Les objectifs de ma thèse étaient d'analyser les avantages et les inconvénients de l'approche bayésienne et développer leur utilisation dans le contexte de l'évaluation médico-économique. Deux projets ont été développés en utilisant l'approche bayésienne. Le premier projet était une revue systématique de la littérature et méta-analyse en réseaux pour estimer l'efficacité et la tolérance mirabegron 50 mg par rapport aux autres traitements anti-muscarinique dans la gestion de l’hyperactivité vésicale. Une revue de littératures a permis d’identifier les données puis des modèles à effet fixes et effet aléatoires ont été utilisés. Le deuxième projet est le développement de la méthode de calibration bayésienne pour estimer les probabilités de transition d’un modèle de Markov dans la maladie de Parkinson. Une étude de simulation a été effectuée pour comparer les résultats de l'approche bayésienne et l'approche classique. Toutes les analyses ont été effectuées en utilisant les logiciels libres WinBUGS et R. Résultats: La méta-analyse bayésienne en réseaux est un outil très utile pour gérer la comparaison directe et indirecte de traitements. la calibration Bayésiennes, les résultats de l’approche bayésiens sont distribués de façon similaire par rapport aux résultats de l'étude de référence.Conclusion: En comparaison avec l’approche classique, l’approche bayésienne à une meilleure base mathématique et philosophique, offre une plus grande flexibilité, et fournit des résultats sous une forme plus naturelle et intuitive. L'utilisation de l'approche bayésienne devrait se poursuivre et se développer dans le domaine de l'économie de la santé. / The objectives of this thesis were to review and develop the use of Bayesian approach in the context of heath economic evaluation. Methods: Two projects of health economics were developed to assess the benefit of Bayesian approach in health economics. The first project was a systematic literature review and mixed treatment comparison to estimate the relative efficacy and safety of mirabegron compared to antimuscarinics treatments in the management of over active bladder (OAB). The second project was the development of Bayesian calibration method to estimate the transition probabilities from cost-effectiveness model. A simulation study was performed to compare the results of Bayesian approach and classical approach. All the analyses were performed using free software WinBUGS and R.Results: The Bayesian approach is widely used in health economic evaluation and it is accepted tool to analyse the data from most of health technology agencies (e.g. HAS, NICE). The Bayesian mixed treatment comparison found to be a very useful framework to handle the comparison of treatments using the information from direct and indirect treatment comparison. The Bayesian calibration method provided results similarly distributed compared to the results of reference study (i.e. simulation study). Conclusion:Bayesian approach has better mathematical and philosophical foundation, offers greater flexibility, and provides results in a more natural and intuitive form. The use of Bayesian approach is expected to continue and grow in the field of health economics and outcomes research, because failing of frequentist to reply to some difficult question and due to the development of high-power computers.

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

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

Bakgrund: Kostnadsökningen inom hälso- och sjukvården har varit ett stort problem sedan 1980-talet och kommer att fortsätta vara ett problem i framtiden. Det har lett till en mer ansträngd ekonomisk situation som hälso- och sjukvården fått förhålla sig till. Därför är det av stor vikt att de disponibla medel som finns inom hälso- och sjukvården allokeras till de områden där de skapar som mest nytta. Syfte: Syftet med studien är att göra en hälsoekonomisk utvärdering av ett nytt arbetssätt för behandling av knäskador på ortopedkliniken på Länssjukhuset i Kalmar. Metodval: Studien är utformad som en utvärderingsstudie med en abduktiv utgångspunkt. Datainsamlingen har skett utifrån semistrukturerade intervjuer på LSK med personal som är involverad i projektet. Utöver de semistrukturerade intervjuerna har även numeriska data utgjort en del av studiens empiri. Slutsats: Efter genomförd studie kan vi konstatera att fler hälsoekonomiska utvärderingar behövs på icke-nationella nivåer för att stödja verksamheter inom hälso- och sjukvården. Det är viktigt att det aktivt arbetas med effektiviseringsarbeten inom offentliga verksamheter som främst finansieras av skattemedel. Vi kan även konstatera att det nya arbetssättet är mer lönsamt än det gamla både monetärt och icke-monetärt där förändringen bidragit med flera vinningar som exempelvis kortare ledtider, bättre samarbete och utökad kompetens. / Background: The cost increase in healthcare has been a major problem since the 1980s and will continue to be a problem in the future. This has led to a more strained economic situation for the healthcare sector. Therefore, it is of the utmost importance that the available funds in the healthcare sector are allocated to the areas where they are most beneficial.  Purpose: The purpose of this study is to make a health-economic evaluation of a new work procedure for the treatment of knee injuries at the orthopedic clinic at Kalmar’s County Hospital. Method: The study is designed as an evaluation study with an abductive outset. The data collection has been based on semi-structured interviews at LSK with the staff that was involved in the project. In addition to the semi-structured interviews, numerical data has also been part of the study for the empirical data collection. Conclusion: After completion of the study, we can state that more health-economic evaluations are needed at non-national levels to support healthcare organisations. It is important to actively work with efficiency initiatives in public organisations that are primarily funded by tax assets. We can also state that the new work procedure is more profitable than the old one, both monetary and non-monetary, where the change of work procedure has resulted in several non-monetary gains, such as shorter lead times, better cooperation and increased competence.

