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Narcolepsy is a lifelong disorder that is usually diagnosed in early adulthood, however most often symptoms begin in the childhood years.It is characterized by excessive day time sleepiness (EDS) and features of rapid eye movement (REM) sleep. It is under diagnosed in both children and adults due to the clinical heterogeneity. We report a case of a child presenting with excessive day time sleepiness, who was diagnosed with narcolepsy, which although is a rare disease, is very treatable, and early intervention will reduce its psychosocial impact., peer-reviewed

Introduction: Australia’s systems for registering and funding medicines are well-established and internationally respected for their success in facilitating access while also protecting patients from harm and ensuring system sustainability. However, in recent years, there have been increasing concerns about whether these systems hinder access to medicines that patients want or need. In response, a number of “accelerated access” initiatives have been implemented to facilitate faster access. However, the risk-benefit balance of such schemes is currently unclear—while they may provide patients with a wider range of treatment options and earlier access to therapies, there is also increased uncertainty surrounding the safety, efficacy and cost-effectiveness of medicines made available via these pathways. Their impacts for stakeholders—including patients, physicians, members of the pharmaceutical industry and the general public—are likely to be significant and stakeholder engagement is therefore essential to determine the best way to proceed. Methods: This ethically-informed stakeholder analysis consisted of two phases. The first involved semi-structured interviews with physicians about their views on and experiences with accelerated access and standards of efficacy, safety and cost-effectiveness that they want to be applied by regulatory and funding bodies. The second was a mixed methods phase exploring consumer perspectives on this phenomenon. This consisted of an in-depth investigation of media debates surrounding three different forms of accelerated access (coverage with evidence development, cancer drugs funds and initiatives to promote access to medicines used to treat rare diseases) and semi-structured interviews with patient advocates and focus group interviews with patients. Main findings: Through this study, I have generated an-depth description of the diverse views of patients and physicians regarding accelerated access. In phase 1, we identified three “archetypes” of physicians: “confident accelerators”, “cautious accelerators” and “decelerators”. Although all acknowledged the potential risks and benefits of accelerated access, they disagreed on their magnitude and extent and how they should be balanced in both policy formation and clinical practice. In phase 2, analysis of media debates illustrated an emotive discourse, with stakeholders emphasising the importance of timely access to new therapies and barriers that current regulatory and reimbursement systems pose and little discussion of issues such as uncertainty surrounding safety and efficacy and cost-effectiveness of new therapies and the opportunity costs of funding these. However, interview participants were more cautious. While they noted a number of reasons for alternative access mechanisms, they were also cognisant of potential risks and viewed ongoing data collection and disinvestment and market withdrawal as ways to address these. Participants in both phases also identified a number of factors—such as transparent decisions made by people with relevant expertise, based on a thorough consideration of both scientific evidence and stakeholder perspectives—required to ensure appropriate decision-making processes. Overall, stakeholder views are varied, nuanced, and largely reasonable, with consideration of both benefits and risks and respect for and understanding of perspectives that differ from their own. Discussion and Conclusions: Stakeholder analysis suggests that there are a number of reasonable views on how we should respond to increasing calls for accelerated access to medicines. I draw on theories of protopianism, pragmatism and incrementalism to argue that the best way to proceed here is to focus on incremental changes to our current systems. While not perfect, these have evolved into something stable, workable and, on the whole, just. While small changes might at times be warranted, calls to drastically change our standards of evidence—as advocated by some—are unjustified and could have serious consequences. However, policy makers cannot simply ignore these calls and must respond to them. I argue that they can do this by focusing on legitimate policy and decision-making (looking at both the structure of the system and specific decisions made about particular therapies). I use theories of legitimate decision-making—such as Daniels and Sabin’s Accountability for Reasonableness, Gutmann and Thompson’s Standards for Assessing the Process of Health Policy Decision-Making and Clark and Weale’s Process Values for Healthcare Priority Setting—to identify both forces that may undermine legitimacy in this area and actions that policy makers can take to address this. I conclude with a number of practical suggestions to ensure both that the benefits of our current systems are maintained and harms to individual patients and the broader healthcare system are minimised.

There are increasing concerns about the ability of current systems to provide timely access to medicines; a number of policy approaches have been suggested to address these. Such initiatives may provide patients with a wider range of treatment options and earlier access to medicines but can also increase uncertainty surrounding safety, efficacy and cost-effectiveness. Stakeholder engagement is essential to determine the appropriate risk-benefit balance of such initiatives.

The current discourse surrounding accelerated access to medicines is an emotive one that is placing increased pressure on policy makers to both register and fund medicines even when there are significant questions about their safety, efficacy and cost-effectiveness. Policy makers need to offer alternative messages (such as community solidarity or nonexploitation) and, more importantly, alternative forms of access (such as publically-funded clinical trials) that address stakeholder concerns about current regulatory and reimbursement processes while protecting the interests of both current and future patients and the broader community.

Title A Population Study of the Epidemiology of Invasive Bloodstream Infections in Hospitalised Children in MaltaAuthors Jessica Pace, Ruth Farrugia, David PaceIntroduction: Invasive bloodstream infections are a significant cause of morbidity and mortality in children. Identification of the causative pathogen from blood cultures is crucial in determining antibiotic susceptibility and rationalising treatment. We aimed to determine the incidence and epidemiology of blood stream infections in hospitaliszed children more than 3 months old. Methodology: Results of all blood cultures taken from children aged 3 months-3 months old. Despite being recognised as a significant cause of invasive disease in children, its importance as a source of community acquired blood stream infection is increasing in contrast to the decline in the incidence of vaccine preventable invasive bacterial infections.

Objectives Indigenous older peoples’ voices and experiences remain largely absent in the dominant models and critical scholarship on aging and late life. This paper examines the relevance of the model of successful aging for Indigenous peoples in North America. Method This paper presents the results of a review of the published conceptual literature on successful aging among Indigenous peoples. Our intent was to explore the current state of the field of successful aging among Indigenous peoples and suggest dimensions that may be more reflective of Indigenous voices and experiences that leads to a more inclusive model of successful aging. Results Based on our review, we suggest four dimensions that may broaden understandings of successful aging to be more inclusive of Indigenous older people: Health and wellness, Empowerment and resilience, Engagement and behavior, and Connectedness. Discussion Our review suggests that Indigenous peoples’ voices and experiences are beginning to be included in academic literature on successful aging. However, we suggest that understandings of successful aging be broadened based on our summative findings and a process of community involvement. Such processes can lead to the development of models that are more inclusive to a wide range of older people, including Indigenous older peoples. This research was supported by an Alzheimer Society of Canada Research Program Postdoctoral Fellowship (15-23 to JP)