM. Le Principisme et le Principe de Précaution pourraient servir de repères 1) pour définir les mesures nécessaires pour éliminer les lacunes observées dans pratiques associées aux processus de réglementation, et 2) pour mieux cerner et documenter les enjeux socio-éthiques spécifiques aux DM à risque élevé. / Socio-ethical and regulatory issues about drug development have received substantial coverage in the scientific literature over the years; this has not been the case of medical devices (MD). This latter sector comprises a vast array of products with a multitude of applications: diagnostics, treatment, symptom management for severe physical and psychiatric conditions, restoring physical functioning, surgery, etc. There is a mistaken view that MD are regulated in the same manner as pharmaceutical drugs, whether this be requirements regarding premarket assessment or post-market surveillance. Not only has the extensive use of MD in clinical practice and the resulting impact on healthcare expenditures raised concerns in the scientific and medical communities, but so too have the many recalls for high risk devices in recent years. Regulatory authorities have been challenged to exercise greater vigilance in both the premarket assessment and post-market surveillance of new high risk MD. A more rigorous evaluation strategy for new MD would permit better monitoring of the risks associated with their use, better understanding of the significance of the various socio-ethical issues arising from the use of MD, and thus preserve public confidence. From the outset, it should be noted that national authorities do not have a mandate to assess the significance of socio-ethical issues or the costs associated with the MD that are the subject of an application for marketing approval. This assessment is based primarily on risk-benefit analysis reports generated following the use of MD for a given indication. Evaluation of socioethical impact and cost-benefit analysis are the remit of Health Technology Assessment (HTA) agencies. But research shows that the MD are not only infrequently assessed by HTA agencies, but the consideration of socio-ethical issues is often incomplete; the recommendations of HTA reports are based mostly on a calculation of cost-benefit. However, the high risk MD sector is particularly problematic; many carry risks not only for patients, but their extended use has significant impact on healthcare systems. I argue that Principlism, associated to biomedical ethics, as much to research ethics and clinical ethics, could be adapted to facilitate the recognition and evaluation by HTA agencies of socio-ethical issues involved in high risk MD. The Precautionary Principle could also serve as a tool to help regulatory agencies to enforce understanding, recognition, analysis and management of the risks associated with the use of MD, especially high risk devices. Principlism and the Precautionary Principle could serve as benchmarks to 1) define necessary steps to eliminate gaps observed in the regulatory process, and 2) better understand and document the socio-ethical issues specific to high risk MD.

Health technology assessment: case study on breast carcinoma treatment in the Czech Republic Bc. Lenka Šlegerová January 4, 2019 Abstract This thesis proposes an original method for assessing total costs of med- ical treatment. It defines the semi-Markov model with four states that are associated with specific costs of the treatment, and not with patients' health statuses. This method is applied to individuals' treatment data drawn from the Czech clinical practice in the treatment of the metastatic HER2+ breast cancer. The aim is to assess the cost-effectiveness of adding medication per- tuzumab to the combination of trastuzumab+docetaxel within first-line therapy and to examine whether using individual data on Czech patients and the economic conditions leads to different results from foreign stud- ies. Furthermore, employing censored data from the clinical practice in the thesis complicates the estimation of patients' overall survival in compari- son to clinical-trials data that form random samples. Therefore, survival functions were not only estimated by the Kaplan-Meier estimator but also using the Cox proportional hazard model and the Accelerated failure time model that both control for the effects of included covariates. The addition of pertuzumab does not result in significantly longer pa- tients'...

Deux thématiques importantes des technologies de la santé: la pratique médicale fondée sur des preuves probantes et l’évaluation des interventions en médecine sont fondées sur une approche positiviste et une conception mécaniste des organisations en santé. Dans ce mémoire, nous soulevons l’hypothèse selon laquelle les théories de la complexité et la systémique permettent une conceptualisation différente de ces deux aspects de la gouvernance clinique d’une unité de Soins Intensifs Chirurgicaux (SIC), qui est considérée comme un système adaptatif dynamique non linéaire qui nécessite une approche systémique de la cognition. L’étude de cas d’une unité de SIC, permet de démontrer par de nombreux exemples et des analyses de micro-situations, toutes les caractéristiques de la complexité des patients critiques et instables et de la structure organisationnelle des SIC. Après une critique épistémologique de l’Evidence-Based Medicine nous proposons une pratique fondée sur des raisonnements cliniques alliant l’abduction, l’herméneutique et la systémique aux SIC. En nous inspirant des travaux de Karl Weick, nous suggérons aussi de repenser l’évaluation des modes d’interventions cliniques en s’inspirant de la notion d’organisation de haute fiabilité pour mettre en place les conditions nécessaires à l’amélioration des pratiques aux SIC. / In Health Technology Assessment and Management, Evidence-Based Medicine and many tools available for clinical assessment reflect a positivistic and mechanistic approach to Health Care Organizations and scientific knowledge. We argue that the Complexity Theories and the Systemic decision-making process give a different insight on those two aspects of Clinical Governance in a Surgical Intensive Care Unit (SICU). In a case-study, we describe the nature of critically ill and unstable patients and the organizational structure of a SICU in a university based hospital. We demonstrate all the characteristics of complexity in that setting, through the use of many examples and micro-situational analysis. After an epistemological critical appraisal of EBM, we suggest that if a SICU is conceptualized as a dynamic non-linear adaptative system, then clinical knowledge and scientific thought processes must include hermeneutical, systemic and abductive types of reasoning. Finally, we draw upon Karl Weick’s work and suggest that a SICU must be considered as a High Reliability Organization in order to aim for improving patient care and create better conditions for quality and performance in this complex environment.

