OBJECTIF : L’hypertension est une maladie chronique pour laquelle seulement 68 % des patients étaient contrôlés en 2013. Les pharmaciens de la province de Québec ont récemment obtenu l’autorité législative pour ajuster la dose des antihypertenseurs lorsqu’une cible thérapeutique est entendue avec le médecin. Cette étude économique vise à estimer le ratio coût- utilité incrémental (RCUI) de ce nouveau modèle de soin au Québec. MÉTHODE : Un modèle de Markov a été développé pour extrapoler l’impact de cette pratique pharmaceutique sur les accidents vasculaires cérébraux (AVC), sur les infarctus du myocarde aigus et sur la mortalité. Les équations du modèle de risque de Framingham ont été utilisées pour estimer l’impact du contrôle de la pression artérielle sur la survenue d’AVC et d’infarctus. L’efficacité de l’intervention a été estimée à partir des résultats de l’essai clinique RxAction, conduit en Alberta. L’efficacité de l’intervention pharmaceutique est exprimée via la proportion de patients contrôlés. La perspective du payeur public a été utilisée et seulement les coûts directs des soins de santé ont été intégrés. L’issue principale observée par le modèle est le gain en années de vie ajustées par la qualité (AVAQ). Les AVAQ et les coûts ont été actualisés à un taux annuel de 1,5 %. Le modèle a simulé une cohorte de 1000 patients âgés de 65 ans, sur un horizon temporel de 20 ans en utilisant des cycles d’un an. RÉSULTATS: Le modèle a généré 0,051 AVAQs de plus par patient dans le groupe d’intervention pour une dépense supplémentaire de 268 $ par patient. Le RCUI est de 5 223 $/AVAQ gagnée. Si le seuil de volonté à payer du tiers public se tient à 50 000 $/AVAQ, cette intervention serait dite coût-efficace. Les résultats de cette simulation étaient surtout sensibles à l’efficacité de l’intervention pharmaceutique ainsi qu’à l’utilité attribuée à l’hypertension. CONCLUSION: Permettre aux pharmaciens d’ajuster la dose des traitements antihypertenseurs à l’intérieur du modèle de rémunération actuel apparaît coût-efficace. L’obtention de données d’efficacité issue d’une étude clinique conduite au Québec nous permettrait d’augmenter la validité externe de cette modélisation économique. / OBJECTIVE: Hypertension is a chronic disease for which only 68% of treated patients were controlled in Canada in 2013. Pharmacists in the province of Québec recently received legislative authority to adjust the dosage of antihypertensive drugs if there is an agreement with the prescriber of the therapeutic target. This research aims to estimate the incremental cost- utility ratio (ICUR) of this new model of care in Québec. METHODS: A Markov model was developed to extrapolate the impact of this pharmacy practice on strokes, myocardial infarctions and mortality. Framingham Risk Equations were used to derive the impact of blood pressure control on strokes and myocardial infarctions. The efficacy of the intervention was derived from the RxAction clinical trial, conducted in Alberta. Efficacy was expressed as the proportion of patients with controlled blood pressure. The model used the payer perspective and direct costs only. The main clinical outcome was expressed as the number of quality-adjusted life years (QALYs) gained. Both QALYS and costs were discounted at a 1.5% annual rate. A cohort of 1000 patients entered the model at 65 years old. The model used a 20 years time horizon, with a 1-year cycle length. RESULTS: The model yielded 0.051 QALYs per patient in the intervention group for an incremental expense of $268 per patient. The ICUR was 5,223 $/QALY. At a willingness-to- pay threshold of $50,000/QALY, the intervention is cost-effective. The results were sensitive to the comparative efficacy of the pharmacist intervention against usual care and to the utility of hypertension. CONCLUSION: Providing pharmacists the ability to adjust the dosage of antihypertensive drugs within the actual fee-for-service rational appears to be cost-effective. Obtaining data on the efficacy of this pharmacy practice from a trial conducted in Québec would provide better data to inform this economic evaluation.

