OBJECTIVES: Acute otitis media (AOM) is characterized by rapid onset of signs and symptoms of inflammation in the middle ear causing pain, fever, changes in the sleep or behavior pattern in children younger than 5 years. The aim of this study was to assess suitability of the patient-reported outcomes (PRO) and observer-reported outcome assessment (ObsRO) measures for inclusion in future clinical trials assessing new potential therapies.

METHODS: A comprehensive literature review was conducted on Ovid until April 2017 to retrieve QoL studies in patients with AOM. AOM trials with PRO endpoints were searched on clinical trials.gov. For the PROs and ObsROs identified, studies describing the tool development process and performance were examined against FDA and EMA PRO guidance.

RESULTS: A total of 624 abstracts were reviewed and the full text screening was performed for 76 articles. From the literature search and clinical trials assessment, 16 tools were identified and these were classified as generic or disease-specific, self-reported or proxy-reported, and patient burden or caregiver burden assessment measures. Although OM-6 was the most frequently used ObsRO, there was insufficient evidence to support its content and construct validity. AOM severity of symptom (AOM-SOS) scale was identified as most suitable disease-specific ObsRO, whereas among generic tools, PedsQL Generic Core Scales (for 2-18 years old) and the PedsQL Infant Scales (for 1-24 months), possessed strong evidence of reliability and validity. Faces pain scale-revised (FPS-R) was found to be a psychometrically sound self-report tool/PRO in children of 2 years of age or older.

CONCLUSIONS: Based on the analysis, it would be preferable to capture QoL outcomes using simultaneously a parent proxy disease specific scale like AOM-SOS, generic scale such as PedsQL and a self-report tool like FPS (keeping in line with regulatory preference for patient self-report over proxy report wherever feasible).