Gene Therapy is Here, Now What?
by Joe Pugliese, President and CEO

Since its inception in 1999, the Hemophilia Alliance has seen significant growth in the research and development of novel therapies for the treatment of patients with hemophilia, von Willebrand disease, and other related bleeding disorders. The first infusion of an FDA approved gene therapy product, done at a federally funded hemophilia treatment center of excellence, outside of a clinical trial, is a milestone towards finding an eventual cure for these patients. We would like to congratulate our member center Hemophilia Outreach Center, Green Bay, WI, for their successful infusion (see article under Community News) and our manufacturing partners for their commitment to finding durable therapies for the patients our member centers support.

On the heels of this historic infusion and after a long review process, BioMarin received approval for Roctavian, their gene therapy product for Factor VIII deficient patients. The week before the FDA accepted Pfizer’s application for their factor IX gene therapy product.

Since the introduction of monoclonal purified products in the late 1980’s, our patients have been the beneficiaries of a seemingly endless stream of new and innovative products. These products have miraculously changed the lives of people with hemophilia. A survivor of the AIDs crisis in our community summarized the change very succinctly, “If I had known I was going to live this long I would have planned better.” This is a message that all individuals living with bleeding disorders should follow as they can look forward to potentially having heart disease, high cholesterol, and a plethora of other diseases you generally only experience by living into your 60s, 70s and beyond.

With the introduction of gene therapy, it has never been more apparent that the key to bringing innovative therapies to market and have them successfully brought to the community is through the federally supported United States Hemophilia Treatment Center Network (USHTCN). The centers are uniquely positioned to evaluate, counsel, administer, and assure the patient is correctly managed in the critical 6–12-month window post-infusion.

With the development of new and improved products, the advances in diagnosis, and a better understanding of the nuances of being a carrier or a mild to moderate patient, the demand for services is continuing to expand at the federally supported hemophilia treatment centers. The Alliance hears from the organizations representing patients in these various groups that they seek greater access to specialized care.

The most expensive patients are always the patients who are mismanaged and often are also the patients with the lowest quality of life and the highest mortality and morbidity. It is critically important that all HTCs in the USHTCN are included in health plan networks for both clinical and pharmacy services. It is the key to ensuring the best patient outcomes at the lowest possible price.