Drugs

CDER Patient-Focused Drug Development

Patient-Focused Drug DevelopmentPatient-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. 

 

21st Century Cures and PDUFA VI21st Century Cures and PDUFA VI
FDA will develop a series of guidance on the collection of patient experience data, and the use of such data and related information in drug development. FDA will develop 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-led PFDD MeetingsFDA-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. 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 PFDD MeetingsExternally-led PFDD Meetings
FDA welcomes patient organizations to identify and organize patient-focused collaborations to generate public input on other disease areas. Meeting reports from externally-led PFDD meetings that have been shared with the FDA can be found here

 

External Resources or InformationExternal 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.

 

 

More in Development & Approval Process (Drugs)

Clinical Data Summary Pilot Program Drug Development Tool Programs and Initiatives Guidance Documents for Drug Applications Laws, Regulations, Policies and Procedures for Drug Applications

Page Last Updated: 06/12/2018
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