Voice of the Patient Report Delivered to FDA

  • MPNRF | January 18, 2022

    "One of the biggest things that has changed in my life is I've had to give things up. I have visual problems (it's like a blind spot) so I really cut down on my driving. I had to quit cooking because I made so many mistakes. I'll walk into a room looking for something and it can be right in front of me and I can't see it."

    MPNRF’s Voice of the Patient report is now live on the FDA website as a resource for biopharmaceutical companies working to develop new treatments for MPNs, and for FDA staff and others to keep patient perspective top of mind at every stage of drug development, including the design of clinical trials. Specifically, it will be helpful to FDA staff involved in the review of new treatments.

    This comprehensive report, including compiled data on some 300 patients’ symptoms and experiences, is a critical tool to understand that many symptoms are not well controlled with current treatments, drug therapy options are currently limited, and treatments often can be difficult for patients to tolerate. The report reflects the detailed results of an intensive Patient-Focused Drug Development meeting held by the MPNRF in September 2019 to highlight the daily challenges faced by people living with ET, PV and MF.