Common Patient-Reported Outcomes Within the Food and Drug Administration Voice of the Patient Reports

Abstract

Objectives: Proponents of disease-specific patient-reported outcome measurements (PROMs) often argue disease-agnostic measures do not adequately capture their patient population's experience. Patient-Reported Outcomes Measurement Information System (PROMIS) provides a disease-agnostic domain set that may adequately cover many diseases. This study seeks to investigate whether PROMIS's quality of life domain coverage can span patient-reported outcomes (PROs) elicited from patients across unrelated diseases.

Methods: The Food and Drug Administration Voice of the Patient reports were an initiative to elevate patient voices regarding their condition and associated treatments. Two reviewers extracted patient-reported health-related (quality of life) domains from the reports and categorized them into PROMIS domains or non-PROMIS domains. Domain coverage was summarized for each report. Any extracted PROs not covered by PROMIS domains were placed in an "other" category and analyzed for common themes.

Results: Across 26 reports, PROMIS covered 216 of 374 (70%) of the reports' PRO domains. The heritable bleeding disorders report had the highest coverage (82%). Human immunodeficiency virus had the lowest coverage (50%). The most common PROMIS domain, "ability to participate in social roles," appeared in 25 reports (96%). The most common domains not included in PROMIS were stigma, sensitivities, and sensory deficits as evident in 19 (73%), 18 (69%), and 18 reports (69%), respectively. If the top 3 unincluded domains were amended into PROMIS, the total domain coverage would increase to 84%.

Conclusions: PRO domains elicited in the Food and Drug Administration Voice of the Patient reports were widely captured by PROMIS, suggesting domains patients experience contain enough overlap to be recorded by appropriate PROMIS domains. PROMIS could increase its coverage by adding domains.

Keywords: Patient-Reported Outcomes Measurement Information System; core outcome set; outcome measurement; patient-reported outcome; patient-reported outcome measurement.