Background: Variation exists in quality of life (i.e., utility) estimates depending on the utility elicitation method utilized. Using the EQ-5D, VAS, and HUI-III, the aim of this thesis was to determine whether these measures adequately capture HRQOL in a CRC population and assess the relationship between utility estimates generated. Methods: A mixed methods study design was employed to collect health status scores and interview data from a sample of 50 CRC patients in Toronto, Ontario. Results: Mean utility scores between the EQ-5D and HUI-III were identical at 0.76 (95% CI), with an overall VAS score of 0.72 (95% CI). Conclusion: The fact that the EQ-5D and HUI-III resulted in identical mean utility scores provides assurance for future studies using these tools in CRC. However, many factors that CRC patients identify as important to their HRQOL are not captured by these instruments. These findings have implications for informing economic evaluations.

Background: Variation exists in quality of life (i.e., utility) estimates depending on the utility elicitation method utilized. Using the EQ-5D, VAS, and HUI-III, the aim of this thesis was to determine whether these measures adequately capture HRQOL in a CRC population and assess the relationship between utility estimates generated. Methods: A mixed methods study design was employed to collect health status scores and interview data from a sample of 50 CRC patients in Toronto, Ontario. Results: Mean utility scores between the EQ-5D and HUI-III were identical at 0.76 (95% CI), with an overall VAS score of 0.72 (95% CI). Conclusion: The fact that the EQ-5D and HUI-III resulted in identical mean utility scores provides assurance for future studies using these tools in CRC. However, many factors that CRC patients identify as important to their HRQOL are not captured by these instruments. These findings have implications for informing economic evaluations.

This thesis aims to fill the gaps in the literature of the theory of constraints (TOC) in publicly funded health systems. While TOC seems to be a natural fit for this resource-constrained environment, there are still no reported application of TOC’s drum-buffer-rope tool and inadequate customizations with regards to defining system-wide goal and performance measures. The “Drum-Buffer-Rope for an Outpatient Cancer Facility” chapter is a real world case study exploring the usefulness of TOC’s drum-buffer-rope scheduling technique in a publicly funded outpatient cancer facility. With the use of a discrete event simulation model populated with historical data, the drum-buffer-rope scheduling policy is compared against “high constraint utilization” and “low wait time” scenarios. Drum-buffer-rope proved to be an effective mechanism in balancing the inherent tradeoff between the two performance measures of instances of delayed treatment and average patient wait time. To find the appropriate level of compromise in one performance measure in favor of the other, the linkage of these measures to system-wide performance measures are proposed. In the “Theory of Constraints’ Performance Measures for Publicly Funded Health Systems” chapter, a system dynamics representation of the classical TOC’s system-wide goal and performance measures for publicly traded for-profit companies is developed, which forms the basis for developing a similar model for publicly funded health systems. The model is then expanded to include some of the factors that affect system performance, providing a framework to apply TOC’s process of ongoing improvement in publicly funded health systems. The “Connecting Low-Level Performance Measures to the Goal” chapter attempts to provide a framework to link the low-level performance measures with system-wide performance measures. It is claimed that until such a linkage is adequately established, TOC has not been fully transferred to publicly funded health systems.

This thesis aims to fill the gaps in the literature of the theory of constraints (TOC) in publicly funded health systems. While TOC seems to be a natural fit for this resource-constrained environment, there are still no reported application of TOC’s drum-buffer-rope tool and inadequate customizations with regards to defining system-wide goal and performance measures. The “Drum-Buffer-Rope for an Outpatient Cancer Facility” chapter is a real world case study exploring the usefulness of TOC’s drum-buffer-rope scheduling technique in a publicly funded outpatient cancer facility. With the use of a discrete event simulation model populated with historical data, the drum-buffer-rope scheduling policy is compared against “high constraint utilization” and “low wait time” scenarios. Drum-buffer-rope proved to be an effective mechanism in balancing the inherent tradeoff between the two performance measures of instances of delayed treatment and average patient wait time. To find the appropriate level of compromise in one performance measure in favor of the other, the linkage of these measures to system-wide performance measures are proposed. In the “Theory of Constraints’ Performance Measures for Publicly Funded Health Systems” chapter, a system dynamics representation of the classical TOC’s system-wide goal and performance measures for publicly traded for-profit companies is developed, which forms the basis for developing a similar model for publicly funded health systems. The model is then expanded to include some of the factors that affect system performance, providing a framework to apply TOC’s process of ongoing improvement in publicly funded health systems. The “Connecting Low-Level Performance Measures to the Goal” chapter attempts to provide a framework to link the low-level performance measures with system-wide performance measures. It is claimed that until such a linkage is adequately established, TOC has not been fully transferred to publicly funded health systems.

