 Key clinicians and patient leaders have been appointed to a research committee, which was established after discussion of the Strategic Plan extension at a World Federation of Hemophilia (WFH) Executive Committee meeting in 2009. Research had been identified in the WFH’s Strategic Plan as an area where the WFH has a role to play in improving treatment and access to care for people with bleeding disorders. Dr. David Lillicrap, from Kingston, Canada, chairs the group, whose mandate is to explore the role the WFH could play in advancing research to achieve this vision. In March 2011, the WFH organized its first Global Research Forum, sponsored by Baxter, Bayer, Biogen Idec Hemophilia, CSL Behring, Grifols, and Novo Nordisk, to define and debate the most pressing research questions that affect the bleeding disorders community, with the ultimate goal of identifying how the WFH can help foster research in these areas to improve treatment and care.
The Global Research Forum represented the first time that all “players” were involved in such discussions: researchers and research organizations, clinicians and healthcare providers, patients, regulators, and manufacturers of treatment products. It was also the first time that there was such widespread participation in in-depth discussions of key challenges to research, such as patient engagement and protection and standardization issues when defining a bleed, an inhibitor, or a clinical end-point. Dr. Alok Srivastava summarized the essence of the meeting when he said, “I have never sat in a room and heard so many people discussing so many issues critical to people with hemophilia.”
Global Research Forum participants fully supported the WFH in its aims to foster research relevant to the global bleeding disorders community. The WFH is uniquely placed to promote multinational research collaboration and mentorship, and to work in close collaboration with research organizations such as the International Society on Thrombosis and Haemostasis (ISTH), the International Society of Blood Transfusion (ISBT), the European Association for Haemophilia and Allied Disorders (EAHAD), the Hemostasis and Thrombosis Research Society (HTRS), and others. As a patient organization, the WFH has supreme responsibility in its role to ensure ethical practice and protect patients, and to encourage and foster patient participation in clinical research.
Many patients and treaters equate research with replacement product trials, but research is much broader than this. We must expand our definition and practice of research to include:
- Systematic clinical observation, data collection, and analysis to document treatment outcomes
- Study of non-product treatments
- Multicentre, multinational participation
- Collaboration to enhance education and opportunities for research for patients and clinicians
As governments are increasingly seeking to justify spending on health programs, the importance of supportive data (evidence-based medicine), not only to advance care, but also to sustain and protect it, increases. Evidence-based medicine may be defined as the integration of the best research evidence with clinical expertise and patient values. Thus performance and presentation of research data play a key role in answering government/payer demands for the evidence which supports treatment practices and justifies costs of treatment and care.
Research is not only driven by product trials—its practice is part of every clinical encounter between a clinician and patient. It needs recognition as the driver for improvement through relevant and systematic observation and data collection, which is essential for analysis and accountability.
There are currently many challenges and barriers for clinicians to participate in bleeding disorders research. Before the Global Research Forum, the WFH distributed a simple survey to hemophilia treatment centres (HTCs) globally and received 113 responses. Some of the challenges identified included lack of interest or awareness of research activities, insufficient training, time, or human and financial resources within centres, and lack of understanding and support for research from hospital administrators and funders outside of centres.
Global Research Forum participants were asked if the WFH should promote education, mentorship, and clinical training programs in the prosecution of good clinical practice as well as research programs to document treatment outcomes. An overwhelming majority (more than 90 per cent) of respondents agreed. When asked what areas should be promoted that target the strengths of the WFH, international studies with participation from both developing and developed countries, and clinical outcome evaluation received the most support. Similarly, when asked what activities should be incorporated, clinical outcome studies and health economic analyses ranked the highest. When asked for potential research objectives, identification of international research priorities, promotion and education to support patient and clinician participation within an ethical framework, and enhanced data collection and outcome analysis were seen as the highest priorities.
The next step, which will be managed by the now formalized Research Committee, is to recommend to the WFH Executive Committee a research framework based on the discussions that took place. Essential components of this will be the development of predominantly clinical research programs, securing international collaboration with other research organizations, and the promotion and training of clinicians in good clinical practice.
Alison Street
VP Medical
Hemophilia World, August 2011
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