The World Federation of Hemophilia (WFH) held its first Global Research Forum on March 22–23, 2011, in Montreal, Canada. Physicians and researchers from around the world gathered to discuss how WFH could incorporate bleeding disorders research into its current activities.
The vision of WFH is “Treatment for All.” To achieve this, WFH needs to understand where it could be most helpful in ensuring the future development of treatment for all bleeding disorders. The forum included discussion on clinical trial challenges, patient and clinician involvement, definition issues and WFH research objectives.
In the clinical trial challenges section, Dr. Alok Srivastava from India discussed the clinically relevant endpoints in treatment, particularly as they relate to diagnosis, prophylaxis, immune tolerance induction and individualized treatment. Dr. Anthony Hubbard, from the United Kingdom, presented on the new extended half-life clotting factor concentrates and how their potencies may have to be measured differently than existing products. Dr. Flora Peyvandi from Italy discussed the difficulties of conducting clinical research in rare bleeding disorders with the limited number of patients, many who often have chronic progressive life-threatening conditions with unmet medical needs.
WFH Conducts Survey About Hemophilia Research Worldwide
In the patient and clinical discussion, Dr. Johnny Mahlangu from Johannesburg, South Africa, presented the challenges of conducting international clinical trials, such as ethical guidelines, cultural norms, social factors, linguistic challenges and economic status. Dr. Alison Street from Australia addressed missed recruitment opportunities. She presented results of a WFH survey of 113 hemophilia treatment centers (HTCs) from around the world that showed a lack of awareness of the definition and methods of research, and a lack of training, time, resources and support within and outside the treatment centers.
WFH President Mark Skinner presented the patient perspective of the WFH research survey. The responses showed that people outside North America and Western Europe are not offered an opportunity to participate in clinical research. However, the majority believed it should be a part of treatment at their HTC. There is a great need for patient participation in research because study results are essential to help governments make decisions regarding treatment.
The final group of speakers addressed definitions of hemophilia and bleeding disorders terms. Dr. Nigel Key, from the US, discussed the definitions regarding severity of hemophilia and the imperfect correlation between phenotype and factor levels: how patients’ severity, based on their factor level, relates to their actual bleeding experience. Dr. Victor Blanchette from Canada described how a target joint is currently defined based on the number of recurring bleeds. He also discussed definitions of prophylaxis treatment, specifically the terms “primary and secondary prophylaxis.” Dr. Manuel D. Carcao of Canada discussed the difficulty of determining “responsive” vs. “nonresponsive” once treatment is administered.
The WFH Research Committee now plans to begin discussions regarding collaborative research partnerships, with a focus on clinical research. Forum attendees agreed there is a need for good clinical research training. Thanks to the information collected at the forum, the committee can now address how WFH can best assist in research efforts around the world.