BACKGROUND & OBJECTIVES Overarching and decision-making frameworks may be used to facilitate the evaluation of prescription drug technologies to enable Health Technology Assessment (HTA) agency’s reassessment recommendations. The objectives of this thesis were to; 1) identify overarching and qualitative decision-making reassessment framework challenges and methodological gaps and; 2) develop and/or modify a framework to address challenges/gaps. The focus was on Canadian public prescription drug reimbursement with the hope that the findings may inform other jurisdictions. METHODS The first paper systematically identified drug disinvestment frameworks to describe framework components, challenges and solutions. A qualitative descriptive study was conducted in the second paper to explore whether a qualitative benefit-risk framework (Universal Methodology for Benefit-Risk Assessment (UMBRA)) could be used or modified to further enable Health Technology Reassessment (HTR) recommendations. The last research paper assessed the Canadian Agency for Drugs and Technologies in Health’s (CADTH’s) Therapeutic Review Process. Enhancements to this process were developed based on previous research and published frameworks. RESULTS Qualitative framework components were identified, disinvestment terms captured and challenges and solutions to drug disinvestment were compiled in Chapter 2. The participants interviewed in chapter 3 recognized that the Therapeutic Review assessment process did not include a qualitative deliberative framework. However, participants did not consider that all steps of the UMBRA framework were transferable to the assessment phase of HTR. Assessment of CADTH’s Therapeutic Review process, conducted in Chapter 4, found three areas for process enhancement: Therapeutic Review topic prioritization criteria; a qualitative assessment framework, and; publically accessible mechanisms for decision monitoring and performance measurement. CONCLUSION This thesis has identified reassessment framework enhancements that are hypothesized to address HTR challenges and specific solutions to enhance CADTH’s Therapeutic Review Framework. Next steps include further evaluation and pilot testing of these proposed enhancements to enable additional Canadian stakeholder feedback. / Thesis / Doctor of Philosophy (PhD) / This thesis focuses on overarching and decision-making assessment frameworks whose purpose is to aid policy-makers in recommending which prescription drugs should continue to be government-funded and/or if modifications to funding should occur. The goal of this work is to; 1) identify challenges and gaps in these frameworks and 2) develop or modify a framework to address findings. This thesis focuses on the Canadian public prescription drug reimbursement environment. Results identified Canadian reassessment framework enhancements which could address challenges to ultimately aid in maintaining financial and institutional stability of public health care systems.

US corporations have long recognized university related scientific research as an important source of long term economic growth and technological innovation. This dynamic involvement with industry has drastically increased the university technology transfer and licensing activities, and has stretched the human and financial resources of Technology Management and Licensing Offices of many US universities. This research provides a mechanism that can aid in the complex process of properly assessing university-owned technologies and intellectual properties, to identify those with licensing and commercialization potential for the pursuit of truly important breakthrough discoveries. This research focuses on the university technology licensing and commercialization process from the perspectives of those licensing professionals whose firms' activities are engaged in licensing-in university technologies. The objectives of this research are to: 1.Identify the decision factors and licensing determinants that influence or impact the licensing and commercialization of university technologies. 2.Build and conduct a survey among those licensing professionals involved in the technology licensing process to determine the relative importance of each of the licensing determinants identified in the literature review, and their most current and up to date selection criteria for technologies they license. 3.Develop a framework to assist the University Technology Management & Transfer Office's personnel and other stakeholders in the assessment of the potential viability of the university technologies for licensing and commercialization.

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)

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.

Congress has become infamous for its lack of understanding of technology, particularly with the Facebook and Google hearings in 2018. To improve this understanding, this thesis argues for the return of the Office of Technology Assessment (OTA), a congressional support agency created in 1972 that provided science and technology expertise to Congress until its termination in 1995. It also considers potential changes that might be made to the old OTA model and the political environment in which a new OTA would need to survive.

