In my VP Medical Column in the April 2016 issue of Hemophilia World, I announced that the World Federation of Hemophilia (WFH) would launch a World Bleeding Disorders Registry (WBDR) pilot program.
This program has now begun and the WBDR is currently being piloted in 31 countries around the world to assess the feasibility of data collection in centres at all levels of development. With patient enrolment almost complete, the pilot project will finish by the end of 2016. The results of the pilot project to date are very promising and will be presented in poster format at the European Association for Haemophilia and Allied Disorders (EAHAD) Congress in February 2017. If the pilot is successful, the full scale WBDR—to be conducted in more than 50 countries—will be implemented in 2017.
The WBDR will generate real world data on more than 15,000 patients, from regions around the world, in the context of routine clinical care. It will be an invaluable resource for researchers to use, for both clinical research and advocacy purposes.
The WBDR is an important endeavour because despite great advances made in hemophilia care in the past 50 years, care is still suboptimal in many instances. Patients around the world continue to suffer from increased morbidity and mortality due to bleeding into joints, muscles, the brain and other sites.
The advancement of evidence-based care of hemophilia and other rare bleeding disorders is limited by factors inherent to research in rare diseases: small samples sizes, geographical dispersion of patients and heterogeneity in the clinical course observed in patients. Combined, these factors diminish a study’s statistical power, making the generation of high quality evidence in clinical and treatment outcomes in rare diseases challenging. This has given rise to a call for global rare-disease registries.
Registries—with international collaboration between centres and countries—are are an effective way to pool enough data in order to achieve a sufficient sample size to enable epidemiological and clinical research for rare disorders. The data generated from registries allows for the analysis of patient outcomes in the context of routine clinical care and real world setting. These data have the potential to improve our current quality of care, examine the patient experience longitudinally, assess the effectiveness of medical treatments and identify significant unmet needs. Increasingly, regulatory bodies around the globe are relying on real world data, including patient registry data, to inform their regulatory and reimbursement decisions, health technology assessments, and treatment guidelines.
The WBDR is the vehicle the WFH is using to respond to a call for a global patient registry to enhance data and strengthen evidence in bleeding disorders. It is intended to fill the gap in evidence by collecting an unprecedented amount of real world data, and it will be tremendously useful for generating evidence to improve the quality of care for patients with bleeding disorders worldwide.
To find out more about the WBDR, please visit www.bleedingdisordersregistry.org.