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

Abstract 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 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.