Les nouvelles technologies médicales contribuent aux dépenses en santé qui ne cessent de croître, alors que les budgets se trouvent limités. L’évaluation économique des technologies devraient permettre d’identifier quelles sont celles qui sont les plus rentables. Malgré cela, plusieurs technologies dont le rapport coût-efficacité reste plutôt limite ou défavorable sont utilisées en médecine moderne et remboursées par notre système public de santé. Ce mémoire se concentre sur deux technologies en santé cardiovasculaire dont le rapport coût-efficacité est plutôt limite mais qui sont fréquemment utilisées au Canada; les tuteurs médicamentés ou pharmaco-actifs et les défibrillateurs cardiaques implantables (DCI). Nous avons fait une évaluation contingente de ces technologies dans le but d’examiner si ce type d’évaluation économique complémentaire pouvait procurer un point de vue nouveau sur la valeur économique et sociétaire des ces technologies. Les résultats de ces deux évaluations indiquent que les patients accordent une grande importance aux bénéfices que procurent ces deux technologies. Nos résultats soutiennent les politiques de santé actuelles de rembourser de façon libérale ces deux technologies. / Technological innovations have greatly contributed to the rising costs in healthcare, while budgets have remained limited. Economic evaluations of technologies should identify which technologies are cost-effective. However, several technologies used in modern medicine are either borderline cost-effective or even not cost-effective according to many studies. This thesis focuses on two technologies in cardiovascular medicine which are considered borderline cost-effective; drug-eluting stents and implantable cardioverter defibrillators. We conducted a contingent valuation of these technologies in hopes of determining if this alternative type of economic evaluation could give a novel point of view on the economic and societal value of these technologies. Results indicated that patients greatly valued benefits provided by these two technologies. Our result support our public healthcare system policies’ of liberal reimbursement of these two technologies.

The most frequent intervention performed by physicians is the writing of a prescription. Modern medicine has been remarkably effective in managing diseases. Medicines play a fundamental role in the effectiveness, efficiency and responsiveness of health care systems. However, health care expenditure is a great cause for concern and many nations around the world struggle to contain rising health care costs. Pharmaceutical benefit management programmes such as pharmacoeconomics, drug utilisation review (DUR) and disease management have emerged as control tools to ensure cost effective selection and use of medicine. These managed care instruments are often used to determine whether new strategies or interventions, such as the implementation of a managed medicine reference price list, are appropriate and have "value". The general objective of this study was to investigate the influences of the implementation of a managed medicine reference price list on the usage and cost of antibiotic medicine in the private health care sector of South Africa. The research design used in this study was retrospective, non-experimental and quantitative. The data used for the analysis were obtained over a two-year study period (1 May 2001 to 31 April 2003) from the central medicine claims database of Medschem&. Data was analysed according to prevalence, cost and original (innovator) or generic medicine items. For the purpose of this study antibiotics referred to beta-lactams (penicillins, cephalosporins and "others"), erythromycin and other macrolides, tetracyclines, sulphonamides and combinations, quinolones, chloramphenicol and aminoglycosides. The results of the empirical investigation showed the total number of medicine items claimed during the study period amounted to 49098736 medicine items having a total expenditure of R7150344897.00. There was a decrease in the prevalence of original (innovator) products during the two-year period. The prevalence of generic products increased from 25.87% to 32.47%. A total of 4092495 antibiotic medicine items were claimed with a total cost of R526309279.43 representing 7.36% (n = R7150344897.00) of all pharmaceutical products purchased during the two-year period. Original antibiotics had a prevalence of 42.32%, while generic antibiotics constituted 57.68% of all antibiotic products claimed (n = 4092495). However, original (innovator) products contributed 62.32% and generic products 37.68% to the total cost of all antibiotics claimed. It was concluded that the beta-lactam antibiotics represented 56.99% of all antibiotics claimed (n = 4092495) and contributed 52.51% to the total antibiotic expenditure (n = R526309279.43) for the two-year period. The average cost of beta-lactam items ranged between R112.88 * 69.95 and R122.18 + 81.42. The Medschema Price List (MPL) was implemented in May 2001. The aim of this reference pricing system was to allocate a ceiling price to a group of drugs, which are similar in terms of composition, clinical efficacy, safety and quality, with the ultimate goal to reduce medicine expenditure. During the year of implementation of the MPL 62.24% of beta-lactam antibiotics claimed (n = 1303464) were MPL listed. These products contributed 43.25% to the total cost of all beta-lactam antibiotics (n = R157142778.38). Medical aid companies reimbursed R61649211.86 for penicillins claimed and MPL listed. If all penicillin products were claimed at the ceiling price set by the MPL, a cost saving of 2.79% could have been achieved. Cost analysis indicated that it is possible to reduce health care costs by implementing strategies with the aim to reduce medicine cost. Further research, however, is necessary and in this regard recommendations for further research were formulated. / Thesis (M.Pharm. (Pharmacy Practice))--North-West University, Potchefstroom Campus, 2005.

