FDA Releases "Voice of the Patient" Summary from Patient-Focused Drug Development Initiative

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In September 2014, the U.S. Food and Drug Administration hosted a public meeting as part of it’s Patient-Focused Drug Development initiative, a 5 year initiative to learn more from patients across a variety of disease and disorders. We were proud to be a part of this meeting and provided support to several of the patient voices represented to help them have their voice heard.
The official report was recently released and highlights many of the topics discussed that day, proving that the bleeding disorders community’s voice was heard on a variety of topics from inhibitor treatment and development, access to medications, data collection before, during, and after clinical trials, and the ongoing management of bleeding disorders and it’s effects on quality of life.

Read the Report

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.

  • 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.