June 25, 2019
3 min read

FDA-approved cancer drugs take more than 6 years to reach children

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Steven G. DuBois, MD
Steven G. DuBois

FDA-approved oncology agents took a median 6.5 years to proceed from first-in-human to first-in-child clinical trials, according to research published in European Journal of Cancer.

Additionally, only six of 117 drugs in the systematic analysis of oncology agents approved by the FDA between 1996 and 2017 had initial pediatric indications.

“There have been several recent initiatives to improve early access to novel agents for children with cancer,” Steven G. DuBois, MD, associate professor of pediatrics at Harvard Medical School and director of experimental therapeutics for pediatric oncology at Dana-Farber Cancer Institute, told HemOnc Today.

“To my knowledge, our field has lacked data that could be used to understand if those initiatives are working,” DuBois added. “Our research helps to fill that gap.”

DuBois, one of the study co-authors, told HemOnc Today that the 6.5-year lag between first-in-human and first-in-children trials is time lost that could be spent treating patients with proven therapies. All the agents included in his group’s study received FDA approval, “so these are real medicines and not drugs that have failed to show promise as anticancer therapies,” he said.

DuBois and colleagues complied an FDA-approved oncology drug list using online reference materials and the FDA’s Cancer Approvals & Safety Notifications website.

The researchers used other online sources, including the ASCO abstract database, clinicaltrials.gov and PubMed, to determine the start dates of corresponding clinical trials.

Time in years between the first-in-human trial for a drug and the first trial with eligibility for patients aged younger than 18 years served as the study’s primary endpoint. The analysis included nonhormonal drugs only.

Researchers identified 126 drugs initially approved by the FDA for an oncology indication between 1997 and 2017.

Nearly half (47%) of FDA-approved oncology drugs were small molecules, 22% were antibodies and 14% were conventional cytotoxic agents. Nine of the drugs were hormonal modulators that were excluded from the study, leaving 117 drugs for final analysis.

The median time between the first-in-human clinical trial and a first-in-child trial was 6.5 years (range, 0-27.7). Fifteen of the 117 drugs (12.8%) had not yet been subject to a first-in-children clinical trial, and six of the drugs (5.1%) had initial pediatric indications, including five indicated for acute lymphoblastic leukemia, which also affects adults.

Twenty-seven (26%) of the 102 first-in-children clinical trials were industry sponsored, whereas the remaining were sponsored by the public sector, academia or cooperative interest groups.


“We expected a significant delay between first-in-human trials and first-in-child trials, although the degree of delay was surprising because these are actual cancer medicines,” DuBois told HemOnc Today.

The median time from initial FDA approval of an oncology agent until it was tested in a first-in-child trial was –0.66 years (range, –43 to 19).

When asked what could be done to encourage more trials that include pediatric populations sooner in the development process, DuBois told HemOnc Today that the recently passed RACE Act should help reduce the time lag between when drugs are available for adults and when they are available for children.

The RACE (Research to Accelerate Cures and Equity) for Children Act of 2017 is part of the FDA Reauthorization Act to amend the Pediatric Research Equity Act (PREA), and it gives the FDA authority to require that companies developing targeted cancer drugs conduct pediatric studies.

“That legislation will be implemented as of August 2020, and we will be eager to track this same metric (time from first-in-human to first-in-child trial) in the RACE Act era,” DuBois said.

“Children with cancer need access to innovative therapies. Our study focused on access via clinical trials. If patients cannot access these therapies via clinical trials or as an approved indication, then clinicians turn to off-label prescribing or access via single-patient protocols. These approaches allow the patient to be treated, but they do not necessarily contribute broader knowledge to our field.” – by Drew Amorosi

For more information:

Steven G. DuBois, MD , can be reached at Dana-Farber Cancer Institute, 450 Brookline Ave., Boston, MA 02215, email: steven_dubois@dfci.harvard.edu.

Reference: FDA. Pediatric Research Equity Act (PREA). Available at: www.fda.gov/drugs/development-resources/pediatric-research-equity-act-prea. Accessed on June 24, 2019.


Disclosures: DuBois reports fees for consultant/advisory board roles with Loxo Oncology and travel expenses from Loxo Oncology and Roche/Genentech. The other authors report no relevant financial disclosures.