Rökning är ett utbrett samhällsproblem världen över och sjukdomar relaterade till rökning leder till mer än åtta miljoner dödsfall årligen. Effektiva rökavvänjningsmetoder spelar därför en viktig roll i samhället då de besparar samhället sjukvårdskostnader och främjar folkhälsan. I detta arbete genomförs en hälsoekonomisk utvärdering av ett SMS-baserat stöd för rökavvänjning. Syftet med arbetet är att fylla en kunskapslucka vad det gäller kostnadseffektivitet av digitala rökavvänjningsinsatser och samtidigt ge ett underlag tillbeslutsfattare som ansvarar för att prioritera folkhälsofrämjande insatser. Den hälsoekonomiska utvärderingen använder sig av kostnadseffektivitetsanalys som metod och baseras på en klinisk studie, där långsiktiga kostnader och hälsoeffekter skattas med hjälpav en beslutsanalytisk modell primärt baserad på en Markovmodell. De tre vanligaste följdsjukdomarna av rökning; KOL (kronisk obstruktiv lungsjukdom), lungcancer och hjärt- och kärlsjukdom modelleras mot bakgrund av hur många som slutar röka med hjälp av SMS-stödet. Data från olika källor används i modellen och i de fall data saknas görs antaganden baserade på tidigare studier för att kunna skatta kostnader och hälsoeffekter. Arbetet påvisar att en implementering av ett SMS-baserat stöd skulle generera ökade hälsoeffekter till lägre kostnader i jämförelse med att inte implementera ett sådant stöd. Resultatet från grundscenariot visar att interventionen skulle leda till kostnadsbesparingar på 63 539 kr per individ som tar del av interventionen och 0,30 vunna år i perfekt hälsa, sett över ett livstidsperspektiv. Resultaten bör tolkas med viss försiktighet då de är förknippade med osäkerheter, men kan användas som en del av det underlag beslutsfattare behöver för att fattabeslut om hur hälso- och sjukvårdens resurser ska prioriteras. Trots osäkerheterna i underlagetförefaller ett SMS-baserat stöd för rökavvänjning ha positiva hälsoeffekter och samtidigt spara resurser och därmed framstår metoden som en potentiellt viktig insats i framtida folkhälsoarbete. / Smoking is a globally widespread societal problem and diseases related to smoking account for more than eight million deaths annually. Effective smoking cessation methods therefore play an important role in society as they save society´s healthcare resources and promote public health. In this work, a health economic evaluation of an SMS-based smoking cessation is performed. The purpose of the work is to fill a knowledge gap in terms of cost-effectiveness of digital smoking cessation initiatives and at the same time provide a basis for decision-makers who are responsible for prioritizing public health promotion initiatives. The health economic evaluation uses cost-effectiveness analysis as a method and is based on a clinical study, where long-term costs and health effects are estimated using a decision-analytical model with a Markov structure. The three most common sequelae of smoking; COPD (chronic obstructive pulmonary disease), lung cancer and cardiovascular disease are modeled based on how many people stop smoking with the help of the SMS support. Data from different sources are used in the model and required assumptions based on previous studies are used to be able to estimate costs and health effects. The work shows that the implementation of an SMS-based support would generate increased health effects at lower costs in comparison with not implementing such support. The results from the basic scenario show that the intervention would lead to cost savings of SEK 63,539 per individual who takes part in the intervention and a gain in 0.30 years in full health, seen over a lifetime perspective. The results should be interpreted with some caution as they are associated with uncertainties but can be used as part of the basis on which decision-makers need to make decisions about how health care resources should be prioritized. Despite these uncertainties the SMS-based support for smoking cessation appears to save resources and lead to positive health effects and the method should be considered a potentially important addition in future public health policy.

