The bleeding disorders community is in a unique period of drug development. Almost every pharmaceutical company currently producing factor products is developing new products that are being examined in humans in clinical studies. The advances of these products range from increased safety to longer half-lives and different formulations. All are being developed to improve the lives of people with bleeding disorders. (See “Treatment Breakthroughs.)
The National Hemophilia Foundation (NHF) has been supporting novel ideas in research since 1972. Through the Judith Graham Pool Postdoctoral Fellowship, NHF has funded close to 80 researchers in basic science to understand the genetics of hemophilia and develop better testing.
Since 2000, NHF has provided Career Development Awards to more experienced researchers, many of whom have become directors at hemophilia treatment centers (HTCs) and established their own laboratories to search for a cure for bleeding disorders.
The NHF/Baxter Clinical Fellowship has been awarding fellowships to physicians since 2003. This program provides academic hospitals the funding and time to develop the future of clinical physicians who specialize in bleeding disorders treatment. To provide the continued expert care patients receive from their hematologists, NHF has graduated 17 physicians from the program who have continued to work in the field of bleeding disorders.
NHF also supports drug advancements through its Medical and Scientific Advisory Council (MASAC). MASAC held a Consensus Conference on the Impediments to Clinical Research in November 2010 to discuss how to make sure new products are made available to consumers.
A questionnaire was also distributed to patients at NHF’s Washington Days in February 2011 about their involvement in a clinical research study. About half the patients had not been involved in clinical trials because none were offered at their HTC. The other half had participated in a study and took pride in having helped develop better treatments.
In 2012, NHF will publish an article in a peer-reviewed professional journal on the discussion that took place at the Consensus Conference. This will allow all members of the bleeding disorders community to provide input that ensures that more advanced products can make it to market and improve the lives of people with bleeding disorders.
Neil Frick, MS, is the vice president for research and medical information at the National Hemophilia Foundation.