Les nouvelles technologies médicales contribuent aux dépenses en santé qui ne cessent de croître, alors que les budgets se trouvent limités. L’évaluation économique des technologies devraient permettre d’identifier quelles sont celles qui sont les plus rentables. Malgré cela, plusieurs technologies dont le rapport coût-efficacité reste plutôt limite ou défavorable sont utilisées en médecine moderne et remboursées par notre système public de santé. Ce mémoire se concentre sur deux technologies en santé cardiovasculaire dont le rapport coût-efficacité est plutôt limite mais qui sont fréquemment utilisées au Canada; les tuteurs médicamentés ou pharmaco-actifs et les défibrillateurs cardiaques implantables (DCI). Nous avons fait une évaluation contingente de ces technologies dans le but d’examiner si ce type d’évaluation économique complémentaire pouvait procurer un point de vue nouveau sur la valeur économique et sociétaire des ces technologies. Les résultats de ces deux évaluations indiquent que les patients accordent une grande importance aux bénéfices que procurent ces deux technologies. Nos résultats soutiennent les politiques de santé actuelles de rembourser de façon libérale ces deux technologies. / Technological innovations have greatly contributed to the rising costs in healthcare, while budgets have remained limited. Economic evaluations of technologies should identify which technologies are cost-effective. However, several technologies used in modern medicine are either borderline cost-effective or even not cost-effective according to many studies. This thesis focuses on two technologies in cardiovascular medicine which are considered borderline cost-effective; drug-eluting stents and implantable cardioverter defibrillators. We conducted a contingent valuation of these technologies in hopes of determining if this alternative type of economic evaluation could give a novel point of view on the economic and societal value of these technologies. Results indicated that patients greatly valued benefits provided by these two technologies. Our result support our public healthcare system policies’ of liberal reimbursement of these two technologies.

