MPN Research Foundation Releases Voice of the Patient Report on MPNs


The MPN Research Foundation is thrilled to share with you the "Voice of the Patient Report on Myeloproliferative Neoplasms."

The MPN Research Foundation is thrilled to share with you the "Voice of the Patient Report on Myeloproliferative Neoplasms." We recently submitted this detailed report to the US Food and Drug Administration (FDA), resulting from an intensive externally-led Patient-Focused Drug Development meeting we hosted in September 2019, with our collaborating advocates in MPN.

The report reflects the comprehensive results of a revealing dialogue with the MPN patient community. The purpose is to inform representatives of the FDA and biopharmaceutical companies, as well as academic researchers, about the daily challenges faced by people living with essential thrombocythemia (ET), polycythemia vera (PV) and myelofibrosis (MF).

MPN patients put a voice to what it means to have an MPN diagnosis, which of their symptoms they would like to see prioritized, and how their disease impacts their quality of life, all in hopes of improving drug development for these types of blood cancers. Nearly 300 patients participated, either in person, via live stream, or in surveys before and during the daylong event to share their experiences openly and thoughtfully.

How will the report be used?

It is critical for FDA staff involved in the review of new treatments to understand that many symptoms are not well controlled with current treatments and that patients find treatment options to be limited, and often difficult to tolerate. Patient perspectives can serve as a base in helping to identify appropriate clinical outcome measures that may be used in trials to assess treatment benefits that are meaningful to patients.

"Don't let anybody tell you that pruritis is only itching . . . it was nearly body-wide pain . . . and punctuated by stabbing horse fly bites for one to three hours, day or night" -David

"I'd say my biggest wish other than a cure is that the treatment I ultimately end up with to manage my disease also reduces the symptom burden without the risk of additional side effects." -Diane

Their insight offers an intimate look into how health events and living with a chronic cancer disrupts their lives, both physically and emotionally. View the Voice of the Patient Report on MPNs for more unique stories that illustrate how different each person's symptom burden can be, along with the strategies used to manage them. Many patients also shared their experiences with various medications and clinical trials.

One clear and consistent message came through: participants were resolute on their desire for therapies that positively impact progression-free survival, with fewer side effect burdens.

Almost half of the participants indicated that severe symptom burden is their biggest struggle. Throughout the meeting, 51 symptoms were identified, including several that patients had not formerly attributed to their MPNs.

We look forward to bringing these insights to light and advancing the conversation to address meaningful treatment benefits and clinical outcomes for MPN patients.

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