PHP154 - Patient-Reported Outcome Measures in the FDA Pilot Compendium: Meeting Today’s Standards for Patient Engagement in Development?

Abstract

In 2016, the Food and Drug Administration (FDA) released its Pilot Clinical Outcome Assessment (COA) Compendium, to foster patient-focused drug development (PFDD) by summarizing in a single resource COA information for various diseases and conditions to: (1) facilitate communication; (2) provide clarity and transparency; and (3) serve as a starting point for early drug development. For included patient-reported outcome (PRO) measures, it is unclear if patient perspectives were solicited during development or validation, a recognized step in FDA’s Roadmap to Patient-Focused Outcome Measurement in Clinical Trials. This review sought to identify level of patient engagement in development of included PRO measures. PROs included in Compendium Chapters 1 or 2 were extracted. Three reviewers independently searched PubMed and Google to identify information on measure pedigree (development, validation, reliability, etc.). Data on method and stage of measure development where patient engagement took place were abstracted. Among 26 evaluated PRO measures, no information on development or validation was found for nearly half the sample (n=12). Among remaining measures (n=14), five had no evidence of patient engagement; two noted engagement during concept elicitation; one noted engagement during psychometric validation; and six noted engagement during both concept elicitation and cognitive interviewing. Measures either previously qualified or submitted for qualification were more likely to have documented patient engagement. For the FDA Pilot COA Compendium to fulfill its purpose of fostering PFDD, it needs to reflect today’s standards by improving transparency and facilitating clear identification of included measures so level of patient engagement, among other factors, can be properly assessed. Suggested improvements include: more clearly identifying specific measure by name or with citations; identifying clinical trials corresponding to COA use in trials; and including only measures already qualified or undergoing qualification.