The most frequent intervention performed by physicians is the writing of a prescription. Modern medicine has been remarkably effective in managing diseases. Medicines play a fundamental role in the effectiveness, efficiency and responsiveness of health care systems. However, health care expenditure is a great cause for concern and many nations around the world struggle to contain rising health care costs. Pharmaceutical benefit management programmes such as pharmacoeconomics, drug utilisation review (DUR) and disease management have emerged as control tools to ensure cost effective selection and use of medicine. These managed care instruments are often used to determine whether new strategies or interventions, such as the implementation of a managed medicine reference price list, are appropriate and have "value". The general objective of this study was to investigate the influences of the implementation of a managed medicine reference price list on the usage and cost of antibiotic medicine in the private health care sector of South Africa. The research design used in this study was retrospective, non-experimental and quantitative. The data used for the analysis were obtained over a two-year study period (1 May 2001 to 31 April 2003) from the central medicine claims database of Medschem&. Data was analysed according to prevalence, cost and original (innovator) or generic medicine items. For the purpose of this study antibiotics referred to beta-lactams (penicillins, cephalosporins and "others"), erythromycin and other macrolides, tetracyclines, sulphonamides and combinations, quinolones, chloramphenicol and aminoglycosides. The results of the empirical investigation showed the total number of medicine items claimed during the study period amounted to 49098736 medicine items having a total expenditure of R7150344897.00. There was a decrease in the prevalence of original (innovator) products during the two-year period. The prevalence of generic products increased from 25.87% to 32.47%. A total of 4092495 antibiotic medicine items were claimed with a total cost of R526309279.43 representing 7.36% (n = R7150344897.00) of all pharmaceutical products purchased during the two-year period. Original antibiotics had a prevalence of 42.32%, while generic antibiotics constituted 57.68% of all antibiotic products claimed (n = 4092495). However, original (innovator) products contributed 62.32% and generic products 37.68% to the total cost of all antibiotics claimed. It was concluded that the beta-lactam antibiotics represented 56.99% of all antibiotics claimed (n = 4092495) and contributed 52.51% to the total antibiotic expenditure (n = R526309279.43) for the two-year period. The average cost of beta-lactam items ranged between R112.88 * 69.95 and R122.18 + 81.42. The Medschema Price List (MPL) was implemented in May 2001. The aim of this reference pricing system was to allocate a ceiling price to a group of drugs, which are similar in terms of composition, clinical efficacy, safety and quality, with the ultimate goal to reduce medicine expenditure. During the year of implementation of the MPL 62.24% of beta-lactam antibiotics claimed (n = 1303464) were MPL listed. These products contributed 43.25% to the total cost of all beta-lactam antibiotics (n = R157142778.38). Medical aid companies reimbursed R61649211.86 for penicillins claimed and MPL listed. If all penicillin products were claimed at the ceiling price set by the MPL, a cost saving of 2.79% could have been achieved. Cost analysis indicated that it is possible to reduce health care costs by implementing strategies with the aim to reduce medicine cost. Further research, however, is necessary and in this regard recommendations for further research were formulated. / Thesis (M.Pharm. (Pharmacy Practice))--North-West University, Potchefstroom Campus, 2005.

Dans un système de santé avec budget limité, l’efficience des différentes options thérapeutiques doit être comparée afin d’assurer une allocation optimale des ressources. À cette fin, l’analyse coût-utilité (ACU) est privilégiée par les agences d’évaluation des technologies de la santé et requiert des données pharmacoéconomiques telles que les coûts et les scores d’utilité. Toutefois, les données pharmacoéconomiques ne sont pas systématiquement collectées lors des essais contrôlés randomisés (ECR). Lorsqu’elles le sont, elles sont soumises aux limites des ECR affectant ainsi la validité externe des résultats. Cela a mené à l’émergence des études en pratique réelle (real-world evidence studies) comme évidence complémentaire aux ECR aux fins de remboursement. Les données en pratique réelle peuvent être des données primaires (recueillies spécifiquement pour répondre à un objectif particulier) ou des données secondaires (données déjà collectées pour répondre à d’autres objectifs). D’importantes données pharmacoéconomiques, telles que l’utilité, la perte de productivité et les coûts non médicaux, étant généralement manquantes dans les banques de données traditionnelles, les chercheurs en pharmacoéconomie doivent parfois générer leurs propres données. Néanmoins, il n’existe pas de lignes directrices spécifiques pour guider les chercheurs désirant générer des données