Introduction Identifying key differences between the manufacturer's submitted analysis and the Canada's Common Drug Review (CDR) economic reanalysis is a crucial step toward creating more appropriate models by manufacturers. We compared manufacturers’ submitted analysis to CDR reanalysis in order to identify any variations in incremental costs utility ratio (ICUR) and 3-year Budget impact analysis (BIA). We assessed the willingness to pay (WTP) threshold and CDR critiques on manufacturers’ clinical and economic reports. Method A pair of reviewers extracted data regarding therapeutic category, percent price reduction requested by CDR, WTP, and the critiques on the manufacturers' clinical and economic reports in publicly available CDR reports from 2018 to 2022. We used Wilcoxon rank test to assess the difference between mean incremental QALY, ICUR, and BIA in manufacturers and CDR reanalysis reports and chi-square tests and logistic regression to assess the relationship between the variables and the final CDR recommendation. Results Of 178 reports assessed, 31 received "do not reimburse" recommendation and 147 received "reimburse with criteria or conditions". The median ICUR in manufacturer's analysis was $138,658/QALY and significantly lower than ICUR reanalyses by CDR of $380,251/QALY. The ICUR in manufacturers' submitted reports was 2.5-fold lower than in the CDR reanalysis ($138,658/QALY versus $380,251/QALY). The CDR reanalysis median for 3-year BIA was $4,575,102 which was 27% higher than the manufacturers submitted 3-year BIA (p value<0.001). The most frequent CDR critiques were clinical effectiveness and the uncertainty of evidence in cost-effectiveness analysis and miscalculations in the population of patients and the percentage of market share in BIA. From 2018 to 2020, $100,000 was the most frequent WTP threshold followed by a $50,000 threshold, but during 2021 and 2022, the CDR only used $50,000 as a WTP threshold. Conclusion Manufacturers may tend to underestimate the costs or overestimate the effect of their medications. / Thesis / Master of Science (MS)

Objectives: The use of interactive public engagement methods to elicit public values is becoming routine practice in health system planning, policy and evaluation; however, little systematic attention has been given to the analysis of how these values are articulated. This process will be examined with the use of deliberative discourse methods in the context of health technologies. Approach: The deliberations of a 14-person Citizens’ Reference Panel on Health Technologies were audiotaped and transcribed. The panel provided input to the Ontario Health Technology Advisory Committee in developing its recommendations. Discussion transcripts were analyzed using Gee’s (2005) ‘building tasks’ framework with a focus on identities, relationships, and politics. In depth language-context analysis was then used to study ‘situated meanings’ of social and ethical citizen values. Both levels of discourse analysis were then used to elicit the meso-level dynamics within the citizen panel deliberations. Results: Panel members used the provided materials, personal experience and other sources of information to express their values toward the technologies under review. In the group, members used their occupational, personal and cultural identities and adopted in-group citizen panel roles that involved summarizing small group discussions, challenging other members, providing information, providing expertise, interpreting information and facilitating. These individual roles were similar across meetings and members began to form relationships with their fellow citizens and make connections between the values involved in similar technologies. Conclusion: Discourse analysis methods can be used to draw in-depth insights from public engagement deliberations which contribute important new knowledge to the field of public deliberation and health policy. Further use and refinement of deliberative discourse methods will allow public values to be better understood and more adequately portrayed in the health technology assessment process. / Master of Science (MSc)

