Generating evidence to support clinical management and ensure access

We have entered an era of unprecedented growth in treatment options for hemophilia. A number of new treatments have, or will soon, enter the market, changing the way we manage the disease—and potentially even cure it.

Especially for hemophilia B, the promise is tremendous. While these developments are bringing new hope to patients and healthcare professionals, the lack of strong evidence for current treatment protocols continues to threaten access to care in all parts of the world. Even in the developed world, authorities demand evidence-based support for the outcome of treatment regimens. With its Research Program, the World Federation of Hemophilia (WFH) is working to provide the community with the tools it needs to meet this challenge.

The WFH Research Program was established in 2013 to support and facilitate clinical research in inherited bleeding disorders. While there are a many questions that still need to be addressed, the WFH’s Research Committee has identified a number of priority areas they believe would have the greatest impact on improving patient care.

WFH Research Priorities

  • Cost-effectiveness of different prophylaxis schedules
  • Inhibitor diagnosis and management
  • Optimal use of adjunctive therapies
  • Care of the neonate with hemophilia
  • Management of chronic hemophilic arthropathy
  • Care of hemophilia carriers
  • Pain management
  • Role of physical activity in hemophilia care
  • Management of special bleeds (eg, iliopsoas, renal)
  • Role of arthrocentesis in hemophilia care

One of the ways the WFH helps the community answer these important questions is by funding innovative clinical research. Since 2014, the organization has distributed US$ 400,000 to support ten research projects. To learn more about these projects and the WFH Clinical Research Grant Program, visit the WFH website.

Another way we can support research is to facilitate epidemiological data collection. The WFH has been collecting and publishing national data on people with inherited bleeding disorders for over a decade through the WFH Annual Global Survey. While this information is very useful for advocacy and program planning, it doesn’t allow us to draw any real conclusions about how best to manage hemophilia. Through the WFH Epidemiological Research Program, we aim to collect data on individual patients and how they are treated in centres around the world.

A number of national, regional, and international registries have already been established for the purpose of monitoring specific aspects of hemophilia care. As a global organization with access to a vast network of hemophilia treatment centres (HTCs), the WFH is uniquely positioned to do this on a global scale, while keeping the rights and interests of patients at the forefront. The World Bleeding Disorder Registry will provide an unprecedented amount of data, which will be tremendously useful for generating evidence to establish standards of care and demonstrate the value of existing practices.

This is not an easy endeavor. It will require a significant investment in infrastructure to ensure that patient information is safe and secure, treaters from around the world to invest time and energy in collecting and recording data, and to pool that data into the registry. It will involve researchers and scientists working together to share the data that is already being collected in registries around the world and have patients’ consent to participate.

The WFH is committed and well-placed to overcome these barriers. In fact, we have already made significant progress. We have just published a comprehensive data collection questionnaire, the Universal Case Report Form (U-CRF), which outlines all the data that would ideally be collected in the clinic, at diagnosis and at each follow-up visit. A number of centres are already collecting this data, or developing systems to do so. The advantage of the WFH U-CRF is that the fields have been selected based on published recommendations and definitions, by consensus of internationally recognized experts.

A pilot project, using a minimal amount of data from the U-CRF in a limited number of centres will be initiated shortly. Participating centres, are selected based upon their interest, willingness, and capability to collect the data. This project will allow us to assess the feasibility of data collection in centres of all levels of development, evaluate the requirements for data validation and management as well as training/education of HTC staff. Following successful completion of the pilot project, it is our intention to expand the program gradually, both in terms of the amount of data that is collected and the number of centres in which it is collected. The first results will be presented at the WFH 2016 World Congress in Orlando.

The global bleeding disorders community must come together to generate the evidence we need to support the clinical management of patients and to ensure access to those who still do not have treatment. We are counting on patients and healthcare professionals around the world to make this vision a reality, and on our partners to continue to support our efforts.