pharmacoéconomiques en pratique réelle. Le but de cette thèse était d’étudier et contribuer à l’amélioration des pratiques méthodologiques pour la génération de données pharmacoéconomiques en pratique réelle. Précisément, l’objectif du volet 1 était donc de caractériser les pratiques méthodologiques des études collectant des données de pharmacoéconomie et d’évaluer la qualité des résultats obtenus. Une revue systématique de la littérature a permis de confirmer certaines craintes face aux études de pratique réelle, c’est-à-dire le manque de transparence et de crédibilité. Le volet 1 a mis de l’avant le besoin d’améliorer les méthodologies actuelles qui ne produisent pas toujours des résultats robustes, pouvant ainsi affecter la validité et la pertinence des évaluations économiques. Un cadre de recherche spécifique à la collecte de données pharmacoéconomiques en pratique réelle pourrait augmenter la qualité des résultats générés. L’objectif du volet 2 était donc d’identifier les lignes directrices spécifiques à la génération de données d’utilité. Ensuite, d’incorporer les bonnes pratiques méthodologiques dans une boîte à outils destinée spécifiquement aux investigateurs cherchant à générer des valeurs d’utilité en pratique réelle. Bien que cette thèse englobe plus largement l’ensemble des données pharmacoéconomiques, telles que les coûts et l’utilité, la boîte à outils se concentre sur les scores d’utilité uniquement. Il s’agit d’un premier pas vers des lignes directrices officielles fournissant des bonnes pratiques méthodologiques pour la conception et conduite d’études en pharmacoéconomie. Pour ce qui est du volet 3, l’objectif était de mettre en pratique la boîte à outils en rédigeant un protocole de recherche de qualité, pertinent et applicable à la réalité des chercheurs en pratique réelle. Précisément, d’utiliser les recommandations de la boîte à outils afin de mettre en place un devis d’étude et des procédures d’étude qui limitent le risque de biais. Cette thèse contribue à l’avancement des bonnes pratiques et sensibilise la communauté de chercheurs à viser un niveau hiérarchique d’évidence plus élevé. La standardisation des méthodologies pour générer des données pharmacoéconomiques en pratique réelle est un besoin qui demeure non comblé. / In a resource-constrained healthcare system, the effectiveness of various therapeutic interventions must be compared by the means of economic evaluations to ensure optimal resource allocation. The cost-utility analysis (CUA) is favored by health technology assessment (HTA) agencies, and requires health economics and outcomes research (HEOR) data such as costs and utilities. Although they have gained prominence, HEOR data are not systematically collected in randomized controlled trials (RCTs). Even if they were, RCTs are well-known to have inherent limitations leading to a low external validity. Consequently, real-world evidence (RWE) studies have been used as complementary evidence in reimbursement decision-making. Real-world data can be primary data (specifically collected to achieve a certain objective) or secondary data (data that have already been collected for other purposes). However, important HEOR data, such as utility scores, productivity and non-medical costs, are not routinely collected in traditional databases. Therefore, HEOR investigators may need to generate their own pharmacoeconomic data. Nevertheless, there is no single guideline for investigators seeking guidance on methodological steps to estimate HEOR data in a real-world setting. The goal of this thesis was to study and improve good practices for HEOR data generation in a real-world setting. Specifically, the objective of the first article was to characterize methodological practices for collecting HEOR data as well as critically appraise the quality of the results. The systematic literature review confirmed concerns about RWE studies, namely the lack of transparency and credibility. The first article highlighted the need for good practices as current methodologies may not generate robust estimates, thereby affecting the validity and relevance of economic evaluations. A research framework specifically designed for HEOR data collection in a real-world setting could help improve the quality of results. Thus, the objective of the second article was to review current recommendations for health state utility (HSU) generation, and incorporate methodological standards into a single toolbox intended for investigators seeking to collect HSU in a real-world setting. Although this thesis broadly encompasses HEOR data, such as costs and utilities, the toolbox focuses on utility scores only. This is a first step toward official guidelines providing good practice recommendations for designing and conducting HEOR studies. As for the third article, the objective was to use the toolbox to develop a study protocol that is relevant, realistic and of good methodological quality. The recommendations of the toolbox were followed to design the study and limit bias. This thesis provides good practices to help generate high quality HEOR estimates, and motivate investigators to improve the level of evidence to be generated. Standardization of RWE studies collecting HSU has yet to come.