MPN RESEARCH FOUNDATION VISITS THE FDA

September 18, 2017

The FDA is working hard to incorporate the patient voice in their mission of approving safe and effective new drugs. The FDA has newly formed the Oncology Center of Excellence that has, as one of its departments, the Office of Patient Outcomes.  This office reached out through the FDA’s Patient Liaison Program to 3 MPN patient advocate groups including the MPN Research Foundation, had discussions about the concerns of MPN patients, and asked them to send representatives to an FDA Mini Symposium:  “Myeloproliferative Neoplasms (MPNs):  A Conversation with Patient Advocates” to discuss the topics in depth. 

Dr. Paul Kleutz, Acting Director of Patient Outcomes, led the FDA contingent.  He was accompanied by 7 other speakers including the Director of Orphan Products Development and several Medical Officers who are hematologist / oncologists in the Center for Drug Evaluation and Research.   Additionally, two physicians who had worked extensively on approving Jakafi, the one new drug approved for MPNs since the discovery of the JAK2 mutation in 2005, were also at the table.

Michelle Woehrle, Executive Director, (pictured, left, with FDA’s Office of Health and Constituent Affairs) and Dave Boule, Board member of the MPN Research Foundation attended the meeting.  Two other advocacy groups sent 3 others.  Of the 5 advocates, 3, including Dave, are also MPN patients.

The Mini Symposium was essentially “FDA 101” as it relates to the MPNs.   It was 4 hours long and highly interactive.  They only had to say once: “Ask your questions any time”.  Three hours into our session, they had only covered 2 hours of the material they planned to present, so, even though the remaining speakers sprinted to the finish line, we went overtime by 30 minutes.

We learned about the regular and accelerated drug approval processes and what differentiates drugs that go through one or the other.  Importantly for the MPN community, we learned all about the Orphan Drug Designation for rare diseases  and the process that provides enormous financial incentives to pharmaceutical companies to develop drugs for diseases where there are 200,000 or fewer patients across the US. All 3 of the MPNs, PV, ET, and MF are rare diseases by this standard.  We also had interesting presentations on patient access outside of clinical trials to investigational drugs pre approval and personal importation of drugs that are approved for use only outside the US.

While the opportunity to learn the fundamentals of “FDA 101” at the meeting was extremely important and useful, Dave and Michelle also came to the meeting with a mission…to promote the interests of the MPN patient community they serve in two specific regards: (1) to determine how the patient advocates can best help to get interferon approved as a front line “on label” therapy for patients with MPNs and (2) to determine how the data being gathered by the brand new MyMPN Patient Registry sponsored by the MPN Research Foundation might be helpful to the FDA.  
Dave had a conversation with Dr. Richard T. Silver, the pioneer in the use of interferon in treating MPNs and a member of the MPN RF Advisory Board on its research project to determine the mechanism of action of interferon.   Interferon is the only effective drug on the market in reversing the progression of MPNs as demonstrated by a reduction of fibrosis in the bone marrow, normalization of blood counts including elimination of anemia in early stage myelofibrosis, a reduction in mutated JAK2, and an alleviation of symptoms including shrinking enlarged spleens.  Dr. Silver noted that the FDA has never been asked to approve it as therapy for MPNs by the pharmaceutical companies that manufacture it (interferon is an approved drug for treatment of Hepatitis C) because their perception that the MPN market is too small to warrant the expense of a Phase III clinical trial (the final and very expensive phase in the FDA’s drug approval process).