The need for more clinical trials of pediatric drugs has long been debated, as many drugs are given to children who have not been formally tested in the population and safety and efficacy information is not available to clinicians making medication choices. On the other hand, there are clinical trials of drugs in pediatric populations that are never published and result in the loss of potentially useful information. A research letter in JAMA Network Open offers a look at the information that will be lost if those studies are not published
The researchers conducted a review of pediatric studies registered in ClinicalTrials.gov. Trials were included if they met the following criteria: they used a randomized trial design, registered between January 2014 and June 2016, tested a drug intervention in children under the age of 18, and were completed or discontinued on June 30, 2018. A The results of The studies were considered published if they were described in a peer-reviewed medical journal, which researchers found by searching PubMed, GoogleScholar, Embase, and company websites. When no publication was found for a drug trial, the researchers looked for reports about the trial in conference summaries, press releases, dissertation papers, trial registers, and preprint servers. The unpublished reports were examined for findings on mortality, adverse events, and efficacy of the drug intervention.
The researchers found 189 pediatric drug studies. One hundred and twenty of the routes had an international trial site and 92 of the trials were funded by academic institutions. After a median follow-up of 3.6 years, 79 were still unpublished. Those unpublished studies accounted for 8,395 of 24,338 pediatric participants. Thirty of the studies were discontinued and only 6 of those studies were published. The most common reasons for discontinuation of studies included scientific reasons, business decisions, and insufficient enrollment of patients. Completed studies were published more often, but only 65.4% were published. Reports were found on unpublished studies for 49 of the 79 unpublished studies. In these studies, 2 contained reports of deaths; 14 had reports of serious adverse events; and 31 had non-serious adverse events. Safety or efficacy information was available for 44 of the unpublished studies.
The researchers concluded that a significant number of pediatric drug traces were not published two years after the study ended. Much of the studies include safety and efficacy findings that remain largely inaccessible to clinicians and the scientific community alike. They insisted on creating additional efforts and incentives to promote the timely publication of pediatric drug studies.
1. Srivastava A, Bourgeois F. Evaluation of the publication of pediatric drug studies. JAMA Netw Open. 2021; 4 (4): e215829. doi: 10.1001 / jamanetworkopen.2021.5829