ABSTRACT Clinical evidence technologies (CETs) are information sources derived from medical research literature that may assist health care providers in continued learning, decision-making, and patient care. Examples of CETs include: MEDLINE/PubMed and Cochrane Reviews, research journal literature, print and electronic medical texts, clinical topic summaries, guidelines, and interactive decision tools. Clinicians utilize CETs to find answers to questions that arise during patient care. However, it was unclear if CETs had a measurable impact on provider practice or patient outcomes. A literature review identified twenty-two articles evaluating CETs’ impact. Study designs included surveys, observational studies, randomized controlled trials and quasi-experimental methods. The review revealed mixed evidence of CET impact on provider-level outcomes such as improved diagnoses and treatments, and on patient level outcomes such as length of hospital stay and mortality. Additional research was needed to determine whether certain CETs or CET types have impact on patient care outcomes in clinically targeted areas. We conducted a cluster-randomized controlled trial (CRCT) to evaluate the effect of a dermatology-focused CET (VisualDx) when used by primary care providers. We found no difference in the patient skin disease outcomes of resolution of symptoms and return visits for the same problem in that trial. Thirty-two PCPs and 433 patients participated. In proportional hazards modelling adjusted for provider clusters, the time from index visit to skin problem resolution was similar in both groups (Hazard Ratio=0.92; 95% Confidence Interval (CI)=0.70, 1.21; P=0.54). Patient follow-up appointments did not differ significantly between groups (Odds Ratio=1.26; CI=0.94, 1.70; P=0.29). In a follow up mixed-methods study, we sought to understand why VisualDx did not make a difference. All CRCT provider participants were surveyed about their experience in the trial. VisualDx users (intervention arm) were interviewed about their experience using the CET. Ease of access and usefulness for patient communication facilitated successful use while irrelevant search results and use of other sources were barriers. Although PCPs reported benefits, they did not perceive the CET as useful often enough to motivate using it frequently or exclusively, thereby reducing the likelihood of it making a difference in the problem resolution and return appointment outcomes. There was no difference in skin problem resolution or number of follow-up visits when PCPs used VisualDx. PCPs did not perceive VisualDx as “useful” often enough for to use it frequently, or exclusively, thereby reducing the likelihood of this CET making a difference in patient-level outcomes.

Engineering research and development work is undergoing a reorientation from focusing on specific parts of different systems to a broader perspective of systems level, albeit at a slower pace. This reorientation should be further developed and enhanced with the aim of organizing and structuring our technical systems in meeting sustainability requirements in face of global ecological threats that have far-reaching social and economic implications, which can no longer be captured using conventional approach of research. Until a list of universally acceptable, clear, and measurable indicators of sustainable development is developed, the work with sustainability metrics should continue to evolve as a relative measure of ecological, economic, and social performance of human activities in general, and technical systems in particular. This work can be done by comparing the relative performance of alternative technologies of providing the same well-defined function or service; or by characterizing technologies that enjoy different levels of societal priorities using relevant performance indicators. In both cases, concepts and methods of industrial ecology play a vital role. This thesis is about the development and application of a systematic approach for the assessment of the performance of technical systems from the perspective of systems analysis, sustainability, sustainability assessment, and industrial ecology. The systematic approach developed and characterized in this thesis advocates for a simultaneous assessment of the ecological, economic, and social dimensions of performance of technologies in avoiding sub-optimization and problem shifting between dimensions. It gives a holistic picture by taking a life cycle perspective of all important aspects. The systematic assessment of technical systems provides an even-handed assessment resulting in a cumulative knowledge. A modular structure of the approach makes it flexible enough in terms of comparing a number of alternatives at the same time, and carrying out the assessment of the three dimensions independently. It should give way to transparent system where the level of quality of input data can be comprehended. The assessment approach should focus on a selected number of key input data, tested calculation procedures, and comprehensible result presentation. The challenge in developing and applying this approach is the complexity of method integration and information processing. The different parts to be included in the same platform come in with additional uncertainties hampering result interpretations. The hitherto tendency of promoting disciplinary lines will continue to challenge further developments of such interdisciplinary approaches. The thesis draws on the experience from ORWARE, a Swedish technology assessment tool applied in the assessment of waste management systems and energy systems; and from the EcoEffect tool used in the assessment of building properties; all assessed as components of a larger system. The thesis underlines the importance of sustainability considerations beginning from the research and development phase of technical systems. The core message of this thesis is that technical systems should be researched as indivisible parts of a complex whole that includes society and the natural environment. Results from such researches can then be transformed into design codes and specifications for use in the research and development, planning and structuring, and implementation and management of technical systems. / QC 20100505

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