FDA Unveils New Cancer Trial Guidance to Back ‘Moonshot’ Initiative

The FDA issued three final guidances to industry for cancer clinical trials that align with the goals set by President Biden in the relaunch of his “Cancer Moonshot” initiative, which was announced last month.

According to the White House, the goal of this revamped endeavor is to cut the age-adjusted death rate from cancer by a least 50% over the next 25 years, and improve on the experiences of people living with and surviving cancer, as well as those of their families and caregivers.

“With today’s actions the FDA is recommending important principles that involve addressing inequities, targeting the right treatments to the right patients, speeding progress against the most deadly and rare cancers, and learning from the experience of all patients,” said Richard Pazdur, MD, director of the FDA’s Oncology Center of Excellence, in a press release. “All of these are tenets of Cancer Moonshot’s mission.”

The first guidance, “Inclusion of Older Adults in Cancer Clinical Trials,” provides recommendations on the inclusion of older adults (65 years and older) — with specific emphasis on including adults over the age of 75 — in clinical trials of cancer drugs in order to ensure that this historically under-represented group is adequately studied.

The guidance includes recommendations for trial design, recruitment strategies, data collection, and developing and reporting more discrete age groups to encourage enrollment among older adults.

The FDA noted that it is important to include older adults in clinical trials due to the many differences between this age group and younger adults, such as comorbidities and use of certain medications that could affect the study drug’s efficacy or the incidence of adverse events.

The second guidance, “Expansion Cohorts: Use in First-in-Human Clinical Trials to Expedite Development of Oncology Drugs and Biologics,” provides recommendations on designing and conducting trials with multiple expansion cohorts that allow for concurrent accrual of patients into different cohorts in order to assess safety, pharmacokinetics, and antitumor activity of first-in-human cancer drugs.

According to the FDA, trials with expansion cohort designs can assess many different aspects of a drug in a single clinical trial and expedite the clinical development of the drug more efficiently.

The third guidance, “Master Protocols: Efficient Clinical Trial Design Strategies to Expedite Development of Oncology Drugs and Biologics,” directs trial sponsors on the design and conduct of clinical trials that are intended to evaluate multiple drugs, disease types, and/or patient populations in multiple substudies, with the goal of accelerating drug development.

It also advises sponsors on how they should interact with the FDA to ensure efficient review and prevent risks to patients.

“Because of the complexity of these trials and the potential regulatory impact, it’s important that the FDA is providing this guidance on how to conduct well designed trials that protect patient safety and obtain quality data needed to support drug approval,” the agency noted.