Does Cost-Effectiveness Analysis Have a Role in US Managed Care Drug Formularies? An Empirical Study of Utilization, Costs, Outcomes, and Elasticity of Demand of a Value-Based Formulary

Thesis (Ph.D.)--University of Washington, 2015

Contributo para a validação da versão portuguesa do instrumento de avaliação VOICE (service users perceptions of impatient care, Views on Inpatient Care)

RESUMO: Nos últimos anos assistiu-se a um reposicionamento das pessoas portadoras de doença mental na sociedade, no sentido de viverem em pleno os seus direitos, sem restrições. Esta tendência acompanhou as transformações que se têm vivido na forma como os utentes dos serviços de saúde interagem com os mesmos e com os profissionais de saúde, de forma a permitir uma maior autonomização e responsabilização no que concerne ao tratamento da sua doença, a relação que estabelecem com o seu médico, e a participação na avaliação e monitorização da qualidade dos serviços. Mais recentemente, também no mundo científico, esta afirmação se fez sentir, com o surgimento de investigação liderada por utentes, no sentido de estudar de forma adequada questões que partem do seu ponto de vista, e que possibilitem a produção de conhecimento significativa no contexto das suas experiências. Com o presente trabalho pretende-se contribuir para a validação da versão portuguesa do VOICE (Service Users’ Perceptions of Inpatient Care, Views on Inpatient Care) (Evans et al., 2012), instrumento para a avaliação dos serviços de internamento de agudos em psiquiatria, construído a partir de um investigação liderado por utentes e partindo das suas perspectiva. O VOICE é constituído por 19 questões, agrupadas em sete domínios: admissão; cuidados e tratamento; medicação; equipa de técnicos do internamento; terapia e atividades; ambiente e diferenças. O presente estudo envolveu uma amostra de 85 utentes de um serviço de internamento de agudos de uma instituição psiquiátrica do Norte de Portugal. A versão portuguesa do VOICE apresentou boa aceitação por parte dos utentes e boas características psicométricas - a consistência interna foi alta (α = 0,87) e todos, exceto um item (item 6), apresentam elevadas correlações item-total (variando de 0,18 - item 6 a 0,71 - item 11; M = 0,54, DP = 0,15), sugerindo ser um instrumento útil na avaliação dos serviços de internamento de agudos. No futuro torna-se necessário alargar o estudo a outros contextos de internamento e envolvendo amostras mais alargadas.-------------- ABSTRACT: In recent years there has been a gradual process to help people with the experience of mental illness regaining their full rights. Following the advances in the understanding of mental health problems, and the use of medication to help patients overcome symptoms, service-users have become more autonomous and responsible in the way they deal with health professionals, and are now called to participate in assessing and monitoring mental health services and policies. In the context of these transformations we have assisted to the emergence of research led by service-users (in this case of psychiatric and mental health services) in order to emphasize their point of view, and to enable the production of significant knowledge resulting from their experiences, and perceptions. The present study aims to contribute to the dissemination of service-user led research, based on the adaptation and validation of the Portuguese version of the VOICE - Service Users' Perceptions Questionnaire, Views on Inpatient Care. (Evans et al., 2012). The VOICE is composed of 19 questions, grouped in seven domains: admissions, care and treatment, medication, team of technicians during hospitalization, therapy and activities, environment and diversity. The present study involved a sample of 85 inpatient of a psychiatric institution in Northern Portugal. The Portuguese version of the VOICE showed good psychometric properties and was well accepted by patients [high internal consistency (α = 0,87); and high correlation of each item, except item 6, with the total score (ranging from 0.18 on item 6 to 0.71 on item 11; average=0,54; SD=0,15), suggesting it to be a useful tool for assessing inpatient services. In the future there is a need to extend the study to other contexts and include larger samples.

Diabetes care in Switzerland: good, but perfectible: a population-based cross-sectional survey.

