Background and objectives - Addressing ethical issues in health technology assessment (HTA) can increase transparency and accountability of the HTA process and lead to better-informed healthcare decisions. Despite its importance, integration of ethics into HTA remains challenging. The objective of this thesis was to develop a process-based framework to support ethical evaluations in HTA and increase their applicability. Methods - Project 1: A systematic literature review was conducted with the purpose of identifying and mapping the methodological features of the existing frameworks for ethics in HTA. Project 2: A systematic literature review and an international survey of HTA agencies were conducted to explore how ethical evaluations may be encouraged or discouraged in the HTA practice. Project 3: A procedural framework was drafted based on the operational features of the identified guidance documents as well as barriers and facilitators for incorporating ethics into HTA. Project 4: The framework was applied to a hypothetical case study, with the aim of helping HTA practitioners touch on key points of the steps outlined by the proposed framework. Results - Project 1: The identified ethical frameworks vary in their purpose, philosophical approach, structure, and comprehensiveness. The review results suggest that the choice of a method for collection and analysis of ethical data depends on the context, purpose of analysis, and availability of resources. Project 2: The results of this study emphasize the importance of simplification of ethics methodology and development of good practice guidelines in HTA, as well as capacity-building for engaging HTA practitioners in ethical analyses. Project 3: The proposed framework consists of an algorithmic flowchart, showing different steps of an ethical evaluation throughout the HTA process; a stepwise guide, which focuses on the tasks and potential questions that are required to be addressed at each step; and a list of some commonly recommended tools to facilitate the evaluation process. Project 4: The case study outlines the key tasks, recommended by the framework, and provides examples of process outputs that could be considered when attempting to perform an ethical evaluation. Conclusions - The outputs of this thesis can be used to support and promote a more consistent practice of ethical evaluation among HTA professionals. However, further validation of the proposed framework is required to establish its utility for HTA practice. / Dissertation / Doctor of Philosophy (PhD)

To fill a gap in the literature and to better inform decision making in oncology, this doctoral thesis investigates the role and impact of analytical methods in the economic evaluation of oncology medications through three main chapters which have been recently published. Chapter 2 presents a systematic literature survey of published economic evaluations in oncology over a 10-year period in order to identify, examine, and describe analytical methods that have been utilized (published in Pharmacoeconomics Open in 2021). This chapter demonstrated that greater detail in reporting of extrapolation methods, statistical techniques, and validation procedures is needed in order to conform with best practices outlined in existing economic evaluation guidelines. Chapter 3 complements the work of chapter 2 but takes a different perspective through an examination of the methods reported in economic evaluations published by HTA agencies in Canada, the UK, and Australia (published in Current Oncology in 2022). This chapter revealed significant reporting discrepancies across the agencies and concluded that common standards for reporting the results of HTAs should be implemented. Building on chapters 2 and 3, chapter 4 provides a model-based health technology re-assessment of an oncology drug approved on the basis of interim trial data using recently published long-term follow up data (published in Current Oncology in 2023). The findings from this chapter highlight the importance of transparency in the reporting of methods, the impact of using a life-cycle approach to HTA, and demonstrate the existence of a tradeoff between clinical/economic uncertainty and the value of the incremental cost-effectiveness ratio (ICER). The final chapter provides the overall conclusions of the research and presents avenues for future research. / Thesis / Doctor of Philosophy (PhD)

