WFH Global Forum: Securing the safety and efficacy of products

This year the World Federation of Hemophilia (WFH) will organize the 9th Global Forum on Research and Treatment Products for Bleeding Disorders on October 22-23, in Montreal.

The WFH is uniquely positioned to bring together government regulators, physicians, researchers, pharmaceutical firm representatives, and people with bleeding disorders to discuss challenges in the areas of translational research and research methodology and issues relating to the global supply of treatment products for bleeding disorders and the safety and efficacy of current and new clotting products.

Although much progress has been made to ensure the safety of products, supply remains a major problem — how can we improve access to care and treatment products for those in need?  From the data that the WFH is collecting in the Annual Global Survey, the majority of patients do not have access to sufficient amounts of treatment products and it is a challenge to close this gap. In the developed world the medical community has only recently accepted that the preferential treatment for all patients with severe hemophilia is primary prophylaxis.

Although the evidence for prophylaxis is no longer debated, the high cost of lifelong therapy is still an important issue.  Only a few countries have a healthcare system that reimburses all treatment costs thereby making the decision for prophylaxis possible for physicians and families. Even though many new products are in the pipeline and soon will be registered in the major markets, they will remain inaccessible to many patients. Most countries will still have little or no access to any clotting products, which reduces the incentive to diagnose new patients and hinders advocacy for the patients in need.

Another important issue that will be addressed during the Global Forum meeting is the safety and efficacy of products. Hemophilia is a rare disease and therefore new products are marketed after clinical trials that have included only a few patients globally. Although the data are carefully evaluated, the results only give some information on dosing and efficacy of new products. Immunogenicity is still a large problem; 25-30 per cent of patients infused with factor VIII develop inhibitory allo-antibodies during the first 50 exposure days.  Much research has focused on the perceived different inhibitor risk between plasma and recombinant products. The general opinion is that neo-immunogenicity can preferably be studied in adults with more than 150 exposure days with factor VIII. Accepting the fact that all products inherently have a high risk for inhibitors, do we want to prolong this attitude or can new products be developedwith a lower risk?

The WFH bares a responsibility to ensure that all knowledge of rare bleeding disorders is improved globally. In 2011, the Global Research Forum was introduced with the aim to build better evidence to improve the management of bleeding disorders. Collection of clinical data concerning treatment and joint outcome is crucial to building knowledge and to supporting advocacy. Only well-collected clinical data of large groups of patients will improve our understanding and help us ask the right research questions for translational research. Clinical studies need to be well defined, patient data has to be protected, and research methodology must carefully considered to ensure that treatment outcomes are assessed with realistic endpoints.

The WFH recognized that combining the Global Forum on Safety and Supply of Treatment Products and the Global Research Forum involves all stakeholders involved in the safety and marketing of new products, interested in access to products, and invested in the collection of real-life data on safety and efficacy after marketing authorization.This is an opportunity to survey both the current and future state of global bleeding disorders diagnosis and care. With new categories of products becoming available, along with ongoing developments in gene therapy and other clinical research, there is a natural convergence of research, safety, and supply topics. This newly combined meeting is now open to all interested parties and we hope to welcome many of you to Montreal in October.