Bleeding disorders research has been growing at a fast pace over the past few years. This is great news for the bleeding disorders community, as it brings hope of enhanced diagnosis, improved clinical practices and better treatments. However, certain challenges exist, and addressing them requires global cooperation. That is why the World Federation of Hemophilia (WFH) is developing a program to facilitate international collaboration in research.
Because the bleeding disorders patient population is small, it is often difficult to enroll enough patients in a clinical trial. In countries where care is good, patients may not understand the benefit or the need to participate in research. “Too often, research studies languish due to the lack of patient recruitment by the hemophilia treatment center and the lack of patients consenting to enroll in the research study,” says Mark Skinner, WFH president.
However, at a time when governments are examining the cost-effectiveness of treatment regimens, research can help answer fundamental questions to protect access to care. “There is a need to gather evidence to support the routine hemophilia care that all clinicians practice,” says Alison Street, MD, WFH vice president, medical. Research can also help improve our understanding of the minimum and maximum dosage and duration of treatment in both on-demand care and prophylaxis.
Research is key to understanding and managing inhibitor formation, and developing treatments for rare factor deficiencies, too. The creation of new products can improve supply, making treatment available to the 75% of people with bleeding disorders around the world who do not have access.
“Advancing the necessary research to achieve the WFH’s vision of ‘treatment for all’ is a challenge that cannot be met by the efforts of one individual, organization, company or country,” Skinner says. “As patients, we play an important role in this research framework. Without our collaboration and participation, research will not advance.”
Global Research Forum
The WFH aims to foster research relevant to the global bleeding disorders community; promote multinational research, collaboration and mentorship; establish strategic partnerships; and ensure patient protection and ethical practices.
As a first step, the WFH organized the first Global Research Forum, which took place March 22–23, 2011, in Montreal. Seventy-six scientific experts, agency regulators, patient leaders, health professionals, ethicists, researchers and industry representatives attended to discuss and debate some of the critical research issues. These included:
- Patient and clinician involvement issues, including recruitment, safety and ethics, and post-trial obligations
- Clinical trial challenges, such as clinically relevant outcomes
- Agreement on definitions, such as whether disease severity should be based on bleeding, not factor level
The forum identified the most pressing challenges and unmet research needs globally. The outcomes will form the basis for the WFH’s new research agenda, which will be launched at its 2012 International Hemophilia Congress in Paris, as part of its 50th anniversary celebration.