What’s lost when pediatric drug trials go unpublished

The need for more clinical trials of drugs in pediatrics has been long discussed, as many drugs are given to children that haven’t been formally tested in the population and information on safety and efficacy are 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 helpful information. A research letter in JAMA Network Open offers a look at the information that is lost when those trials aren’t published.¹

The investigators performed an evaluation of pediatric trials that had been registered in ClinicalTrials.gov. Trials were included if they met the following criteria: used a randomized trial design, registered between January 2014 and June 2016, were trialing a drug intervention in children aged younger than 18 years, and were either completed or discontinued by June 30, 2018. A trial’s results were considered published if they were described in a peer-reviewed medical journal, which investigators found by searching PubMed, GoogleScholar, Embase, and company websites. If no publication was found for a drug trial, the investigators searched for reports on the trial in conference abstracts, press releases, thesis documents, trial registries, and preprint servers. The unpublished reports were examined for findings on mortality, adverse events, and efficacy of the drug intervention.

The investigators found 189 pediatric drug trials. One hundred twenty of the trails had an international trial site and 92 of the trials were funded by academic institutions. After a median follow-up period of 3.6 years, 79 were still unpublished. Those unpublished studies accounted for 8395 of 24,338 pediatric participants. Thirty of trials were discontinued and only 6 of those trials were published. Most common reasons for discontinuing trials included scientific reasons, business decisions, and insufficient patient enrollment. Completed trials were more likely to be published, but only 65.4% were published. Reports on unpublished studies were found for 49 of the 79 unpublished trials. In these trials, 2 included reports of deaths; 14 had reports of serious adverse events; and 31 had nonserious adverse events. Safety or efficacy information was available for 44 of the unpublished trials.

The investigators concluded that a sizeable number of pediatric drug trails were unpublished 2 years after the trial ended. Many of the trials included safety and efficacy findings that remain mostly inaccessible to both clinicians and the scientific community. They urged the creation additional efforts and incentives to promote the timely publication of pediatric drug trials.

Reference

1. Srivastava A, Bourgeois F. Evaluation of publication of pediatric drug trials. JAMA Netw Open. 2021;4(4):e215829. doi:10.1001/jamanetworkopen.2021.5829