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The Predictive Safety Testing Consortium: safety bio-markers, collaboration, and qualification

John-Michael Sauer, Elizabeth G Walker, Amy C Porter

Abstract

The Predictive Safety Testing Consortium (PSTC) is one of nine consortia comprising the Critical Path Institute (C-Path), a non-profit organisation launched in 2005 and dedicated to playing the role of a catalyst in the development of new approaches that advance medical innovation and regulatory science. C-Path achieves this by lead-ing teams that share data, knowledge and expertise resulting in sound, consensus-based science. PSTC is a unique, public-private partnership that brings pharmaceutical companies together to share and validate safety testing methods under the advisement of worldwide regulatory agencies, including the US Food and Drug Administration (FDA), the European Medicines Agency (EMA), and the Japanese Pharmaceuticals and Medical Devices Agency (PMDA). The eighteen corporate members of PSTC share a common goal: to find improved safety testing methods and approaches utilizing fluid-based safety biomarkers which accurately predict drug-induced tissue injury. Specifically, the primary goal of PSTC is the qualification of novel translational safety biomarkers for use in early clinical trials in order to ena-ble safer investigations and development of new drug candidates. This manuscript describes the critical importance of improved safety biomarkers to the drug development process and the present state of the biomarker qualification process with regulatory agencies. In addition, the work that the PSTC and its collaborative partners have done and con-tinue to do to identify and qualify more selective and specific safety biomarkers is highlighted. Finally, successes in-cluding the recently adopted regulatory Letter of Support and ongoing efforts to better define the regulatory qualifica-tion process and an integrated translational safety strategy are also discussed.

Keywords

safety biomarker; biomarker qualification; letter of support; translation; target organ; therapeutic index; drug development tool; context of use

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DOI: http://dx.doi.org/10.18063/jmds.v1i1.116
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