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.
On this page, you will be able to link to more information on FDA’s PFDD efforts as well as other external resources. To get updates about CDER's Patient-Focused Drug Development programs, subscribe to our free email subscription service by clicking on the button near the top of the page. To contact FDA’s CDER Patient-Focused Drug Development Program Staff, please email firstname.lastname@example.org.
21st Century Cures and PDUFA VI
FDA is developing a series of of methodological guidance on the collection of patient experience data, and the use of such data and related information in drug development. 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).
Under the 21st Century Cures Act, FDA issued a draft guidance on developing and submitting proposed draft guidance relating to patient experience data.
CTTI and FDA held a public workshop on enhancing the incorporation of patient perspectives in clinical trials. This workshop is intended to meet an FDA commitment included in PDUFA VI.
COA Grant Program
As part of PFDD efforts, FDA had 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-led PFDD Meetings
From 2012 to 2017, under the fifth authorization of PDUFA (PDUFA V), FDA conducted disease-specific PFDD meetings to obtain the patient perspective. Although there are no further commitments to PDUFA V, FDA recognizes the value of continuing to gather patient input through PFDD meetings. 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.
External Resources or Information Related to Patients’ Experience
FDA welcomes patient stakeholders and others to submit information or patients experience data related to their patient community. The resources on this page are for your convenience and do not indicate endorsement of their content by FDA.
To get updates about CDER's Patient-Focused Drug Development programs, subscribe to our free email subscription service using the button at the top of the page.
- FDA Patient-Focused Drug Development Guidance Series for Enhancing the Incorporation of the Patient's Voice in Medical Product Development and Regulatory Decision Making
- The Voice of the Patient: A Series of Reports from FDA-led PFDD Meetings under PDUFA V
- Guidelines for Developing a Letter of Intent (LOI) for externally-led PFDD meetings
- CDRH's Patient Preference Initiative
- Oncology Center of Excellence Patient-Focused Drug Development Program