FDA Hosts Patient-Focused Meeting

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Members of the bleeding disorders community sharing their experiences before the FDA on Monday, September 22, 2014. Photo from right to left of the morning panel, Amanda Heisey, Mark Skinner, Daniel Bond, and Sonji Wilkes.

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. To read some of the testimonies given, please see below.
There is still time to have your voice heard! The FDA is accepting comments until November 28, 2014. For details on how to submit your official statements to the FDA, please click here. Additionally, you can help shape the future of bleeding disorders treatments by taking this short survey!
If you would like assistance in crafting your statement, please reach out to our Advocacy team at advocacy@hemophiliafed.org.


  • Debbie, whose twenty-year-old son has Hemophilia A and an inhibitor, made a powerful stand for the inhibitor community, shedding light on the limited access to treatment available to patients like her son.
  • Donald explained his experiences across seven decades of hemophilia treatment, from receiving whole blood to factor, while detailing his hopes for contemporary Hepatitis C therapy trials. Read his powerful call for a cure, so that his great-grandchildren will not have to grow up with hemophilia.
  • Sonji offered insights both as a female hemophilia patient and the mother of a 13-year-old boy growing up with an inhibitor. She shared hardships she’s faced in seeking care for her condition, as well as the technological advancements that allow her son to attend school while staying home to recover from bleeds. Follow along as she advocates for her son’s safety as the Administration considers new inhibitor therapy trials.
  • Mark, a 34-year-old co-infected Hemophilia A patient and father of two carriers, voiced his hopes for new advancements in treatment, like subcutaneous injections, long-lasting infusions, and a world without inhibitors. Mark also joined his fellow speakers in calling on the FDA to prioritize cure research.
  • Dan is a 60 year old Hemophilia B patient with AIDS who has been fortunate enough to spontaneously clear his hepatitis C infection. Dan used the cost of his hemophilia treatments to urge cure research. See how much Dan’s treatments have cost over his lifetime.
  • Kimberly, HFA’s Executive Director and the mother of a twelve-year-old patient, stressed the need for easy access to safe, affordable, and various treatment methods. She cautioned that progress should not come at the cost of risk factors like inhibitors, and asked the the FDA push for a change in diagnosis guidelines to ensure that women get the treatment they need and deserve. Read Kimberly’s moving statement.

*As part of the renewal of the Prescription Drug User Fee Act (PDUFA V) in 2012, the Food and Drug Administration (FDA) must incorporate the patient perspective into their regulatory decision-making. To accomplish this, the FDA is holding a series of Patient-Focused Drug Development meetings where they seek the input of patients in 20 disease groups to assess drugs and treatment options. Patients provide an important and unique context for the FDA as they develop their new approach to rulemaking using the Benefit-Risk Assessment Framework.