Timing of first-in-child trials of FDA-approved oncology drugs

Aim: The lag time between initial human studies of oncology agents and the first-in-child clinical trials of these agents has not been defined. Methods: We conducted a systematic analysis of time from first-in-human trials to first-in-child trials (age of eligibility < 18 years) of agents first approved by the US Food and Drug Administration (FDA) for any oncology indication from 1997 to 2017. We used clinical trial registry data, published literature and oncology abstracts to identify relevant trials and start dates. Results: From 1997 to 2017, 126 drugs received initial FDA approval for an oncology indication. Of these, 117 were non-hormonal agents used in subsequent analyses. Fifteen of 117 drugs (12.8%) did not yet have a paediatric trial, and six of 117 drugs (5.1%) had an initial approval that included children. The median time between the first-in-human trial and first-in-child trial was 6.5 years (range 0-27.7 years). The median time from initial FDA approval to the first-in-child clinical trial was -0.66 years (range -43 to +19 years). These values were stable regardless of year of initial FDA approval, drug class and initial approved disease indication. Conclusion: The median lag time from first-in-human to first-in-child trials of oncology agents that were ultimately approved by FDA was 6.5 years. These results provide a benchmark against which to evaluate recent initiatives designed to hasten drug development relevant to children with cancer. (C) 2019 Elsevier Ltd. All rights reserved.