On September 16, the U.S. Food and Drug Administration (FDA) issued a draft guidance to provide sponsors with recommendations for conducting multiregional clinical trials in support of applications for drugs intended to treat cancer, according to a notice published in the Federal Register.
Background
A multiregional clinical trial is a study that is conducted in more than one region under a single protocol, with the region defined as a geographic region, country, or regulatory region.
There have been decreasing proportions of U.S. participants included in multiregional oncology clinical trials—potentially limiting the ability to determine whether an observed treatment outcome in the study is consistent between enrolled U.S. participants and the overall study population in the trial. For instance, known differences in the prevalence, presentation, causes, or severity of a cancer type may exist across countries or regions. These differences can impact how the data may 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. As a result, foreign clinical data may not be appropriate to support an FDA regulatory decision.
The new draft guidance seeks to expand on current principles for multiregional clinical trials and, when finalized, is expected to provide additional recommendations to improve the planning, design, conduct, and analysis of future multiregional oncology clinical trials. The guidance may also 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 United States and to U.S. standard oncologic care.
Conclusions
“The FDA encourages sponsors to pursue multiregional clinical trials but stresses that such trials should be conducted within the appropriate context. 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 oncologic care,” emphasized Richard Pazdur, MD, Director of the Oncology Center of Excellence and Acting Director of the Office of Oncologic Diseases at the Center for Drug Evaluation and Research at the FDA. “The new draft guidance, when finalized, will not only support the agency’s review of data generated from multiregional clinical trials, but [will] also help sponsors improve the generalizability and applicability of results from these trials to the U.S. population and to U.S. medical practice,” he concluded.
Disclosure: To view the full draft guidance, visit federalregister.gov.
