Rare use of Patient Reported Outcomes (PROs) in childhood cancer clinical trials – a systematic review of clinical trial registries

Abstract

Background: patient-reported outcomes (PROs) are the gold standard to assess the patients’ subjective health-status. While both the FDA and EMA recommend the use of PROs as endpoints 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. Methods : in June 2020 ClinicalTrials and EudraCT were systematically searched for all trials including children and adolescents (21 years) with cancer registered between 2007-2020. 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. Results: 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 endpoints; 0.6% as the primary endpoint. The most commonly used questionnaire was the PedsQLTM (32.8%), followed by the PROMIS 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. Conclusion: 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.

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ORCID for Samantha Sodergren: orcid.org/0000-0001-8755-146X

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