Advocating for better outcomes—using data to build the evidence base

The first day of the World Federation of Hemophilia (WFH) 2018 World Congress in Glasgow opened with a session on the importance of data and evidence, led by the Chair of the Data & Demographics Committee, Dr. Alfonso Iorio.

Jamie O’Hara of The Haemophilia Society, U.K. started with a beginner’s guide to evidence in the context of hemophilia. Clinical trials generally provide the strongest evidence, but “we need to be more holistic when considering hemophilia,” said O’Hara. “It is such a rare disease that often we do not get statistically significant trial results.” Good examples of evidence-based advocacy, such as the Patient Reported Outcomes, Burdens, and Experiences (PROBE) study illustrate how patients’ perspectives and experience contribute to defining and measuring key health outcomes.

Regional experiences of successful advocacy programs were presented. Both India and Nigeria had originally struggled to reach their hemophilia population. “India was not successful in its national hemophilia data registration until 2010 because they did not have the numbers,” said Vikash Goyel, President of the Hemophilia Federation of India. Initiatives such as forming state-wide coordinated hemophilia chapters and youth and women groups have boosted national registration.

A similar situation was seen in Nigeria. Theresa Nwagha, MD, Consultant Hematologist at the University of Nigeria said, “Prior to 2005, there was no central register for bleeding disorders in Nigeria. Policy makers did not understand the problem.” In 2016, the Hemophilia Federation Nigeria established a goal to engage the Federal Ministry of Health with a step-by-step approach to implement a national program. Following a stakeholder meeting in 2017, the number of registered people with hemophilia across Nigeria has increased from 50 to 347, and three Nigerian states have now appointed hemophilia desk officers.

Evidence-based data has been key to overcoming the crisis facing China where an estimated 30,000 young boys with hemophilia were only receiving on-demand treatment for joint bleeds due to decades of cost constraints. By leveraging outcome data from 11 clinical studies, China demonstrated the treatment benefits and cost-effectiveness of low-dose prophylaxis.

Glenn Pierce, MD, PhD, WFH Medical board member highlighted the gaps in current evidence and the considerable unmet needs in hemophilia treatment. “In the U.S.A, the burden of the disease is high—it is expensive and non-adherence is common”. Furthermore, there are several undesirable and not fully understood complications, such as pseudotumours. There is a clear need for one standardized data collection system to explore these issues, to make comparisons between countries and treatment centres, and to understand the natural history of complications.

Updates were shared on the accomplishments of the WFH Annual Global Survey (AGS) and the WFH World Bleeding Disorders Registry (WBDR). The AGS 2016 survey, discussed by Christine Herr, WFH Data and Research Manager, represents data collected from 113 countries and 184,723 people with hemophilia. Donna Coffin, WFH Research & Public Policy Director, discussed the global implementation strategy of the WBDR. The minimal data set opened in January 2018, and there are plans to release a patient app later this year. The five-year enrollment goals are expected to meet the target number of intended countries.

The session concluded with an interactive exercise facilitated by Alfonso Iorio, MD. Through a practical example of low-dose prophylaxis, Dr. Iorio synthesized the messages of the session speakers and demonstrated how to use data to support advocacy efforts.

Mitch Semienchuk, Editor, Hemophilia World Online, wishes to thank Georghia Michael. PhD, for her contributions to this article.