By Gary Evans, Medical Writer

IRB members have until Aug. 3, 2018, to submit comments on FDA draft guidance that would open adult oncology clinical trials to children ages 12 to 17 years.

“The purpose of this draft guidance is to provide the pharmaceutical industry, clinical investigators, and institutional review boards with information to facilitate the inclusion of adolescent patients in relevant adult oncology clinical trials,” the FDA stated.1

The guidelines seek comment on three principal areas:

• appropriate criteria for inclusion of children in adult trials at various stages of drug development;

• considerations for dosing, pharmacokinetic evaluations, safety monitoring;

• ethical requirements.

“Although most cancers in children and adults are distinctly different entities, there are some diseases that occur in both and span the adolescent age groups,” said FDA Commissioner Scott Gottlieb, MD. “If there’s no evidence that an investigational drug might have exaggerated toxicity in younger patients, then we’re encouraging sponsors to enroll adolescents into disease-appropriate trials.”

Speaking June 2, 2018, at the American Society of Clinical Oncology meeting in Chicago, Gottlieb said the move is part of a general trend to include more “underrepresented” research subjects.

“Since the pharmacological parameters of adults and adolescents for most agents are comparable, early access to innovative drugs is warranted where there’s proof of principle and adequate dosing information to maximize potential for clinical benefit,” he said.

Under ethical considerations, the FDA draft guidance states that “Under 21 CFR 50.50, IRBs reviewing adult oncology clinical trials that allow for the enrollment of adolescents must ensure that the provisions of 21 CFR part 50, subpart D, Additional Safeguards for Children in Clinical Investigations, and, specifically, 21 CFR 50.52, clinical investigations involving greater than minimal risk but presenting the prospect of direct benefit to individual subjects, are satisfied before approving the studies.”

The FDA mission to balance risk and access is not a “zero-sum” game, Gottlieb said.

“People try to paint any change we make in our regulatory policy, or to the policy requirements we impose, as a binary choice between speed and safety,” he said. “That’s a false dichotomy. This isn’t a zero-sum game.”

Oncology research is leading the “precision medicine revolution,” with FDA approving 16 new cancer drugs and biologics in 2017. However, “the generalizability of traditional clinical trials to real-world patients at the point of care is increasingly hard,” he said.

“We know that traditional eligibility criteria often exclude the very patients most likely to be treated once the drug is on the market: the elderly, patients with poor performance status, organ dysfunction, brain metastasis, or other comorbidities,” he said. “In 2018, a cancer patient’s hope for recovery shouldn’t hinge on their socioeconomic status or a ZIP code lottery.”


1. FDA. Considerations for the Inclusion of Adolescent Patients in Adult Oncology Clinical Trials: Draft Guidance for Industry. Fed Reg June 4, 2018. Available at: