By: Theresa M. Mullin, Ph.D.
Last year, FDA began the Patient-Focused Drug Development (PFDD) program to more systematically obtain the patient perspective on certain diseases and their treatments. The effort is part of an FDA commitment under the fifth authorization of the Prescription Drug User Fee Act (PDUFA V).
After conducting a public process to nominate disease areas for fiscal years 2013-2015, FDA held the first PFDD meeting on April 25, 2013. This meeting focused on chronic fatigue syndrome (CFS) and myalgic encephalomyelitis (ME), sometimes called CFS-ME, a debilitating disease for which there are currently no FDA-approved treatments.
Here, we heard directly from patients, patient advocates, and caretakers about the symptoms that matter most to them, the impact the disease has on patients’ daily lives, and the patient experience with currently available treatments. FDA staff, including members of FDA’s Division of Pulmonary, Allergy, and Rheumatology Products, listened carefully to the personal accounts of this devastating condition.
After the meeting, we released a report titled The Voice of the Patient: Chronic Fatigue Syndrome and Myalgic Encephalomyelitis, a detailed summary of the meeting. In this report we documented, in the patients’ own words, what mattered most to them in terms of impacts of the disease and treatment approaches. This summary included the patient testimony at the meeting, perspectives shared in 230 docket comments, as well as unique views provided by those who joined the meeting webcast.
These reports serve an important function in communicating, to both FDA review staff and the regulated industry, what improvements patients would most like to see in their daily life. FDA believes that the long-term impact of this program will be a better, more informed understanding of how we might find ways to develop new treatments for these diseases.
Soon after the CFS and ME meeting, in June 2013, we conducted similar meetings on HIV and lung cancer, and these summary reports are now available on our website. The reports for our recent meetings on narcolepsy and sickle cell disease will be posted soon. Our most recent meeting was held on fibromyalgia on March 26, 2014. On May 13, we held the meeting on pulmonary arterial hypertension, and coming up next is the meeting on inborn errors of metabolism, on June 10.
By the end of FY 2015, we plan to have conducted at least 16 PFDD meetings to hear from patients suffering from a wide range of conditions. These are currently identified on our web page. For the remaining two years in PDUFA V, we will conduct another public process to identify the diseases that will be addressed during that time.
We are gratified by the enthusiastic response within the patient community to PFDD, and we look forward to continued success with these meetings — and the long-term benefit they can offer for drug development in important therapeutic areas.
Theresa M. Mullin, Ph.D., is Director of FDA’s Office of Strategic Programs in the Center for Drug Evaluation and Research