Members of the community may remember that in September 2014, the US Food and Drug Administration (FDA) held a novel meeting devoted to hearing directly from people with bleeding disorders about their lives and treatment needs. The meeting was part of the FDA’s Patient-Focused Drug Development Initiative, a series of 20 disease-specific meetings held to provide the FDA with input from individuals affected by a given condition and their caregivers. NHF advocated for bleeding disorders to be on the list of conditions for the meetings. This pilot program is important for two reasons: it allows FDA officials who consider bleeding disorders treatments for approval to have a deeper understanding of what it’s like to live with a bleeding disorder, and it gives the FDA insight in how to collect patient preferences and concerns.
At the meeting, people affected by hemophilia, von Willebrand disease (VWD) and rare bleeding disorders explained how their daily lives are affected by having a bleeding disorder, how their disorder and treatments have changed over time, and what they desire from the ideal treatment. Participants told powerful stories of fear of developing inhibitors, living with chronic pain, and dealing with depression and anxiety. There were also hopeful stories of the freedom that has come from home treatment, prophylaxis and the new longer-lasting products. Further, there was a discussion of what “normal” is and should be for people with bleeding disorders. In addition to the perspectives shared at the meeting, NHF also conducted a survey of the community.More than 140 people completed the survey, adding their thoughts and stories. A summary of the survey was provided to the FDA in writing.
This fall, the FDA released a “Voice of the Patient” report summarizing the bleeding disorders meeting and written comments that the agency received. In the report, the FDA explains how it will use the information received from the bleeding disorders community to inform its interactions with drug manufacturers. It also highlights the discussion of inhibitors at the meeting and that consistent with this concern, the FDA joined with the National Institutes of Health (NIH), NHF and the Plasma Protein Therapeutics Association (PPTA) to hold a workshop on the subject in September 2015.
NHF is committed to working with the FDA and all stakeholders to ensure that people with bleeding disorders have access to safe and effective treatments. The FDA continues to seek input from NHF and the broader bleeding disorders community. Just this month, NHF participated in a research project to evaluate FDA’s benefit/risk frameworks for bleeding disorders treatments. NHF will continue to update the community on the patient-focused drug development process and its work with the FDA.