BACKGROUND: While Switzerland invests a lot of money in its healthcare system, little is known about the quality of care delivered. The objective of this study was to assess the quality of care provided to patients with diabetes in the Canton of Vaud, Switzerland. METHODS: Cross-sectional study of 406 non-institutionalized adults with type 1 or 2 diabetes. Patients' characteristics, diabetes and process of care indicators were collected using a self-administered questionnaire. Process indicators (past 12 months) included HbA1C check among HbA1C-aware patients, eye assessment by ophtalmologist, microalbuminuria check, feet examination, lipid test, blood pressure and weight measurement, influenza immunization, physical activity recommendations, and dietary recommendations. Item-by-item (each process of care indicator: percentage of patients having received it), composite (mean percentage of recommended care: sum of received processes of care / sum of possible recommended care), and all-or-none (percentage of patients receiving all specified recommended care) measures were computed. RESULTS: Mean age was 64.4 years; 59% were men. Type 1 and type 2 diabetes were reported by 18.2% and 68.5% of patients, respectively, but diabetes type remained undetermined for almost 20% of patients. Patients were treated with oral anti-diabetic drugs (50%), insulin (23%) or both (27%). Of 219 HbA1C-aware patients, 98% reported ≥ one HbA1C check during the last year. Also, ≥94% reported ≥ one blood pressure measurement, ≥ one weight measurement or lipid test, and 68%, 64% and 56% had feet examination, microalbuminuria check and eye assessment, respectively. Influenza immunization was reported by 62% of the patients.The percentage of patients receiving all processes of care ranged between 14.2%-16.9%, and 46.6%-50.7%, when considering ten and four indicators, respectively. Ambulatory care utilization showed little use of multidisciplinary care, and low levels of participation in diabetes-education classes. CONCLUSIONS: While routine processes-of-care were performed annually in most patients, diabetes-specific risk screenings, influenza immunization, physical activity and dietary recommendations were less often reported; this was also the case for multidisciplinary care and participation in education classes. There is room for diabetes care improvement in Switzerland. These results should help define priorities and further develop country-specific chronic disease management initiatives for diabetes.

Appropriateness of respiratory care: evidence-based guidelines.

PRINCIPLES: Respiratory care is universally recognised as useful, but its indications and practice vary markedly. In order to improve the appropriateness of respiratory care in our hospital, we developed evidence-based local guidelines in a collaborative effort involving physiotherapists, physicians and health service researchers. METHODS: Recommendations were developed using the standardised RAND appropriateness method. A literature search was conducted based on terms associated with guidelines and with respiratory care. A working group prepared proposals for recommendations which were then independently rated by a multidisciplinary expert panel. All recommendations were then discussed in common and indications for procedures were rated confidentially a second time by the experts. The recommendations were then formulated on the basis of the level of evidence in the literature and on the consensus among these experts. RESULTS: Recommendations were formulated for the following procedures: non-invasive ventilation, continuous positive airway pressure, intermittent positive pressure breathing, intrapulmonary percussive ventilation, mechanical insufflation-exsufflation, incentive spirometry, positive expiratory pressure, nasotracheal suctioning and non-instrumental airway clearance techniques. Each recommendation referred to a particular medical condition and was assigned to a hierarchical category based on the quality of the evidence from the literature supporting the recommendation and on the consensus among the experts. CONCLUSION: Despite a marked heterogeneity of scientific evidence, the method used allowed us to develop commonly agreed local guidelines for respiratory care. In addition, this work fostered a closer relationship between physiotherapists and physicians in our institution.

Weight status and gender-related differences in motor skills and in child care - based physical activity in young children.

Over the last decades, a decline in motor skills and in physical activity and an increase in obesity has been observed in children. However, there is a lack of data in young children. We tested if differences in motor skills and in physical activity according to weight or gender were already present in 2- to 4-year-old children. Fifty-eight child care centers in the French part of Switzerland were randomly selected for the Youp'là bouge study. Motor skills were assessed by an obstacle course including 5 motor skills, derived from the Zurich Neuromotor Assessment test. Physical activity was measured with accelerometers (GT1M, Actigraph, Florida, USA) using age-adapted cut-offs. Weight status was assessed using the International Obesity Task Force criteria (healthy weight vs overweight) for body mass index (BMI). Of the 529 children (49% girls, 3.4 ± 0.6 years, BMI 16.2 ± 1.2 kg/m2), 13% were overweight. There were no significant weight status-related differences in the single skills of the obstacle course, but there was a trend (p = 0.059) for a lower performance of overweight children in the overall motor skills score. No significant weight status-related differences in child care-based physical activity were observed. No gender-related differences were found in the overall motor skills score, but boys performed better than girls in 2 of the 5 motor skills (p ≤ 0.04). Total physical activity as well as time spent in moderate-vigorous and in vigorous activity during child care were 12-25% higher and sedentary activity 5% lower in boys compared to girls (all p < 0.01). At this early age, there were no significant weight status- or gender-related differences in global motor skills. However, in accordance to data in older children, child care-based physical activity was higher in boys compared to girls. These results are important to consider when establishing physical activity recommendations or targeting health promotion interventions in young children.

