The resources listed on this page are provided for informational purposes only. They are not created, maintained, or formally endorsed by the PARCR team. We offer them to help individuals explore additional materials that may support their understanding of patient experience data, patient‑focused drug development, and related topics.
General Resources
FDA Patient-Focused Drug Development Guidance Series overview: FDA Patient-Focused Drug Development Guidance Series for Enhancing the Incorporation of the Patient’s Voice in Medical Product Development and Regulatory Decision Making | FDA
FDA, Patient-Focused Drug Development: Workshop to Discuss Methodologic and Other Challenges Related to Patient Experience Data Dec 13 2024
Video:
Part 1: PFDD Workshop to Discuss Methodologic & Other Challenges Related to Patient Experience Data – Part 1
Part 2: PFDD Workshop to Discuss Methodologic & Other Challenges Related to Patient Experience Data – Part 2
Patient-Focused Drug Development: Workshop #2 to Discuss Methodologic and Other Challenges Related to Patient Experience Data Sept 18-19 2025
Guidance 1
Final guidance : Patient-Focused Drug Development: Collecting Comprehensive and Representative Input | FDA
FDA, Public Meeting: Using Methods from PFDD Guidance 1 and Guidance 2 Meeting #1, June 30, 2022
FDA, Public Meeting: Public Meeting: Using Methods from PFDD Guidance 1 and Guidance 2 Meeting #2, July 25, 2022
Guidance 2
Final guidance : Patient-Focused Drug Development: Methods to Identify What Is Important to Patients | FDA
Guidance 3
Final guidance: Patient-Focused Drug Development: Selecting, Developing, or Modifying Fit-for-Purpose Clinical Outcome Assessments | FDA
Relevant Manuscripts by PARCR Personnel
Edwards, M. C., Slagle, A., Rubright, J. D. & Wirth, R. J. Fit for purpose and modern validity theory in clinical outcomes assessment. Quality of Life Research 27, 1711–1720 (2018). https://doi.org/10.1007/s11136-017-1644-z
Weinfurt, K. P. Constructing arguments for the interpretation and use of patient-reported outcome measures in research: an application of modern validity theory. Qual Life Res 30, 1715–1722 (2021). https://doi.org/10.1007/s11136-021-02776-7
Weinfurt, K. P. Constructing and evaluating a validity argument for a performance outcome measure for clinical trials: An example using the Multi-luminance Mobility Test. Clin Trials 19, 184–193 (2022). https://doi.org/ 10.1177/17407745211073609
Houts, C. R., Bush, E. N., Edwards, M. C. & Wirth, R. J. Using validity theory and psychometrics to evaluate and support expanded uses of existing scales. Qual Life Res 31, 2969–2975 (2022). https://doi.org/10.1007/s11136-022-03162-7
Oehrlein, E. M. et al. An Interview with the Food & Drug Administration about Draft Patient-Focused Drug Development Guidance 3: Selecting, Developing, or Modifying Fit-for-Purpose Clinical Outcome Assessments. Value Health 26, 791–795 (2023). https://doi.org/10.1016/j.jval.2023.04.006
Weinfurt, K. P. Developing, Selecting, and Modifying Performance Outcome Assessments. Value Heal. 26, 957–958 (2023). https://doi.org/10.1016/j.jval.2023.04.011
Weinfurt, K. P. The Argument-Based Approach to Validity Applied to Clinical Outcome Assessments: Some History and Notable Features. Value Heal. 28, 997–1000 (2025). https://doi.org/10.1016/j.jval.2025.03.012
Guidance 4
Public Webinar: https://www.bing.com/videos/riverview/relatedvideo?q=PFDD+guidance+one+webinar+FDA&mid=6FBD6D4AC3F593F9713F6FBD6D4AC3F593F9713F&FORM=VIRE
Relevant Manuscripts by PARCR Personnel
Weinfurt, K. Interpreting the meaningfulness of treatment effects estimated in parallel groups designs: comment on Trigg et al. Qual. Life Res. 34, 1885–1889 (2025). https://doi.org/10.1007/s11136-025-03952-9
Patient Engagement Resources
ISPOR: ISPOR - Patient Engagement in HEOR
The International Society for Quality of Life Research (ISOQOL): Patient-reported outcomes in randomized clinical trials: development of ISOQOL reporting standards - PubMed
Clinical Trials Transformation Initiative (CTTI): Patient Engagement - CTTI