Background and Objectives: Economic evaluation and decision analysis provide a framework to evaluate incremental costs and effects associated with alternative health interventions. These methods can also be used as a tool to evaluate alternative clinical behaviours or practice patterns. The objective of this thesis was to investigate the impact of current Canadian practices in human epidermal growth factor receptor-2 (HER2) testing to target trastuzumab in early-stage breast cancer (BC). Methods: Project 1: A systematic review of previous trastuzumab and HER2 testing economic analyses was conducted to identify methodological gaps and key lessons. Project 2: A population-level, retrospective cohort was studied to determine HER2 testing and trastuzumab treatment patterns in Ontario early-stage BC patients. Project 3: A cost-utility analysis of alternative test-treat strategies was conducted using a Markov model of BC calibrated to the Canadian setting, and incorporating Project 2 findings. Results: Project 1: Previous economic evaluations demonstrated that HER2 test accuracy and sequencing were key considerations when modelling the cost-effectiveness of trastuzumab treatment. Consideration of local testing and treatment practices was lacking. Project 2: HER2 testing and treatment practice differed from guidelines, where documentation was available. Only 88% of equivocal results were confirmed, while 57% of HER2 positive patients received trastuzumab. Project 3: Calibration of the BC model minimised gaps between trial-based survival and expected Canadian survival patterns. Deviations from guidelines in practice suggest that primary testing with fluorescence in situ hybridization (FISH) would produce greater health gains at a reduced cost vs. primary immunohistochemistry with FISH confirmation. This finding was more apparent as the prevalence of HER2 positive disease increased. Introduction of newer in situ hybridisation tests may be cost-effective as well. Conclusions: Practice deviations from guidelines are an important consideration when modelling the cost-effectiveness of trastuzumab therapy. Underlying local disease progression and prevalence can also significantly impact outcomes. / Dissertation / Doctor of Philosophy (PhD)

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Cost-utility analysis is one method of determining the cost effectiveness of nursing interventions. It is heralded by the World Health Organization as the measure to determine allocation of resources. This method of measurement includes calculation of both the cost of quality-adjusted life years (QALY) and the cost of disability-adjusted life years (DALY). The purpose of this article is to present cost-utility analysis as a relevant measure for describing the value of registered nurses. First the article will present a short overview of cost effectiveness, along with a discussion of two cost-effectiveness measures, cost-effective analysis and cost-utility analysis. Then the measurement of quality-adjusted life years and disability-adjusted life years will be presented. The article will conclude by challenging nurses to develop cost-utility analyses into a meaningful and useful methodology that can provide nursing with a process to measure the economic outcomes of our nursing interventions.

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

<p>The goals of this thesis are to evaluate quality of life (QoL) in adult patients with growth hormone deficiency (GHD) in relation to population normative data, to construct a preference-weighted index (utility) from a disease-specific QoL measure and to assess it in a clinical context.</p><p>The study included samples from the general population and patients with GHD from four European populations: England & Wales, the Netherlands, Spain and Sweden. The country-specific patient cohorts were retrieved from KIMS (Pfizer International Metabolic Database). </p><p>A questionnaire was developed that contained items from existing QoL questionnaires including, among others, Quality of Life Assessment in Growth Hormone Deficiency in Adults (QoL-AGHDA) and the EQ-5D. The QoL-AGHDA is a disease-specific measure for use in adults with GHD. The EQ-5D is a generic instrument which describes health states for which country-specific preference-based weights are available. Thus, it was possible to generate preference-weighted indices (utilities) based on data generated by both instruments. </p><p>This thesis reports QoL-AGHDA normative values for the populations of England & Wales, the Netherlands, Spain and Sweden, and confirms the extent of QoL impairment in patients with GHD in comparison with the general population. Long-term GH replacement resulted in sustained improvements in overall QoL towards normative country-specific values, as well in most of the dimensions that were impaired before treatment. </p><p>For use in health economic evaluations, models for generating utilities (QoL-AGHDA_utility) from QoL-AGHDA were developed. It is believed that these models may facilitate medical decision making, given that they provide a tool for obtaining utilities in the absence of directly collected preference-weighted indices.</p><p>QoL-AGHDA_utility effectively monitored treatment effects in patients with GHD. Moreover, this study confirmed a QoL-AGHDA_utility deficit before treatment and a gain after starting GH replacement. </p><p>The novel aspect of the present approach was to apply preference-weighted indices derived from a disease-specific measure to assess QoL in the clinical context, together with patient demographic and clinical characteristics. The robustness of this analysis is reinforced by the fact that utilities in both general and patient populations were generated using the same methodology. </p>

