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.

No description available.

In Anbetracht steigender Inzidenzraten für Tumorerkrankungen, bei gleichzeitig steigenden Überlebensraten, gewinnt die Frage nach der verbleibenden gesundheitsbezogenen Lebensqualität (HRQOL) im klinischen Alltag immer größere Bedeutung. Für einige HRQOL- Domänen bzw. Symptom-Items konnte bereits gezeigt werden, dass diese mit der Überlebenszeit von Patienten korrelieren bzw. Prädiktoren für längeres Überleben sind. Ziel der vorliegenden Studie war es die gesundheitsbezogenen Lebensqualität (HRQOL) bei einer großen Kohorte (N=1879) deutscher Tumorpatienten zu untersuchen. Mit Hilfe des Fragebogens EORTC QLQ-C30 (Version 3.0) wurde die physische Funktion, Rollenfunktion, emotionale Funktion, kognitive Funktion, soziale Funktion, Fatigue und die globale Lebensqualität gemessen. Aus der Krankenakte der betreffenden Patienten wurden relevante soziodemographische Merkmale (Alter, Geschlecht, Nationalität, Body-Mass-Index) und klinische Merkmale (Diagnose, TNM- Stadium, Therapieform, Intervall seit Diagnose, Rehabilitationsmaßnahme) extrahiert und statistisch ausgewertet. Wir stellten fest, dass die Tumorpatienten in allen HRQOL-Domänen starke Beeinträchtigungen im Vergleich zur gesunden deutschen Allgemeinbevölkerung zeigen. In besonderem Maße zeigten sich jüngere Tumorpatienten (≤59Jahre) beeinträchtigt. Weiterhin sahen wir klinisch relevante Beeinträchtigungen bei Patienten mit einer Therapiekombination aus Radio- und Chemotherapie, Patientinnen mit Brustkrebs und Tumoren des weiblichen Genitaltrakts und Patienten mit Migrationshintergrund aus Südosteuropa. Im gesamten Patientenkollektiv stellten wir eine überdurchschnittlich hohe Ausprägung von Fatigue (CRF) fest. Aufgrund der gemessenen Beeinträchtigungen der HRQOL, sowie der hohen Ausprägung von CRF sollten den betroffenen Patienten möglichst frühzeitig zusätzliche spezifische Therapie-Angebote unterbreitet werden, wie z.B. eine onkologische Rehabilitation und psycho-onkologische Therapiemaßnahmen, denn diese Intervention zeigte eine klinisch relevante Verbesserung in allen HRQOL-Domänen. Die routinemäßige Erfassung der subjektiven Lebensqualität der Betroffenen stellt in unseren Augen eine wichtige Zielgröße dar, welcher in der Routineversorgung neben objektiven Befunden (wie Tumoransprechen usw.) viel mehr Beachtung geschenkt werden sollte.

One of the great challenges facing human systems today is how to prepare for, manage, and adapt successfully to the profound and rapid changes wreaked by disasters. Wellington, New Zealand, is a capital city at significant risk of devastating earthquake and tsunami, potentially requiring mass evacuations with little or short notice. Subsequent hardship and suffering due to widespread property damage and infrastructure failure could cause large areas of the Wellington Region to become uninhabitable for weeks to months. Previous research has shown that positive health and well-being are associated with disaster-resilient outcomes. Preventing adverse outcomes before disaster strikes, through developing strengths-based skill sets in health-protective attitudes and behaviours, is increasingly advocated in disaster research, practise, and management. This study hypothesised that well-being constructs involving an affective heuristic play vital roles in pathways to resilience as proximal determinants of health-protective behaviours. Specifically, this study examined the importance of health-related quality of life and subjective well-being in motivating evacuation preparedness, measured in a community sample (n=695) drawn from the general adult population of Wellington’s isolated eastern suburbs. Using a quantitative epidemiological approach, the study measured the prevalence of key quality of life indicators (physical and mental health, emotional well-being or “Sense of Coherence”, spiritual well-being, social well-being, and life satisfaction) using validated psychometric scales; analysed the strengths of association between these indicators and the level of evacuation preparedness at categorical and continuous levels of measurement; and tested the predictive power of the model to explain the variance in evacuation preparedness activity. This is the first study known to examine multi-dimensional positive health and global well-being as resilient processes for engaging in evacuation preparedness behaviour. A cross-sectional study design and quantitative survey were used to collect self-report data on the study variables; a postal questionnaire was fielded between November 2008 and March 2009 to a sampling frame developed through multi-stage cluster randomisation. The survey response rate was 28.5%, yielding a margin of error of +/- 3.8% with 95% confidence and 80% statistical power to detect a true correlation coefficient of 0.11 or greater. In addition to the primary study variables, data were collected on demographic and ancillary variables relating to contextual factors in the physical environment (risk perception of physical and personal vulnerability to disaster) and the social environment (through the construct of self-determination), and other measures of disaster preparedness. These data are reserved for future analyses. Results of correlational and regression analyses for the primary study variables show that Wellingtonians are highly individualistic in how their well-being influences their preparedness, and a majority are taking inadequate action to build their resilience to future disaster from earthquake- or tsunami-triggered evacuation. At a population level, the conceptual multi-dimensional model of health-related quality of life and global well-being tested in this study shows a positive association with evacuation preparedness at statistically significant levels. However, it must be emphasised that the strength of this relationship is weak, accounting for only 5-7% of the variability in evacuation preparedness. No single dimension of health-related quality of life or well-being stands out as a strong predictor of preparedness. The strongest associations for preparedness are in a positive direction for spiritual well-being, emotional well-being, and life satisfaction; all involve a sense of existential meaningfulness. Spiritual well-being is the only quality of life variable making a statistically significant unique contribution to explaining the variance observed in the regression models. Physical health status is weakly associated with preparedness in a negative direction at a continuous level of measurement. No association was found at statistically significant levels for mental health status and social well-being. These findings indicate that engaging in evacuation preparedness is a very complex, holistic, yet individualised decision-making process, and likely involves highly subjective considerations for what is personally relevant. Gender is not a factor. Those 18-24 years of age are least likely to prepare and evacuation preparedness increases with age. Multidimensional health and global well-being are important constructs to consider in disaster resilience for both pre-event and post-event timeframes. This work indicates a need for promoting self-management of risk and building resilience by incorporating a sense of personal meaning and importance into preparedness actions, and for future research into further understanding preparedness motivations.

