The MPN Research Foundation held an externally-led Patient-Focused Drug Development meeting on September 16, 2019, in Hyattsville, MD. This meeting was a long-awaited opportunity for the MPN patient community to stand in front of decision-makers including FDA regulators, academic researchers, and biopharma representatives to convey the challenges that ET, PV, and MF patients face every day. Our objective was to give MPN patients a platform to share with the world what it means to have an MPN diagnosis, which of their symptoms should be prioritized, and how their disease impacts their quality of life in hopes of improving drug development around this group of blood cancers.


The Patient-Focused Drug Development (PFDD) initiative aims to more systematically obtain the patient perspective on specific diseases and their treatments. The patient perspective is critical in helping the FDA understand the context in which regulatory decisions are made for new drugs. PFDD meetings give FDA and other key stakeholders, including medical product developers, health care providers, federal partners, an important opportunity to hear directly from patients, their families, caregivers, and patient advocates about the symptoms that matter most to them, the impact the disease has on patients’ daily lives, and patients’ experiences with currently available treatments. This input can inform FDA’s decisions and oversight both during drug development and during our review of a marketing application. Click here for more.

There are examples of other meetings that have already taken place and resulting reports ( MPN community has the benefit of publications and studies already focusing on this topic. However, we believe that there will be a positive benefit to presenting all of this information in the form of a “Voice of the Patient” report that will capture relevant information pertaining to the issues they would like addressed by drug development shared by those with PV, ET, and MF.


Michelle Woehrle, Executive Director of the MPN Research Foundation

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 (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.




The PFDD was hosted by the MPN Research Foundation in collaboration with other advocacy organizations in the MPN space such as MPN Advocacy and Education International, The Leukemia & Lymphoma Society, MPN Cancer Connection, MPN Education Foundation as well as UT Health San Antonio MD Anderson Cancer Center’s Drs. Ruben Mesa and Robyn Scherber.

Take the steps you need to help change your prognosis.