The U.S. Food and Drug Administration (FDA) has issued draft guidance for multiregional clinical trials, which include pivotal trials, conducted in more than one geographical region, country, or regulatory region under a single protocol.
The guidance to be published September 17 is intended for sponsors who are planning global oncology clinical development programs for drugs to support a marketing application. The FDA emphasized that evidence generated should be derived from study populations that enable the results to be interpretable in the context of U.S. patients with the disease or condition and U.S. medical practice.
"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," said Dr. Richard Pazdur in an FDA statement. Pazdur is 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.
"The new draft guidance [Considerations for Generating Clinical Evidence from Oncology Multiregional Clinical Development Programs], when finalized, will not only support the agency’s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," Pazdur said.
Known differences in the prevalence, presentation, causes, or severity of a cancer may exist across countries or regions, the FDA further explained. 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.
FDA is providing more detailed recommendations to support the safe and effective use of cancer drugs in the U.S. population. The guidance will expand on principles described in FDA’s existing guidance documents but is not binding, FDA noted. Comments on the draft guidance must be submitted by November 16.
