Consideration of useful patient-reported outcome measures to identify unmet medical needs of children and adults with autoimmune and autoinflammatory diseases.

Abstract

Patients with autoimmune and autoinflammatory diseases experience difficult physical, mental, and social situations and have various unmet medical needs (UMNs). To provide appropriate solutions for these patients, an accurate understanding of their UMNs is necessary. Patient-reported outcomes (PROs) reflect the problems of patients and are highly likely to be useful in understanding patient needs. This article reviews established PRO measures from the perspective of determining those appropriate for identifying the UMNs of patients with autoimmune and autoinflammatory diseases. To consider appropriate PRO measures, discussions were held by experts at the Ministry of Health, Labor and Welfare group meetings. The possibility of developing a method to collect PRO data electronically using information and communication technology was also considered. The experts proposed 28 measures, both disease-specific and non-disease-specific, as candidates. It was confirmed that linguistic validation was important and that measures obtained from adults and children could not be considered together. A migration from paper to digital PRO measures was not conducted due to the need for ensuring accuracy and the shortage of technical and financial support. Appropriate non-disease-specific PRO measures were considered to be the KINDL® and EQ-5D-Y for children and the SF-36v2® and EQ-5D™ for adults.