FDA: Data from multiregional cancer trials must be applicable to US citizens

Cancer drug developers seeking US approval must ensure trial data is applicable to US patients, according to the U.S. Food and Drug Administration (FDA), which has warned data from multiregional clinical trials (MRCTs) may not be appropriate to support a regulatory decision.

The agency included the recommendation in draft guidelines published in September - downloadable here - citing declining trial participation rates in the US and a reliance on clinical data generated elsewhere as a concern.

“There have been decreasing proportions of US participants included in oncology MRCTs. This may limit the ability to determine if an observed treatment outcome in the study is consistent between US enrolled participants and the overall study population in the MRCT.”

The agency pointed out that because differences in the prevalence, presentation, causes, or severity of a cancer exist in different countries, the findings from any study, including sites in those countries, may be hard to generalize.

“These differences can impact how the data can be interpreted in the context of the US population and US medical practice. In addition, the distribution of demographic or clinical characteristics of participants enrolled in these trials may differ significantly from the US population such that foreign clinical data may not be appropriate to support an FDA regulatory decision,” the agency said.

US involvement

To mitigate these differences, the FDA advises sponsors planning an MRCT to include a representative number of US citizens and study sites in the program.

“[The] FDA recognizes that a thoughtfully designed multiregional CDP [clinical development program] can facilitate enrollment of foreign participants with diverse genetic ancestral backgrounds that may be scientifically and clinically relevant to the US population.

“However, because there may be limitations or challenges in characterizing an individual on the basis of genetic ancestry, sponsors should aim to enrol an adequately representative subgroup of US participants in an MRCT to allow for a robust assessment of the drug’s safety and efficacy in this subgroup relative to the overall MRCT study population.”

The agency also noted that “sponsors should prospectively plan the distribution of clinical sites in an MRCT to achieve enrolment of a population that adequately represents the US population affected with the cancer indication being studied.”

The draft guidelines are open for public comment for the next 60 days.

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