FDA Issues 3 Final Pieces of Guidance Regarding Oncology-Related Clinical Trials

The agency addresses the inclusion of older individuals in cancer-studies, the designing and conducting of multiple expansion cohorts, and the expedited development of biologics and drugs.

The FDA has issued 3 pieces of final guidance regarding cancer clinical trials, which parallels the goal of President Joseph R. Biden’s effort to renew and build upon the 2016 Cancer Moonshot initiative, the agency said in a statement.

The Cancer Moonshot initiative intends to continue advancement in cancer detection, prevention, patient care, and research.

Biden’s new goals are to reduce the death rate from cancer by at least 50% over the next 25 years and to improve the experience of individuals and their families living with and surviving cancer.

“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 of the agency’s Oncology Center for Excellence, said in the statement.

The first piece of guidance released by the FDA addresses the inclusion of older individuals in cancer-related clinical trials. It includes recommendations to sponsors and institutional review boards to include individuals who are aged 65 years and older in the clinical trials for cancer drugs.

The recommendation states to include older individuals in early phases of clinical trials, if appropriate, so that the information can better help inform decisions about later-phase studies. It also includes recommendations for developing and reporting more discrete age groups, information collection, recruitment strategies and trial sizecollection.

This should help encourage more enrollment, especially, because different patient populations could have different drug responses and toxicities.

Older individuals often have other conditions or diseases or take other medications that could affect efficacy or the severity of adverse events.

The second piece of guidance provides advice on designing and conducting trials that include multiple expansion cohorts. This would allow for concurrent accrual of individuals into different cohorts that help assess different aspects of cancer drugs, such as anti-tumor activity, pharmacokinetics, and safety.

These recommendations include guidance on the characteristics of drug products that are best suited for development under multiple expansion cohorts, information about investigational new drug application submissions to include support of the cohorts, safeguards to protect individuals enrolled in the cohorts, and when to interact with the FDA on planning and conducting these cohorts.

Finally, the final piece of guidance addresses a master protocol design for the expedited development of oncology biologics and drugs. The master protocol design includes information about what sponsors should submit to the FDA as part of these trial designs.

Additionally, it also directs sponsors about how they should interact with the FDA to facilitate an efficient review and mitigate risk to individuals.

The clinical trials are designed to expedite the clinical development of drugs to treat cancer and can help allow more than 1 disease type, more than 1 investigational biologic or drug, or more than 1 patient population, to be evaluated under a single clinical trial structure.


FDA clinical trial guidances share Biden Administration’s goals for advancing development of cancer treatments. FDA. News release. March 1, 2022. Accessed March 1, 2022. https://www.fda.gov/news-events/press-announcements/fda-clinical-trial-guidances-share-biden-administrations-goals-advancing-development-cancer

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