Ventilator-associated pneumonia (VAP) is the most common healthcare-associated infection in the intensive care unit, resulting in a high burden of illness, mortality and increased cost. The literature around the cost-effectiveness of probiotics in prevention of health-care associated infections has not been previously well-described, and a definitive health economic evaluation alongside a well-designed randomized control trial assessing probiotic prophylaxis has not been previously performed. This thesis consists of 3 separate manuscripts (with 2 published in peer-reviewed journals and 1 pending). The theme of this thesis was to: (1) describe the literature about the cost-effectiveness of probiotics in hospitalized patients in preventing healthcare-associated infections; (2) design a protocol for an economic evaluation alongside a randomized control trial (RCT) examining probiotic prophylaxis of VAP; and then (3) perform and analyze the health economic evaluation presented in the protocol. The first component of this thesis is a systematic review of probiotic prophylaxis of healthcare-associated infections in hospitalized patients. We performed an extensive search including multiple databases which found 7 studies. Probiotics demonstrated favourable cost-effectiveness in 6 of 7 (86%) economic evaluations, with 3 studies being manufacturer-supported, all suggesting cost-effectiveness. Certainty of cost-effectiveness evidence was very low due to risk of bias, imprecision and inconsistency using the GRADE approach. Hence further RCTs with economic evaluations were stated as a solution. The second component of this thesis is a study protocol for an economic evaluation alongside the Probiotics to Prevent Severe Pneumonia and Endotracheal Colonization Trial (PROSPECT), which assessed the efficacy of probiotic prophylaxis in the prevention of healthcare-associated infections (specifically VAP). The third component of this thesis is the cost-effectiveness analysis performed utilizing the individual patient data from PROSPECT to produce the economic evaluation (E-PROSPECT). As of the date of thesis submission, PROSPECT is still pending publication, and hence E-PROSPECT is also pending analysis and publication. However, I have prepared a draft manuscript (along with figures and tables) that will be produced at the conclusion of E-PROSPECT for thesis committee review. / Thesis / Master of Health Sciences (MSc) / Ventilator-associated pneumonia (VAP) is the most common healthcare-associated infection in the intensive care unit, resulting in a high burden of illness, mortality and increased cost. The literature around the cost-effectiveness of probiotics in prevention of health-care associated infections has not been previously well-described, and a definitive health economic evaluation alongside a well-designed randomized control trial assessing probiotic prophylaxis has not been previously performed. This thesis consists of 3 separate manuscripts (with 2 published in peer-reviewed journals and 1 pending). The theme of this thesis was to: (1) describe the literature about the cost-effectiveness of probiotics in hospitalized patients in preventing healthcare-associated infections; (2) design a protocol for an economic evaluation alongside a randomized control trial (RCT) examining probiotic prophylaxis of VAP; and then (3) perform and analyze the health economic evaluation presented in the protocol. The first component of this thesis is a systematic review of probiotic prophylaxis of healthcare-associated infections in hospitalized patients. We performed an extensive search including multiple databases which found 7 studies. Probiotics demonstrated favourable cost-effectiveness in 6 of 7 (86%) economic evaluations, with 3 studies being manufacturer-supported, all suggesting cost-effectiveness. Certainty of cost-effectiveness evidence was very low due to risk of bias, imprecision and inconsistency using the GRADE approach. Hence further RCTs with economic evaluations were stated as a solution. The second component of this thesis is a study protocol for an economic evaluation alongside the Probiotics to Prevent Severe Pneumonia and Endotracheal Colonization Trial (PROSPECT), which assessed the efficacy of probiotic prophylaxis in the prevention of healthcare-associated infections (specifically VAP). The third component of this thesis is the cost-effectiveness analysis performed utilizing the individual patient data from PROSPECT to produce the economic evaluation (E-PROSPECT). As of the date of thesis submission, PROSPECT is still pending publication, and hence E-PROSPECT is also pending analysis and publication. However, I have prepared a draft manuscript (along with figures and tables) that will be produced at the conclusion of E-PROSPECT for thesis committee review.

