In 2004, FDA and EMA laid the groundwork for joint-agency biomarker reviews when they developed a framework called the Voluntary Exploratory Data Submission review process, which allowed public-private partnerships (PPPs) such as C-Path to submit a single biomarker data application to both regulatory agencies, and then to meet jointly with scientists from both agencies to discuss it in detail and to address additional scientific questions posed by the regulators. Each regulatory agency would then review the application separately and make independent decisions on use of the new biomarkers.
In 2007, C-Path’s Predictive Safety Testing Consortium (PSTC) became the first consortium to utilize this process, when it submitted biomarker data applications for seven nonclinical kidney safety biomarkers. Merck & Co., a PSTC member in the United States, and Novartis AG, a European member of the PSTC, identified the new biomarkers, tested them to prove their accuracy and usefulness, and then shared their findings with the other consortium members for further study. The consortium then submitted applications for use of the biomarkers to FDA and EMA.
In May 2008, EMA issued a qualification statement for these nephrotoxicity biomarkers. It was the first of several EMA qualifications C-Path has achieved to date.
C-Path has also received EMA qualifications or letters of support for:
- AD biomarker: hippocampal volumetric measures (November 2011)
- Disease model of mild to moderate AD (June 2013)
- Kidney safety biomarkers (November 2014)
- Hollow Fiber System model of tuberculosis (February 2015)
- Skeletal muscle safety biomarkers (March 2015)
- TKV imaging as enrichment biomarker in ADPKD (November 2015)
- Liver injury biomarker (November 2017)
- PD biomarker (March 2018)
C-Path not only engages with EMA in the formal submission process for regulatory qualification, but many of its consortia regularly interact with EMA for scientific advice and informed opinions concerning novel methodologies as well. In addition, C-Path has participated in or co-sponsored numerous workshops with EMA, including:
- EU Science: Global Challenges, Global Cooperation (March 4-8, 2013)
C-Path was invited to present on challenges in bioscience and models of cooperation.
- European Medicines Agency workshop on the clinical investigation of medicines for the treatment of Alzheimer’s disease (November 24-25, 2014)
- Applying Regulatory Science to Neonates: launch of the International Neonatal Consortium (May18-19, 2015)
- Scientific Advice Meeting (October 7, 2015)
- Applying Regulatory Science to Neonates: Second Annual Scientific Workshop at European Medicines Agency (EMA) (September 12-13, 2016)
- Workshop on qualification and reporting of physiologically-based pharmacokinetic (PBPK) modelling and simulation (November 21, 2016)
- Workshop on update of TB Guideline (November 25, 2016)
Presentation: “Selection of Agents, Doses and Regimens for Clinical Study”
Programs utilizing EMA scientific advice/opinion completed or underway: