Median Lag Time for First-in-Human to First-in-Child Oncology Trials
Cancer drugs approved by the U.S. Food and Drug Administration (FDA) took a median of 6.5 years to advance from the first clinical trial in adults to the first trial in children, according to a study published by Neel et al in the European Journal of Cancer.
“Despite knowing that these agents are effective anticancer drugs, it’s taking too long to even start studying these therapies in children,” said corresponding author Steven G. DuBois, MD, of Dana-Farber/Boston Children’s Cancer and Blood Disorders Center, in a statement.
Analysis
Researchers conducted a systematic analysis of the time from first-in-human trials to first-in-child trials of agents first approved by the FDA for any oncology indication from 1997 to 2017. The investigators utilized clinical trials registry data, published literature, and oncology abstracts to identify relevant trials and start dates.
Findings
In that time frame, 126 drugs received initial FDA approval for an oncology indication. After excluding hormonal modulators (not relevant to children’s cancers), 117 agents remained for analysis. Of 117 drugs, 15 (12.8%) had not yet had a pediatric trial, whereas 6 (5.1%) included children in the initial FDA approval.
The data showed a median 6.5-year lag between first-in-human and first-in-child clinical trials, with a range of 0 years to 27.7 years. The median time from initial approval by the FDA to the first-in-child clinical trial was −0.66 years (range = −43 to +19 years). These values were stable, regardless of the year of initial approval by the FDA, the class of treatment, and initial approved disease indication.
“Some may argue that this lag is appropriate to ensure safety of a vulnerable pediatric population and to only study agents in children that are on a path to FDA approval, based upon activity in adults with cancer,” said Dr. DuBois. “Others may argue that this lag is too long for children with life-threatening diseases, and that some agents that fail in adult indications may nevertheless prove to be important drugs for pediatric indications.”
The authors concluded, “These results provide a benchmark against which to evaluate recent initiatives designed to hasten drug development relevant to children with cancer.”
Disclosure: For full disclosures of the study authors, visit ejcancer.org.
The content in this post has not been reviewed by the American Society of Clinical Oncology, Inc. (ASCO®) and does not necessarily reflect the ideas and opinions of ASCO®.
