CDER Patient-Focused Drug Development
What is Patient-Focused Drug Development?
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. As experts in what it is like to live with their condition, patients are uniquely positioned to inform the understanding of the therapeutic context for drug development and evaluation.
The primary goal of patient-focused drug development is to better incorporate the patient’s voice in drug development and evaluation, including but not limited to:
- Facilitating and advancing use of systematic approaches to collecting and utilizing robust and meaningful patient and caregiver input to more consistently inform drug development and regulatory decision-making
- Encouraging identification and use of approaches and best practices to facilitate patient enrollment and minimizing the burden of patient participation in clinical trials
- Enhancing understanding and appropriate use of methods to capture information on patient preferences and the potential acceptability of tradeoffs between treatment benefit and risk outcomes
- 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.
What We Do
The Patient-Focused Drug Development Program Staff leads initiatives and provides strategic, regulatory, program, and policy assistance within the Center for Drug Evaluation and Research (CDER) to facilitate the incorporation of patient input into decision-making.
Connect with FDA’s CDER Patient-Focused Drug Development Program Staff
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On This Page
On this page, you will be able to link to more information on FDA’s PFDD efforts as well as other external resources.
PFDD Methodological Guidance Series
FDA is developing a series of methodological guidance on the collection of patient experience data, and the use of such data and related information in drug development.
FDA developed a COA Pilot Grant Program to support the development of publicly available core set(s) of Clinical Outcome Assessments (COAs) and their related endpoints for specific disease indications.
FDA’s PFDD meetings have provided key stakeholders, including FDA, patient advocates, researchers, drug developers, healthcare providers, and others, an opportunity to hear the patient’s voice.
Externally-led Patient-Focused Drug Development Meetings
FDA welcomes patient organizations to identify and organize patient-focused collaborations to generate public input on other disease areas.
Condition-Specific Meeting Reports
This webpage hosts an alphabetical listing of condition-specific meeting reports and other information related to patients’ experience. These meetings include FDA-led Patient-Focused Drug Development (PFDD) meetings, Externally-led PFDD meetings, and Patient Listening Sessions.
- eCTD Technical Conformance Guide
- Assessment of the Use of Patient Experience Data in Regulatory Decision-Making