393 - Loss of Participants in Neonatal Clinical Trials

Poster Number: 393
Publication Number: 393.134

Allison N. Lyle, SEATTLE CHILDREN'S HOSPITAL, Seattle, WA, United States; Henna Shaikh, Perelman School of Medicine at the University of Pennsylvania, Caleta, La Romana, Dominican Republic; Megan Gray, University of Washington School of Medicine, Seattle, WA, United States; Elliott Weiss, University of Washington School of Medicine, Seattle, WA, United States

Background: Clinical trials are the bedrock for safe and effective therapeutics, yet many neonatal trials fail due to insufficient participation. Infants not included in research may differ from those who are included in meaningful ways, biasing the sample and limiting the generalizability of findings.

Objective: To describe the proportion of eligible neonates that were included in US trial results from a single calendar year as well as the reasons for non-inclusion.

Design/Methods: We performed a systematic search of Cochrane CENTRAL to retrieve articles meeting the following inclusion criteria: full-length, peer-reviewed articles describing clinical trial results in at least 20 human neonates from US NICUs, published in English, and added to Cochrane CENTRAL in 2019. Retrieved articles were screened for inclusion by two independent researchers.

Results: Twenty-two articles met inclusion criteria. Sixteen articles reported the number of neonates eligible for participation. Drawing from these, we found an aggregate 44.3% of eligible neonates included in reported results (2306 included out of 5185 eligible). Among all eligible neonates, most reasons for not being included in results were classified as “modifiable” or “potentially modifiable” by the research team. Modifiable reasons included factors related to study logistics, such as unmet staffing needs or failure to appropriately provide or collect data on the intervention for enrolled patients (11.5%), or “other” (4.1%). Potentially modifiable reasons included parents who declined (25.4%) or were not approached (3.4%), clinicians who declined (2.5%), patients participating in another study (2.3%), and consent withdrawn (0.2%).Conclusion(s): This synthesis of reporting on eligible neonates included and not included in clinical trials may help future trialists designing recruitment protocols. We found serious gaps in reporting of reasons for non-inclusion of eligible infants. Reporting must be improved so that clinical trialists know which potential participants are being missed. Improved reporting can lead to a better understanding of limits to the generalizability of research findings. Further, as we found that most non-inclusion was due to modifiable or potentially modifiable factors, our report suggests potential targets for the development of interventions to optimize broader inclusion in neonatal clinical trials.
Figure 1Aggregate flow of participants included and not included in neonatal clinical trials.
Figure 2Percent of eligible patients not included in results of individual neonatal trials.