August 10, 2018
“As part of our ongoing efforts to advance more efficient ways to develop medical products, the FDA is encouraging pioneering new ways that innovators can modernize the way they conduct clinical trials,” said FDA Commissioner Scott Gottlieb, M.D. “The approach we’re describing in new guidance today is to help innovators to evaluate drugs in trials that are potentially lower cost, more efficient, and could enable us to learn more about the safety and efficacy when compared to traditional trial designs. We’ve outlined how drug developers can use an innovative seamless trial design in early stages of oncology drug development – specifically, the first time they’re tested in humans – that compresses the traditional three phases of trials into one continuous trial, called an expansion cohort trial. A lot of the time and cost of clinical development is spent waiting in between the start and end of the phases of trials. Expansion cohort trials can bring efficiency to drug development, potentially reducing development costs and time. These clinical trial improvements can help ensure that innovative new therapies can be advanced efficiently to patients confronting a cancer diagnosis.”
The draft guidance for industry, Expansion Cohorts: Use in First-In-Human Clinical Trials to Expedite Development of Oncology Drugs and Biologics, provides advice on designing and conducting adaptive trial designs in which pharmaceutical companies and researchers can assess many different aspects of a drug in development in a single clinical trial while enrolling the minimum number of study participants necessary to obtain this information.
This guidance provides FDA’s recommendations regarding: (1) characteristics of drug products best suited for consideration for development under a multiple expansion cohort trial; (2) information to include in investigational new drug application submissions to support the design of individual expansion cohorts; (3) when to interact with FDA on planning and conduct of multiple expansion cohort studies; and (4) safeguards to protect patients enrolled in these expansion cohort studies.
The FDA, an agency within the U.S. Department of Health and Human Services, protects the public health by assuring the safety, effectiveness, and security of human and veterinary drugs, vaccines and other biological products for human use, and medical devices. The agency also is responsible for the safety and security of our nation’s food supply, cosmetics, dietary supplements, products that give off electronic radiation, and for regulating tobacco products.