Health Systems Sustainability and Rare Diseases

Abstract

The paper is addressing aspects of health system sustainability for rare diseases in relation to the current economic crisis and equity concerns. It takes into account the results of the narrative review carried out in the framework of the Joint Action for Rare Diseases (Joint RD-Action) "Promoting Implementation of Recommendations on Policy, Information and Data for Rare Diseases", that identified networks as key factors for health systems sustainability for rare diseases. The legal framework of European Reference Networks and their added value is also presented. Networks play a relevant role for health systems sustainability, since they are based upon, pay special attention to and can intervene on health systems knowledge development, partnership, organizational structure, resources, leadership and governance. Moreover, sustainability of health systems can not be separated from the analysis of the context and the action on it, including fiscal equity. As a result of the financial crisis of 2008, cuts of public health-care budgets jeopardized health equity, since the least wealthy suffered from the greatest health effects. Moreover, austerity policies affected economic growth much more adversely than previously believed. Therefore, reducing public health expenditure not only is going to jeopardise citizens' health, but also to hamper fair and sustainable development.

How can the healthcare system deliver sustainable performance? A scoping review

“…The importance of political stability and bridging the jurisdictional-federal divide in federated healthcare systems (such as in the USA, Canada and Australia) was important for effective unified healthcare system functioning 24 69 70 104–106. It is not only organisational culture in healthcare,107 but the broader organisation, governance and regulation of the healthcare system that are important for SPHS 108–110…”

“…A recurring sentiment in the articles reviewed was the importance of support for the continuation of interventions from leaders and stakeholders 57 98 103 107 118 119. Leaders and managers have a clear role in supporting staff throughout the processes of reforms and changes, by providing opportunities for codesign, education including e-learning, and building peer networks89 120 while creating open communication to involve front-line staff in planning and implementation 103 121.…”

Challenges Affecting Access to Health and Social Care Resources and Time Management among Parents of Children with Rett Syndrome: A Qualitative Case Study

“…The distribution of health expenditure varies among countries, dependent on factors such as the burden of diseases and the priorities of each health system [15,16]. In the case of rare diseases, due to their low prevalence, high variability and numerous subtypes, a more global and comprehensive approach is required on behalf of various public and private organizations aimed at preventing significant morbidity and premature mortality, and improving the quality of life and socioeconomic resources of affected people [17][18][19].…”