People living with a condition are uniquely positioned to inform the understanding of the therapeutic context for drug development and evaluation. In 2012, the U.S. Food and Drug Administration (FDA) established the Patient-Focused Drug Development (PFDD) initiative to more systematically obtain the patient perspective on specific diseases and their currently available treatments. PFDD meetings are unique among FDA public meetings, with a format designed to engage patients and elicit their perspectives on two topic areas: (1) the most significant symptoms of their condition and the impact of the condition on daily life; and, (2) their current approaches to treatment.
From 2012 to 2017, under the fifth authorization of PDUFA (PDUFA V), FDA conducted 24 disease-specific patient-focused drug development (PFDD) meetings to more systematically obtain the patient perspective on specific diseases and their treatments. PFDD meetings provide key stakeholders, including FDA, patient advocates, researchers, drug developers, healthcare providers, and others, an opportunity to hear the patient’s voice. The lessons learned include but are not limited to specific experiences that matter most to patients, patient perspectives on meaningful treatment benefits and how patients want to be engaged in the drug development process.
The list of the 24 disease-area PFDD meetings conducted under PDUFA V can be found below, along with links to meeting materials, including transcripts, webcast recordings and presentation slides that are publicly available. Following each PFDD meeting, FDA summarized the input shared by patients and patient representatives in a Voice of the Patient report. The links to these summary reports can also be found below.
More information on the selection of disease areas for fiscal years 2013-2015 can be found in the Federal Register Notice (PDF – 111KB) published on April 11, 2013. More information on the selection of disease areas for fiscal years 2016-2017 can be found in the Federal Register Notice published on July 2, 2015.
FDA recognizes the value of gathering patient input through PFDD meetings and continues to host disease-specific PFDD meetings. More information on FDA-led PFDD meetings hosted after PDUFA V and any upcoming FDA-led PFDD meetings can also be found below.