Health technology assessment (HTA) frameworks appraise the value of technologies – be they drugs, devices, procedures or services – to inform policy decision-making and resource allocation amongst alternatives within publicly funded health systems. The prevailing principles and metrics by which HTA is conducted were designed with adult health conditions and treatments in mind. The evidentiary and normative dimensions of HTA frameworks may have unique repercussions for drug policy and coverage decisions in children, but their relevance to child health has received almost no critical scrutiny in either academic or policy circles. Approaches to paediatric drug coverage approval and access currently lack child-specific data on social values and priorities, a core component of HTA in most countries with public drug funding programs, including Canada. This thesis presents a mixed methods study of social values relevant to child HTA and drug policymaking in publicly funded health systems, comprised of three original scientific contributions. The first of these is a critical interpretive synthesis (CIS) of the academic literature on the moral dimensions of child health and social policymaking across a range of disciplines and policy domains. The second is a grounded theory analysis of qualitative interviews with diverse health system stakeholders on the social values and health system factors relevant to child HTA and drug funding policy in Canada. The third is a stated preference survey of the general public that assesses societal preferences for health resource allocation to children as compared to adults, to generate evidence for priority setting on health technologies within Canada’s publicly funded health system. Together, these studies yield specific knowledge about the policy landscape for child health technologies in Canada, broad conceptual insights into the normative and methodological dimensions of child HTA, and a foundational understanding of the social values relevant to drug policy decisions for children. / Thesis / Doctor of Philosophy (PhD) / Drug research, development and policy have historically excluded children. One area of persistent neglect is public policy on funding for paediatric medicines. In most publicly funded health systems, including Canada’s, decisions about which drugs to cover are made through a formal process called health technology assessment (HTA). This dissertation examines the role and challenges of HTA as applied to child health technologies, with a focus on the social values that inform drug policy for children. It addresses existing gaps in knowledge through the integration of insights from: 1) a comprehensive review of the academic literature on the moral dimensions of child health and social policymaking; 2) in-depth qualitative analysis of the HTA and drug policy environments for children in Canada, employing Ontario as a case study; and 3) a survey of the Canadian public on health system resource allocation for children. Together, these studies generate a detailed picture of the Canadian policy landscape for child health technologies, insights into the fit of current HTA approaches to the realities of child health and illness, and a foundational understanding of the social values relevant to drug policy decisions for children.

This thesis addresses the question of the design, production and use of information graphics in health technology assessment (HTA). Drawing on previous research in both information design and health policy, it describes a comprehensive design process for creating new visual presentations that can inform health policy-makers. The thesis begins by introducing, and functionally defining the terms ‘information graphics’ and ‘health technology assessment’ in Chapter 1. It then offers a methodological discussion of how research can be performed at the intersection between these two diverse fields. This discussion forms Chapter 2 of the thesis. The context of use is surveyed in two studies, which are presented in Chapter 3. These assess the current use of information graphics in HTA, and the information needs of health policy decision-making bodies. This enables a needs-based approach to the design of 10 information graphics, that could be used in hta. These are shown in Chapter 4. Finally, two of these information graphics are empirically tested with two further research studies, forming Chapter 5 and Chapter 6. The thesis is aimed at giving practical advice to those wanting to produce graphical presentations of information in HTA, and to provide the foundation for further original research in information design and HTA. Chapter 7 draws together the research from the rest of the thesis, to make recommendations in light of the combined findings.

Les développements récents en économie du bien-être ouvrent la voie à des méthodes d’évaluations reposant sur d’autres modèles de justice sociale que l’utilitarisme. Leur faisabilité dans les pratiques quotidiennes d’évaluation des interventions de santé pose question : l’objectif de cette thèse est de contribuer d’y répondre. Pour cela nous nous sommes intéressés à trois cas pratiques. L’objectif du Chapitre I est de comparer les méthodologies d’évaluation de trois agences publiques d’évaluation, le NICE (Grande-Bretagne), l’IQWiG (Allemagne) et le KCE (Belgique), pour identifier les positions en matière de justice sociale qui en découlent. Le Chapitre II propose d’étudier le dilemme moral que suscite le phénomène d’adaptation des préférences dans l’évaluation deux dispositifs de compensation du handicap. Trois options sont avancées résoudre ce dilemme, elles s’appuient sur les théories de la justice sociale égalitaristes. Enfin, le Chapitre III démontre la faisabilité de l’approche revenu équivalent-santé, développée par Fleurabey, dans l’aide à la décision publique, au sujet des traitements antihypertenseurs en prévention primaire / Welfare economics encompasses other public choice theories besides utilitarianism, like egalitarian social justice theory. Whether these economic frameworks provide practical tools that could be used in health technology assessment is an ongoing debate, and this study aim to contribute to answer this question. The aim of the first chapter is to review health economic evaluation guidelines and compare methods from three national health technology assessment agencies - NICE (England and Wales), IQWiG (Germany) and KCE (Belgium) - and to assess what social justice principles are implied by their methodological choices. The second chapter studies the moral dilemma raised by adaptive preferences in growth hormones treatment and bilateral cochlear implants. Three options are put forward to solve this dilemma: they are grounded on egalitarian social justice theories. The objective of the third chapter is to provide evidence about the feasibility of assessment based on equivalent income approach, developed by M. Fleurbaey, in the context of public decision making. For this application case, we focussed on antihypertensive treatments in primary prevention

