FDA Issues 3 Cancer Clinical Trial Final Guidance Documents

FDA recently issued three final guidance documents to the industry regarding cancer clinical trials that parallel the goals of President Biden’s effort to renew and rebuild upon his 2016 Cancer Moonshot initiative.

Biden’s goal for the iteration is to reduce the death rate from cancer by at least 50% over the next 25 years, improve the experience of people and their families living with and surviving cancer, and end cancer as we know it.

The first guidance, “Inclusion of Older Adults in Clinical Trials,” provides recommendations to sponsors and institutional review boards for older adult patients aged 65 years and older in clinical trials for cancer treatment.  

The guidance recommends enrolling older adults in early phase studies of cancer clinical trials to obtain information that better informs later phase studies. It also includes recommendations for trial design, recruitment strategies, information collection, and developing and reporting more discrete age groups to encourage enrollment for this population.

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

Pharmaceutical companies and researchers can use trials with expansion cohort design to assess different aspects of drugs in a single clinical trial and efficiently expedite drug development.

Additionally, the guidance provides the FDA’s recommendations regarding characteristics of drug products best suited for development under multiple expansion cohort trials, information to include in investigational new drug applications, and submissions to support the design of individual expansion cohorts.

Finally, the “Master Protocols: Efficient Clinical Trial Design Strategies to Expedite Development of Oncology Drugs and Biologics” guidance addresses master protocol design, including information on what sponsors should submit to the FDA as part of trial design approaches.

It also directs how sponsors should interact with the FDA to facilitate review and mitigate patient risks. These clinical trials can help expedite the clinical development to treat cancer, as they allow more than one investigational drug or biologic, more than one disease type, or more than one patient population to be evaluated under a single trial structure.

“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,” Richard Pazdur, MD, director for the FDA’s Oncology Center for Excellence, said in the announcement. “All of these are tenets of Cancer Moonshot’s mission.”