Background: Great strides have been made in combating malaria, however, the indicators in sub Saharan Africa still do not show promise for elimination in the near future as malaria infections still result in high morbidity and mortality among children. The abundance of the malaria-transmitting mosquito vectors in these regions are driven by climate suitability. In order to achieve malaria elimination by 2030, strengthening of surveillance systems have been advocated. Based on malaria surveillance and climate monitoring, forecasting models may be developed for early warnings. Therefore, in this thesis, we strived to illustrate the use malaria surveillance and climate data for policy and decision making by assessing the association between weather variability (from ground and remote sensing sources) and malaria mortality, and by building malaria admission forecasting models. We further propose an economic framework for integrating forecasts into operational surveillance system for evidence based decisionmaking and resource allocation.  Methods: The studies were based in Asembo, Gem and Karemo areas of the KEMRI/CDC Health and Demographic Surveillance System in Western Kenya. Lagged association of rainfall and temperature with malaria mortality was modeled using general additive models, while distributed lag non-linear models were used to explore relationship between remote sensing variables, land surface temperature(LST), normalized difference vegetation index(NDVI) and rainfall on weekly malaria mortality. General additive models, with and without boosting, were used to develop malaria admissions forecasting models for lead times one to three months. We developed a framework for incorporating forecast output into economic evaluation of response strategies at different lead times including uncertainties. The forecast output could either be an alert based on a threshold, or absolute predicted cases. In both situations, interventions at each lead time could be evaluated by the derived net benefit function and uncertainty incorporated by simulation.  Results: We found that the environmental factors correlated with malaria mortality with varying latencies. In the first paper, where we used ground weather data, the effect of mean temperature was significant from lag of 9 weeks, with risks higher for mean temperatures above 250C. The effect of cumulative precipitation was delayed and began from 5 weeks. Weekly total rainfall of more than 120 mm resulted in increased risk for mortality. In the second paper, using remotely sensed data, the effect of precipitation was consistent in the three areas, with increasing effect with weekly total rainfall of over 40 mm, and then declined at 80 mm of weekly rainfall. NDVI below 0.4 increased the risk of malaria mortality, while day LST above 350C increased the risk of malaria mortality with shorter lags for high LST weeks. The lag effect of precipitation was more delayed for precipitation values below 20 mm starting at week 5 while shorter lag effect for higher precipitation weeks. The effect of higher NDVI values above 0.4 were more delayed and protective while shorter lag effect for NDVI below 0.4. For all the lead times, in the malaria admissions forecasting modelling in the third paper, the boosted regression models provided better prediction accuracy. The economic framework in the fourth paper presented a probability function of the net benefit of response measures, where the best response at particular lead time corresponded to the one with the highest probability, and absolute value, of a net benefit surplus.  Conclusion: We have shown that lagged relationship between environmental variables and malaria health outcomes follow the expected biological mechanism, where presentation of cases follow the onset of specific weather conditions and climate variability. This relationship guided the development of predictive models showcased with the malaria admissions model. Further, we developed an economic framework connecting the forecasts to response measures in situations with considerable uncertainties. Thus, the thesis work has contributed to several important components of early warning systems including risk assessment; utilizing surveillance data for prediction; and a method to identifying cost-effective response strategies. We recommend economic evaluation becomes standard in implementation of early warning system to guide long-term sustainability of such health protection programs.