A Avaliação Tecnológica em Saúde (ATS) é uma sistemática que avalia os impactos clínicos, sociais e econômicos das tecnologias em saúde e tem como finalidade auxiliar os gestores na tomada de decisão quanto ao desenvolvimento, incorporação e descontinuação destas tecnologias. Agências de ATS têm destacado a importância da existência de métodos que visem padronizar e implantar esta sistemática em diferentes contextos. Assim, o objetivo geral desta tese é propor procedimentos para apoiar a avaliação tecnológica de equipamentos médico-hospitalares, por meio de uma pesquisa construtiva (Constructive Research) aplicada à terapia de hemodiálise. Os procedimentos propostos compõem um modelo adaptado do EuroScan, modelo desenvolvido por agências Europeias e indicado como boa prática pelo Ministério da Saúde do Brasil. Também são procedimentos ajustados às necessidades de pesquisadores de centros regionais de avaliação de equipamentos médico-hospitalares. Inicialmente, foi desenvolvida uma coleta de dados sobre os problemas de desenvolvimento e incorporação das tecnologias da saúde por meio de duas fontes: uma revisão sistemática de literatura sobre oportunidades de melhorias do EuroScan no mundo e discussões com pesquisadores no país. A partir destas investigações foi proposto um modelo preliminar de ATS, aplicado para o domínio Operacional e de Inovação na hemodiálise. Finalmente a aplicação foi validada e examinada por especialistas para a geração de um modelo final. Os principais procedimentos resultantes foram: (i) validação da relevância das avaliações para novos desenvolvimentos e incorporações tecnológicas do ponto de vista de diferentes especialistas, integrando diferentes áreas do conhecimento na ATS; (ii) operação de Observatórios Tecnológicos, auxiliando na definição das tecnologias e domínios a serem avaliadas; (iii) definição de técnicas para análise dos domínios de ATS, como o uso de séries temporais e o Technology Roadmap (TRM) para previsões sobre o futuro das inovações e o uso das dimensões da usabilidade e de incidentes críticos de riscos para os aspectos operacionais; e (iv) integração das diretrizes de ATS no Brasil em um único modelo, amparando pesquisadores não relacionados a saúde na compreensão sobre ATS e no desenvolvimento ágil de novos estudos tanto para equipamentos emergentes como em uso. Já a avaliação dos domínios selecionados para a hemodiálise resultou: (i) na identificação de um crescimento médio anual de 7% dos indicadores da terapia; (ii) no diagnóstico de usabilidade de máquinas eficazes, porém com possibilidades de gerarem maior satisfação aos usuários quanto: ao espaço entre elas, a redução dos desperdícios de água e materiais, aos planos de manutenção para evitar temperaturas elevadas, a capacitação visando mais atenção e treinamento para tarefas de apoio e aos aspectos de display (realimentação de dados e visualização); e (iii) na consolidação entre profissionais sobre um futuro promissor, para os próximos 10 anos no Brasil, da assistência homecare com máquinas de hemodiálise portáteis, mais seguras e sustentáveis. / The Health Technology Assessment (HTA) is a systematic evaluation of clinical, social and economic impacts of health technologies aiming to assist managers in the decision making process related to the development, consolidation and discontinuation of these technologies. HTA agencies have been highlighting the importance of methods to standardize and apply this systematic evaluation in different contexts. Thus, the general objective of this thesis is to propose procedures to support the technology assessment of medical-hospital equipment through a constructive research applied to hemodialysis therapy. The proposed procedures compose a EuroScan adapted model, which was developed by European agencies and is recommended as a good practice by the Brazilian Ministry of Health, and also are procedures adjusted to the needs of researchers in regional centers of medical equipment assessment. Initially, a data collection on the problems of developing and incorporating health technologies was carried out using two sources: a systematic review of the literature on opportunities to improve the EuroScan around the world and discussions with Brazilian researchers. Based on these investigations, it was proposed a preliminary HTA model applied to the hemodialysis’ Operational and Innovation domain. The application of the model was reviewed and evaluated by experts to produce a final version. The main resulting procedures were: (i) to validate the importance of assessments for new technological developments and incorporations considering the point of view of different experts from diverse knowledge areas; (ii) to operate Technological Observatories, assisting to define the technologies and domains to be evaluated; (iii) to set techniques for the analysis of HTA domains such as the use of time series and the Technology Roadmap (TRM) to make predictions about the future of innovations and the use of usability dimensions and critical incidents of risks to the operational aspects; and (iv) to integrate HTA Brazilian guidelines in a single model, supporting researchers not related to health in the understanding of HTA and fast development of new studies for both emerging and in use equipment. The assessment of the domains selected for hemodialysis led to: (i) the identification of an annual growth in therapy indicators (7% in average); (ii) the usability diagnosis of efficient machines, however with the potential to generate greater user satisfaction considering: distance between machines, reduction in the waste of water and materials, maintenance plans to avoid high temperatures, training aiming more attention to supporting tasks as well as focus on display aspects (data feedback and visualization); and (iii) the consolidation among professionals of a future, for the next 10 years in Brazil, based on homecare with portable machines, safer and more sustainable.

Systematic reviews - appraisal and synthesis of all primary research - are increasingly being used to inform policy and practice in health care. Therefore, it is important to understand how the key questions in systematic reviews are identified and prioritised and whether they are relevant to policy makers, practitioners and members of the public. Research priority setting (RPS) is usually defined as any interpersonal activity that leads to the selection of topics and/or choices of key questions to investigate . Diverse approaches to setting research priorities are used in different countries, regions and organisations. There is no consensus in the literature on the most effective processes with which to set these priorities. However, these decisions define the quality and implications of the evidence, and syntheses of it, available to patients, public and policy makers to help them make informed decisions. My initial scoping work, was to design and conduct a survey across an influential international systematic review organisation (Cochrane Collaboration ) on how they set priorities for their reviews. We identified 13 structured approaches to setting priorities. As part of the project, we developed an evaluation framework that demonstrated whether the priority setting processes meet the values and principles of the Cochrane Collaboration. Subsequently, we developed an equity lens for research priority setting exercises to inform the design of research priority setting processes to ensure that they consider the priorities of disadvantaged groups along with advantaged groups. We used the equity lens to do a second evaluation on the priority setting processes in the Cochrane Collaboration. Both evaluation frameworks demonstrated that the Cochrane Collaboration requires better designed priority setting approaches and must be more transparent in reporting those processes. The evaluation of research priority setting exercises in the Cochrane Collaboration, along with the wider literature, demonstrates that research priority setting exercises cannot be evaluated in isolation from organisational cultures, values and context. Therefore, the next step of the project focused on a specific stakeholder group (major research funders) with significant influence on research, including support for systematic reviews. We selected 11 national research agencies in the UK, Netherlands, France, Norway, Denmark, Germany, Australia, Canada, and the USA. We devised and used a checklist based on Chalmers and Glasziou’s “avoidable research waste” framework (and evaluated the processes and policies of these agencies using this checklist). As previous evaluations had demonstrated, this second evaluation found a lack of transparency in the process of setting priorities for research and other related organisational and policy issues. Increased funding is needed for methodological research to evaluate research practices and to monitor how funding research projects is done and reported. My evaluation of funding agencies and the Cochrane Collaboration found a similar lack of transparency and accountability in the context of conflicting values among stakeholders that decreases accountability and scrutiny of researchers and their institutions. However, the projects have led to organisational and policy changes in the two key stakeholder groups (the Cochrane Collaboration and selected funding agencies). Officials of national health research funding agencies have approached me to collaborate with them to address the issues raised by my work on reducing research waste. This led to the establishment of Funders Forum - the Ensuring Value in Research (EViR) Funders’ Collaboration and Development Forum - to enable agencies in various countries to exchange their experience in addressing issues and creating work groups to address them. The Forum is chaired by individuals from three major research funders: NIHR (UK), ZonMW (Netherlands) and Patient-Centered Outcomes Research Institute (PCORI; USA). The Forum organises several meetings to establish common principles, standards and work plans to achieve the common objective around reducing research waste and adding value for research for a national research funder.

