Assessment of the Use of Patient Experience Data in Regulatory Decision-Making
Section 3004 of the 21st Century Cures Act directs FDA to report on the use of patient experience data in regulatory decision-making, especially focusing on the review of patient experience data and information on Patient-Focused Drug Development tools as part of applications approved under section 505(c) of the Federal Food, Drug, and Cosmetic Act (21 U.S.C. 355(c)) or section 351(a) of the Public Health Service Act (42 U.S.C. 262(a)).
The 2026 Report
FDA contracted with Booz Allen Hamilton (Booz Allen) to conduct the 2026 report, the second of three assessments, and to produce the report. Booz Allen assessed document reviews for New Drug Applications (NDAs), Biologics License Applications (BLAs), and efficacy supplements and conducted interviews with FDA staff to describe use of patient experience data in regulatory decision-making, and to understand FDA staff perspectives on advising and evaluating patient experience data throughout product development.
The report provides details regarding the methods used to identify, collect, and analyze data for this assessment, results, and areas for continued focus for sponsors and the FDA. The report noted reviewer feedback for sponsors seeking pre-IND or IND meetings to provide detailed meeting requests so that FDA can properly engage the appropriate review disciplines and SMEs and, when submitting a market application, for sponsors to adhere to the eCTD Technical Conformance Guide Technical Specifications document. During the interviews, staff reported a need to clarify roles, responsibilities, and best practices of when to engage specialized expertise across disciplines as areas for potential improvement. The document review noted that efficacy supplements that mentioned patient experience data were less likely to include a brief statement about the patient experience data submitted and reviewed as part of the application.
To view the 2026 report please see 2026 FDA Assessment of Use of Patient Experience Data in Regulatory Decision-Making.
The 2021 Report
FDA contracted with Eastern Research Group, Inc. (ERG) to conduct this first assessment and to produce the report. ERG assessed document reviews for New Drug Applications (NDAs), Biologics License Applications (BLAs), and efficacy supplements and conducted interviews with FDA staff, applicants, and external stakeholders (patients, caregivers, clinicians, representatives of advocacy/research organizations).
The assessment gathered information to answer three questions:
- How does FDA use patient experience data in regulatory decision-making?
- How do FDA staff, applicants, and external stakeholders characterize (a) use of patient experience data in regulatory decision-making and (b) FDA communication about use of these data?
- What good practices and opportunities for improvement exist for (a) use of patient experience data in regulatory decision-making and (b) FDA communication about use of these data?
The report provides details regarding the methods used to identify, collect, and analyze data for this assessment, results, and key findings. The report includes recommendations to the FDA on how to better communicate and expand the use of patient experience data in regulatory decision- making, and to external stakeholders regarding the use of good practices to aid in the acceptance of patient experience data by the FDA. FDA will use this report to support its efforts to enhance the use of patient experience data in regulatory decision-making. FDA is committed to incorporating the patient’s voice in drug development and evaluation.
To view the report, please see 2021 FDA Assessment of Use of Patient Experience Data in Regulatory Decision-Making.
If you have any questions, please contact FDA’s CDER Patient-Focused Drug Development Program Staff by emailing patientfocused@fda.hhs.gov.