Three Final FDA Guidance Documents Released to Support Cancer Research

The U.S. Food and Drug Administration (FDA) recently issued three final guidance documents regarding cancer clinical trials. The announcement supports the goals of the so-called “Cancer Moonshot” initiative to facilitate research and advances in cancer prevention, diagnosis, and treatment. Although the guidance is directed primarily at industry sponsors, research sites should be aware of them as they may impact areas of patient recruitment, informed consent, and so on.

Inclusion of Older Adults in Cancer Clinical Trials

While much of the national dialogue about diversity in clinical trials has focused on the inclusion of underserved populations, communities of color, and women, the recent FDA guidance turned its attention to the inclusion of older adults — those over 65 years of age — in clinical research. Older adults are more likely the population that will be utilizing cancer treatments, yet they remain underrepresented in clinical trials.

The guidance encourages the enrollment of older adults in earlier phases of clinical trials. It also includes recommendations for trial design, recruitment strategies, and information collection. It also discusses developing and reporting more discrete age groups to encourage enrollment of this historically excluded population.

Expansion Cohorts: Use in First-in-Human Clinical Trials to Expedite Development of Oncology Drugs and Biologics

This guidance provides advice for “First in Human” (FIH) trials, with recommendations for designing and conducting expansion cohort trials. These are trial designs that employ multiple, concurrently accruing subject cohorts, where individual cohorts assess different aspects of the safety, pharmacokinetics, and activity of the studied drug.

The guidance offers considerations for which kinds of interventions are best suited for a multiple expansion cohort design, as well as what information must be included in the new drug application submission. The guidance also addresses issues for institutional review boards (IRBs) and the needed safeguards to protect participants in these expansion cohort trials.

Master Protocols: Efficient Clinical Trial Design Strategies to Expedite Development of Oncology Drugs and Biologics

This guidance document addresses “master protocols.” In contrast to traditional trial designs, where a single drug is tested in a single disease population in one clinical trial, master protocols use a single infrastructure, trial design, and protocol to simultaneously evaluate more than one investigational drug and/or more than one cancer type in multiple sub-studies.

The goal is for investigators and sponsors to address critical research questions more quickly and efficiently.

Each of these guidance documents include possible implications for IRBs and research sites in the start-up and conduct of oncology clinical trials. Over the next few weeks, we will unpack each of them in a more detailed review.