The value to consider user needs throughout the development of medical devices has been acknowledged in the field of health technology assessment. Yet, user needs are narrowly conceptualised and are mainly examined from an ergonomic perspective. By focusing on the user-device interaction per se with a view to detect use errors and to create design solutions that promote intended use, the dominant approach to user needs research fails to adequately elaborate upon symbolic and practice-related dimensions in the user-technology relationship. Moreover, whilst the examination of user needs from a User standpoint is clearly required, it is also crucial to investigate how the medical device industry understands and addresses this issue, since it is these understandings that will eventually be projected onto the technology. The present research sought to provide a cross-actor account on the issue of user needs by examining the perspectives of two key stakeholders: the users and the medical device manufacturer. Using the example of home blood pressure (BP) monitoring, a qualitative programme of research explored, on the one hand, the process of integrating home blood pressure monitors (HBPMs) into daily life as well as the elements that are conducive to building trust in this technology, and on the other, the practices the medical device manufacturer adopts to capture its users and their needs. The results suggest that people engage with home BP monitoring in an effort to develop an experiential understanding of their health condition reproducing the dominant discourse around the benefits of self-care. Nevertheless, communicating this practice outside the home was not always without tension since concerns around the ascription of undesired identities were expressed. Home measurements were occasionally performed to check the dependability of technology – arguably an unintended device use – indicating the importance of establishing trust in the artefact. Building trust in HBPMs appeared to be a multifaceted phenomenon that was not limited to the perceived trustworthiness of the technology but implicated a network of other trustworthy relationships with humans, institutions and technologies. Medium-to-large medical device manufacturers appear to appreciate the value of a user needs-informed approach to medical device development employing a series of routes, more or less direct and formalised, to reach their user. The challenge for the industry is to synthesise the evidential base deriving from individual user studies to create a higher order knowledge base. The term ‘symbolic’ – also employed in the title of this thesis – signifies people’s representations, reasoning and meanings constructed around the use or production of home medical technology whilst the term ‘practical’ (or practice-related) refers to actions, activities, and routines pertaining to these two aspects.

A Avaliação Tecnológica em Saúde (ATS) é uma sistemática que avalia os impactos clínicos, sociais e econômicos das tecnologias em saúde e tem como finalidade auxiliar os gestores na tomada de decisão quanto ao desenvolvimento, incorporação e descontinuação destas tecnologias. Agências de ATS têm destacado a importância da existência de métodos que visem padronizar e implantar esta sistemática em diferentes contextos. Assim, o objetivo geral desta tese é propor procedimentos para apoiar a avaliação tecnológica de equipamentos médico-hospitalares, por meio de uma pesquisa construtiva (Constructive Research) aplicada à terapia de hemodiálise. Os procedimentos propostos compõem um modelo adaptado do EuroScan, modelo desenvolvido por agências Europeias e indicado como boa prática pelo Ministério da Saúde do Brasil. Também são procedimentos ajustados às necessidades de pesquisadores de centros regionais de avaliação de equipamentos médico-hospitalares. Inicialmente, foi desenvolvida uma coleta de dados sobre os problemas de desenvolvimento e incorporação das tecnologias da saúde por meio de duas fontes: uma revisão sistemática de literatura sobre oportunidades de melhorias do EuroScan no mundo e discussões com pesquisadores no país. A partir destas investigações foi proposto um modelo preliminar de ATS, aplicado para o domínio Operacional e de Inovação na hemodiálise. Finalmente a aplicação foi validada e examinada por especialistas para a geração de um modelo final. Os principais procedimentos resultantes foram: (i) validação da relevância das avaliações para novos desenvolvimentos e incorporações tecnológicas do ponto de vista de diferentes especialistas, integrando diferentes áreas do conhecimento na ATS; (ii) operação de Observatórios Tecnológicos, auxiliando na definição das tecnologias e domínios a serem avaliadas; (iii) definição de técnicas para análise dos domínios de ATS, como o uso de séries temporais e o Technology Roadmap (TRM) para previsões sobre o futuro das inovações e o uso das dimensões da usabilidade e de