Patient-reported outcomes (PROs) are the gold standard to assess the patients’ subjective health status. While both the Food and Drug Administration and European Medicines Agency recommend the use of PROs as end-points in paediatric clinical trials to support claims for medical product labelling, it is not known how often PROs are actually used. The aim of this study was to assess the usage of PRO instruments in childhood cancer clinical trials investigating anti-cancer medication.
In June 2020 ClinicalTrials and EudraCT were systematically searched for all trials including children and adolescents (≤21 years) with cancer registered between 2007 and 2020. The use of PRO measures and trials characteristics were analysed. To investigate which trial characteristics are associated with the use of PROs, a binary logistic regression was calculated.
Of 4789 identified trials, 711 were included. The most frequent reason for exclusion was age limitation (age >21 years). Of all included trials, only 8.2% used PROs as end-points; .6% as the primary end-point. The most commonly used questionnaire was the PedsQL™ (32.8%), followed by the Patient-Reported Outcomes Measurement Information System scales (12.1%). No association was observed between the use of PROs and trial region, number of centres, trial phase, time period or intervention type (all p > .05). The use of PROs did not substantially increase over time. Only 20.3% of the closed studies had published their results.
Despite recommendations of regulatory agencies, PRO assessment is extremely rare in paediatric oncology clinical trials. More efforts should be undertaken to facilitate implementation of PRO in paediatric trials to guarantee patient-centred research and treatments.

Copyright © 2021 The Authors. Published by Elsevier Ltd.. All rights reserved.

Author