Ensuring maximum outcomes and benefits in comprehensive care for bleeding disorders through surveillance and data collection

Abstract

The challenges of answering government and payer demands for evidence-based medicine and cost justification for the introduction and enhancement of treatment and care are ever-present and growing. To sustain and continue the expansion of access to care globally it is critical to build the body of outcome data for individual patients, within HTCs, nationally, regionally and globally. Doing so will not only improve clinical practices and support the allocation of scare resources, but most importantly, the well-being of patients will improve as well.

The World Federation of Hemophilia Annual Global Survey 1999‐2018

“…The AGS allows benchmarking and tracks achievement in some settings encouraging other countries to attempt the same. 22 Finally, the AGS continues to show the large inequity in the distribution of resources for treating haemophilia across the globe.…”

“…Continued research is needed to improve the care standards and to successfully detect and diagnose the rare bleeding disorders. Although plenty of data exist on the number of identified patients, expected number of patients, survival into adulthood and amount of available treatment; baseline data to compare outcomes and quality of life between national patient groups, countries or treatment regimens over time are limited 22 . In addition to further development of the AGS, and the WBDR, the WFH is committed to setting up a Gene Therapy Registry as well as a tool for patient‐reported outcomes, which can in turn act as companion tools alongside the AGS, allowing for capturing a more comprehensive and complete picture of the impact of treatment and care.…”

“…Therefore, to obtain a more representative sample of information on the global haemophilia population, the WFH Development Model was extended to include a sixth area of focus-tracking national demographic data. 21,22 The additional focus area of the WFH Development Model empowered WFH and NMOs to more effectively mitigate the resistance from government bodies to allocate their scarce resources towards rare bleeding diseases by providing objective data to show the gap in haemophilia care. Also, the collection of the same global data over time enabled the WFH to track advances in care that have resulted, at least in part, from WFH programmes activities.…”

“…Unfortunately, nearly all existing data had been collected from high rather than low‐income countries. Therefore, to obtain a more representative sample of information on the global haemophilia population, the WFH Development Model was extended to include a sixth area of focus—tracking national demographic data 21,22 …”