Contexte : L'absence d'un ensemble de valeurs permettant le calcul d’années de vie ajustées par la qualité (AVAQ) est une limite importante lorsqu'il s'agit d'établir la valeur des traitements émergents pour traiter l'allergie alimentaire. Pour être valables, les AVAQ doivent être calculées à partir de questionnaires adaptés à la population cible en raison des déficits et des préférences variables selon les populations et les maladies, ce qui n'était pas le cas dans les études précédentes en allergie alimentaire. L'objectif de cette étude était d'établir un ensemble de valeurs de préférence pour le questionnaire générique SF-6Dv2 pour le calcul de l'utilité dans la population canadienne atteinte d'allergies alimentaires. Méthodes : Deux cent quatre-vingt-quinze parents de patients âgés de 0 à 17 ans et 154 patients âgés de 12 ans et plus atteints d'allergie(s) alimentaire(s) ont été recrutés en clinique et en ligne. Les participants ont été sollicités à remplir un questionnaire en ligne autoadministré comprenant des questionnaires génériques de qualité de vie reliée à la santé. Différents états de santé décrits par le SF-6Dv2 ont été évalués à l'aide d'exercices d’arbitrage temporel et de choix expérimentaux discrets. Les données issues de ces techniques d'élicitation ont été combinées à l'aide du modèle de régression hybride. Résultats : Deux cent quarante et un parents et 125 patients ont réalisé 3904 exercices d’arbitrage temporel et 5112 choix expérimentaux discrets. La désutilité a été estimée pour chaque niveau de chaque dimension du SF-6Dv2. Les valeurs d'utilité calculées à partir de l’ensemble de valeurs de préférences établi étaient en moyenne inférieures de 0,15 (IC à 95 %: 0,12-0,18) et corrélaient faiblement (R2=0,46) avec celles dérivées du questionnaire générique EQ-5D-5L dans la même cohorte. Conclusion : Un ensemble de valeurs de préférence représentatif des patients et familles atteints d'allergies alimentaires a été déterminé à partir du questionnaire générique SF-6Dv2. Cet ensemble de valeurs de préférence adapté contribuera à améliorer la validité des futures estimations d'utilité dans cette population pour l'évaluation des traitements émergents ayant potentiellement un impact sur la qualité de vie reliée à la santé, mais étant parfois coûteux. / Background: The lack of a value set allowing the calculation of quality-adjusted life-years (QALY) is an important limitation when establishing the value of emerging therapies to treat food allergy. To be valid, QALYs must be calculated from questionnaires adapted to the target population because of varying deficits and preferences across populations and diseases, which was not the case in previous studies. This study aimed to develop a Short-Form Six-Dimension version 2 (SF-6Dv2) preference value set for the calculation of health utilities from the Canadian food allergic population. Methods: Two hundred ninety-five parents of patients aged 0-17 years old and 154 patients aged 12 years old and above with food allergy were recruited in the clinic and online. Participants were asked to complete a self-administered online questionnaire including generic health-related quality of life questionnaires. Various health states described by the SF-6Dv2 were valued with time-trade-off and discrete choice experiments. Data from elicitation techniques were combined using the hybrid regression model. Results: A total of 241 parents and 125 patients performed 3904 time trade-off and 5112 discrete choice experiments. Utility decrements were estimated for each level of each SF-6Dv2 dimension. Utility values calculated based on the validated preference set were on average 0.15 lower (95% CI: 0.12-0.18) and were poorly correlated (R2=0.46) with those derived from the EQ-5D-5L generic questionnaire in the same cohort. Conclusion: A representative preference value set for patients with food allergy was determined using the SF-6Dv2 generic questionnaire. This adapted preference value set will contribute to improve the validity of future utility estimates in this population for the appraisal of upcoming potentially impactful but sometimes costly therapies.

Parkinson's is a chronic, progressive, neurodegenerative disease. The most common treatment is levodopa/carbidopa, which suppresses the symptoms of the disease. In this report, a cost-utility analysis of the MyFID levodopa/carbidopa micro tablet dispenser has been conducted. The method used was a continuous-time Markov chain with states based on the score in MDS-UPDRS II and MDS-UPDRS III. The model was simulated over a time horizon of five years and was started at different disease severity levels. The results obtained from the simulations were a dominant ICER for all model versions except when the simulation was started in the earliest stages of the disease, where it was moderate to high. The conclusion was that the treatment method with the micro / Parkinsons är en kronisk, progressiv, neurodegenerativ sjukdom. Den vanligaste behandlingen är levodopa/carbidopa, som undertrycker symptomen av sjukdomen. I denna rapport har en kostnadsnyttoanalys av MyFID levodopa/ carbidopa-mikrotablettdispensern genomförts. Metoden som användes var en kontinuerlig Markovkedja med tillstånd baserade på poängen i MDS-UPDRS II och MDS-UPDRS III. Modellen simulerades över en tidsperiod av fem år och startades vid olika svårighetsgrader av sjukdomen. Resultaten som erhölls från simuleringarna visade en dominerande ICER för alla modellversioner förutom när simuleringen startades i de tidigaste stadierna av sjukdomen, där den visade en måttligt till hög kostnad. Slutsatsen var att behandlingen med hjälp av mikrotablettdispensern var kostnadseffektiv i de måttliga till senare stadierna av sjukdomen.

