Although

not discussed in this paper, there are other significant research challenges to be addressed if we are to optimize and advance Treatment for All particularly for those living with inhibitors or a rarer factor deficiency. Notably these include:  Improving treatment options for patients with rarer factor deficiencies, such as factor II, factor V, factor VII, factor V+VIII, factor X, factor XI and factor XIII. Health outcomes research – analysis, surveillance and data collection.  Over the past 50 years we have witnessed major improvements to the safety and efficacy of treatment for inherited bleeding disorders, and a greater understanding of their management. The challenge of answering government and payer demands for evidence-based medicine and cost Selleckchem Barasertib justification for the advances, or for the introduction and further enhancement of treatment are ever-present and growing. As a global CAL-101 purchase community we must recognize the challenge and build capacity to produce the necessary outcomes-based data to defend and support continued treatment advances. The need to have a surveillance system becomes more and more important as ministries of health want to know the expected outcomes of investing resources into haemophilia [30]. Even with the improvements of recent decades, further enhancement of the therapeutic options

are needed to improve access to treatments, enhance convenience, elevate quality of life and mitigate treatment complications such as inhibitor development. In order to achieve these goals, detailed and objective clinical outcomes data must be available from

large patient populations to serve as the critical comparator for evaluating the influence of new interventions. Beginning in 2013, the WFH Development 上海皓元 Model will be expanded to include a sixth element – the ability to track and report patient health outcomes through surveillance, enhanced data collection and outcomes analysis. In doing so, the WFH will build on its current data collection and analysis expertise from the WFH Global Survey [7]. Today, the WFH Global Survey, begun in 1998, reports national, regional, global and longitudinal trend data from over 100 countries collected through an annual survey of patient registries for a set of targeted demographic and treatment metrics. Quantifiable data contained in national registries and the WFH Global Survey facilitates the measurement of the effectiveness of healthcare programs. The WFH will continue to promote the establishment of national registries through collaboration between national patient organizations, healthcare professionals, HTCs and ministries of health [29,49]. This new WFH Development Model element will also support a new WFH research initiative to provide access and support of clinical outcomes collation and analysis infrastructures.