The goals of this thesis are to evaluate quality of life (QoL) in adult patients with growth hormone deficiency (GHD) in relation to population normative data, to construct a preference-weighted index (utility) from a disease-specific QoL measure and to assess it in a clinical context. The study included samples from the general population and patients with GHD from four European populations: England &amp; Wales, the Netherlands, Spain and Sweden. The country-specific patient cohorts were retrieved from KIMS (Pfizer International Metabolic Database). A questionnaire was developed that contained items from existing QoL questionnaires including, among others, Quality of Life Assessment in Growth Hormone Deficiency in Adults (QoL-AGHDA) and the EQ-5D. The QoL-AGHDA is a disease-specific measure for use in adults with GHD. The EQ-5D is a generic instrument which describes health states for which country-specific preference-based weights are available. Thus, it was possible to generate preference-weighted indices (utilities) based on data generated by both instruments. This thesis reports QoL-AGHDA normative values for the populations of England &amp; Wales, the Netherlands, Spain and Sweden, and confirms the extent of QoL impairment in patients with GHD in comparison with the general population. Long-term GH replacement resulted in sustained improvements in overall QoL towards normative country-specific values, as well in most of the dimensions that were impaired before treatment. For use in health economic evaluations, models for generating utilities (QoL-AGHDAutility) from QoL-AGHDA were developed. It is believed that these models may facilitate medical decision making, given that they provide a tool for obtaining utilities in the absence of directly collected preference-weighted indices. QoL-AGHDAutility effectively monitored treatment effects in patients with GHD. Moreover, this study confirmed a QoL-AGHDAutility deficit before treatment and a gain after starting GH replacement. The novel aspect of the present approach was to apply preference-weighted indices derived from a disease-specific measure to assess QoL in the clinical context, together with patient demographic and clinical characteristics. The robustness of this analysis is reinforced by the fact that utilities in both general and patient populations were generated using the same methodology.

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

Background Stress urinary incontinence (SUI) is the leakage of urine when coughing, sneezing, or on exertion. It affects 10-35% of women, and can impair quality of life (QOL). First-line treatment is pelvic floor muscle training (PFMT). However, access barriers and embarrassment may prevent women from seeking care. There is a need for new, easily accessible ways to provide treatment. Aim To evaluate the treatment outcome, patient satisfaction, and cost-effectiveness of an Internet- based treatment programme for SUI. Methods We recruited 250 community-dwelling women aged 18-70 years, with SUI ≥1/week via our website. Participants were randomised to 3 months of PFMT with either an Internet-based programme (n=124), or a programme sent by post (n=126). We had no-face-to face contact with the participants, but the Internet group received individually tailored e-mail support from an urotherapist. Treatment outcome was evaluated after 4 months with intention-to-treat analysis. After treatment, we telephoned a strategic selection of participants (Internet n=13, postal n=8) to interview them about their experiences, and analysed the results according to grounded theory principles. We also performed a cost-utility analysis with a 1-year societal perspective, comparing the treatment programmes with each other and with a no-treatment alternative. To scrutinize our measure of QOL, we performed a reliability study of the ICIQ-LUTSqol questionnaire. Results Participants in both intervention groups achieved highly significant improvements (p&lt;0.001) with large effect sizes (&gt;0.8) in the primary outcomes symptom score (ICIQ-UI SF: mean change Internet 3.4 [SD 3.4], postal 2.9 [3.1]), and condition-specific QOL (ICIQ-LUTSqol: mean change Internet 4.8 [SD 6.1], postal 4.6 [SD 6.7]); however, the differences between the groups were not significant. Compared with the postal group, more participants in the Internet group perceived they were much or very much improved after treatment (40.9%, vs. 26.5%, p=0.01), reduced their use of incontinence aids (59.5% vs. 41.4%, p=0.02), and indicated satisfaction with the treatment programme (84.8% vs. 62.9%, p&lt;0.001). Results from the interviews fell into three categories: about life with SUI and barriers to seeking care; about the treatments and the patient-provider relationship; about the sense of empowerment many women experienced. A core category emerged: “Acknowledged but not exposed.” The extra cost per quality-adjusted life year (QALY) gained through use of the Internet-based programme compared with the postal programme was €200. The extra cost per QALY for the Internet-based programme compared with no treatment was €30,935. The condition-specific questionnaire ICIQ-LUTSqol is reliable in women with SUI, with high degrees of agreement between overall scores (Intraclass correlation coefficient 0.95, p&lt;0.001). Conclusion Internet-based treatment for SUI is a new, effective, and patient-appreciated treatment alternative, which can increase access to care in a sustainable way.