Patterns of care in recurrent glioblastoma in Switzerland: a multicentre national approach based on diagnostic nodes.

Despite moderate improvements in outcome of glioblastoma after first-line treatment with chemoradiation recent clinical trials failed to improve the prognosis of recurrent glioblastoma. In the absence of a standard of care we aimed to investigate institutional treatment strategies to identify similarities and differences in the pattern of care for recurrent glioblastoma. We investigated re-treatment criteria and therapeutic pathways for recurrent glioblastoma of eight neuro-oncology centres in Switzerland having an established multidisciplinary tumour-board conference. Decision algorithms, differences and consensus were analysed using the objective consensus methodology. A total of 16 different treatment recommendations were identified based on combinations of eight different decision criteria. The set of criteria implemented as well as the set of treatments offered was different in each centre. For specific situations, up to 6 different treatment recommendations were provided by the eight centres. The only wide-range consensus identified was to offer best supportive care to unfit patients. A majority recommendation was identified for non-operable large early recurrence with unmethylated MGMT promoter status in the fit patients: here bevacizumab was offered. In fit patients with late recurrent non-operable MGMT promoter methylated glioblastoma temozolomide was recommended by most. No other majority recommendations were present. In the absence of strong evidence we identified few consensus recommendations in the treatment of recurrent glioblastoma. This contrasts the limited availability of single drugs and treatment modalities. Clinical situations of greatest heterogeneity may be suitable to be addressed in clinical trials and second opinion referrals are likely to yield diverging recommendations.

Child care by choice or by default? : examining the experiences of unregulated home-based child care for women in paid work and training

This thesis aims to uncover the dynamics, causes and outcomes of women's reliance on unregulated home-based child care in Ontario, Canada, and the implications ofthis form of care for women's equality. Drawing on a longitudinal qualitative study, I examine the diverse experience of 14 women using home-based child care and engaged in both paid work/training and care work for children under the age of six, and draw comparisons with users of other forms of child care. I argue that home-based child care involves high levels of instability for continuity of care and is chosen largely as a default position based on economic considerations. It represents a compromise between the demands of social reproduction and paid work/training that entangles mothers in relations of exploitation with care providers. Doing so leaves both mothers and care providers socially and economically vulnerable and relying on social networks to fill in the gaps.

Managing Risk in a Culture of Rights: Providing Support and Treatment in Community-Based Settings for Persons with Intellectual Disabilities who Sexually Offend

People with intellectual disability who sexually offend commonly live in community-based settings since the closing of all institutions across the province of Ontario. Nine (n=9) front line staff who provide support to these individuals in three different settings (treatment setting, transitional setting, residential setting) were interviewed. Participants responded to 47 questions to explore how sex offenders with intellectual disability can be supported in the community to prevent re-offenses. Questions encompassed variables that included staff attitudes, various factors impacting support, structural components of the setting, quality of life and the good life, staff training, staff perspectives on treatment, and understanding of risk management. Three overlapping models that have been supported in the literature were used collectively for the basis of this research: The Good Lives Model (Ward & Gannon, 2006; Ward et al., 2007), the quality of life model (Felce & Perry, 1995), and variables associated with risk management. Results of this research showed how this population is being supported in the community with an emphasis on the following elements: positive and objective staff attitude, teamwork, clear rules and protocols, ongoing supervision, consistency, highly trained staff, and environments that promote quality of life. New concepts arose which suggested that all settings display an unequal balance of upholding human rights and managing risks when supporting this high-risk population. This highlights the need for comprehensive assessments in order to match the offender to the proper setting and supports, using an integration of a Risk, Need, Responsivity model and the Good Lives model for offender rehabilitation and to reduce the likelihood of re-offenses.

