Pediatric Device Clinical Trials Activity Compared to Drugs and Biologics Trials 1999-2022

Objective This study assessed the state of PMD development by comparing PMD clinical trials to pediatric trials evaluating drugs and biologics, from 1999-2022. We hypothesized changes in numbers and types of PMD trials compared to drugs and biologics represent an indicator of PMD growth. Study Design [www.clinicaltrials.gov][1] was used to identify and quantify both PMD clinical trials and pediatric trials for drugs and biologics. Clinical specialty was also assessed. The institutions included were the seven children’s hospitals primarily affiliated with the FDA PDC grants program between 2018-2023. Results 243 PMD clinical trials were identified based on the year of initiation. The average number of PMD trials initiated per year per institution was 1.5. PMD trials significantly increased (p=0.0083) from 2014 onward compared to pediatric clinical trials for drugs and biologics, which demonstrated no significant change in trial initiation activity. A more than five-fold increase in PMD trials was observed from 2014-2018 compared to previous time periods, and there were 48% more PMD trials from 2019-2022 compared to 2014-2018. PMD trials represented 5% of clinical trials at the institutions studied. Conclusions While clinical trial activity for drug and biologic development remained stable from 1999-2022, initiation of PMD trials significantly increased. The present results suggest that clinical trials growth reflects increased PMD development. Accommodation and promotion of PMD clinical trial activity, which is still relatively small, by relevant programs and policies at the institutional and government levels may foster the advancement of PMD to further address unmet needs. Article Summary This article is an analysis of device trials performed at seven children’s hospitals affiliated with the FDA Consortia grants program between 1999 and 2022. What’s Known on This Subject There have been no prior studies of device trial activity at a cohort of children’s hospitals at academic medical centers. Over the past decade, FDA programs have been initiated to assist stakeholders in advancing the development of pediatric medical devices. What This Study Adds Pediatric device trials account for only 5% of total trials at the institutions studied. Of note, only half of these PMD trials (2.4% of total clinical trials) were sponsored by industry and likely seeking pediatric labeling. ### Competing Interest Statement The authors have declared no competing interest. ### Funding Statement This manuscript is supported by the Food and Drug Administration (FDA) of the U.S. Department of Health and Human Services (HHS) as part of awards totaling $150,000.00 with 0% financed with non-governmental sources. The contents are those of the author(s) and do not necessarily represent the official views of, nor an endorsement, by FDA, HHS, or the U.S. Government. For more information, please visit FDA.gov. The FDA had no role in the design and conduct of this study. ### Author Declarations I confirm all relevant ethical guidelines have been followed, and any necessary IRB and/or ethics committee approvals have been obtained. Yes The details of the IRB/oversight body that provided approval or exemption for the research described are given below: The study used ONLY openly available human data that were originally located at: https://www.clinicaltrials.gov/ I confirm that all necessary patient/participant consent has been obtained and the appropriate institutional forms have been archived, and that any patient/participant/sample identifiers included were not known to anyone (e.g., hospital staff, patients or participants themselves) outside the research group so cannot be used to identify individuals. Yes I understand that all clinical trials and any other prospective interventional studies must be registered with an ICMJE-approved registry, such as ClinicalTrials.gov. I confirm that any such study reported in the manuscript has been registered and the trial registration ID is provided (note: if posting a prospective study registered retrospectively, please provide a statement in the trial ID field explaining why the study was not registered in advance). Yes I have followed all appropriate research reporting guidelines, such as any relevant EQUATOR Network research reporting checklist(s) and other pertinent material, if applicable. Yes All data produced in the present study are available upon reasonable request to the authors * FDA : Food and Drug Administration CDRH : Center for Devices and Radiological Health PMD : Pediatric medical device(s) PDC : Pediatric Device Consortia OOPD : Office of Orphan Products Development CHLA : Children’s Hospital Los Angeles CHOP : Children’s Hospital of Philadelphia CNH : Children’s National Hospital LPCHS : Lucile Packard’s Children’s Hospital Stanford TCH : Texas Children’s Hospital UCSF BCH : University of California San Francisco Benioff Children’s Hospital UPMC CHP : University of Pittsburgh Medical Center Children’s Hospital of Pittsburgh [1]: http://www.clinicaltrials.gov