On September 22nd, the US Food and Drug Administration (FDA) held a 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 providing the FDA with input from people affected by a condition and also their caregivers. FDA wants to better understand how people affected by a condition think about the risks and benefits of potential treatments and cures.
At the meeting, people affected by hemophilia, von Willebrand disease (VWD) and rare bleeding disorders told their own stories and 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 (antibodies to infused factor product), 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. There was a discussion of what “normal” is and should be for people with bleeding disorders.
The good news is that there’s still time for you to share your input. The FDA is accepting written comments on the questions discussed at the meeting until the end of November. Further, the National Hemophilia Foundation (NHF) is finalizing a survey on the same questions, which will be distributed to the community.
The meeting was recorded and will be posted on the FDA website shortly. To learn more about it or to tell your story to the FDA, please click here.
