Regulatory Focus™ > News Articles > FDA Looks to Recalibrate how it Uses Non-Traditional Data to Support Regulatory Decisions

FDA Looks to Recalibrate how it Uses Non-Traditional Data to Support Regulatory Decisions

Posted 04 October 2013 | By Alexander Gaffney, RAC

The government may be partially shut down and the Federal Register all but empty, but that doesn't mean the US Food and Drug Administration (FDA) isn't still announcing upcoming meetings.

On 4 October 2013, the agency announced it will soon hold a public meeting on "meta-randomized controlled clinical trials for the evaluation of risk to support regulatory decisions."


The meeting, it explained, will present various scientific approaches on how to conduct and evaluate meta-analyses of randomized clinical trials-the gold standard for approvals and the linchpin of most product applications reviewed by FDA.

In the past, FDA has expressed considerable skepticism about the use of meta-analysis to support expanded indications or labeling claims, going as far as to issue Untitled Letters for the use of the data in advertising materials.

But more to the point, the agency said it has realized that while it must often "draw conclusions from imperfect data"-meta-analyses included-there are ways of identifying and evaluating sources of that uncertainty so that imperfect data can be used to better inform a product's overall benefit-risk framework.

Issues Being Discussed

In a report issued earlier this year, FDA indicated that the characterization of uncertainty with respect to the use of data "from sources of varying levels of rigor" would be one of its two top areas of focus during the coming years.

"In contrast to the prospective and highly planned studies of effectiveness, safety findings emerge from a wide range of sources, including spontaneous adverse event reports, epidemiology studies, meta-analyses of controlled trials, or in some cases from randomized, controlled trials," FDA wrote. "A systematic approach that specifies the sources of evidence, the strength of each piece of evidence, and draws conclusions that explain how the uncertainty weighed on the decision, can lead to more explicit communication of regulatory decisions."

FDA said that while it anticipated starting work in FY2013, work would continue in the subsequent years as well. For now, the agency anticipates working in any altered benefit-risk framework starting with new molecular entities submitted through new drug applications (NDA) and original biologics license applications (BLAs) in 2014/2015. Efficacy supplements would be assessed starting in 2016, and all original NDAs would be included starting in 2017.

An Upcoming Meeting

With 2014 fast approaching, FDA is now making moves to put its plan into action, announcing a 25 November 2013 meeting at which it plans to discuss its report and the various issues associated with the use of data from meta analyses.

"The purpose of the workshop is to initiate constructive discussion and information sharing among regulators, researchers, healthcare providers, representatives from the pharmaceutical industry and health care organizations, and others from the general public, about the use of meta-analyses of randomized trials as a tool for safety assessment in the regulation of pharmaceutical products," FDA wrote.

The meeting announcement is rather unusual in that it was posted first to FDA's website, likely indicating that it was unable to utilize its usual venue for meeting announcement: the Federal Register.

Additional meeting materials would be forthcoming, the agency added. Under normal circumstances it usually includes a list of questions it wants industry and public participants to be able to provide insight on.

Meeting Announcement

FDA Report on Drug Benefit-Risk Assessment

Federal Register


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