Global Forum of the World Federation of Hemophilia, September 26–27, 2005, Montreal, Quebec, Canada

Abstract

Overview of the varying needs of people with hemophilia around the worldMarkSkinnerMr.PresidentWorld Federation of HemophiliaThe World Federation of Hemophilia (WFH) is making tremendous progress towards determining the prevalence of bleeding disorders, said Mr. Mark Skinner. Diagnoses of patients with hemophilia have increased by 5% globally, and Global Alliance for Progress countries are now diagnosing five new patients daily. Once patients are diagnosed, it is essential to find safe, efficacious, and affordable products with which to treat them. “However, the reality today is that economic capacity drives the availability of concentrates, and 75% of people with hemophilia lack treatment,” Mr. Skinner said.Mr. Skinner reported that, in March 2005, the World Health Organization (WHO) reaffirmed clotting factor concentrates as essential for the treatment of bleeding disorders and as a health care priority for governments around the world. Meeting the worldwide need for safe and affordable factor replacement therapy cannot be accomplished by the WFH alone. It requires a “winning coalition” of patients and their families, health care professionals, governments and health ministries, regulators, and industry, Mr. Skinner said. The WFH builds core expertise in areas such as laboratory diagnosis, data collection and analysis, development of cases for support, and government purchase, tender, or reimbursement. The challenge is whether affordable treatment will follow a diagnosis of hemophilia.Increasing the worldwide supply requires a multivariate approach that involves managing existing resources, using all treatment options available, reducing manufacturing costs, developing new markets, and structuring prices innovatively so as to make products more affordable for some around the world, and providing humanitarian aid.The WFH recently published a monograph with guidelines on treatment, which outlines optimal dosing and describes plasma-derived and recombinant products alike as important treatment options for hemophilia and other bleeding disorders. Both product types have a robust safety record, and the supply of either alone is insufficient to meet the global needs, he emphasized. Moreover, “economic capacity dictates what a country can purchase, so it is important that there is a range of products and prices.” The supply shortages of recombinant products several years ago and disruptions from viral infections before that demonstrate that it is important to have a system that is built on flexibility and that relies on both plasma-derived and recombinant products.In its March 2005 report on essential medicines, WHO cited some safety limitations with regard to cryoprecipitate; however, the reality is that much of the world still uses it, Mr. Skinner said. At the same time, the WFH recognizes that existing therapies have their limitations. “Product innovation is an important part of meeting the global supply.”The WFH is working on a number of initiatives:•Very rare bleeding disorders: Because the population of patients who have FV, FVII, FXI, and FXIII deficiency and do not have hemophilia or von Willebrand disease (vWD) is so small (10,500 patients have been identified), running clinical trials is difficult. This situation makes it very difficult to meet the challenges of bringing products to the market.•Unused plasma proteins: As countries have made the transition to recombinant products, some cryoprecipitate unused for the production of FVIII and FIX concentrates has gone to waste. Because of the vision of executive committee member Mr. David Page, the WFH has been working to bring partners together to stimulate the development and use of this product to benefit others around the world. A pilot project in Canada is working on developing affordable clotting factor concentrates that might be used to support humanitarian aid and access needs in emerging countries.•Regulatory harmonization: The influence of key regulatory bodies—specifically, the US Food and Drug Administration (FDA) and the European Medicines Agency (EMEA)—is paramount. These agencies drive much of what happens around the world and by specifying differences that do not make sense from a safety or efficacy perspective, they can negatively influence access, cost, and portability of products between countries.•Twin-track pricing: Following innovative WHO pricing models related to access to therapies and vaccines for the human immunodeficiency virus (HIV), the WFH is exploring the possibility pricing systems that would allow products to be more affordably sold to meet needs in emerging countries—for example, by offloading investment and development costs.•Von Willebrand disease: The WFH is incorporating vWD into its new strategic plan, with the goal of improving diagnosis and access to treatment options for the estimated 43,000 people with vWD around the world.Mr. Skinner encouraged participants in the Global Forum to have a candid exchange of perspectives so that they can together find solutions for affordable supply and stimulate the development of products and the pursuit of new avenues for treatment.