Children with cancer wait an average of 6.5 years longer than adults to access new targeted drugs, finds study at Dana-Farber/Boston Children’s
May 21, 2019
Researchers call for expanding children’s access to experimental cancer therapies
Cancer drugs approved by the U.S. Food and Drug Administration
(FDA) took a median of 6.5 years to go from the first clinical trial in adults
to the first trial in children, according to a study at the Dana-Farber/Boston
Children’s Cancer and Blood Disorders Center. The study was published in the May issue of 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,”
says Steven G. DuBois, MD, Dana-Farber/Boston Children’s Cancer and Blood
Disorders Center, and the study’s corresponding author. “As a doctor taking
care of young cancer patients, this is tremendously frustrating. If I were a
parent of a child with cancer, I wouldn’t stand for this.”
The team at Dana-Farber/Boston Children’s 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
In that timeframe, 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. Fifteen of 117 drugs
(12.8%) had not yet had a pediatric trial, while 6 of 117 drugs (5.1%) included
children in the initial FDA approval.
showed a median 6.5-year lag between first-in-human and first-in-child clinical
trials, with a range of 0 to 27.7 years.
“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,” says
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.”
In the U.S., the recent RACE for Children Act strengthens the
requirement that new cancer therapies with potential biological relevance to
pediatric cancers be evaluated in children. This study can serve as a benchmark
as this new policy is enacted, says DuBois.
is the corresponding author. Other coauthors are Dylan V. Neel, Harvard Medical
School student, and David S. Shulman, MD, from Dana-Farber/Boston Children’s.
The study was supported by Alex’s Lemonade Stand Foundation and the NIH.