L’insomnie, commune auprès de la population gériatrique, est typiquement traitée avec des benzodiazépines qui peuvent augmenter le risque des chutes. La thérapie cognitive-comportementale (TCC) est une intervention non-pharmacologique ayant une efficacité équivalente et aucun effet secondaire. Dans la présente thèse, le coût des benzodiazépines (BZD) sera comparé à celui de la TCC dans le traitement de l’insomnie auprès d’une population âgée, avec et sans considération du coût additionnel engendré par les chutes reliées à la prise des BZD. Un modèle d’arbre décisionnel a été conçu et appliqué selon la perspective du système de santé sur une période d’un an. Les probabilités de chutes, de visites à l’urgence, d’hospitalisation avec et sans fracture de la hanche, les données sur les coûts et sur les utilités ont été recueillies à partir d’une revue de la littérature. Des analyses sur le coût des conséquences, sur le coût-utilité et sur les économies potentielles ont été faites. Des analyses de sensibilité probabilistes et déterministes ont permis de prendre en considération les estimations des données. Le traitement par BZD coûte 30% fois moins cher que TCC si les coûts reliés aux chutes ne sont pas considérés (231$ CAN vs 335$ CAN/personne/année). Lorsque le coût relié aux chutes est pris en compte, la TCC s’avère être l’option la moins chère (177$ CAN d’économie absolue/ personne/année, 1,357$ CAN avec les BZD vs 1,180$ pour la TCC). La TCC a dominé l’utilisation des BZD avec une économie moyenne de 25, 743$ CAN par QALY à cause des chutes moins nombreuses observées avec la TCC. Les résultats des analyses d’économies d’argent suggèrent que si la TCC remplaçait le traitement par BZD, l’économie annuelle directe pour le traitement de l’insomnie serait de 441 millions de dollars CAN avec une économie cumulative de 112 billions de dollars canadiens sur une période de cinq ans. D’après le rapport sensibilité, le traitement par BZD coûte en moyenne 1,305$ CAN, écart type 598$ (étendue : 245-2,625)/personne/année alors qu’il en coûte moyenne 1,129$ CAN, écart type 514$ (étendue : 342-2,526)/personne/année avec la TCC. Les options actuelles de remboursement de traitements pharmacologiques au lieu des traitements non-pharmacologiques pour l’insomnie chez les personnes âgées ne permettent pas d’économie de coûts et ne sont pas recommandables éthiquement dans une perspective du système de santé. / Insomnia is common in the geriatric population, typically treated with benzodiazepine drugs which can increase the risk of falls. Cognitive behavioral therapy (CBT) is a non-pharmacological intervention with equivalent efficacy and no adverse events. This thesis compares the cost of benzodiazepines versus CBT for the treatment of insomnia in older adults, with and without consideration of the additional cost of falls incurred by benzodiazepine use. A decision tree model was constructed and run from the health payer’s perspective over 1 year. The probability of falls, ER visits, hospitalisation with and without hip fracture, cost data and utilities were derived from a comprehensive literature review. Cost consequence, cost utility and potential cost saving analyses were performed. Both probabilistic and deterministic sensitivity analyses were conducted to account for uncertainty around the data estimates. Benzodiazepine treatment costs 30% less than the price of CBT when the costs of falls are not considered (CAN $231 vs. CAN $335 per individual per year). When the cost of falls is considered, CBT emerges as the least expensive option (absolute cost-saving CAN$ 177 per person per year, CAN $1,357 with benzodiazepines vs. $1,180 for CBT). CBT dominated benzodiazepines, with a mean cost saving of CAN $ 25,743 per QALY gained with CBT due to fewer falls. The cost savings analysis shows that if the CBT were to completely replace benzodiazepine therapy, the expected annual direct cost savings for the treatment of insomnia would be $ 441 million CAD dollars, with a cumulative cost savings of $112 billion CAD dollars over 5-years. The PSA report shows that even at different varying parameters, benzodiazepines cost CAD$ 1,305, S.D $ 598 (range 245-2,625) on average / person / year vs. CAD$ 1,129, S.D $ 514 (range 342-2,526) on average / person / year for CBT. Current treatment reimbursement options that fund pharmacologic therapy instead of non-pharmacologic therapy for geriatric insomnia are neither cost-saving nor ethically recommendable from the health system’s perspective.

No description available.