A tese abordou questões relacionadas à economia da saúde, particularmente à visão econômica dos transplantes renais. Foi conduzida uma análise de custo-utilidade para verificar qual tratamento, transplante renal ou hemodiálise, possui menor razão de custo por anos de vida ajustados por qualidade. O resultado obtido corrobora as evidências internacionais, as quais indicam o transplante renal como estratégia mais custo-efetiva. No caso deste estudo, a razão de custo-utilidade para o transplante renal e hemodiálise foi de R$ 18.161,00/AVAQ e R$ 40.872,00/AVAQ, respectivamente. Apesar de o transplante renal ser uma estratégia dominante, a escassez de órgãos impede que essa estratégia seja amplamente utilizada, reduzindo, assim, os ganhos de eficiência na alocação dos recursos escassos. Nesse contexto, também foi alvo desta tese a questão da escassez de órgãos. Pelo levantamento feito, há tendência de aumento do desequilíbrio entre demanda e oferta de órgãos, visto que a primeira cresce rapidamente, enquanto a segunda mostra pequena tendência de crescimento. Assim, alternativas para contornar esse problema foram analisadas, especialmente as relacionadas a mudanças institucionais na lei de doação de órgãos. Entre elas, foi argüido que a lei de consentimento presumido seria a opção mais factível, por não ferir o pressuposto do altruísmo. Objetivando estimar quanto seria o eventual incremento na doação de órgãos por doador cadáver devido à lei de consentimento presumido, fez-se uso do ferramental da econometria da saúde, aplicando, para uma amostra de 34 países ao longo de cinco anos, o método de regressão quantílica para dados de painel. Os resultados obtidos nessa aplicação indicam que há benefício na adoção da lei de consentimento presumido, que tem um efeito positivo sobre a taxa de doação de órgãos, em torno de 21-26%, comparada à lei de consentimento informado. / The thesis broaches questions related to health economics, particularly the economic vision of renal transplants. A cost-utility analysis was conducted to assess which treatment, renal transplant or hemodialysis, has a lower cost rate per quality-adjusted life years. The result obtained corroborates the international evidence, which indicates renal transplant as the most cost effective strategy. In the case of this study, the cost-utility ratio for renal transplant and hemodialysis was US$ 11,157/QALY and US$ 25,110/QALY, respectively. In spite of renal transplant being the dominant strategy, the scarcity of organs hinders this strategy to be widely used, reducing in this way, the efficiency gain in the allocation of scarce resources. Within this context, the organ shortage was also a target issue of this thesis. Through the survey performed, there is a tendency towards the increase of unbalance between the demand and supply of organs, being that the first grows rapidly while the second shows small tendency towards growth. Within this context, the investigation target of this thesis was to look into possible alternatives to by-pass this problem, especially those related to institutional changes in the organ donation law. Among them, it was argued that the law of presumed consent would be the most feasible option, since it does not harm the presupposition of altruism. With the object of estimating what would be the eventual increase in organ donation, per cadaveric donor, due to the law of presumed consent, the health econometric tool of quantile regression method for panel data was used, applied to a sample of 34 countries during a five-year period. The results obtained in this application indicate that there is benefit in adopting the law of presumed consent, which has a positive effect on the organ donation rate, around 21 – 26%, compared to the law of informed consent.