Les dispositifs médicaux (DM) innovants offrent sans cesse de nouvelles options thérapeutiques et diagnostiques et contribuent à améliorer la prise en charge des malades à l’hôpital. Toutefois, ces innovations, potentiellement onéreuses, ne disposent pas toujours de données probantes garantissant leur efficience. Par conséquent, pour guider le choix des décideurs hospitaliers, une évaluation à l’échelle de l’hôpital est souvent nécessaire afin de lever le maximum d’incertitudes avant leur acquisition. Cette démarche, appelée évaluation des technologies de santé (ETS) en milieu hospitalier, s’est beaucoup développée à l’étranger, mais reste méconnue en France. A travers notre travail, nous avons montré que les CHU français proposaient des modèles d’organisation pour l’évaluation des DM innovants tout à fait comparables à ceux rencontrés dans d’autres pays. Parmi les modèles d’ETS en milieu hospitalier, la mini-ETS semble être le plus généralisable et pourrait concourir à l’harmonisation des pratiques entre CHU. Ce modèle en plein essor existe sous de multiples formes dans le monde. La confrontation des critères d’évaluation identifiés dans les modèles assimilables à la mini-ETS à ceux utilisés dans les CHU français nous a permis d’isoler 26 critères d’évaluation d’intérêt pour l’évaluation des DM innovants. Enfin, nous avons élaboré puis testé, en situation réelle, un outil d’aide multicritère à la décision dérivé des précédents critères identifiés. / Innovative medical devices offer solutions to medical problems. However, medical devices arriving on the European market have generally little evidence about efficiency. To manage the uncertainty surrounding the introduction of innovative medical devices, hospitals and university hospitals have developed worldwide hospital-based health technology assessment (HTA) processes to guide the selection of expensive and innovative products. Nevertheless, little is known about hospital-based HTA activities in France. We demonstrated that French models of hospital-based HTA are comparable to those found elsewhere and presented sometimes a combination of several HTA models. We also underlined that a mini-HTA like model could easily be applied to French university hospitals. Mini-HTA has become widespread in many different forms across the world. We compared criteria of different mini-HTA forms to criteria used in French university hospitals and identified 26 relevant criteria to assess innovative medical devices. Finally, we developed and tested in real conditions a multiple-criteria decision tool derived from these 26 criteria.

Background: Health technology assessment (HTA) is a form of policy analysis to inform recommendations for decision-makers. An equity-focused HTA recommendation consists of one that explicitly addresses the impact of health technologies on individuals disadvantaged in society because of their social conditions. However, there is a need for more evidence on the relationships between patient engagement and the development of equity-focused HTA recommendations. Objectives: The objectives of this dissertation were to examine the association between patient engagement and equity-focused HTA recommendations and identify implementation considerations for patient engagement in HTA. Methods: I used explanatory sequential mixed methods to analyze 60 HTA reports and 11 interviews with patients and analysts from Canadian organizations: the Canadian Agency for Drugs and Technologies in Health (CADTH) and Health Quality Ontario (HQO). Results: Quantitative analysis of the HTA reports showed that patient engagement significantly predicts equity-focused HTA recommendations (OR: 0.26; 95% CI: [0.16 – 0.41]). HTA reviews where HTA analysts directly interviewed patients (OR: 3.85; 95% CI: [2.40 – 6.20]) and where an advisory committee used consensus were more likely to contain equity-focused recommendations (OR: 2.27; 95% CI: [1.35 – 3.84]). Qualitative analysis of the interviews identified strategies for engaging diverse patients in HTA. Conclusion: The findings of this dissertation can inform the designing of patient engagement in HTA.

Ökad kostnadsutveckling inom offentlig hälso- och sjukvård och en ökad en efterfråga dess tjänster har uppmärksammat behov av prioriteringar för resursfördelning, inte minst för avancerad medicinteknisk utrustning. Användning av olika beslutsunderlag kan bidra till lättare prioritering och mer kostnadseffektiva investeringsbeslut i den svenska hälso- och sjukvården. / Increased expenses in the public health care, combined with an increased demand of its services, have led to a higher demand for resource prioritization, not least for advanced medical equipment. Using different types of supporting information enables better prioritizing and more cost-effective investment decisions in Swedish public health care.

Mémoire numérisé par la Division de la gestion de documents et des archives de l'Université de Montréal.