As doenças raras são epidemiologicamente caracterizadas por ocorrerem entre 0,65-1:1.000 indivíduos. Houve importante incremento no surgimento de tratamentos específicos para muitas destas condições a partir da publicação de legislações específicas em diferentes países (sendo o pioneiro em 1983 nos EUA o chamado 'Orphan Drug Act'). Dentre as doenças que dispõem atualmente de tratamento específico está a Doença de Fabry, uma doença genética classificada erro inato do metabolismo do grupo das doenças Lísossômica se caracterizada pelo acúmulo de glicoesfíngol ipídíos no endotélio vascular, o que ocasiona problemas renais, cardíovasculares e neurológicos. Os problemas renais e cardíovasculares são os responsáveis pela maior morbi-mortalídade entre os portadores da doença. Esta ocorre em homens e mulheres, com manifestações heterogêneas entre indivíduos e diferentes gêneros. O tratamento específ ico da doença é através da chamada Terapia de Reposição Enzimática (TRE) havendo duas opções existentes: a agalsidase alfa e a agalsidase beta. Não há, porém, consenso sobre os resultados da TRE sobre desfechos clínicos relevantes. O alto custo associado a esta terapêutica (cerca de 200 mil dólares/ano/por paciente adulto) é o dificulta o acesso dos pacientes à mesma. Uma das formas se racionalizar a incorporação de tecnologias novas na área de saúde, que se caracteriza empreender uma avaliação objetiva de benefícios e custos associados é a chamada Avaliação de Tecnologias de Saúde. Este conjunto de métodos é cada vez mais empregado para auxiliar ou nortear decisões na área de saúde. Existem, no entanto , dificuldades associadas ao seu emprego no campo das doenças raras. Nossa proposta consiste em tendo como modelo a Doença de Fabry, obter um panorama da situação no Brasil a o acesso a tratamentos de alto custo para doenças raras, e, analisar a aplicabilidade dos princípios da avaliação de tecnologias de saúde no campo das doenças raras, tentando por fim colaborar no aprimoramento do processo de acessibilidade a estes tratamentos. OBJETIVOS Objetivo geral : Avaliar o acesso no Brasil a tratamentos de doenças raras e a aplicabilidade da avaliação de tecnolog ias em saúde no contexto específico destas. Objetivos específicos: 1) Caracterizar as políticas existentes no Brasil em relação ao acesso de medicamentos para doenças raras e avaliar as formas empregadas para este acesso. 2) Colaborar no aprimoramento do processo de incorporação destes tratamentos (de al to custo) no segmento das doenças raras, através da aplicação de principias de 'aval iação de tecnologias em saúde', usando como exemplo a Doença de Fabry. 3) Identificar e propor possíveis estratégias que possam colaborar para uma acessibilidade adequada e justa a tratamentos cl inicamente efetivos no campo das doenças raras. MÉTODOS 1) Foi realizada uma revisão da literatura caracterizando as politicas brasileiras na área de medicamentos e aval iando as formas de acesso utilizadas pelos pacientes; 2) Uma segunda revisão, de caráter sistemático, sobre os efeitos da TRE na DF, foca d a nos efe itos sobre a nefropatia e a cardiopatia (sistemas cujo comprometimento em pacientes com DF causa maior morbi-mortalidade) da doença foi feita. Baseado nos dados obtidos elaborou-se um modelo probabilístico (markoviano) para avaliar o efeito da TRE sobre a nefropatia da DF seguido de aval iação do custo-efetividade. A partir de uma análise destes resultados e da li teratura, foram identificados os aspectos que mais influenciariam a acessibilidade ao tratamento, seguida da elaboração de propostas para auxiliar a incorporação e a acessibil idade a estes tratamentos. RESULTADOS 1) A política de assistência farmacêutica brasileira atual é baseada em elencos de medicamentos divididos em atenção básica e do 'componente especiali zado'. Não existe uma política especifica direcionada aos tratamentos (de alto custo) dos portadores de doenças raras. 2) A revisão empreendida mostrou que dentre os dois sistemas aval iados de forma particular (pela morbi-mortalidade associada) havia mais dados quer sobre história natural , quer sobre efeitos da TRE em relação à nefropatia, tendo sido esta escolh ida então como foco da modelagem . O modelo construido foi capaz de identificar um subgrupo de pacientes com DF que quando tratado com TRE tem redução significativa (diferença absoluta de 10%) na probabilidade de ter progressão da nefropatia (evoluir a estágio dialítico). A despeito dos benefícios observados a análise mostrou ser esta opção não custo-efetiva no contexto existente (custo é superior ao limite preconizado pela OMS de s 3 vezes o PIB per capíta do Brasil). 