Abstract : Rare diseases are debilitating conditions often leading to severe clinical manifestations for affected patients. Orphan drugs have been developed to treat these rare diseases affecting a small number of individuals. Incentives in the legal framework aimed to recoup the research and development cost of orphan drugs for pharmaceutical companies have been implemented in the United States and the European Union. At the present time, Canada is still lacking a legal and policy framework for orphan drugs. Several problems at the federal and provincial levels remain: lack of research funds for rare diseases, discrepancies on orphan drug policies between provinces, difficulties to access and reimburse these high price drugs. Recommendations and measures are proposed, such as a pan-Canadian (national) scientific committee to establish evidence-based guidelines for patients to access orphan drugs uniformly in all provinces with a disease specific registry, a formal agreement for a centralized Canadian public funding reimbursement procedure, and increasing the role of “guardian” for prices by the Patented Medicines Review Board in Canada. These recommendations and measures will be beneficial for the implementation of a policy framework for orphan drugs in Canada. / Résumé : Les maladies rares sont des maladies sérieuses pouvant causer des manifestations cliniques sévères chez les patients atteints. Les médicaments orphelins ont été développés pour le traitement de ces maladies rares qui touchent un petit nombre d’individus. Un cadre légal permettant des incitatifs pour les compagnies pharmaceutiques aux États-Unis et au niveau de l’Union Européenne a favorisé la recherche et le développement desdits médicaments. Présentement, il n’existe pas de cadre juridique et de politiques spécifiques au Canada entourant les médicaments orphelins. Ceci a mené à plusieurs problèmes tant au niveau fédéral que provincial dont: un manque de support financier consacré à la recherche pour les maladies rares, des disparités entre les provinces concernant les politiques pour les médicaments orphelins, des difficultés d’accès et de remboursement desdits médicaments dont les coûts sont élevés. Des recommandations et mesures sont proposées, telles l’implantation d’un comité scientifique pancanadien (national) afin d’établir des lignes directrices fondées sur des données probantes pour faciliter un accès uniforme aux médicaments orphelins pour les patients, y compris un registre spécifique élaboré pour chaque maladie, établir une entente formelle centralisée pour tout le Canada pour un financement public de remboursement des médicaments orphelins, augmenter le rôle de « gardien » des prix par le Conseil d’examen du prix des médicaments brevetés au Canada. Ces recommandations et mesures serviront à l’implantation d’un cadre de politiques pour les médicaments orphelins au Canada.

Hundreds of state regulations were passed during the “managed care backlash” of the late 1990s and early 2000s. Many of these anti-managed care regulations eased or eliminated constraints on patient utilization of health care services imposed by managed care organizations. Other regulations gave managed care providers more flexibility in the way they practiced care or helped patients appeal denials of claims. Despite the effort undertaken to pass these regulations, limited research exists on whether the regulations achieved their goal. To fill this gap, this study takes advantage of the variety of regulations enacted during the managed care backlash of the late 1990s and early 2000s to investigate their impact on patient-reported quality of care and mortality for managed care enrollees. The results indicate the regulations did improve patient-reported outcomes, but to varying degrees and only in the latter period of the backlash. Specifically, managed care enrollees who lived in states that adopted moderate-intensity regulations between 2000 and 2004 reported relatively better improvements in access to care and confidence in their provider than did managed care enrollees in states with low-intensity backlash regulations. The positive effect on access to care was similar in states that adopted high-intensity regulations. However, no positive effect was found for any outcome in the first period (1996-2000). These results show that states with the most intense regulatory backlash did not realize better patient-reported outcomes. Instead, states that pursued moderate-intensity backlash regulations experienced relatively better outcomes for their managed care enrollees.

This thesis argues that current treatment in acute public mental health services is counterproductive for the wellbeing of those subject to such services. The consumer/survivor movement activism against the coercive nature of treatment is analysed according to new social movement theory. According to social theorists such as Alaine Touraine, new social movements are characterised by a struggle over identity. Consistent with this theme, what is identified in this thesis as central to the consumer/survivor movement objection to the nature of treatment in acute public mental health services, is the failure of services to respect patient identity as persons. What might account for this failure is analysed in this thesis through an examination of the question of the conceptualisation of the subject in the theory and concepts of psychiatry, in the practice of psychiatry, in mental health law and in government policy. / As a counterposition to the above perspectives, the work of RD Laing, Charles Taylor and Paul Ricoeur are considered in an attempt to develop a conceptualisation of the subject grounded in a historical narrative. Further, Emmanuel Levinas’ and Axel Honneth’s work is drawn on to identify the practical implications of Honneth’s claim for a politics of recognition, which also supports the consumer/survivor movement demand for recognition as subjects in mental health services.