incidentes críticos de riscos para os aspectos operacionais; e (iv) integração das diretrizes de ATS no Brasil em um único modelo, amparando pesquisadores não relacionados a saúde na compreensão sobre ATS e no desenvolvimento ágil de novos estudos tanto para equipamentos emergentes como em uso. Já a avaliação dos domínios selecionados para a hemodiálise resultou: (i) na identificação de um crescimento médio anual de 7% dos indicadores da terapia; (ii) no diagnóstico de usabilidade de máquinas eficazes, porém com possibilidades de gerarem maior satisfação aos usuários quanto: ao espaço entre elas, a redução dos desperdícios de água e materiais, aos planos de manutenção para evitar temperaturas elevadas, a capacitação visando mais atenção e treinamento para tarefas de apoio e aos aspectos de display (realimentação de dados e visualização); e (iii) na consolidação entre profissionais sobre um futuro promissor, para os próximos 10 anos no Brasil, da assistência homecare com máquinas de hemodiálise portáteis, mais seguras e sustentáveis. / The Health Technology Assessment (HTA) is a systematic evaluation of clinical, social and economic impacts of health technologies aiming to assist managers in the decision making process related to the development, consolidation and discontinuation of these technologies. HTA agencies have been highlighting the importance of methods to standardize and apply this systematic evaluation in different contexts. Thus, the general objective of this thesis is to propose procedures to support the technology assessment of medical-hospital equipment through a constructive research applied to hemodialysis therapy. The proposed procedures compose a EuroScan adapted model, which was developed by European agencies and is recommended as a good practice by the Brazilian Ministry of Health, and also are procedures adjusted to the needs of researchers in regional centers of medical equipment assessment. Initially, a data collection on the problems of developing and incorporating health technologies was carried out using two sources: a systematic review of the literature on opportunities to improve the EuroScan around the world and discussions with Brazilian researchers. Based on these investigations, it was proposed a preliminary HTA model applied to the hemodialysis’ Operational and Innovation domain. The application of the model was reviewed and evaluated by experts to produce a final version. The main resulting procedures were: (i) to validate the importance of assessments for new technological developments and incorporations considering the point of view of different experts from diverse knowledge areas; (ii) to operate Technological Observatories, assisting to define the technologies and domains to be evaluated; (iii) to set techniques for the analysis of HTA domains such as the use of time series and the Technology Roadmap (TRM) to make predictions about the future of innovations and the use of usability dimensions and critical incidents of risks to the operational aspects; and (iv) to integrate HTA Brazilian guidelines in a single model, supporting researchers not related to health in the understanding of HTA and fast development of new studies for both emerging and in use equipment. The assessment of the domains selected for hemodialysis led to: (i) the identification of an annual growth in therapy indicators (7% in average); (ii) the usability diagnosis of efficient machines, however with the potential to generate greater user satisfaction considering: distance between machines, reduction in the waste of water and materials, maintenance plans to avoid high temperatures, training aiming more attention to supporting tasks as well as focus on display aspects (data feedback and visualization); and (iii) the consolidation among professionals of a future, for the next 10 years in Brazil, based on homecare with portable machines, safer and more sustainable.

A Avaliação Tecnológica em Saúde (ATS) é uma sistemática que avalia os impactos clínicos, sociais e econômicos das tecnologias em saúde e tem como finalidade auxiliar os gestores na tomada de decisão quanto ao desenvolvimento, incorporação e descontinuação destas tecnologias. Agências de ATS têm destacado a importância da existência de métodos que visem padronizar e implantar esta sistemática em diferentes contextos. Assim, o objetivo geral desta tese é propor procedimentos para apoiar a avaliação tecnológica de equipamentos médico-hospitalares, por meio de uma pesquisa construtiva (Constructive Research) aplicada à terapia de hemodiálise. Os procedimentos propostos compõem um modelo adaptado do EuroScan, modelo desenvolvido por agências Europeias e indicado como boa prática pelo Ministério da Saúde do Brasil. Também são procedimentos ajustados às necessidades de pesquisadores de centros regionais de avaliação de equipamentos médico-hospitalares. Inicialmente, foi desenvolvida uma coleta de dados sobre os problemas de desenvolvimento e incorporação das tecnologias da saúde por meio de duas fontes: uma revisão sistemática de literatura sobre oportunidades de melhorias do EuroScan no mundo e discussões com pesquisadores no país. A partir destas investigações foi proposto um modelo preliminar de ATS, aplicado para o domínio Operacional e de Inovação na hemodiálise. Finalmente a aplicação foi validada e examinada por especialistas para a geração de um modelo final. Os principais procedimentos resultantes foram: (i) validação da relevância das avaliações para novos desenvolvimentos e incorporações tecnológicas do ponto de vista de diferentes especialistas, integrando diferentes áreas do conhecimento na ATS; (ii) operação de Observatórios Tecnológicos, auxiliando na definição das tecnologias e domínios a serem avaliadas; (iii) definição de técnicas para análise dos domínios de ATS, como o uso de séries temporais e o Technology Roadmap (TRM) para previsões sobre o futuro das inovações e o uso das dimensões da usabilidade e de incidentes críticos de riscos para os aspectos operacionais; e (iv) integração das diretrizes de ATS no Brasil em um único modelo, amparando pesquisadores não relacionados a saúde na compreensão sobre ATS e no desenvolvimento ágil de novos estudos tanto para equipamentos emergentes como em uso. Já a avaliação dos domínios selecionados para a hemodiálise resultou: (i) na identificação de um crescimento médio anual de 7% dos indicadores da terapia; (ii) no diagnóstico de usabilidade de máquinas eficazes, porém com possibilidades de gerarem maior satisfação aos usuários quanto: ao espaço entre elas, a redução dos desperdícios de água e materiais, aos planos de manutenção para evitar temperaturas elevadas, a capacitação visando mais atenção e treinamento para tarefas de apoio e aos aspectos de display (realimentação de dados e visualização); e (iii) na consolidação entre profissionais sobre um futuro promissor, para os próximos 10 anos no Brasil, da assistência homecare com máquinas de hemodiálise portáteis, mais seguras e sustentáveis. / The Health Technology Assessment (HTA) is a systematic evaluation of clinical, social and economic impacts of health technologies aiming to assist managers in the decision making process related to the development, consolidation and discontinuation of these technologies. HTA agencies have been highlighting the importance of methods to standardize and apply this systematic evaluation in different contexts. Thus, the general objective of this thesis is to propose procedures to support the technology assessment of medical-hospital equipment through a constructive research applied to hemodialysis therapy. The proposed procedures compose a EuroScan adapted model, which was developed by European agencies and is recommended as a good practice by the Brazilian Ministry of Health, and also are procedures adjusted to the needs of researchers in regional centers of medical equipment assessment. Initially, a data collection on the problems of developing and incorporating health technologies was carried out using two sources: a systematic review of the literature on opportunities to improve the EuroScan around the world and discussions with Brazilian researchers. Based on these investigations, it was proposed a preliminary HTA model applied to the hemodialysis’ Operational and Innovation domain. The application of the model was reviewed and evaluated by experts to produce a final version. The main resulting procedures were: (i) to validate the importance of assessments for new technological developments and incorporations considering the point of view of different experts from diverse knowledge areas; (ii) to operate Technological Observatories, assisting to define the technologies and domains to be evaluated; (iii) to set techniques for the analysis of HTA domains such as the use of time series and the Technology Roadmap (TRM) to make predictions about the future of innovations and the use of usability dimensions and critical incidents of risks to the operational aspects; and (iv) to integrate HTA Brazilian guidelines in a single model, supporting researchers not related to health in the understanding of HTA and fast development of new studies for both emerging and in use equipment. The assessment of the domains selected for hemodialysis led to: (i) the identification of an annual growth in therapy indicators (7% in average); (ii) the usability diagnosis of efficient machines, however with the potential to generate greater user satisfaction considering: distance between machines, reduction in the waste of water and materials, maintenance plans to avoid high temperatures, training aiming more attention to supporting tasks as well as focus on display aspects (data feedback and visualization); and (iii) the consolidation among professionals of a future, for the next 10 years in Brazil, based on homecare with portable machines, safer and more sustainable.

