PPTA Hosts Congressional Staff Briefing
PPTA collaborated with the patient community March 31 to host a Congressional staff briefing that focused on the uniqueness of plasma protein therapies; how they are made; the rare, chronic diseases they treat; and the importance of access.
Presentations were made by patients including Lisa Miller, who has primary immune deficiency; Michelle Rice, who has two sons with hemophilia and works with the National Hemophilia Foundation NHF); and John Walsh, who has alpha-1 and also is the founder, president and CEO of the Alpha 1 Foundation. All of the patient presenters described their disease (or their children's disease), how important access to therapies is to their health and to their community and recognized manufacturers and plasma donors for their contributions to creating lifesaving therapies. Patient representatives were able to speak first hand about how came to learn about how a certain therapy works best for them based on their specific biology and that plasma protein therapies are not one-size-fits all products. The complexities of coping with a rare, chronic disease that requires regular infusion of a biological therapy also was described and how it is like another job to manage the disease.
Dr. Craig Kessler, a hematologist/oncologist with Georgetown University Hospital and chair of NHF's Medical and Scientific Advisory Council also presented at the briefing and described the challenges with treating hemophilia, and reinforced the need for medical treatment decisions to be made by the doctor in consultation with the patient and for continuing the excellent safety and efficacy advancements made in plasma protein therapies.
Julie Birkofer, senior vice president, North America, PPTA, moderated the presentations and provided an overview of the plasma protein therapeutics industry, plasma donation and the uniqueness of the therapies and the diseases they treat.
The informative presentations not only illustrated the diseases treated with plasma protein therapies and how they are best managed, but highlighted issues of concern to each community including access to therapies and specialists; choice in site of service; safety; continuing to create a business environment that supports manufacturer efforts to develop therapies for orphan conditions; proposed cuts in federal funding that affect hemophilia programs; and insurance coverage.
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