The experiences and priorities of young people who care for their siblings in Tanzania and Uganda

This report presents key findings from a small-scale pilot research project that explored the experiences and priorities of young people caring for their siblings in sibling-headed households affected by AIDS in Tanzania and Uganda. Qualitative and participatory research was conducted with 33 young people living in sibling-headed households and 39 NGO staff and community members in rural and urban areas of Tanzania and Uganda. The report analyses the ways that young people manage transitions to caring for their younger siblings following their parents’ death and the impacts of caring on their family relations, education, emotional wellbeing and health, social lives and their transitions to adulthood. The study highlights gendered- and age-related differences in the nature and extent of young people’s care work and discusses young people’s needs and priorities for action, based on the views of young people, NGO staff and community members. Meeting the basic needs of young people living in sibling-headed households, listening to young people’s views, fostering peer support and relationships of trust with supportive adults, raising awareness and advocacy emerge as key priorities to safeguard the rights of children and young people living in sibling-headed households and challenge the stigma and marginalisation they sometimes face.

Access, equity and the role of rights in health care

Modern health care rhetoric promotes choice and individual patient rights as dominant values. Yet we also accept that in any regime constrained by finite resources, difficult choices between patients are inevitable. How can we balance rights to liberty, on the one hand, with equity in the allocation of scarce resources on the other? For example, the duty of health authorities to allocate resources is a duty owed to the community as a whole, rather than to specific individuals. Macro-duties of this nature are founded on the notion of equity and fairness amongst individuals rather than personal liberty. They presume that if hard choices have to be made, they will be resolved according to fair and consistent principles which treat equal cases equally, and unequal cases unequally. In this paper, we argue for greater clarity and candour in the health care rights debate. With this in mind, we discuss (1) private and public rights, (2) negative and positive rights, (3) procedural and substantive rights, (4) sustainable health care rights and (5) the New Zealand booking system for prioritising access to elective services. This system aims to consider: individual need and ability to benefit alongside the resources made available to elective health services in an attempt to give the principles of equity practical effect. We describe a continuum on which the merits of those, sometimes competing, values-liberty and equity-can be evaluated and assessed.

Ownership rights in performance-based contracting: A case study of English law

Clients and contractors need to be aware of the project’s legal environment because the viability of a procurement strategy can be vitiated by legal rules. This is particularly true regarding Performance-Based Contracting (PBC) whose viability may be threatened by rules of property law: while the PBC concept does not require that the contractor transfers the ownership in the building materials used to the client, the rules of property law often lead to an automatic transfer of ownership. But does the legal environment really render PBC unfeasible? In particular, is PBC unfeasible because contractors lose their materials as assets? These questions need to be answered with respect to the applicable property law. As a case study, English property law has been chosen. Under English law, the rule which governs the automatic transfer of ownership is called quicquid plantatur solo, solo credit (whatever is fixed to the soil belongs to the soil). An analysis of this rule reveals that not all materials which are affixed to land become part of the land. This fate only occurs in relation to materials which have been affixed with the intention of permanently improving the land. Five fictitious PBC cases have been considered in terms of the legal status of the materials involved, and several subsequent legal questions have been addressed. The results suggest that English law does actually threaten the feasibility of PBC in some cases. However, it is also shown that the law provides means to circumvent the unwanted results which flow from the rules of property law. In particular, contractors who are interested in keeping their materials as assets can insist on agreeing a property right in the client’s land, i.e. a contractor’s lien. Therefore, the outcome is that English property law does not render the implementation of the PBC concept unfeasible. At a broader level, the results contribute to the theoretical framework of PBC as an increasingly used procurement strategy.

Ethics of care in supporting disabled forced migrants: interactions with professionals and ethical dilemmas in health and social care in the South-East of England

This article explores the interactions between disabled forced migrants with care needs and professionals and the restrictive legal, policy and practice context that health and social care professionals have to confront, based on the findings of a qualitative study with 45 participants in the South-East of England. In-depth interviews were conducted with 15 forced migrants who had diverse impairments and chronic illnesses (8 women and 7 men), 13 family caregivers and 17 support workers and strategic professionals working in social care and the third sector in Slough, Reading and London. The legal status of forced migrants significantly affects their entitlements to health and social care provision, resulting in prolonged periods of destitution for many families. National asylum support policies, difficult working relationships with UK Border Agency, higher eligibility thresholds and reduced social care budgets of local authorities were identified as significant barriers in responding to the support needs of disabled forced migrants and family caregivers. In this context, social workers experienced considerable ethical dilemmas. The research raises profound questions about the potential and limitations of health and social care policies, provision, and practice as means of protection and support in fulfilling the human rights of forced migrants with care needs.