3) A acessibilidade é ainda um obstáculo ao uso destes medicamentos. O custo é o limitante central da acessibilidade aos tratamentos específicos destas patologias. Atuar em aspectos associados a este e às pol íticas vigentes seriam formas de tentar mudar este cenário. CONCLUSÕES 1) Não há em nosso país políticas que sistematizem o processo de incorporação de tratamentos (de alto custo) para doenças raras. O acesso a estes é disperso, no caso da DF não existe disponibil idade da terapia de reposição enzimática via SUS. 2) A revisão sobre o efeito da TRE na nefropatia mostrou resultados muito heterogêneos. Foi possivel, no entanto, elaborar um modelo avaliando TRE na nefropatia da DM, o qual é o primeiro conhecido utilizando este desfecho especifico e elaborado dentro contexto brasileiro. A despeito de identificado um subgrupo de pacientes que poderia beneficiar-se significativamente com o tratamento especifico a estratégia não foi custo-efetiva para este desfecho. Os custos associados foram o limitante central. Novas opções em termos de efetivação da incorporação destas tecnologias devem ser consideradas. / BACKGROUND. According to the WHO definition rare diseases occur between 0.65-1/1,000 individuals. The U.S. 'Orphan Drug Act' in 1983', the first legislation with incentives for the development of therapies for rare diseases had an impact on the development of treatments for these diseases. Fabry Disease (FD) is a rare genetic disease characterized by accumulation of glycosphingolipids in vascular endothelium leading to systemic dysfunction (renal, cardiovascular, and neurologic disease). This disease has specific treatment available, with two options of recombinant enzymes (alfa or betagalsidase) for enzyme replacement therapy (ERT). There are few controlled trials evaluating their effects, indicating some improvements in neuropathic pain, in heart abnormalities and in globotriaosylceramide (GL-3) levels. Nevertheless most of the clinical benefits of ERT are still unclear. Another aspect is the high cost associated with this treatment which makes it not easily accessible. OBJECTIVES: General Objective: Evaluate access to rare disease treatments in Brazil and the applicability of health technology assessment (HTA) in the context of these diseases. Specific objectives: 1) Evaluate the Brazilian governmental policies for rare diseases treatment and how the access to the treatments is actually done. 2) Evaluate the applicability of health technology assessment (HTA) in the context of rare diseases, using the example of Fabry disease. 3) Identify and propose strategies that could contribute to a fair access to clinically effective treatments for of rare diseases. METHODS: An extensive literature review about the Brazilian policies in the area was performed. In the sequence a systematic review about the subject ERT and FD was conducted. After this, a model estimating the likelihood of nephropathy progression with or without ERT was built, followed by a cost-effectiveness analysis. Another literature review focused in the identification of obstacles to accessibility and possible strategies to overcome them was conducted. RESULTS: 1) There is no specific policy in Brazil regarding high cost drugs for the treatment of rare diseases. 2) ERT appears to slow the progression of nephropathy in the Fonly proteinuria subgroup '. However the cost associated is very high making this option not cost-effective. 3) The accessibility to innovative treatments for rare diseases is not adequate and the high cost of these therapies is a major obstacle to change this scenario. CONCLUSION: There are no policies in Brazil to systematize the access to the specific (high cost) treatments for rare diseases. The model evaluating TRE in FD nephropathy was able to identify benefits for a subgroup of patients. It was the first known model using this specific outcome and built focusing the Brazilian context. However the ERT strategy was not cost-effective for this outcome. The costs associated with these therapies are very high and an important limiting factor to the access. the central. New options should be considered to offer adequate access to the (effective) therapies.