Objectif principal: Il n’est pas démontré que les interventions visant à maîtriser voire modérer la médicamentation de patients atteints d’hypertension peuvent améliorer leur gestion de la maladie. Cette revue systématique propose d’évaluer les programmes de gestion contrôlée de la médicamentation pour l’hypertension, en s’appuyant sur la mesure de l’observance des traitements par les patients (CMGM). Design: Revue systématique. Sources de données: MEDLINE, EMBASE, CENTRAL, résumés de conférences internationales sur l’hypertension et bibliographies des articles pertinents. Méthodes: Des essais contrôlés randomisés (ECR) et des études observationnelles (EO) ont été évalués par 2 réviseurs indépendants. L’évaluation de la qualité (de ce matériel) a été réalisée avec l’aide de l’outil de Cochrane de mesure du risque de biais, et a été estimée selon une échelle à quatre niveaux de qualité Une synthèse narrative des données a été effectuée en raison de l'hétérogénéité importante des études. Résultats: 13 études (8 ECR, 5 EO) de 2150 patients hypertendus ont été prises en compte. Parmi elles, 5 études de CMGM avec l’utilisation de dispositifs électroniques comme seule intervention ont relevé une diminution de la tension artérielle (TA), qui pourrait cependant être expliquée par les biais de mesure. L’amélioration à court terme de la TA sous CMGM dans les interventions complexes a été révélée dans 4 études à qualité faible ou modérée. Dans 4 autres études sur les soins intégrés de qualité supérieure, il n'a pas été possible de distinguer l'impact de la composante CMGM, celle-ci pouvant être compromise par des traitements médicamenteux. L’ensemble des études semble par ailleurs montrer qu’un feed-back régulier au médecin traitant peut être un élément essentiel d’efficacité des traitements CMGM, et peut être facilement assuré par une infirmière ou un pharmacien, grâce à des outils de communication appropriés. Conclusions: Aucune preuve convaincante de l'efficacité des traitements CMGM comme technologie de la santé n’a été établie en raison de designs non-optimaux des études identifiées et des ualités méthodologiques insatisfaisantes de celles-ci. Les recherches futures devraient : suivre les normes de qualité approuvées et les recommandations cliniques actuelles pour le traitement de l'hypertension, inclure des groupes spécifiques de patients avec des problèmes d’attachement aux traitements, et considérer les résultats cliniques et économiques de l'organisation de soins ainsi que les observations rapportées par les patients. / Objective: Whether interventions including measurement and correction of patients’ attitude to antihypertensive medication can improve hypertension management is unclear. The review aims to determine the effectiveness of patient compliance measurement-guided medication management (CMGM) programs in essential hypertension. Design: Systematic review. Data sources: MEDLINE, EMBASE, CENTRAL, hypertension meetings abstracts, and bibliographies of identified articles. Methods: Randomized controlled trials (RCT) and observational studies (OS) were assessed by 2 reviewers independently. Quality assessment was performed with the Cochrane risk of bias tool and evaluated in a four-point continuum. A narrative data synthesis was performed due to significant heterogeneity among studies. Results: 13 studies (8 RCT, 5 OS) involving 2150 hypertensives were included. Five trials of CMGM with electronic devices as a sole intervention suggested decrease in blood pressure (BP) but the result may have been due to bias. Short-term BP improvement under CMGM in complex interventions was revealed in 4 studies of low-to-moderate quality. In 4 integrated care studies of higher quality the impact of CMGM component was not possible to distil and may be compromised by medication regimens. Regular feedback to the treating physician seems to be an essential component of CMGM and may be effectively mediated by a nurse or a pharmacist and via telecommunication. Conclusions: No convincing evidence for the effectiveness of CMGM as a health technology was found due to non-optimal study designs and methodological quality. Future research should follow accepted quality standards and current guidelines for the treatment of hypertension, include specific groups of patients with compliance problems and consider clinical, economic, patient-reported and organizational outcomes.

