To advance global knowledge on diagnosis, clinical course, and treatment of bleeding disorders, data are needed that capture the entire patient experience from around the globe. Clinical trials in rare diseases are challenged with small sample sizes, geographical dispersion of patients and short follow up times, limiting the collection of reliable treatment and outcome data.
Patient registries make it possible to collect longitudinal, real world data on large cohorts of patients, allowing researchers to address important research questions. Real world data—or data collected on patients outside of the traditional clinical trial—offer the benefit of providing a picture of the patient experience under routine clinical practice. In some countries, real world data are the only data possible to obtain. In other countries, real world data can be used to supplement clinical trial data. These data are a valuable addition to the arsenal of strategies we need to advance the quality of clinical care and advocacy measures for patients with bleeding disorders.
Currently, the WFH collects aggregate data provided by our national member organizations (NMO) which is published annually in the WFH Annual Global Survey. These data give a global overview of the number of patients with bleeding disorders identified and a snapshot of the level of access to care around the world.
To meet the challenge of increasing the amount and type of data available on patients with bleeding disorders, the WFH is developing the World Bleeding Disorders Registry (WBDR). The WBDR will collect individual patient level data through Hemophilia Treatment Centers (HTC). Uniform data on demographics, diagnosis, clinical, and treatment outcomes on patients will be collected. This registry is intended to collect real world data on the patient clinical experience around the globe. It will provide researchers with the capacity to compare the patient experience from country to country and within countries.
With reach to a network of over 1,000 HTCs and 134 WFH NMOs, and with access to patients in countries with varying levels of care and access to care, the WFH is uniquely positioned to effectively conduct a global registry. Currently a pilot project, the full WBDR is expected to launch in 2017. The aim of the pilot project is to assess the feasibility of conducting a patient registry around the globe. It will assess the interest of HTCs and patients in participating, the ability of HTCs to obtain regulatory approval, and the ability to successfully add their data to a web-based data system. Forty HTCs were invited to participate in the pilot project and 31 have agreed to do so—a response rate of 78%. The countries involved in the pilot project are scattered around the globe, and include: Algeria, Argentina, Australia, Belgium, Brazil, Cameroon, Canada, Egypt, Estonia, Ethiopia, India, Indonesia, Jamaica, Kenya, Kyrgyzstan, Mali, Morocco, Philippines, Senegal, Slovenia, Sri Lanka, Switzerland, Thailand, Uganda, the U.K., U.S.A., Uzbekistan, Venezuela, Vietnam, and Zimbabwe. The pilot project is expected to be completed by the end of 2016.
Chaired by Glenn Pierce, MD, PhD, the WFH Epidemiological Steering Committee will evaluate the results of the pilot project and determine the feasibility of continuing with the full scale WBDR in 2017. The Epidemiological Steering Committee is composed of hematologists, patients, researchers, and methodologists who represent Canada, India, Senegal, the Netherlands, the U.K., and U.S.A.
“There’s a very human element to the WBDR that can’t be overstated,” explains Glenn. “The patient registry is about bringing patients together from around the world in a cooperative fashion. It’s about all of us working as a team to further knowledge in the bleeding disorders community.”
If the pilot is successful, HTCs from all levels of economic development will be invited to join the WBDR starting in mid- 2017, and patient recruitment will begin at HTCs upon ethics approval. Over the first 5-year period, from 2017 to 2022, the WBDR seeks to include HTCs from more than 50 countries, representing over 20% of all HTCs worldwide, and over 15,000 individuals with bleeding disorders.
The value of a large patient registry, such as the WBDR, lies in the utilization of the data collected. Patients who participate in the WBDR will have the opportunity to contribute to the shaping of care around the world. As the data accumulates, it will increasingly reflect the patient experience and the state of care of bleeding disorders around the world. This will be an invaluable resource for research and advocacy purposes. Anonymized data will be available to participating researchers with secure data protection to permit researchers to carry out studies on a larger number of patients than they would have access to outside the model of patient registry. Individual countries will be able to use country-specific data and country-comparative data to advocate for better care.
In the current landscape of many regional, national, and international patient registries, the harmonization of datasets is essential to compare, combine, and make the best use of our data. Despite varying objectives, many of the ongoing patient registries have common core data elements. The task of harmonizing core data elements and linking databases will require collaborative initiatives between governing bodies of existing patient registries. It will require forward thinking on how to best align data collection efforts with existing patient registries. The WFH is taking advantage of the current development period of the WBDR to align our core data set with the larger, existing patient registries around the globe in order to make future linkage easier to implement.
Combining data from the smaller and less developed countries with those of larger and more developed countries will help complete the global picture of people with bleeding disorders. Joined together, data from around the world will provide an unprecedented amount of real world data and a strong and robust platform upon which to advance patient care around the globe. Ultimately, researchers, clinicians—and most of all, patients—will benefit from the World Bleeding Disorders Registry.