Introduction: Quality improvement (QI) encompasses a wide range of healthcare studies and activities with the common goal of improving patient outcomes, healthcare system performance, and professional development. QI is characterized by a diversity of definitions, stakeholders, clinical fields and study designs, which creates challenges for rigorous reporting and evaluation of these studies. Understanding and addressing the methodological issues that arise from conducting QI studies from multiple clinical disciplines is critical for generating good evidence for healthcare improvement to tackle health system challenges. Objectives: This thesis addressed three independent objectives: (i) Determine the quality of reporting of QI studies in neonatology. (ii) Compare different statistical methods that can be used to analyze data from a cluster randomized controlled trial with repeated measures data and examine how the estimate of intervention effects varies between these approaches. (iii) Determine the cost-effectiveness of providing timely surgery or timely rehabilitation for patients with hip fracture. Methods: Objective 1: We conducted a systematic survey of quality improvement studies in neonatology to examine the extent to which these publications adhered to SQUIRE 2.0, the guidelines for reporting studies that sought to improve the quality, safety, and value of healthcare. Using the same set of articles, we examined how various methodological attributes, such as stakeholder engagement, outcome measures, and statistical process controls, are reported in these studies. Objective 2: To compare the differences in the statistical estimates of intervention effects between linear mixed models and Generalized Estimating Equations, for the CP@Clinic Program cluster randomized RCT, which contains routinely collected monthly outcome data aggregated at the cluster level. Objective 3: We constructed a Markov cohort model to estimate the cost-effectiveness of receiving timely surgery within 24 hours of admission to the emergency department, receiving immediate admission to inpatient rehabilitation following acute care discharge, receiving both, or none. Results: Objective 1: In our assessment of reporting quality, we found that adherence to SQUIRE 2.0 guidelines was inadequate and that journals should endorse the SQUIRE 2.0 guideline for improvement publications to alleviate this issue. We found that process measures was the most frequently reported methodological attribute (89%), while stakeholder engagement with leadership (32%) or caregivers (10%) were infrequently reported or conducted. Objective 2: In comparing statistical methods for analyzing a cluster randomized controlled trial with correlated data, we found that it was critical to apply a correction to the variance estimator of generalized estimating equations to produce robust estimates of the intervention effects. Objective 3: In our economic evaluation, both timely surgery alone and the combination of timely surgery and timely rehabilitation yielded cost-effective improvements in the quality-adjusted life-years of patients with hip fracture. However, the combination of receiving timely surgery and timely rehabilitation requires a high willingness-to-pay threshold, above $128,000 per quality-adjusted life-years, to be considered cost-effective. Conclusions: Overall, understanding the state of reporting and the broad spectrum of methods and methodologic issues for evaluating quality improvement initiatives will advance its rigorous research, evaluation, reporting, and contribution towards informed decision-making for tackling pressing healthcare issues. / Thesis / Doctor of Philosophy (PhD) / Quality improvement (QI) is a field of healthcare research that can be defined in many ways, and research in this field is conducted by researchers from various medical disciplines. Consequently, challenges may arise in reporting and evaluating QI interventions. Thus, it is important to examine how QI interventions are reported in academic literature and the methods used to evaluate their effectiveness in improving health. The current thesis aims to address these issues through three independent objectives: (1) examine the details reported in QI studies in neonatology, (2) compare different statistical methods that can be used to analyze data from a community paramedicine cluster randomized controlled trial, and (3) investigate whether providing timely surgery and timely hospital-based rehabilitation is a cost-effective way to improve the quality of life of patients who have experienced hip fracture. The findings of these studies will provide insights into the challenges of reporting and evaluating QI interventions, and suggest ways to improve them.

