MPN EXTERNALLY-LED PATIENT FOCUSED DRUG DEVELOPMENT MEETING
The MPN Research Foundation held an externally-led Patient-Focused Drug Development meeting on September 16, 2019 in Hyattsville, Maryland. This meeting was a long-awaited opportunity for the MPN patient community to educate representatives of the Food and Drug Administration (FDA) and biopharmaceutical companies as well as academic researchers about the challenges that individuals living with essential thrombocythemia (ET), polycythemia vera (PV), and myelofibrosis (MF) face daily. 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.
MPNRF produced a Voice of the Patient report to summarize key findings from the meeting discussion, live polling and True Reply survey done in advance of the meeting. The report will be added to the FDA website as a resource for biopharmaceutical companies working to develop new treatments for MPNs and FDA staff, so that they can keep the perspective of patients top of mind at every stage of drug development including the design of clinical trials. 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, especially since treatments that have been found to be effective can be difficult for patients to tolerate.
The Patient-Focused Drug Development (PFDD) initiative at FDA aims to more systematically obtain the patient perspective on specific diseases and their treatments. PFDD meetings give FDA and other key stakeholders 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 their review of a marketing application.
At the externally-led Patient-Focused Drug Development meeting on MPNs, people living with ET, PV and MF discussed how health events and living with chronic cancer disrupts their lives, their symptom burden, and what strategies they use for managing them as well as their experiences with various medications and clinical trials. Most importantly, each patient spoke about their hopes for the future as it pertains to drug development.
During PFDD meetings, the FDA is in listen mode, absorbing the information people directly affected with a disease are sharing with them. Ann Farrell, Division Director for the Office of Hematology and Oncology Products, her team, and other staff from FDA attended as did biopharmaceutical industry professionals and MPN patients. Invited speakers included Ruben Mesa, MD, FACP and Robyn Scherber, MD, MPH, both of University of Texas Health, San Antonio MD Anderson Cancer Center, and John Mascarenhas, MD of Icahn School of Medicine at Mount Sinai in New York.
The externally-led PFDD was hosted by the MPN Research Foundation in collaboration with MPN Advocacy and Education International, MPN Cancer Connection, MPN Education Foundation and The Leukemia & Lymphoma Society.
Thank you to the following companies for financial support of the externally-led PFDD meeting and resulting Voice of the Patient report: Celgene, Constellation Pharmaceuticals, CTI BioPharma, Genentech, Geron, Incyte, Kartos Therapeutics, PharmaEssentia and Sierra Oncology.