Article
Ashley Woolmore , Dirk Arnold , Jean‐Yves Blay , Christian Buske , Alfredo Carrato , Winald Gerritsen , Marc Peeters , Jesus Garcia‐Foncillas , David Kerr
Timing of first-in-child trials of FDA-approved oncology drugs
Summary
- Aim: To define the lag time between initial human studies and first-in-child clinical trials of oncology agents.
- Methods: Systematic analysis of time from first-in-human trials to first-in-child trials for agents approved by the FDA from 1997 to 2017. Data was sourced from clinical trial registries, literature, and oncology abstracts.
- Results:
- 126 drugs received initial FDA approval for oncology indications between 1997 and 2017.
- 117 non-hormonal agents were used in subsequent analyses.
- 15 of 117 drugs (12.8%) did not have a pediatric trial.
- 6 of 117 drugs (5.1%) had an initial approval that included children.
- Median time from first-in-human to first-in-child trial: 6.5 years (range 0–27.7 years).
- Median time from initial FDA approval to first-in-child trial: -0.66 years (range -43 to +19 years).
- Time intervals were stable across different years of FDA approval, drug classes, and disease indications.
Progress in treating pediatric malignancies over the last forty years has largely been driven by advances in conventional cytotoxic chemotherapy regimens, surgery and radiation. While overall survival for children with cancer now exceeds 80%, outcomes remain poor for many patients. Further, treatment-related morbidity remains high for the majority of children treated with chemotherapy-based regimens. Our understanding of cancer genetics,
molecular drivers of disease and differences between adult and pediatric malignancies continues to grow. In spite of this greater understanding of pediatric cancer biology, few targeted therapies are approved by regulatory agencies for children with cancer.
Opportunities exist to spur pediatric drug development at the level of regulatory bodies, the pharmaceutical industry, and academic clinical investigators. While legislation such as the Pediatric Research Equity Act (PREA) and Best Pharmaceuticals for Children Act (BPCA) in the United States have attempted to mandate and incentivize pharmaceutical companies to develop drugs for children, new strategies are needed to accelerate evaluation of new drugs in children with cancer...
pediatric cancer; phase 1 trials; drug development; targeted therapy; disparity
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