Examining the persistence of disparities over time is an important obligation in terms of rectifying, maintaining, and improving community health and social well-being for all. This study analyzed the individual factors of (a) race/ ethnicity and (b) dual eligibility, as a proxy measure of socioeconomic status, as well as the environmental factor of (c) place of residence, and the organizational factor of (d) Rural Health Clinic (RHC) type on emergency room (ER) utilization of older adult Medicare patients treated by RHCs within the Department of Health and Human Services' (DHHS) Region 4. A prospective, multi-level, longitudinal design was employed to analyze potential health disparities or gaps that may exist among RHC Medicare beneficiary patients (+65) using longitudinal, mixed multilevel modeling in SPSS. The years of investigation were 2010 through 2012. R4 has continually lagged behind other Regions in the Nation in having higher Health Disparities and ER Utilization rates related to Race, Poverty, and Rural Isolation. A key question is: Do these disparities persist? This study's findings support that dual eligible RHC patients utilized ER services at higher rates than non-dual eligible, Medicare only RHC patients at: 77%, 80%, and 66%, in 2010, 2011, and 2012, respectively; and above the White reference group, Black RHC Medicare patients utilized ER services at higher rates of: 18%, 20%, and 34%, in 2010, 2011, and 2012, respectively. These findings support that dual Medicare and Medicaid eligibility, as a proxy measure of socioeconomic status, and race continue to influence higher rates of ER utilization in Region 4. In terms of health and utilization disparities, strikingly and persistently, as recent as 2012, Black, dual eligible RHC Medicare beneficiary patients age 65 and over are twice as likely to utilize ER services for health care than their more advantaged counterparts. Health care leaders and policymakers are seeking evidence-based performance measures as tools for detecting gaps in health care and using those subsequent findings as leverage to implement policy change for the purpose of increasing health care delivery performance system-wide while lowering health disparities across various patient populations. Toward that goal, communicating and disseminating the findings of this study contributes to the body of knowledge and enables policy leaders to better make decisions based on empirical evidence in order to strengthen the health care delivery system for older adults in diverse rural contexts. From a health and public affairs policy perspective, crafting in tandem targeted, top-down, population health and bottom-up, community interventions to curb poor health outcomes and high health care utilization would be in the public interest at-large within this region of the Southeastern United States.

The Patient Protection and Affordable Care Act (ACA) aimed to increase accessibility to medical resources for those previously uninsured. Certainly, the ACA has expanded insurance to millions of Americans, however, the evidence and discourse surrounding health accessibility calls into question why, despite the growth of insured Americans, the increase in health insurance coverage under the ACA has not lead to greater accessibility for low income minorities. I propose that disparities in preventive care, the emergency room, and primary care provider services stand as barriers for low income minorities. Insurance coverage does not necessarily equate to greater accessibility if individuals do not have the means to utilize those resources.

In the aftermath of World War II, the Liberal Democratic Party of Japan followed Germany’s blueprint in fashioning a universal health coverage system. Comparisons to Germany’s welfare state during this same time period reveal markedly different social and mental health policy practices, as Germany’s Christian Democratic Union and Social Democratic Party cooperated toward progressive policies while the Liberal Democratic Party largely neglected social welfare expansion. The effect of these practices is reflected in budgetary provisions, institutionalization practices, and mental health epidemiology. This research finds that a favorable economic climate allowed the Liberal Democratic Party to politically isolate the Social Democratic Party and focus on economic productivity as opposed to welfare expansion. In contrast, West Germany’s competition with East Germany forced cooperation of its two largest political parties to balance economic policy and social progress, which is today reflected in mental health outcomes and policies markedly more favorable than those of Japan.