Economic evaluations and network meta-analyses (NMAs) are complex methodologies. Increasing their transparency and accessibility could enhance confidence in the legitimacy of policy decisions informed by these analyses. Four systematic reviews were conducted to understand policymakers’ informational needs and to determine what guidance researchers have on how to present economic evaluations and NMAs. Qualitative interviews were conducted with Canadian policymakers, i.e., knowledge users, to understand barriers and facilitators to using and communicating economic evaluations and NMAs and with individuals in international health technology assessment organizations, i.e. knowledge producers, to explore current approaches to communicating economic evaluations and NMAs. A toolkit for NMAs and economic evaluations was proposed based on an integrated review of these findings and guided by the Knowledge-to-Action framework. Examples of tools were developed and applied to an economic evaluation and NMA of osteoporosis therapies. Systematic reviews and qualitative interviews found that communication approaches that provide robust content, identify contextual factors relevant to policy decisions and enhance clarity were valued. Twelve tools were proposed that enhance communication, education and access to resources for policymakers. Two of these tools were developed: Economic Guidance for Researchers and NMA Guidance for Researchers.

Interest in the use of medical cannabis for the treatment of drug-resistant epilepsy in children has grown over the last decade; however, little is known about its potential benefits and harms, cost-effectiveness, or the perspectives of key stakeholders. In this thesis, a health technology assessment approach was adopted to assess the intended and unintended consequences of medical cannabis use in the treatment of pediatric drug-resistant epilepsy. This thesis comprises three main sections: (1) a living systematic review of the benefits and harms of medical cannabis for the treatment of pediatric epilepsy, including drug-resistant forms; (2) an economic evaluation of the cost-effectiveness of medical cannabis for the treatment of pediatric drug-resistant epilepsy, and (3) qualitative exploration of the perspectives of neurologists and parents of children with drug-resistant epilepsy about the use of medical cannabis in this population. While neurologists generally perceived medical cannabis as a viable treatment option for drug-resistant epilepsy in children, particularly after other treatments have failed, they identified several gaps in the evidence base, including a lack of long-term studies and a lack of evidence related to cannabinoids other than cannabidiol. This is in keeping with the findings of the living systematic review, which support a beneficial role for medical cannabis in reducing seizures associated with drug-resistant epilepsy, although the certainty of the evidence was moderate at best. Parents described experiencing many barriers to accessing medical cannabis for their children, primarily related to finding a health care provider to authorize its use, the high cost of cannabis-based treatments, and a lack of reimbursement through public or private insurance programs. However, cannabinoid oil may be a more cost-effective treatment for some types of pediatric drug-resistant epilepsy compared with antiepileptic drugs currently reimbursed by some provincial insurance programs. These findings suggest that medical cannabis is a potentially effective and cost-effective treatment for drug-resistant epilepsy that may addresses an unmet need. However, additional studies are needed to address uncertainty related to the long-term benefits and harms of cannabis-based products, particularly with respect to products available in Canada.

Antibiotic resistance is a global health concern. Its increasing development is seemingly due to the irresponsible use of antibiotics and their availability. To ensure the correct treatment of patients and avoid unnecessary strain on the individual and society as a whole, it is vital to ensure access to both new and old antibiotics where there is a distinct need. Temocillin is an antibiotic used in the treatment of febrile UTIs. It is currently unavailable in Sweden, although being well-established and proven effective in other European countries. Hence, this report aims to investigate the introduction of temocillin to the Swedish or Scandinavian market in regards to the economic, technical, medical, and regulatory possibilities and barriers. The research is based on literature, internet, and database reviews and empirical data collection through interviews with people of expertise within the fields of antibiotic resistance and concerned aspects. This study emphasizes the great potential of introducing temocillin to Sweden. The evident conclusion drawn is that a production of the antibiotic is not possible in Sweden in the near future. Despite this, an identified supply chain in Europe can enable a role for Sweden as a distribution country. Through a performed break-even analysis, the calculated break-even point and profit margins indicated a low risk of financial loss for a MAH acting as the supplier of temocillin, such as the company Eumedica S.A.. Only 1 % of the Swedish market would have to be covered to break even, and regardless of the estimated volumes of usage upon introduction being either 50 % or 25 % of the total medical need, a profit is likely. In addition, temocillin holds a great medical and social value. However, the obvious obstacle observed is its out-dated documentation. The dossier is of the wrong format, which complicates the regulatory procedure. Thus, this needs to be taken into consideration for an introduction to be possible.