The European Medicines Agency (EMEA) and the US Food and Drug Administration (FDA) have concluded their first joint qualification process for biomarkers.

Based on pooled data submitted by the Critical Path Institute’s Predictive Safety Testing Consortium (PSTC), the two agencies qualified the use of seven biomarkers of drug-induced renal toxicity in the context of non-clinical drug development. The data were filed with both the EMEA and the FDA, which conducted a joint evaluation using state-of-the-art standards, the European agency said.

According to a recent white paper by Thomson Scientific, while biomarkers are set to become “one of the major drivers of pharmaceutical research and development in the 21st Century”, commitment from innovators is patchy and regulatory authorities remain conservative about accepting biomarkers as primary evidence for drug approval. Without more determined efforts at validation, the report warned, there is a danger researchers will lose faith and turn their attention elsewhere.

After assessing the biomarker data filed by the PSTC, the EMEA and the FDA concluded that:

- the renal biomarkers submitted were acceptable in the context of non-clinical drug development for detection of acute drug-induced renal toxicity;

- the biomarkers provided information that was “additional and complementary” to the currently available standards;

- the use of renal biomarkers in clinical trials should be considered on a case-by-case basis, “in order to gather further data to qualify their usefulness in monitoring drug-induced renal toxicity in man”.

As the EMEA pointed out, under the C-Path PSTC programme the pharmaceutical industry “has for the first time pooled together data from different companies in order to achieve the critical mass of scientific information” that enabled the European agency and the FDA to qualify the renal toxicity biomarkers.

The PSTC is a public/private partnership between industry, academia and regulators, intended as a neutral body for co-ordinating activities related to biomarker qualification in drug development. It was set up by the non-profit Critical Path Institute two years ago, with the FDA serving in an advisory capacity.

A consortium of eight leading pharmaceutical companies took the unprecedented step of agreeing to share preclinical laboratory data in order to improve the testing of medicines before they reached human clinical trials. The FDA has made biomarkers central to the Critical Path programme, launched in March 2004 with the aim of modernising and streamlining medical product development.

Recently one of the PSTC partners, Novartis, announced it had completed a two-year co-operative research and development agreement (CRADA) with the FDA, yielding a process for defining and qualifying safety biomarkers as well as real-world data to support regulatory decision-making on renal safety.

The relevant data were shared with the PSTC and, together with data generated by other partners in the Consortium, were filed with the FDA and the EMEA as part of a Voluntary Exploratory Data Submission (VXDS).

Based on the agency’s experience with the first joint qualification process, the EMEA’s Committee for Medicinal Products for Human Use (CHMP) recently published a draft document, Biomarkers Qualification: Guidance to Applicants, which is out for consultation until 30 June 2008.