A tese abordou questões relacionadas à economia da saúde, particularmente à visão econômica dos transplantes renais. Foi conduzida uma análise de custo-utilidade para verificar qual tratamento, transplante renal ou hemodiálise, possui menor razão de custo por anos de vida ajustados por qualidade. O resultado obtido corrobora as evidências internacionais, as quais indicam o transplante renal como estratégia mais custo-efetiva. No caso deste estudo, a razão de custo-utilidade para o transplante renal e hemodiálise foi de R$ 18.161,00/AVAQ e R$ 40.872,00/AVAQ, respectivamente. Apesar de o transplante renal ser uma estratégia dominante, a escassez de órgãos impede que essa estratégia seja amplamente utilizada, reduzindo, assim, os ganhos de eficiência na alocação dos recursos escassos. Nesse contexto, também foi alvo desta tese a questão da escassez de órgãos. Pelo levantamento feito, há tendência de aumento do desequilíbrio entre demanda e oferta de órgãos, visto que a primeira cresce rapidamente, enquanto a segunda mostra pequena tendência de crescimento. Assim, alternativas para contornar esse problema foram analisadas, especialmente as relacionadas a mudanças institucionais na lei de doação de órgãos. Entre elas, foi argüido que a lei de consentimento presumido seria a opção mais factível, por não ferir o pressuposto do altruísmo. Objetivando estimar quanto seria o eventual incremento na doação de órgãos por doador cadáver devido à lei de consentimento presumido, fez-se uso do ferramental da econometria da saúde, aplicando, para uma amostra de 34 países ao longo de cinco anos, o método de regressão quantílica para dados de painel. Os resultados obtidos nessa aplicação indicam que há benefício na adoção da lei de consentimento presumido, que tem um efeito positivo sobre a taxa de doação de órgãos, em torno de 21-26%, comparada à lei de consentimento informado. / The thesis broaches questions related to health economics, particularly the economic vision of renal transplants. A cost-utility analysis was conducted to assess which treatment, renal transplant or hemodialysis, has a lower cost rate per quality-adjusted life years. The result obtained corroborates the international evidence, which indicates renal transplant as the most cost effective strategy. In the case of this study, the cost-utility ratio for renal transplant and hemodialysis was US$ 11,157/QALY and US$ 25,110/QALY, respectively. In spite of renal transplant being the dominant strategy, the scarcity of organs hinders this strategy to be widely used, reducing in this way, the efficiency gain in the allocation of scarce resources. Within this context, the organ shortage was also a target issue of this thesis. Through the survey performed, there is a tendency towards the increase of unbalance between the demand and supply of organs, being that the first grows rapidly while the second shows small tendency towards growth. Within this context, the investigation target of this thesis was to look into possible alternatives to by-pass this problem, especially those related to institutional changes in the organ donation law. Among them, it was argued that the law of presumed consent would be the most feasible option, since it does not harm the presupposition of altruism. With the object of estimating what would be the eventual increase in organ donation, per cadaveric donor, due to the law of presumed consent, the health econometric tool of quantile regression method for panel data was used, applied to a sample of 34 countries during a five-year period. The results obtained in this application indicate that there is benefit in adopting the law of presumed consent, which has a positive effect on the organ donation rate, around 21 – 26%, compared to the law of informed consent.