FDA Releases Guidelines for Cancer Clinical Trials, Aligning With Goals of the Biden Administration


The FDA issued 3 final guidances to industry regarding cancer clinical trials that parallel the goals of President Biden's 2016 Cancer Moonshot initiative.

Three guidances have been finalized and released by the FDA regarding including older adults in cancer clinical trials, conducting first-in-human cancer trials with expansion cohorts, and developing master protocol designs for efficient review of oncology drugs and biologics.

These guidances align with the goals announced by President Biden's administration in regard to his 2016 Cancer Moonshot initiative. With this renewed effort, continued advancement in cancer prevention, detection, research, and patient care are built upon.

The finalizations were based on medical advancements that have been in the field since 2016. The President's new goals include reducing the death rate from cancer by at least 50% over the next 25 years and improving the experience of patients and their families living with cancer. The end goal is ultimately to end cancer as we know it today.

"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 for the FDA's Oncology Center for Excellence, in the press release. "All of these are tenets of Cancer Moonshot's mission." 

The first guidance, “Inclusion of Older Adults in Cancer Clinical Trials” seeks to provide recommendations in regard to adult patients aged 65 and older and their inclusion in cancer clinical trials.

For the purpose of this final guidance, older adults are defined as those aged 65 years and older. The guidance emphasizes the importance of enrolling older adults in early phase trials earlier as it will help to gather information for later trial phases. Additionally, the guidance will help examine potential drug-drug interactions in order to limit trial exclusion within this patient population.

Recommendations for trial design are also included as having an adequate representation of ages in cancer clinical trials will provide more information for later phase studies. Recruitment strategies, information collection, and ways to encourage enrollment of this population are also discussed within this guidance.

The second guidance, “Expansion Cohorts: Use in First-in-Human Clinical Trials to Expedite Development of Oncology Drugs and Biologics,” consists of advice on the design and ways to conduct first-in-human clinical trials. The guidance specifically focuses on trials with multiple expansion cohorts which allow for concurrent accrual of patients into different cohorts in order to assess safety, pharmacokinetics, and anti-tumor activity of first-in-human cancer drugs.

This guidance is intended for researchers and pharmaceutical companies to use trials with expansion cohort design to assess different aspects of a drug in a single clinical trial to efficiently expedite the clinical development of the drug. 

Some recommendations included in the guidance regard the characteristics of products used for consideration for development under a multiple expansion cohort trial, information to include in investigational new drug application submissions which can support designs, when to interact with the FDA while planning and conducting of these studies, and safeguards to the enrolled patients in these expansion cohort studies.

The final guidance, “Master Protocols: Efficient Clinical Trial Design Strategies to Expedite Development of Oncology Drugs and Biologics," provides advice to the sponsors of drugs and biologics for cancer treatment. The guidance includes recommendations for the design of clinical trials, the way they are conducted, and how it should be executed.

The guidance also lays out the ways in which sponsors should interact with the FDA in order to facilitate efficient review and limit risks to patients. Because they allow more than 1 investigational drug or biologic, disease type, or patient population to be evaluated under a single clinical trial structure, these clinical trials will be able to speed the clinical development process of a drug to treat cancer.

According to the FDA, “[t]he standard approach to generating evidence has become more expensive and challenging to execute and, as a result, answers to important clinical questions are often delayed,”

This guidance hopes to protect patient safety and gather information regarding quality data to support drug approval with well-designed master protocols. These aim to provide answers more quickly and efficiently than within traditional clinical trials.


FDA Clinical Trial Guidances Share Biden Administration’s Goals for Advancing Development of Cancer Treatments. News release. FDA; March 1, 2022. Accessed March 2, 2022. https://bit.ly/3tqqKNM

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