Face aux problématiques d’accès, de désertification médicale et de qualité des soins, la loi « Hôpital, Patients, Santé, Territoires » (HPST), votée en 2009, représente un socle pour la réorganisation des soins primaires en France. L’accent est mis sur la collaboration entre les professionnels de santé et l’optimisation des compétences de chacun. Le pharmacien d’officine se trouve au coeur de cette loi. Grâce à son accessibilité, sa formation et sa proximité avec les patients sains comme malades, il voit l’opportunité d’étendre ses pratiques au-delà de la simple dispensation des médicaments en fournissant directement des soins au patient. Cette proposition n’est pas une exception française mais s’inspire des expériences internationales et du concept de « soins pharmaceutiques ». Le pharmacien écossais peut désormais être payé à la capitation pour délivrer des consultations pharmaceutiques lors du renouvellement d’ordonnance au patient atteint de maladie chronique; le pharmacien anglais peut prescrire des médicaments dans le cadre d’un plan de gestion clinique et recevoir un honoraire de dispensation; le pharmacien suisse peut organiser des réunions de discussion avec les médecins; et le pharmacien portugais peut vacciner son patient au sein de l’officine. Les illustrations de la diversification des pratiques du pharmacien d’officine sont donc variées et se replacent dans leur contexte national. L’enjeu est aujourd’hui de comprendre les conditions et les effets d’un ensemble d’innovations techniques, organisationnelles et sociales qui pourraient être en faveur du développement des soins pharmaceutiques en France. Ce travail de thèse a pour objectif de nourrir les débats autour de cette problématique. Un état des lieux de la profession en France et une enquête d’opinion auprès des futurs pharmaciens montrent que le contexte sanitaire, professionnel, économique est propice au développement des pratiques du pharmacien et que l’avenir des soins pharmaceutiques trouve un écho favorable auprès des pharmaciens de demain. Cependant, nombreux sont les facteurs qui peuvent constituer des obstacles à leur diffusion (Chapitres 1 et 2). D’un point de vue économique, d’après une revue de littérature systématique sur l’efficience des soins pharmaceutiques en Europe, les services de surveillance médicamenteuse, de médication officinale, de collaboration entre les professionnels de santé et de promotion de la santé pourraient être, dans certaines conditions, des interventions coût-efficaces d’un point de vue collectif (Chapitre 3). Mais qu’en est-il en France ? On ne dispose pas de travaux sur le sujet. Deux travaux originaux - une étude de cohorte (exposés/non exposés) et une analyse coût-efficacité -, que nous avons menés, se focalisant sur l’implication du pharmacien d’officine dans le dépistage d’une maladie chronique (le syndrome d’apnées du sommeil) montrent que les coûts engendrés par la mise en place d’une telle intervention sont sous certaines conditions compensés par les gains générés, et les résultats sont en faveur de l’implantation du service en pratique de routine (Chapitre 4). / Facing the issues of access, quality and proximity, the "Hospital, Patients, Health and Territories" (HPST) law, passed in 2009, constitutes a basis for the reorganization of primary care in France. The healthcare reform emphasizes on the collaboration between healthcare professionals and the optimization of their skills. The community pharmacist is on the forefront of this reform. Through its accessibility, its training and its proximity with healthy and sick patients, community pharmacists have the opportunity to broaden the scope of their practices beyond the merely dispensing of medication by delivering patient care. This proposal is not a French exception but takes inspiration from international experiences and the concept of "pharmaceutical care." The Scottish pharmacist can now be paid by capitation to renew a prescription for a patient with chronic illness; the English pharmacist can prescribe medication as part of a clinical management plan and receive a dispensing fee, the Swiss pharmacist can organize meetings with general practitioners and in Portugal, pharmacists can vaccinate a patient in the pharmacy. Pharmaceutical care practices are diversified and depend on national context. The challenge today is to understand the conditions and consequences of technical, organizational and social innovations that could be in favor of the development of pharmaceutical care in France. The objective of the thesis is to feed into the debates around this problematic. An overview of profession in France and an original opinion survey of future pharmacists show that the healthcare, professional and economic contexts are suitable to develop pharmaceutical care and future pharmacists are in favor of it. However, there are many barriers to their spread in routine practice (Chapters 1 and 2). From an economical perspective, a systematic review of the literature on the efficiency of pharmaceutical care in Europe show that drug monitoring services, prescription for minor ailments, collaboration between health professionals and health promotion interventions could be cost-effective from a collective point of view under certain conditions (Chapter 3). What about France? There is no study on this topic. We conducted two original studies - a cohort study (exposed / unexposed), and a cost-effectiveness analysis which focused on the involvement of the pharmacist in screening for chronic disease (sleep apnea syndrome). We show that the costs of implementing such an intervention are offset by the gains under certain conditions, and the results are in favor of the implementation of the service in routine practice (Chapter 4).

Le diabète de type 2 (DT2) est une maladie chronique associée à de graves et coûteuses complications. Dans un contexte de restriction budgétaire, il est nécessaire de pouvoir estimer les ressources à affecter à la prise en charge des maladies chroniques et donc de suivre l’évolution épidémiologique et économique d’une telle maladie. Une base de données a été construite à partir des données médico-administratives de l’assurance maladie luxembourgeoise. Elle comprenait les consommations de soins, associées au diabète et ses complications, des patients diabétiques de type 2 traités entre 2000 et 2006. L’objectif était d’étudier les champs d’utilisation de ces données et leurs applications possibles pour les décisions en santé publique. Cette thèse en donne quelques exemples. En 2006, la prévalence du DT2 au Luxembourg était de 3,79% (N= 17 070). Un algorithme a permis d’identifier trois stades de la néphropathie diabétique (3,77% des cas de DT2 en 2006). L’analyse de l’adhérence aux recommandations européennes de bonnes pratiques médicales a mis en évidence une situation critique associée à certains facteurs (médecin traitant, type de traitement, région de résidence…). Les dépenses moyennes d’un patient en hémodialyse a été estimé à 116 647€/patient en 2006. Enfin, une analyse médico-économique a montré la dominance coût-efficace d’une stratégie d’implémentation de la dialyse péritonéale sur la situation actuelle. Malgré les difficultés à évaluer leur qualité, les données médico-administratives offrent une source d’informations précieuses pour les décideurs publics et les professionnels de la santé, dans le but d’améliorer la prise en charge des patients. / Type 2 diabetes (T2D) is a chronic disease associated with many severe and costly complications. In a context of budgetary constraint, it is necessary to obtain an estimate the amount of resources to allocate to the management of chronic diseases. This includes monitoring the epidemiologic and economic evolutions. A database was built from medico-administrative databases of the national health insurance of Luxembourg. It included the healthcare consumptions associated with diabetes and its complications, of all type 2 diabetic patients treated in Luxembourg between 2000 and 2006. The objectives were to study the fields of use of this database and the possible applications for public health decision-making. This thesis gives some examples. In 2006, T2D prevalence in Luxembourg was 3.79% (N= 17070). An algorithm was built and permitted to identify three stages of diabetic nephropathy (3.77% of T2D cases in 2006). The analysis of the adherence to European follow-up guidelines showed a critical situation associated to several factors (treating physician, type of treatment, living region…). The mean costs associated with patients in dialysis were estimated at 116 647€/patient in 2006. Finally, a health-economic evaluation showed the dominance of a strategy promoting peritoneal dialysis in Luxembourg over the present situation.

L’insomnie, commune auprès de la population gériatrique, est typiquement traitée avec des benzodiazépines qui peuvent augmenter le risque des chutes. La thérapie cognitive-comportementale (TCC) est une intervention non-pharmacologique ayant une efficacité équivalente et aucun effet secondaire. Dans la présente thèse, le coût des benzodiazépines (BZD) sera comparé à celui de la TCC dans le traitement de l’insomnie auprès d’une population âgée, avec et sans considération du coût additionnel engendré par les chutes reliées à la prise des BZD. Un modèle d’arbre décisionnel a été conçu et appliqué selon la perspective du système de santé sur une période d’un an. Les probabilités de chutes, de visites à l’urgence, d’hospitalisation avec et sans fracture de la hanche, les données sur les coûts et sur les utilités ont été recueillies à partir d’une revue de la littérature. Des analyses sur le coût des conséquences, sur le coût-utilité et sur les économies potentielles ont été faites. Des analyses de sensibilité probabilistes et déterministes ont permis de prendre en considération les estimations des données. Le traitement par BZD coûte 30% fois moins cher que TCC si les coûts reliés aux chutes ne sont pas considérés (231$ CAN vs 335$ CAN/personne/année). Lorsque le coût relié aux chutes est pris en compte, la TCC s’avère être l’option la moins chère (177$ CAN d’économie absolue/ personne/année, 1,357$ CAN avec les BZD vs 1,180$ pour la TCC). La TCC a dominé l’utilisation des BZD avec une économie moyenne de 25, 743$ CAN par QALY à cause des chutes moins nombreuses observées avec la TCC. Les résultats des analyses d’économies d’argent suggèrent que si la TCC remplaçait le traitement par BZD, l’économie annuelle directe pour le traitement de l’insomnie serait de 441 millions de dollars CAN avec une économie cumulative de 112 billions de dollars canadiens sur une période de cinq ans. D’après