Possibilities and limits of patient-reported outcome exemplified by systemic lupus erythematosus and the LuLa study

Abstract

Over the last three decades patient reported outcomes (PRO) have become increasingly more important for the assessment of the course of diseases and therapy response. It represents the generic term for instruments which elicit information on various disease dimensions by direct questioning of patients and thereby collate subjective estimations and perceptions. This article presents the current situation with respect to the utilization of PRO. The advantages and disadvantages of individual instruments are discussed exemplified by systemic lupus erythematosus (SLE) and the LuLa study. The PRO instrument collates subjective estimations of various aspects of diseases directly from the patients. The PRO is mainly used to survey (health-related) quality of life but can also be used to reflect other disease-associated aspects. Various institutions promote the use of PRO in clinical trials for assessment of approval for pharmaceutical and medical products because ultimately only benefits for the patients are of relevance. The development of PRO instruments requires the same extensive psychometric testing as physicians instruments to measure clinical endpoints. Because of the heterogeneous sociodemographic composition of patient collectives, particular attention has to be given to cultural and linguistic adaptation. In addition to common challenges, such as missing values, under-reporting, over-reporting and response shift, subjective opinions can be influenced by other contextual factors, such as life situations unrelated to the disease. Alteration of internal standards or conceptualization can lead to interpretation difficulties in the long-term. Determinants of discordant estimations between physicians and patients need to be considered when using PRO but none of the aspects is generally better than the other.