Contexte : La quantification des subjectivités associées aux états de santé peut fournir des informations sur la qualité de vie reliée à la santé (QVRS). Pour rendre compte de ces subjectivités dans la mesure de la QVRS, on utilise des instruments d'utilité multi attribut comme le Short-Form 6-Dimension Version 2 (SF-6Dv2). Ces instruments permettent de décrire les états de santé, de dériver les utilités correspondantes et d’estimer le changement minimal important (CMI) qui est spécifique à différents groupes de populations, afin de tirer des inférences sur l'état de santé de la population. Le CMI est ici un point central correspondant à la variation minimale considérée comme importante dans la QVRS par les répondants. Le but de cette étude est de déceler le seuil de CMI du SF-6Dv2 pour un échantillon de la communauté québécoise. Méthodes : Une enquête en ligne auprès de la population générale adulte du Québec a été menée entre mars 2020 et septembre 2021 pendant six cycles ou intervalles de temps. Le questionnaire SF-6Dv2 a été administré à chaque cycle, ainsi qu’une question sur la perception d’un changement dans l’état de santé (‘Global Rating of Change’) à partir du cycle 2. Les scores d’utilité en santé ont été calculés pour chaque participant à l’aide de deux ensembles de valeurs de référence (‘value set’), soit pour le Royaume-Uni et le Québec. Actuellement il n'existe pas de value set canadien pour le SF-6Dv2 et le value set québécois n'était disponible qu'en 2023. Une analyse des données avec des méthodes basées sur la distribution et l’ancrage a été effectuée pour déterminer les valeurs de CMI. Résultats : En considérant que les participants avec les réponses 2 ou 4 à la question du ‘Global Rating of Change’ ont connu un changement minimal dans leur état de santé, le CMI calculé avec la pondération du Royaume-Uni était de 0,030 (IC 95% [0,023 ; 0,038], valeur p < 0,001) et pour le Québec de 0,029 (IC 95% [0,023 ; 0,036], valeur p < 0,001) pour le SF-6Dv2. Les méthodes basées sur la distribution ont permis d'obtenir une réponse moyenne standardisée (SRM) de 0,134 et 0,152 avec les ensembles de valeurs du Royaume-Uni et du Québec, respectivement. La taille de l'effet et le demi-écart-type ont également été calculés (0,106 et 0,143 avec Royaume-Uni) (0,115 et 0,127 avec Québec). Conclusion : Le CMI calculé valide la bonne sensibilité (réactivité) du SF-6Dv2 dans la population générale du Québec et fournit une estimation du seuil minimalement important pour les participants. Le CMI permet d’interpréter la QVRS calculée et l’utiliser pour comprendre si la santé s'est significativement améliorée ou détériorée du point de vue des répondants. Les études d’évaluation des technologies de la santé et de prise de décision deviennent ainsi plus valables, car elles tiennent compte de la subjectivité individuelle ressentie et associée à un état de santé particulier, en plus des résultats cliniques objectifs. / Background: Quantifying subjectivities associated with health states can offer information on the health-related quality of life (HRQoL). To account for such subjectivities on HRQoL we use multi attribute utility instruments such as the Short-Form 6-Dimension version 2 (SF-6Dv2). They allow health state description, derive corresponding health utilities, and provide an estimate of minimal important change (MIC) which is specific to different population groups, for inferences on population health. MIC is a central point corresponding to the minimal change in HRQoL that is considered important by the participant. The aim of this study was to find the minimal important change of a generic preference-based instrument, the SF-6Dv2 for a Quebec community sample. Methods: An online survey of the adult general population in Quebec was conducted between March 2020 and September 2021 over six time intervals. SF-6Dv2 was administered at each cycle and a global rating of change question was also administered in the follow-up cycles starting from cycle 2. Health utilities were calculated for each participant using a United Kingdom (UK) based value set (preference weights) and a Quebec value set. At present a Canadian value set is not available for SF-6Dv2 and the Quebec value set was developed in 2023. Data analysis was performed with both anchor and distribution-based methods to ascertain the MIC values. Results: Considering the participants who chose 2 or 4 in the Global Rating of Change question as having experienced a minimal change in health state, the MIC calculated with UK value set was 0.03 (95% CI [0.023 ; 0.038], p-value < 0.001) and with Quebec value set 0.0293 (95% CI [0,023 ; 0,036], p-value < 0,001) for SF-6Dv2. Distribution-based methods yielded a standardized response mean (SRM) of 0.134 and 0.152 with the UK and Quebec value sets, respectively. Effect size and half a standard deviation were 0.106, 0.143 for UK and 0.115, 0.127 for Quebec, respectively. Conclusion: The MIC calculated hence validates SF-6Dv2’s responsiveness in the general population and estimates a utility score minimally important to the participants. This MIC enables HRQoL calculated from such instruments to be appropriately interpreted and to understand if health has improved or deteriorated from the participant’s perspective. Further rendering health technology evaluation and decision-making studies more valid by accounting for the subjectivity felt and associated with a particular health state, apart from the objective clinical results.