Bringing the voice of MPN to the FDA

On Monday, September 16th, twelve courageous people living with Polycythemia Vera, Essential Thrombocythemia and Myelofibrosis took the stage in Bethesda, MD at the Externally-Led Patient-Focused Drug Development Meeting on Myeloproliferative Neoplasms. They discussed how health events and living with chronic cancer continues to disrupt their lives, their symptom burden, and what strategies they use for managing them as well as their experiences with various medications and clinical trials. But most importantly, each patient spoke about their hopes for the future as it pertains to drug development. YOU have a chance to weigh in as well. For the next 30 days, we will be collecting YOUR feedback on what issues you are living with, what benefits you would like to experience with further drug development. Just send an email with your name and diagnosis to communications@mpnrf.org (we will not share your name or email). 

This meeting framework, created by the Food and Drug Administration (FDA) was designed to obtain the patient perspective on specific diseases and their treatments. The FDA is "responsible for advancing the public health by helping to speed innovations that make medical products more effective, safer, and more affordable and to help the public get the accurate, science-based information they need to use medical products and foods to maintain and improve their health." Essentially, this means the FDA works with sponsors to design clinical trials, determine outcome measures (assessment of how a patient is doing), endpoints, and whether to approve or revoke a therapy. 

As funders of global MPN research and advocates for the patient community, MPNRF felt it was imperative to seek an audience with FDA decision-makers. The opportunity to hold an externally-led PFDD meeting was a natural first step. During PFDD meetings, the FDA is in listen mode, absorbing the information people directly affected with an MPN are sharing with them. We were pleased that Ann Farrell, Division Director for the Office of Hematology and Oncology Products, her team, as well as other staff from the agency, were present. The meeting was also attended by Drs. Robyn Scherber, Ruben Mesa and John Mascarenhas, industry professionals and many MPN patients. 

Our panelists, David K, Vivienne, Bridget, Vicki, Ruth, Morgan, Ned, Nancy, David A, Diane, Karrie, and Stephanie, put a face to the name myeloproliferative neoplasms and validated for the world what their experiences with ET, PV, and MF have been. Our job now is to ensure their efforts and openness materialize into a tangible benefit for all MPN patients. That is our mission, and we will remain faithfully on that path. 

Author: Michelle Woehrle

Michelle Woehrle joined the MPN Research Foundation in 2007 after being inspired by their mission to fund research that will improve the lives of people living with the rare blood cancers PV, ET and MF. In 2015 she was promoted to Executive Director. She has assisted with the expansion of the grants program, grassroots initiatives to connect with more patients internationally and optimize operations for the Foundation.

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