le rapport sensibilité, le traitement par BZD coûte en moyenne 1,305$ CAN, écart type 598$ (étendue : 245-2,625)/personne/année alors qu’il en coûte moyenne 1,129$ CAN, écart type 514$ (étendue : 342-2,526)/personne/année avec la TCC. Les options actuelles de remboursement de traitements pharmacologiques au lieu des traitements non-pharmacologiques pour l’insomnie chez les personnes âgées ne permettent pas d’économie de coûts et ne sont pas recommandables éthiquement dans une perspective du système de santé. / Insomnia is common in the geriatric population, typically treated with benzodiazepine drugs which can increase the risk of falls. Cognitive behavioral therapy (CBT) is a non-pharmacological intervention with equivalent efficacy and no adverse events. This thesis compares the cost of benzodiazepines versus CBT for the treatment of insomnia in older adults, with and without consideration of the additional cost of falls incurred by benzodiazepine use. A decision tree model was constructed and run from the health payer’s perspective over 1 year. The probability of falls, ER visits, hospitalisation with and without hip fracture, cost data and utilities were derived from a comprehensive literature review. Cost consequence, cost utility and potential cost saving analyses were performed. Both probabilistic and deterministic sensitivity analyses were conducted to account for uncertainty around the data estimates. Benzodiazepine treatment costs 30% less than the price of CBT when the costs of falls are not considered (CAN $231 vs. CAN $335 per individual per year). When the cost of falls is considered, CBT emerges as the least expensive option (absolute cost-saving CAN$ 177 per person per year, CAN $1,357 with benzodiazepines vs. $1,180 for CBT). CBT dominated benzodiazepines, with a mean cost saving of CAN $ 25,743 per QALY gained with CBT due to fewer falls. The cost savings analysis shows that if the CBT were to completely replace benzodiazepine therapy, the expected annual direct cost savings for the treatment of insomnia would be $ 441 million CAD dollars, with a cumulative cost savings of $112 billion CAD dollars over 5-years. The PSA report shows that even at different varying parameters, benzodiazepines cost CAD$ 1,305, S.D $ 598 (range 245-2,625) on average / person / year vs. CAD$ 1,129, S.D $ 514 (range 342-2,526) on average / person / year for CBT. Current treatment reimbursement options that fund pharmacologic therapy instead of non-pharmacologic therapy for geriatric insomnia are neither cost-saving nor ethically recommendable from the health system’s perspective.

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The Healthy Alberta Communities (HAC) is a community-based chronic disease prevention project that draws on a wide spectrum of community-initiated interventions undertaken as a cluster in four Alberta communities since 2005. HAC-funded collaborative projects are undertaken with local stakeholders. Community stakeholders who buy in contributed their own resources in kind in the collaborative process. These in kind resources are considered HAC's indirect cost from a societal perspective since stakeholders forgo the benefit of using these resources for themselves, a forgone best alternative. This study proposes a methodology to identify, catalogue and count these in kind resources, called indirect cost, which will be used in HAC economic evaluation. Methodological challenges of identifying, cataloguing and counting both direct anad indirect costs for a cluster of diverse interventions, and the manner with which these challenges were addressed, are explained. Both direct and indirect cost data that span up to the first 24 months in two HAC communities were analyzed. Some results included are: (1)in kind resources are counted in number of in kind person-hours; (2) a combined total of 11,483 in kind person-hours from community stakeholders were catalogued and counted over an eight-month period; (3) in a monetary context, a suggested typical operating expenditure to generate one in kind person-hour using a HAC model (one head office, two community offices) was $15.58. This is the first study to directly measure resources donated in kind in public health. / Epidemiology