Submitted by Fabiana Gonçalves Pinto (benf@ndc.uff.br) on 2015-12-09T19:00:22Z No. of bitstreams: 1 Simone Pereira Lermontov.pdf: 27639825 bytes, checksum: 8e739b8c382c9f2fc847de9ba00bbec9 (MD5) / Made available in DSpace on 2015-12-09T19:00:22Z (GMT). No. of bitstreams: 1 Simone Pereira Lermontov.pdf: 27639825 bytes, checksum: 8e739b8c382c9f2fc847de9ba00bbec9 (MD5) Previous issue date: 2010 / Mestrado Acadêmico em Ciências do Cuidado em Saúde / Este estudo objetivou avaliar os custos e consequências do tratamento da pseudartrose com aplicação de células-tronco. A metodologia correspondeu à avaliação econômica parcial do custo e das consequências desse tratamento. O cenário do estudo foi o Hospital Universitário Antonio Pedro e os sujeitos foram oito pacientes operados entre setembro de 2008 e maio de 2009. A perspectiva do estudo foi a do Sistema Único de Saúde, principal fonte de financiamento da assistência médica no Brasil. Para o tratamento estatístico dos dados foram utilizados o software STATISTICA 6.0 e o teste não paramétrico de Spearman. A análise estatística apresentou resultados satisfatórios dentro de uma margem aceitável. O custo total médio do tratamento foi de R$ 1243.199 e o tempo de consolidação foi em média 14.71429 semanas. Observamos através do teste não paramétrico de Spearman que a correlação entre o custo total e o tempo de consolidação é - 0, 5078, uma indicação fraca de que o custo total é inversamente proporcional ao tempo de consolidação. A partir deste estudo, podemos concluir que o custo com o tratamento não foram exorbitantes. O pequeno tamanho da amostra não afetou os resultados de forma significativa, sendo que os achados em relação à idade são parecidos com os de outros estudos, o que nos leva a concluir que a população acometida pela pseudartrose é a economicamente ativa. A criação de um protocolo foi primordial para o levantamento do custo com o tratamento, possibilitando a consolidação dos dados e nos permitindo visualizar de forma mais abrangente o tratamento. O tempo de consolidação foi igual aos melhores resultados vistos em estudo internacionais e o procedimento se mostrou seguro e eficaz. Constatamos que o questionário SF36 é um instrumento de fácil aplicação e foi de confiabilidade no acompanhamento ambulatorial de pacientes submetidos ao tratamento. / The purpose of this study was to evaluate the costs and consequences of the use of stem cells in the treatment of pseudarthrosis. The methodology involved a partial economic assessment of the cost and consequences of this treatment. The study scenario was the Hospital Universitário Antonio Pedro and the subjects were eight patients operated between September 2008 and May 2009. The study perspective was that of the Sistema Único de Saúde, the main financial resource for medical assistance in Brazil. Statistical treatment of the data was performed using the STATISTICA 6.0 application and the Spearman nonparametric test. The results were statistically significant within an acceptable margin of error. The average treatment cost was R$ 1.247,21 and the average time for consolidation was 12.875 weeks. The Spearman nonparametric test showed that the correlation between the total cost and the time for was -0.25, a weak indication that the total cost is inversely proportional to the time for consolidation. From this study, we can conclude that the treatment costs were not prohibitive. The small size of the sample had no significant influence on the results; the agerelated findings are similar to those of other studies and lead to the conclusion that population affected by pseudarthrosis is economically active. The creation of a protocol was essential for cost evaluation, providing a framework for data consolidation and yielding a more general view of the treatment. The time for consolidation was about the same as for the best results reported in the international literature and the procedure was observed to be safe and effective. We concluded that the SF36 questionnaire is an easily applied tool that can be reliably used in the post-treatment follow-up of the patients.