FDA issues guidance on multiple end-point cancer trials

Nov 21, 2018
FDA issues guidance on multiple end-point cancer trials

Reassessing how we “do” clinical research will make sure new drugs make it to the people who need them as soon as possible.

The Food and Drug Administration (FDA) has issued draft guidance on the use of expansion cohort trials – in which researchers assess more than one aspect of a drug during a single study using the minimum number of patients.

Speaking about the oncology-specific guidelines on the agency’s website, Dr. Richard Pazdur said: “Over the past decade, advances in the understanding of cancer biology have led to the development of targeted treatments that are more effective than the chemotherapies of the past century.”

“These therapies are demonstrating response rates large in magnitude or response durations prolonged in early trials, or both. Patient demand to enter these trials has increased, and so have calls to expedite the drug development and approval processes, all while maintaining high standards for safety and efficacy.”

Traditionally, clinical trials have been conducted in phases with one or two main objectives per study, he explained.

Phase one studies help determine safety and dosing for future trials, while phase two studies provide preliminary evidence of safety and activity in a single setting. All this takes time and money.

Trials with multiple expansion cohorts, however, can be “inherently more efficient,” which speeds up the drug development process. They allow for multiple questions to be asked in a single trial. This is made possible by designing them to be flexible enough to be amended as new objectives are identified.

The guidelines set out how researchers and pharmaceutical companies can conduct such trials in a cost-effective and timely fashion, while maintaining the progress that has been made to date.

In 2017, the FDA approved 16 new cancer treatments, including the first two CAR-T cell therapies, as well as 30 new indications for existing cancer drugs and biologics, said Dr. Pazdur.

The first cancer treatment that works based on a genetic feature of a tumor, rather than where in the body it originated, and the first two oncology biosimilars were also approved last year.

As a result of these and other developments, the overall cancer death rate in the United States fell by 25% between 1990 and 2014.1 And by 2024, there will be an estimated 19 million people in the country who have survived cancer.1

Dr. Pazdur said: “The principal advantage of expansion cohort trials is efficiency in drug development, with the goal of making highly effective drugs widely available to the public as quickly as possible.

“Well-designed and well-conducted clinical trials help ensure patient safety while also obtaining quality data that may support drug approval. This new draft guidance describes our proactive steps to help industry design clinical trials for today’s highly complex cancer therapies.”

To read the guidance, please click here.


Richard Pazdur (August 14, 2018). FDA Advances Efficient Approaches to Designing and Conducting Cancer Clinical Trials. [blog post]. Available from https://blogs.fda.gov/fdavoice/ (accessed November 2018).

FDA issues guidance on multiple end-point cancer trials

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