Indiana University-Purdue University Indianapolis (IUPUI) / Little is known regarding the psychosocial outcomes of adult siblings of adults with autism. Accordingly, the current study sought to: (1) illuminate factors that predict health-related quality of life, caregiver burden, and reported benefits in adult siblings of adults with autism, using a stress and coping framework and (2) compare outcomes of siblings of adults with autism (n = 31) to siblings of adults with Down syndrome (DS) (n = 54). For the within subject hypotheses, analyses were repeated within the DS group and an overall Disability group (n = 97). The Disability group consisted of participants in the Autism and Down syndrome groups plus twelve individuals in a mixed group of adult siblings of adults with DS who presented with co-morbid symptoms of autism. Variables were organized using The Adult Sibling Caregiver Conceptual Model (ASCCM) into three categories: antecedents (e.g., sibling relationship quality, problem behaviors of the disabled sibling), mediators (e.g., coping strategies, cognitive appraisal types), and psychosocial outcome variables (i.e., mental and physical health-related quality of life, caregiver burden, and reported benefits). For the within subject primary hypotheses, I posited a series of relationships between the antecedents and outcomes based on prior literature on demographic and individual difference predictors (e.g., siblings of adults with autism with fewer problem behaviors would have increased health-related quality of life [HRQOL], decreased caregiver burden, and increased reported benefits) and on stress and coping factors related to the burden of providing care for an individual with autism (e.g., greater use of avoidant coping strategies would be related to lower HRQOL, increased caregiver burden, and decreased reports of benefits). Exploratory hypotheses also were examined (e.g., being married would be associated with increased HRQOL, decreased burden, and increased reported benefits). The within-subject results indicated support for eight of the ten primary hypotheses and five of the six exploratory hypotheses when examined within at least one of the study groups: Autism, DS, or Disability. Overall, sibling caregivers, regardless of their sibling’s disability, reported more favorable psychosocial outcomes when demands were lower (e.g., less severe levels of problem behaviors, fewer autism symptoms exhibited by their disabled sibling, decreased additional pile-up stress), when resources were available to cope with stress (e.g., social support, respite care), and when healthy responses to stress were reported (e.g., use of emotion focused vs. avoidant coping strategies, use of challenge vs. threat appraisal types). Of note, reported benefits, which have rarely been examined in the autism literature, were strongly related to the quality of a sibling relationship across all study groups, and with the helpfulness of received services and perceived social support within the Autism group. The between subject hypotheses also were largely supported. As expected, compared to siblings of those with Down syndrome, siblings of those with autism demonstrated greater levels of caregiver burden and lower mental HRQOL. Moreover, there was a rank ordering in levels of caregiver burden across disability groups; siblings of adults with DS reported the lowest burden, siblings of adults with DS with symptoms of autism reported significantly higher levels of burden, and siblings of those with autism reported the most burden. The results imply that autism, either alone or co-morbid with another diagnosis, presents an increased risk of stress and caregiver burden, not only in parent caregivers, but also in sibling caregivers. Interestingly, there was also evidence for higher levels of stress related growth within the Autism group compared to the DS group. Future research will be needed to generalize the results of this study to broader samples of adult siblings while taking a life course perspective to assess changes in non-disabled siblings’ outcomes over time.

