A Possibility of Administering Selected Domains of Existing Patient-Reported Outcome Questionnaires to Reduce Patient Burden and Increase Clinical Relevance

ISSUE:

FDA has been a proponent of the idea of administering selected domains of Patient-reported outcome questionnaires, while other stakeholders insist on using only the validated measures as they were originally developed. Sponsors are caught in the middle needing to satisfy stakeholder desires while also being incentivized to reduce respondent and operational burden and optimize the study costs.

OVERVIEW:

Patient-reported outcomes (PROs) are used for a variety of purposes including to describe the course of disease or condition, inform treatment decisions, and understand treatment tolerability and benefit in clinical research. Some frequently used PRO assessments have limitations, however, and are often critiqued as, among other things, too long and burdensome (or too short and not comprehensive) or too generic and not relevant for a patient population (or too restrictive and not relevant for a patient population). For these reasons, outcomes researchers are interested in selecting and administering only the relevant “core domains” from existing validated assessments to patients participating in clinicals trials. This is to minimize redundancy (and potentially bad or inconsistent data because of it) as well as burden and costs while still collecting sufficient information on which treatment tolerability and efficacy conclusions can be drawn. Such a “modular approach” to assessment has appeal, although some stakeholders posit that there is an unknown consequence to content validity, psychometric performance, and score interpretation when administering only selected domains from an existing tool. In other words, the goal of this panel is to discuss whether a subset of domains from an existing instrument may be considered “fit for purpose” and to gain a deeper understanding of the impact this modular approach may have in clinical trial research particularly in the context of selecting, implementing, and using these assessments in pivotal studies as key endpoints to support product approval and labeling objectives.