Addressing Bias in Responder Analysis of Patient-Reported Outcomes.

ABSTRACT:

Introduction

Quantitative patient-reported outcome (PRO) measures ideally are analyzed on their original scales and responder analyses are used to aid the interpretation of those primary analyses. As stated in the FDA PRO Guidance for Medical Product Development (2009), one way to lend meaning and interpretation to such a PRO measure is to dichotomize between values where within-patient changes are considered clinically important and those that are not. But even a PRO scale with a cutoff score that discriminates well between responder and non-responders is fraught with some misclassification.

Methods

Using estimates of sensitivity and specificity on classification of responder status from a PRO instrument, formulas are provided to correct for such responder misclassification

SUBMITTER: Cappelleri JC 

PROVIDER: S-EPMC8332587 | biostudies-literature | 2021 Sep

REPOSITORIES: biostudies-literature