This dissertation consists of six essays in health economics.The first essay, “Economic evaluations in health care: Basic principles and special topics”, serves as an introduction to economic evaluations in health care, including estimations of costs, health effects, and the discount rate. Special topics of interest for the rest of the studies are also discussed, e.g. the role of modelling in cost-effectiveness analysis, and methods for dealing with incomplete observations in clinical trial data. The main theme of the second essay, “Consumption and production by age in Sweden: Basic facts and health economic implications”, is a fairly detailed compilation of consumption and production figures by age in Sweden. The purpose of this is to use the difference between consumption and production in each age group as a measure of the average costs of added years of life in the general population. In economic evaluations of health care interventions, only future costs for related ill­nesses have typically been included in the analysis. However, the health economist David Meltzer has argued that future costs for un­related illnesses and general consumption should also be in­cluded in eco­nomic evaluations. Otherwise, the analysis will not be consistent with expected utility maximiza­tion. The third essay is entitled “The possibility of predicting health care costs in the future from predicted changes in age structure and age specific mortality: The case of Sweden”. Changes in the age structure, especially the growing number of elderly people, have raised concerns about increasing costs for health and elderly care in the future. However, the number of elderly per se is not the main problem, since the growing number of elderly people is a result of better health and hence lower morta­lity. The main purpose of the study is to investigate if future health care costs can be predicted based on forecasts of future changes in age structure and mortality rates. It is shown here that at least in Sweden and in the U.S., there is a linear relationship between age-specific mortality and age-specific health care costs. When these relationships are applied retrospectively to old data, however, the predictions are underestimates of the actual costs. These results are in line with earlier studies, which show that the future age structure is not likely to have a great impact on the future health care costs. The fourth essay is called “Cost effectiveness of bisoprolol in the treatment of chronic congestive heart failure in Sweden: Analysis using data from the Cardiac Insufficiency Bisoprolol Study II” (with Niklas Zethraeus and Bengt Jönsson). Treatment of heart failure with beta blockers was introduced in Sweden already in the 1970s, but it was not until the 1990s that large-scale clinical trials established the efficacy of beta blockers in reducing heart failure mortality. The study consists of an economic evaluation of the beta blocker bisoprolol added to standard treatment of chronic heart failure, compared with placebo added to the same standard treatment. The study raises a number of methodological issues. At the forefront are the inclusion of costs of added years of life, and the question of how to model health effects that extend beyond the clinical trial on which the economic evaluation is based. The results indi­cate that treatment with bisoprolol is cost-effective. A drawback of the analysis in the fourth study was that the expected survival after the end of follow-up was modelled deterministically. This makes it impossible to assess the uncertainty of the cost-effectiveness estimate in a realistic way. The fifth essay is entitled “Assessing uncertainty in cost-effectiveness analysis by combining resampling of clinical trial data with stochastic modelling: The economic evaluation of bisoprolol for heart failure revisited”. Here, the drawback with the fourth study that was mentioned above is addressed by using resamp­ling of the clinical trial data in combination with stochastic modelling of the expected survival after the end of follow-up in the clinical trial. The methodology is inspired by the bootstrap method, which is a simulation technique whereby various statistics, like the mean and variance, can be estimated through repeated resampling from the original sample. The difference from the traditional boot­strap method is that resampling of observations from the clinical trial data is combined with stochastic modelling of the expected remaining lifetime of the patients who were alive at the end of the clinical trial. Cost-effectiveness acceptability curves for treatment of heart failure with bisoprolol were obtained as a result of the analysis. The sixth essay, “Survival analysis techniques for estimating the costs attributable to head and neck cancer in Sweden”, concerns the estimation of average treatment cost attri­butable to a disease when the data contain censored, i.e. incomplete, observations. For various reasons, censored observations are common in medical and epidemiological studies. As a result, the length of the survival time or the size of the costs for those who are alive at the end of follow-up are not exactly known. This is of course problematic if we want to estimate the average survival time or the average cost for all patients, both survivors and non-survivors included. In this study, the Kaplan-Meier sample-average estimator is used for overcoming the problem with censored observations. It is a method that has been proposed specifically for handling censored cost data. / Diss. Stockholm: Handelshögsk., 2002