Este estudo exploratório-descritivo tem como objetivo avaliar o desempenho funcional e a qualidade técnica de um registro eletrônico do processo de enfermagem junto a especialistas em informática, enfermeiros docentes e enfermeiros assistenciais. O Hospital Universitário da Universidade de São Paulo em parceria com a Escola de Enfermagem da Universidade de São Paulo, desenvolveu um registro eletrônico denominado Sistema de Documentação Eletrônica do Processo de Enfermagem da Universidade de São Paulo (PROCEnfUSP), para que pudesse ser utilizado na automação das informações do Processo de Enfermagem, visando à adoção de um Sistema de Apoio à Decisão que pudesse contribuir para a melhoria da gestão, da qualidade da assistência e da satisfação dos pacientes. A garantia da qualidade desse sistema é um importante desafio e meta devido à responsabilidade com os usuários e pacientes. Para assegurar essa qualidade foi realizada uma avaliação do sistema PROCEnf-USP utilizando-se do Modelo de Qualidade proposto pela norma ISO/IEC 25010 e do Processo de Avaliação definido na norma ISO/IEC 25040. Na avaliação dos especialistas em informática, adequação funcional obteve 91% de respostas positivas, confiabilidade 76%, usabilidade 62%, eficiência de desempenho 84%, compatibilidade 86%, segurança 89%, manutenibilidade 93% e portabilidade 92%. Os enfermeiros docentes avaliaram positivamente todas as características, adequação funcional obteve 92%, confiabilidade 89%, usabilidade 84%, eficiência de desempenho 81%, compatibilidade 75% e segurança 100%. Os enfermeiros assistenciais das clínicas médica e cirúrgica do hospital avaliaram adequação funcional com 82% de respostas positivas, confiabilidade 69%, usabilidade 85%, eficiência de desempenho 47% compatibilidade 62% e segurança 98%. Os enfermeiros assistenciais de outras unidades avaliaram adequação funcional com 88%, confiabilidade 61%, usabilidade 80%, eficiência de desempenho 46%, compatibilidade 69% e segurança 100%. Conclui-se que o PROCEnf-USP atingiu mais de 70% de respostas positivas na maioria das características de qualidade avaliadas por todos os especialistas. Entretanto, eficiência de desempenho, confiabilidade e compatibilidade obtiveram índices abaixo do parâmetro estabelecido na avaliação dos enfermeiros assistenciais. Os resultados obtidos neste estudo serão utilizados para a realização de melhorias no sistema. Essa pesquisa propiciará a disseminação do conhecimento de uma área emergente, agregando mais uma iniciativa aos crescentes esforços na área de informática em saúde e em enfermagem / This descriptive exploratory study aims to evaluate functional performance and technical quality of an electronic record for the nursing process with technology experts, teaching staff and nurses. The University Hospital in the University of São Paulo in partnership with the School of Nursing of the University of São Paulo, has developed an electronic record called Electronic Documentation System for the Nursing Process of the University of São Paulo (PROCEnf-USP), to be used for the automation of information of the Nursing Process, aimed at the adoption of a Decision Support System that could contribute to the improvement of management, quality of care and patient satisfaction. The quality guarantee of this system is an important challenge and target, due to responsibility with users and patients. To ensure such quality an assessment of the PROCEnf-USP system was carried out using the Quality Model proposed by ISO/IEC 25010 and the Evaluation Process defined in ISO/IEC 25040. In the evaluation of technology experts the characteristic functional suitability had 91% of positive responses, reliability 76%, usability 62%, performance efficiency 84%, compatibility 86%, security 89%, maintainability 93% and portability 92%.The teaching staff positively evaluated all the characteristics, functional suitability had 92%, reliability 89%, usability 84%, performance efficiency 81%, compatibility 75% and security 100%. The nurses from general medical and surgical hospital evaluated functional suitability with 82% of positive responses, reliability 69%, usability 85%, performance efficiency 47%, compatibility 62% and security 98%. The nurses from other units evaluated functional suitability with 88%, reliability 61%, usability 80%, performance efficiency 46%, compatibility 69% and security 100%. It is concluded that the PROCEnf-USP reached more than 70% of positive responses in the majority of quality characteristics evaluated by specialists. However, performance efficiency, reliability and compatibility found rates below the parameter established in the assessment of nurses. The results of this survey will be used to implement improvements in the system. Such research will allow the spreading of knowledge in an emerging area, thus adding a further initiative to the growth efforts made in the information technology areas of health and nursing.