What is a Patient-Focused Drug Development (PFDD) meeting?
Patient-Focused Drug Development (PFDD) is a systematic approach to help ensure that patients’ experiences, perspectives, needs, and priorities are captured and meaningfully incorporated into drug development and evaluation. PFDD meetings follow a town hall style discussion format where the majority of the meeting is dedicated to hearing from patients and caregivers about their perspectives on their condition.
Why should I care?
Patient input from meetings and meeting summary reports can support FDA staff in conducting benefit-risk assessments for products under review by informing the therapeutic context, and advising drug sponsors on their development programs. Patient input can support drug development more broadly by identifying areas of unmet need in the patient population, identifying or developing tools that assess benefit of potential therapies, and raising awareness and increasing engagement within the patient community.
Who can participate in these meetings?
The key participants in PFDD meetings are patients, patient representatives, patient advocates, caregivers, loved ones, and anyone with a lived experience with the disease or condition. While patients and caregivers share their perspectives, key stakeholders are in listening mode as part of the audience. These stakeholders may include FDA and other regulatory/federal agencies, medical product developers, academic researchers, and clinicians and healthcare professionals.
What will I be asked to talk about during the meeting?
Participants are asked to share their perspectives during two panels followed by open discussion. The first panel focuses on the symptoms and daily impacts of the condition, while the second panel focuses on the current treatment approaches and what participants would look for in an ideal treatment. Panel two may also include a discussion of what patients consider when determining whether or not to participate in clinical trials, and a discussion of benefit-risk to better understand what tradeoffs patients may perceive as acceptable. Each topic starts with a panel of patients and caregivers who each speak for a few minutes at a time to share their experiences and help set the tone for the rest of the discussion. Following each panel, the discussion is open to all patients and caregivers in the audience.
How does my input actually influence drug development?
Patient input from meetings and meeting summary reports can support FDA staff in conducting benefit-risk assessments for products under review by informing the therapeutic context, and advising drug sponsors on their development programs. Patient input can support drug development more broadly by identifying areas of unmet need in the patient population and identifying or developing tools that assess benefit of potential therapies.
What happens after the meeting?
Following each meeting, a meeting summary report called the Voice of the Patient report is developed by the host patient organization to capture patient experience data and perspectives shared during the EL-PFDD meeting. Links to these reports can be found on the Condition-Specific Meeting Reports and Other Information Related to Patients’ Experience webpage.
What are the benefits for GAN patients?
EL-PFDD meetings are a great networking and community-building opportunity for the patient community and can help to channel patient engagement and create momentum in the patient community. Several host patient organizations have hosted follow-up meetings to build on this momentum and have synergized their efforts with other patient organizations in the space. EL-PFDD meetings may also serve to identify opportunities for further discussions with patients about their condition and may also generate new research questions. EL-PFDD meetings offer an opportunity to foster relationships between patient organizations and industry/medical product developers.
Where can I get more information?
For more details about EL-PFDD, click the following link: Externally-led Patient-Focused Drug Development Meetings | FDA