Hearing the Patient’s Voice
The US Food and Drug Administration (FDA) has come up with a patient-focused drug development guidance series to enhance the incorporation of the patient’s voice in medical product development and regulatory decision making.
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, according to the FDA. As experts in what it is like to live with their condition, patients are in a unique position to inform the understanding of the therapeutic context for drug development and evaluation. The major objective of patient-focused drug development is to better incorporate the patient’s voice in drug development and evaluation.
Part of that process is facilitating and advancing the use of systematic approaches to collecting and using robust and meaningful patient and caregiver input to more consistently inform drug development and regulatory decision-making. Another aspect is encouraging the identification and use of approaches and best practices to facilitate patient enrollment and minimizing the burden of patient participation in clinical trials.
PFDD is also designed to enhance the understanding and appropriate use of methods to capture information on patient preferences and the potential acceptability of tradeoffs between treatment benefit and risk outcomes. Finally, it will assist in identifying the information that is most important to patients related to treatment benefits, risks, and burden, and how to best communicate the information to support their decision making.
Under the fifth authorization of the Prescription Drug User Fee Act (PDUFA V), FDA conducted 24 disease-specific PFDD meetings to more systematically gather patients’ perspectives on their condition and available therapies to treat their condition. FDA recognizes the value of gathering patient input through PFDD meetings and continues to host disease-specific PFDD meetings.
The agency is in the process of developing a series of four methodological PFDD guidance documents. These documents are designed to address, step by step, the ways in which stakeholders can collect and submit patient experience data and other relevant information from patients and caregivers for medical product development and regulatory decision making.
The collection of guidelines is part of the FDA’s PFDD efforts in accordance with the 21st Century Cures Act and The Food and Drug Administration Reauthorization Act of 2017 Title I. FDA is developing these guidances over a period of five years to implement provisions of the 21st Century Cures Act and to fulfill commitments under the sixth authorization of the Prescription Drug User Fee Act (PDUFA VI).
FDA has a webpage that contains information and documents related to the agency’s development of the methodological PFDD guidances. It includes public workshops, draft guidances and hypothetical scenarios. All of the information is designed to serve as a basis for dialogue. To contact FDA’s CDER Patient-Focused Drug Development Program Staff, please email patientfocused@fda.hhs.gov.
