USA – FDA Unveils 5 Guidances on Broadening Cancer Clinical Trial Eligibility

As part of a push to transform clinical trial eligibility criteria that have been accepted over time without a clear scientific or clinical rationale, the US Food and Drug Administration (FDA) on Tuesday published four draft guidance documents on cancer clinical trial eligibility criteria and one final guidance on including adolescents in adult oncology trials.

The four drafts, developed by FDA with input from the American Society of Clinical Oncology and Friends of Cancer Research, focus on minimum age for pediatric patients; patients with HIV, hepatitis B or C viruses; patients with organ dysfunction or prior or current malignancies; and patients with brain metastases.

“Overly restrictive eligibility criteria may slow patient accrual, limit patients’ access to clinical trials and lead to trial results that don’t fully represent treatment effects in the patient population that will ultimately receive the drug,” FDA Commissioner Scott Gottlieb said, noting how the draft guidances offer new recommendations for broadening cancer trial eligibility criteria.

Minimum Age for Pediatric Patients

This seven-page draft guidance explains how sponsors seeking to include pediatric patient populations in adult cancer trials should evaluate pediatric formulations, taking into account the age, size, physiologic condition and the treatment needs of pediatric patients.

The draft notes the types of evidence that could support inclusion of patients from two years of age to under age twelve, clarifying that children under the age of two “may be particularly vulnerable to expected and unanticipated toxicity due to developmental concerns and age-dependent maturation of metabolic enzyme systems and organ function, children < 2 years should not be included in adult cancer trials. In rare instances, infants beyond the neonatal period may be appropriate candidates for select new drugs. However, enrollment of children < 2 years of age is best reserved for exceptional cases and only after consultation with the FDA.”…