Submitted by Bruno Araujo Ramalho (brunrama@hotmail.com) on 2017-03-14T18:02:34Z No. of bitstreams: 1 Dissertação - BRUNO ARAUJO RAMALHO - (versão final).pdf: 2077332 bytes, checksum: 0dd8154f4be3890a0c30271468fdb13f (MD5) / Approved for entry into archive by Publicação Direito Rio (publicacao.direitorio@fgv.br) on 2017-03-15T18:39:56Z (GMT) No. of bitstreams: 1 Dissertação - BRUNO ARAUJO RAMALHO - (versão final).pdf: 2077332 bytes, checksum: 0dd8154f4be3890a0c30271468fdb13f (MD5) / Made available in DSpace on 2017-03-23T12:17:19Z (GMT). No. of bitstreams: 1 Dissertação - BRUNO ARAUJO RAMALHO - (versão final).pdf: 2077332 bytes, checksum: 0dd8154f4be3890a0c30271468fdb13f (MD5) Previous issue date: 2017-02-07 / Procedural requirements related to decision making and justification of regulatory choices may have limited application under uncertainty, informational limitation and other obstacles that lead the regulator to adopt strategies to tailor decision making to reality. Based on this hypothesis, and considering the uncertainties and issues of high technical complexity related to the 'list of health care procedures and events in health care', the objective of the research was to carry out an exploratory analysis intended to identify limitations, obstacles and observable strategies in the elaboration and justification of these norms. For this purpose, it was adopt a methodology of qualitative analysis based on documentary research, which included different documents available through the National Regulatory Agency for Private Health Insurance and Plans (ANS) website in public consultations, in the Technical Group for reviewing the list of procedures and in the Committee on Regulation of Health Care (COSAÚDE). As a result, it was verified the hypothesis mentioned above, since the dynamics of the choice presents restrictions that lead the regulator to adopt different strategies to deal with. The obstacles related to 'decision-making' dimension arise from the complexity and uncertainties related to the medical evidences, informational limitations and from interferences of the Legislative Branch and the Judiciary. Regarding the giving reasons requirement, although it is possible to see a greater density of justification starting in 2013, the reasons and replies presented in a synthesized format often do not suffice to clarify all the issues that support complex choices. Among the proposals of the study, the regulator should to provide a more detailed record about the valuation and the operationalization of its methodology criteria. / Exigências procedimentais relacionadas ao processo decisório e à justificação de escolhas regulatórias podem ter sua aplicação limitada em cenários de incerteza, limitação informacional e demais obstáculos que levam o regulador a adotar estratégias para adequar a tomada de decisão aos recursos disponíveis. Partindo-se desta hipótese, a pesquisa teve por objetivo a realização de uma análise exploratória com vistas a identificar limitações, obstáculos e estratégias observáveis na elaboração e justificação do rol de procedimentos e eventos em saúde – eis que a elaboração do 'rol' é permeada por incertezas e por questões de alta complexidade técnica. Para tanto, adotou-se a metodologia de análise qualitativa baseada em pesquisa documental, que englobou diferentes anexos disponibilizados a partir do sítio eletrônico da Agência Nacional de Saúde Suplementar (ANS) em consultas públicas, no Grupo Técnico de revisão do rol de procedimentos e no Comitê Permanente de Regulação da Atenção à Saúde (COSAÚDE). Ao final, foi possível evidenciar importantes elementos que moldam ou restringem a tomada de decisão e a sua respectiva justificação. No âmbito dos resultados, os obstáculos relacionados à dimensão 'processo decisório' decorrem de diferentes fontes de incerteza, do volume (e complexidade) das informações e de interferências promovidas pelo Poder Legislativo e Judiciário – sendo observáveis diferentes providências do regulador para cada caso. No que tange ao dever de motivação, embora se percebam melhorias a partir de 2013, a apresentação de justificativas e réplicas segue um formato mais compactado e não contempla algumas questões metodológicas que servem como base para premissas elencadas pelo regulador. Dentre outras propostas do estudo, sugeriu-se que a agência disponibilize o amplo acesso a documentos ou relatórios que registrem, de forma pormenorizada, a operacionalização dos quesitos que integram a metodologia de decisão.

Made available in DSpace on 2011-05-04T12:36:28Z (GMT). No. of bitstreams: 0 Previous issue date: 2009 / As atividades de rastreamento do horizonte tecnológico, uma forma de avaliação de tecnologia em saúde realizada no início do ciclo de vida das tecnologias, vêm sendo difundidas em países desenvolvidos desde a década de 90, visando tornar mais eficientes os processos de tomada de decisão relativos à incorporação de novas tecnologias. Trata-se de uma abordagem sistemática para a identificação e avaliação de tecnologias novas / emergentes, com o objetivo de alertar os tomadores de decisão quanto à sua existência e potenciais conseqüências de sua incorporação para o sistemade saúde, antes da difusão do seu uso. Considerando a relevância de operacionalizar as atividades de rastreamento do horizonte tecnológico para a atenção à saúde no Brasil, apresente dissertação teve o objetivo de produzir subsídios para a elaboração de diretrizes de um sistema de rastreamento do horizonte a ser criado no País. Para auxiliar o processo de elaboração, foi realizado um exercício de rastreamento do horizonte, no qual foi utilizado o caso das tecnologias envolvidas no manejo da osteoporose em mulheres na pós-menopausa, um problema de saúde considerado relevante, para o qual não existe solução razoável. Inicialmente, procedemos a uma revisão do conceito e das experiências internacionais com sistemas de rastreamento do horizonte tecnológico (SRH) em saúde. As fontes consultadas e os trabalhos analisados indicam um consenso de que um SRH deve sempre basear seu trabalho na melhor evidência preliminar disponível sobre o efeito das tecnologias, na saúde das pessoas e sobre o sistema de saúde, e atuar de maneira transparente, explicitando ao máximo a metodologia adotada no processo de trabalho. A revisão também permitiu a identificação de muitos desafios e pontos críticos para o desenvolvimento e operacionalização de tal sistema. O exercício de rastreamento realizado possibilitou a identificação de uma nova tecnologia medicamentosa, o denosumab, para o tratamentoda osteoporose, cujos ensaios clínicos de fase II e III, recentemente publicados, foram analisados. As limitações enfrentadas na realização do exercício permitiram reconhecer algumas dificuldades básicas específicas da avaliação tecnológica no início do ciclo devida. A avaliação das experiências internacionais e o exercício acima referido indicam que, para o desenvolvimento e operacionalização de um SRH no Brasil, o intercâmbio e a colaboração institucional com as unidades governamentais de monitoramento do horizonte de países desenvolvidos será fundamental, tendo em vista o corpo qualificado e a longa experiência daquelas unidades nessas atividades e, ainda, a quantidade de trabalho e de tempo envolvidos na análise de cada tecnologia e a urgência inerente a essas atividades. Além disso, deverá haver uma articulação entre os atores-chave, especialmente os do âmbito governamental, com o objetivo de discutir e estabelecer as diretrizes para a criação do sistema. Com isso, espera-se que as informações produzidas pelo SRH brasileiro sejam adequadamente utilizadas no processo de tomada de decisão do sistema de saúde. / Activities related to horizon scanning of technologies for health care, a form of health technology assessment carried out at the beginning of the life cycle of those technologies, have been adopted in several developed countries since the 90s, with the purpose of improving the efficiency of decision making related to the incorporation of new technologies. Horizon scanning consists of a systematic approach to identify and assess new and emerging technologies in order to warn health care decision makers about the existence and potential consequences of their incorporation to individuals and to the health system, before the diffusion stage. Given the importance of developing horizon scanning activities for health care technologies in Brazil, the present work had the objective of producing subsidies for the elaboration of guidelines to develop a horizon scanning system in Brazil. To aid that elaboration process, the case of technologies designed for the secondary prevention of osteoporotic fractures after menopause, a relevant public health problem far from being solved, was analyzed. Initially, a review of the concept of horizon scanning in health care and of the related international governmental experiences was carried out. The available sources and studies indicate a consensus on that: a) recommendations made by a horizon scanning 8 system must be supported by the best available early scientific evidence of the consequences on the health of the individuals and on the health system of the adoption of new/emerging technologies; b) the horizon scanning system must act in a transparent way, making explicit the criteria and methodology it uses. The review also showed that there remain several challenges to be dealt with in order to further develop such monitoring system. The horizon scanning exercise related to the secondary prevention of osteoporotic fractures enabled the identification of a new drug, called denosumab; phase II and phase III trials of that technology have been recently published and were analyzed. Limitations faced in such exercise made it possible the identification of some specific difficulties associated to technology assessment at the beginning of the life cycle. The review of the international experiences with horizon scanning of health care technologies, at central government level, along with the mentioned exercise, indicate that, for developing a horizon scanning system in Brazil, collaboration with developed countries’ units is essential, taking into account the qualified and experienced staff of such units as well as the amount of work and time involved in the analysis of each technology and the inherent urgency of such activities. Finally, key actors, specially those at central government level, must be articulated for the discussion and elaboration of the guidelines towards the creation of such system. In this way, the chances of a successful effort, with an adequate utilization of produced information in the process of decision-making, are increased.

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