On Monday, September 22, several members of the bleeding disorders community came out to share their experiences with bleeding disorders treatment with the FDA.
HFA worked with partners like NORD, PPTA, and NHF to ensure that our community was effectively represented. Patients and advocates of every age, every background, and with several different conditions came out to share with the FDA their hopes, fears, highlights, and trials in treating their conditions. So many great opinions were shared, covering two open-ended topics set by the FDA.
Below is the statement delivered by Kimberly Haugstad, HFA’s Executive Director. Kimberly explained to the FDA that no two patients treat their bleeding disorders the same way, making choice and safety paramount in the therapy approval process. Kimberly also called for the FDA to make it easier to diagnose women, and advocated for the inhibitor community. We hope that you will join Kimberly and share your own treatment experiences with the FDA.
My name is Kimberly Haugstad. I am the Executive Director of the Hemophilia Federation of America. HFA serves as a patient advocate for safe, affordable, and obtainable blood products for bleeding disorders patients.
Through our high-touch national programs and patient surveys, we collect qualitative data from a broad range of community members – many of whom do not have a voice in arenas like this. Consequently, we have a unique perspective from which to comment on the questions posed today.
We find that patients living with bleeding disorders use a range of treatment options, including a variety of recombinant, monoclonal, and plasma-derived products, as well as other alternative coping and pain-management strategies. We do appreciate that we have access to a range of options.
The use of prophylaxis treatment and the ability for patients to infuse at home are significant for the bleeding disorders community. This allows for greater flexibility in treatment schedules and the ability to lead a more active and productive life.
Individuals who have developed inhibitors have significant and vital additional needs, as do older adults as they experience common aging issues in tandem with their bleeding disorders. Women clearly are still undiagnosed and underserved.
Access to multiple treatments are critical for the hemophilia community and personalization of treatment is needed.
Hemophilia treatment is simply not one size fits all. Based on anecdotal evidence, one patient might develop allergies or an inhibitor while on one product where another patient will not. It is often reported that different products have varying levels of hemostatic efficacy.
Inhibitors impact approximately 30% of previously untreated individuals. Much is still unknown in our community about how or why our treatments do or do not work. With these uncertainties, there is the need for patients to continue to have access to a variety of treatments and a need for a transparent and open dialog about any data collected on the impact of treatments.
The most critical treatment concern for members of our community is that treatments are safe. We do not forget the staggering impact HIV- and hepatitis C-tainted products had on this community. The emotional impact was not only on the health of those affected but also on the entire family unit.
Problems definitely still exist that need attention. Frankly, an inhibitor rate of 30% in previously untreated patients (PUPs) today is simply not acceptable. Families and HFA believe in the importance of a stringent, constant, and demonstrable dedication to the safety of treatments to insure the well-being of our community.
Thank you for your time and the opportunity to speak on behalf of people with bleeding disorders today.