Reflections on the U.S. Food & Drug Administration (FDA) Patient-Focused Drug Development (PFDD) Public Workshop on Guidance 1, Collecting Comprehensive and Representative Input (December 18, 2017)

We were very happy to have Farrah Pompilus represent Modus Outcomes at the inaugural FDA PFDD public workshop as an attendee in December 2017. It is encouraging to see that the FDA will be developing four guidance documents to further ensure greater patient-focused input throughout the regulatory process.

We trust that these new documents will provide evidence-based recommendations that build on the previously published Guidance for Patient Reported Outcome instruments (2009) and Qualification Process for Drug Development Tools (2014). These foundational guidance documents helped to shape, structure, and formalize the use of patient-centered outcome measures (PCOM) in industry drug and medical device therapeutic trials.[1]

We hope that the central tenets of context of use, concept of interest, and content validity will remain the cornerstones of evaluating whether existing (or new) PCOMs are fit-for-purpose for use in clinical trial settings. Since its inception in 2014, Modus Outcomes has heavily emphasized this important triad of considerations. Simply speaking, we need to ask the right questions about the right things to the right people; this is the foundation of what we call concept-driven measurement.[2],[3]

The relevance, importance, and appropriate targeting of PCOM content to patients is vital to ensure interpretable measurement. These interpretations aid not only in drawing conclusions about meaningful outcomes in a clinical trial setting, but also in generalizing those findings to provide real world evidence.

Fully incorporating the voice of the patient, and that of all key stakeholders is a vital component of this partnership approach; we applaud the FDA PFDD’s efforts to this end.

Modus Outcomes will be watching the developments at the FDA closely in the coming months and years. We look forward to contributing to future development, where and when we can.


[1] Walton MK, Powers JH, 3Hobart J, Patrick D, Marquis P, Vamvakas S, et al. Clinical Outcome Assessments: Conceptual Foundation-Report of the ISPOR Clinical Outcomes Assessment – Emerging Good Practices for Outcomes Research Task Force. Value Health. 2015;18(6):741-52.

[2] Morel T, Cano SJ. Measuring what matters to rare disease patients – reflections on the work by the IRDiRC taskforce on patient-centered outcome measuresOrphanet Journal of Rare Diseases. 2017;12:171.

[3] Patrick, D.L., Burke, L.B., Gwaltney, C.J. et al. Content validity—establishing and reporting the evidence in newly developed patient-reported outcomes (PRO) instruments for medical product evaluation: ISPOR PRO Good Research Practices Task Force Report: part 1—eliciting concepts for a new PRO instrument. Value Health. 2011; 14: 967–977.



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