The national tragedy of HIV/AIDS in the hemophilia community catapulted NBDF into the spotlight as the prime advocate for a safer blood supply and blood products. While blood and blood product safety has dramatically improved over the last 20 years, the risk remains for known and unknown potential infectious agents and pathogens that threaten the blood supply and blood products. Throughout its work on blood safety, NBDF's stance has never wavered.
NBDF's commitment to blood product safety drives a strong nationwide research effort into improved treatments, including improved protein-free recombinant clotting factor products and gene therapy. Blood safety must and will always be a top NBDF priority.
In more recent years, our advocacy and monitoring have also focused on the need for all of the treatments used by people with bleeding disorders – plasma-derived, recombinant, non-factor products, as well as potential gene and novel therapies that could soon come to market.
Federal Advisory Committees
NBDF monitors the activities of several federal advisory committees, including the Department of Health and Human Services Advisory Committee on Tissue and Blood Safety and Availability and the FDA's Blood Products Advisory Committee.
MSM Donation Policies
Working with other members of the American Plasma Users Coalition (APLUS), NBDF has been actively engaged in recent debates relating to donor deferral policies, including whether men who have had sex with another man (MSM) should be permitted to donate blood. NBDF continues to advocate for donor deferral criteria based on scientific and epidemiological evidence.
The bleeding disorders community was rocked by an unexpected number of recalls and other product safety issues in 2019. These events were unsettling to some in our community, echoing tragedies of the recent past – and, at the same time, highlighting the need to articulate standards that will serve us well as we enter into a new and uncharted era of gene therapy and other novel treatments. NBDF worked with our sister organization, the Hemophilia Federation of America, to plan and host a summit to bring together diverse stakeholders from across the community – including patients, representatives of local, national and international patient organizations, providers, specialty pharmacies, pharmaceutical companies, the Patient Notification System and federal partners – to discuss and reach consensus on a set of “best practices” related to the communications around safety issues for each subset of stakeholders.
Patient-focused Drug Development
NBDF successfully advocated for hemophilia and other inherited bleeding disorders to be included on the list of conditions for which there will be Patient-Focused Drug Development meetings, as required by the Food and Drug Administration Safety and Administration Act (FDASIA).
NBDF advocated for the creation of a regulatory pathway for the approval of biosimilars in the Affordable Care Act, provided it included important patient protections surrounding interchangeability and clinical trials.