(009) Completion and reporting rates of paediatric clinical trials conducted as part of an EU Paediatric Investigation Plan.

Background: The European Paediatric Regulation strives to enhance the availability of information regarding the use of medications for children through Paediatric Investigation Plans (PIPs). These plans are mutually agreed upon by sponsors and the Paediatric Committee of the European Medicines Agency (PDCO). However, recent data indicates that a significant number of these PIPs are not fully executed. Additionally, previous studies have suggested that paediatric trials are frequently prematurely halted and remain unpublished, leading to a potential waste of resources and valuable information.

Objectives: This study aimed to assess completion and discontinuation rates of pediatric clinical studies under agreed PIPs. Additionally, it investigated the frequency of posting results from completed studies to the EU Clinical Trial Register (EUCTR) or publishing them in scientific literature.

Methods: We identified all paediatric clinical trials (at least one participant below the age of 18) conducted as part of a PIP and registered in EudraCT from January 2010 to December 2017. For all identified trials, we collected information about involved countries, therapeutic areas, age groups of paediatric participants, study design, and trial status at the end of follow-up (November 2023). Data on the reporting of trial completion were collected from EUCTR, PubMed and Embase.

Results: A total of 751 clinical trials conducted as part of a PIP were identified. The large majority of trials was conducted in children (2 – 11 years of age) or adolescents (12-17 years of age), only few trials 9% included newborn ( < 27 days). The most frequently studied therapeutic area relates to congenital, familial and genetic disorders (20%), followed by infections and infestations (14%). Most trials were randomized (51,3%), controlled (52,1%), designed as blinded studies (54,5%), and the most common trial phase was phase 3 (55,0%). Germany accounted for the largest portion of registered trials within the EU/EEA (40%). Additionally, 46% of the trials were conducted in at least one country outside the EU. 52 trials (6,9%) were prematurely ended and 536 trials (71,4%) were completed after a median follow-up of 10,4 years (IQR 8,6-11,8). Almost all completed trials had reported results at EUCTR or in the scientific literature (97,8%). In addition, it is worth noting that multiple discrepancies within study reporting in EUDRACT were identified.

Conclusions: Preliminary results suggest that instances of discontinuation and nonreporting are rare. Moreover, nearly three-quarters of the clinical trials are completed. However, this study also highlights data deficiencies within the EU Clinical Trials Register (EUCTR), emphasizing the importance of improving the quality of data in the registry.