Background InVietnam, the proportion of people aged 60 and above has increased rapidly in recent decades. The majority live in rural areas where socioeconomic status is more disadvantaged than in urban areas.Vietnam’s economic status is improving but disparities in income and living conditions are widening between groups and regions. A consistent and emerging danger of communicable diseases and an increase of non-communicable diseases exist concurrently. The emigration of young people and the impact of other socioeconomic changes leave more elderly on their own and with less family support. Introduction of user fees and development of a private sector improve the coverage and quality of health care but increase household health expenditures and inequalities in health care. Life expectancy at birth has increased, but not much is known about changes during old age. There is a lack of evidence, particularly in rural settings, about health-related quality of life (HRQoL) among older people within the context of socioeconomic changes and health-sector reform. Knowledge of long-term elderly care needs in the community and the relevant models are still limited. To provide evidence for developing new policies and models of care, this thesis aimed to assess general health status, health care needs, and perspectives on future health care options for community-dwelling older people. Methods An abridged life table was used to estimate cohort life expectancies at old age from longitudinal data collected by FilaBavi DSS during 1999-2006. This covered 7,668 people aged 60 and above with 43,272 person-years. A 2007 cross-sectional survey was conducted among people aged 60 and over living in 2,240 households that were randomly selected from the FilaBavi DSS. Interviews used a structured questionnaire to assess HRQoL, daily care needs, and willingness to use and to pay for models of care. Participant and household socioeconomic characteristics were extracted from the 2007 DSS re-census. Differences in life expectancy are examined by socioeconomic factors. The EQ-5D index is calculated based on the time trade-off tariff. Distributions of study subjects by study variables are described with 95% confidence intervals. Multivariate analyses are performed to identify socioeconomic determinants of HRQoL, need of support, ADL index, and willingness to use and pay for models of care. In addition, four focus group discussions with the elderly, their household members, and community association representatives were conducted to explore perspectives on the use of services by applying content analysis. Results Life expectancy at age 60 increased by approximately one year from 1999-2002 to 2003-2006, but tended to decrease in the most vulnerable groups. There is a wide gap in life expectancy by poverty status and living arrangement. The sex gap in life expectancy is consistent across all socioeconomic groups and is wider among the more disadvantaged populations.  The EQ-5D index at old age is 0.876. Younger age groups, position as household head, working, literacy, and belonging to better wealth quintiles are determinants of higher HRQoL. Ageing has a primary influence on HRQoL that is mainly due to reduction in physical (rather than mental) functions. Being a household head and working at old age are advantageous for attaining better HRQoL in physical rather than psychological terms. Economic conditions affect HRQoL through sensory rather than physical functions. Long-term living conditions are more likely to affect HRQoL than short-term economic conditions. Dependence in instrumental or intellectual activities of daily living (ADLs) is more common than in basic ADLs. People who need complete help are fewer than those who need some help in almost all ADLs. Over two-fifths of people who needed help received enough support in all ADL dimensions. Children and grand-children are confirmed to be the main caregivers. Presence of chronic illness, age groups, sex, educational level, marital status, household membership, working status, household size, living arrangement, residential area, household wealth, and poverty status are determinants of the need for care. Use of mobile teams is the most requested service; the fewest respondents intend to use a nursing centre. Households expect to use services for their elderly to a greater extent than did the elderly themselves. Willingness to use services decreases when potential fees increase. The proportion of respondents who require free services is 2 to 3 times higher than those willing to pay full cost. Households are willing to pay more for day care and nursing centres than are the elderly. The elderly are more willing to pay for mobile teams than are their households. ADL index, age group, sex, literacy, marital status, living arrangement, head of household status, living area, working status, poverty and household wealth are factors related to willingness to use services.   Conclusions                                                                                         There is a trend of increasing life expectancy at older ages in ruralVietnam. Inequalities in life expectancy exist between socioeconomic groups. HRQoL at old age is at a high level, but varies substantially according to socioeconomic factors. An unmet need of daily care for older people remains. Family is the main source of support for care. Need for care is in more demand among disadvantaged groups.  Development of a social network for community-based long-term elderly care is needed. The network should focus on instrumental and intellectual ADLs rather than basic ADLs. Home-based care is more essential than institutionalized care. Community-based elderly care will be used and partly paid for if it is provided by the government or associations. The determinants of elderly health and care needs should be addressed by appropriate social and health policies with greater targeting of the poorest and most disadvantaged groups. Building capacity for health professionals and informal caregivers, as well as support for the most vulnerable elderly groups, is essential for providing and assessing the services. / Aging and Living Conditions Program / Vietnam-Sweden Collaborative Program in Health, SIDA/Sarec