FDA Issues Draft Guidance on Conducting Multiregional Clinical Trials in Oncology

The U.S. Food and Drug Administration issued a draft guidance for industry that, when finalized, will provide sponsors with recommendations for conducting multiregional clinical trials (MRCT) in support of applications for drugs intended to treat cancer. An MRCT is a trial that is conducted in more than one region under a single protocol, with region defined as a geographical region, country, or regulatory region.

“The FDA encourages sponsors to pursue multiregional clinical trials, but stresses that such trials should be conducted within the appropriate context,” said Richard Pazdur, M.D., director of the FDA’s Oncology Center of Excellence and acting director of the Office of Oncologic Diseases in the FDA’s Center for Drug Evaluation and Research. “It is important that data from multiregional clinical trials are applicable to patients in the United States who may use the drug and our current standards of oncological care. The new draft guidance, when finalized, will not only support the agency’s review of data generated from multiregional clinical trials, but also help sponsors improve the generalizability and applicability of results from these trials to the U.S. population and to U.S. medical practice.”

There have been decreasing proportions of U.S. participants included in oncology MRCTs. This may limit the ability to determine if an observed treatment outcome in the study is consistent between U.S. enrolled participants and the overall study population in the MRCT. For example, known differences in the prevalence, presentation, causes, or severity of a cancer may exist across countries or regions. These differences can impact how the data can be interpreted in the context of the U.S. population and U.S. medical practice. In addition, the distribution of demographic or clinical characteristics of participants enrolled in these trials may differ significantly from the U.S. population such that foreign clinical data may not be appropriate to support an FDA regulatory decision.

The new draft guidance, “Considerations for Generating Clinical Evidence from Oncology Multiregional Clinical Development Programs,” expands on current principles for MRCTs and, when finalized, will provide additional recommendations to improve the planning, design, conduct and analysis of future oncology MRCTs. It also will aid sponsors in planning multiregional clinical development programs that consider the agency’s evaluation of trial results that can be applied to the intended use population in the U.S., and to U.S. standard oncological care.

Comments on the draft guidance must be submitted within 60 days after publication in the Federal Register to Regulations.gov to ensure the agency considers them.

• Considerations for Generating Clinical Evidence from Oncology Multiregional Clinical Development Programs

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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, radiation-emitting electronic products, and for regulating tobacco products.