Background: Sexual consent is often defined as the voluntary agreement to participate in a sexual act, though the differing definitions across and within countries make legal consensus difficult. In recent years, due to popularization through social media, nonconsensual condom removal, termed stealthing, is becoming common, especially among young adults. Yet, little to no empirical evidence exists on this sexual behavior. Methods: In this exploratory sequential mixed methods approach, we aimed to address the current perception of stealthing among young adults. College students were recruited from general education courses at a medium-sized four- year public university. Focus groups were conducted to understand the current perception of stealthing, including knowledge, perceived influence, and outcome, followed by quantitative assessment of knowledge, attitude, and self-efficacy of sexual consent. Results: Results demonstrated central theme of health-decision making with associated themes of consent, which further included subthemes of privacy, trust, and violation, followed by consideration of stealthing as sexual assault and social norm and acceptance of stealthing. Quantitative assessment showed that knowledge and awareness of stealthing remains low, though sex differences exist on the perception of stealthing being considered sexual assault; with higher rates among males as compared to females. Conclusion: The act of stealthing has been popularized in social media. Our results demonstrate that there is a need for health educators to assess the prevalence of such a behavior among young adults and policy makers to assess the legal implications of nonconsensual condom removal.

Early diagnosis is a promising strategy to reduce premature mortalities and for optimal use of resources. But the absence of mathematical models specific to the data settings in LMIC’s impedes the construction of economic analysis necessary for decision-makers in the development of cancer control programs. This thesis presents a new methodology for parameterizing the natural history model of breast cancer based on data availabilities in low and middle income countries, and formulation of a control optimization problem to find the optimal screening schedule for mammography screening, solved using dynamic programming. As harms and benefits are known to increase with the increase in the number of lifetime screens, the trade-off was modeled by formulating the immediate reward as a function of false positives and life-years saved. The method presented in thesis will provide optimal screening schedules for multiple scenarios of Willingness to Pay (numeric value assigned for each life-year lived), including the resulting total number of lifetime screens per person, which can help decision-makers evaluate current resource availabilities or plan future resource needs for implementation.

The unprecedented growth of the aging population in Tennessee is a significant demographic trend that highlights the necessity for healthcare policy that tackles end-of-life issues. This study examined the perceived quality of end-of-life healthcare in Tennessee, areas that are in need of improvement, policies that have the potential to influence improvements, and the role of politics in end-of-life healthcare policy. It also assessed the support for end-of-life healthcare policy that would advance quality of care and expand end-of-life choices for Tennesseans, while evaluating the policy-making process that legislators employ. For this study, data were collected using semi-structured, in-depth interviews with an open interview guide. These interviews were conducted in a two-tiered format. Tier 1 included ten leading experts in various areas of end-of-life healthcare and aging in Tennessee. Tier 2 included nine legislators who were members of the health committees of either the Tennessee House of Representatives or the Tennessee Senate. Qualitative data were organized into numerous categories, and an initial phase of open coding was completed. From this phase of coding, emergent themes and focuses were discovered. This was followed by focused coding on all nineteen interviews using coding software to organize subtleties. Findings of the Tier 1 interviews indicated that there are many areas of end-of-life healthcare that need improvement including increased funding, expanded caregiver support, improved doctor-patient communication, and increased use of advance directives. Emergent themes included the influence of money, having difficult conversations, the stigma of death and dying, and supporting the caregiver. Tier 2 findings highlighted the motivations behind voting decisions and the level of awareness legislators have in areas of end-of-life healthcare policy. Both tiers also explored the support and opposition of physician-assisted death. The findings of this dissertation are intended to inform health professionals and state legislators. The data gathered through this exploratory research and the knowledge gained will lead to a greater understanding of end-of-life healthcare in Tennessee, and what needs to be done to improve its quality. This will allow individuals and families faced with end-of-life decisions to navigate the process with expanded options, access, and support.

Mental health treatment in state prisons is revealed to be highly variable, under-funded, and systematically inadequate. Existing literature exposes this injustice but fails to provide a comprehensive proposal for reform. This paper attempts to fill that gap, outlining a cost-effective, evidence-based treatment proposal, directly addressing the deficits in care revealed through analysis of our current system. In addition, this paper provides historical overviews of the prison system and mental health treatment, utilizing theoretical perspectives to contextualize this proposal in the present state of affairs. Lastly, the evidence is provided to emphasize the potential economic and social benefits of improving mental health treatment in state prisons. Significant findings suggest a clear financial, legal, and moral incentive for states to address this issue, while the proposal provides a viable method of doing so.