Educational Resources to Support FDA's Patient‐Focused Drug Development Guidance Series

CERSI Collaborators: Triangle CERSI: Kevin Weinfurt, PhD

FDA Collaborators: Robyn Bent, RN, MS

Project Start Date: September 1, 2024

Regulatory Science Framework:

Modernize development and evaluation of FDA-regulated products, Clinical Outcome Assessment

Regulatory Science Challenge:

The FDA’s Patient-Focused Drug Development (PFDD) guidance series was developed to address in a stepwise manner, how stakeholders can collect and submit patient experience data and other relevant information from patients and caregivers for medical product development and regulatory decision-making. These guidance documents discuss scientific topics and methods that may not be well understood by all users. Therefore, this project will develop educational materials to help users better understand and incorporate the recommendations and advice presented in the guidance series.

Project Description and Goals:

This project has four goals. Goal 1 is to develop education modules that explain the Roadmap to Patient-Focused Outcome Measurement in Clinical Trials and how the four PFDD guidance documents inform each stage. Goal 2 is to create modules that discuss approaches for identifying, modifying, or developing measures of how patients feel or function for use in clinical trials.  These measures are called Clinical Outcome Assessments (COAs). Goal 3 is to develop modules that discuss how to use COAs in clinical trials to understand the effects of medical products on people’s health. Goal 4 is to develop training materials on special topics in patient-focused outcome measurement, including considerations for use in pediatric patients, populations affected by rare diseases, and/or conditions where patients cannot reliably self-report.

Anticipated Outcomes/Impact:

This project will produce (1) training materials on the PFDD guidance series for FDA staff and (2) training materials on the PFDD guidance series for external stakeholders, including regulated industries, academic researchers, and patient‐powered research groups. Additional outcomes may include presentations internally at FDA or externally (e.g., NIH Collaboratory Grand Rounds) to raise awareness about the educational materials.