EMA qualification of novel methodologies: are we ready for M&S ?
Objectives: Engage industry, academia and the European Medicines Agency in discussions on how to qualify modelling & simulation (M&S) platforms to a level of standards that could be used as an integral part of drug development and subsequently in the regulatory submissions.
Methods: Since the launch of the qualification process in 2009 (1), the CHMP reviewed/is reviewing 62 requests for qualification advice or opinion (as of Mar 2014) related to biomarkers (BM) or other novel drug development tools (e.g. patient reported outcome measures, modelling and statistical methods). The qualification opinions are available on the EMA website (2). Also there is a trend of increasing numbers of qualification requests to CHMP, indicative of the pace that targeted drug development and personalised medicine is gaining and the need to bring the new tools from research to drug development and clinical use. The focus of the presentation is the regulatory experience gained so far from the CHMP qualification procedure. The different methodologies/biomarkers submitted for qualification are mapped against the drug lifecycle.
Different M&S methods have already been discussed in different contexts of use resulting in two positive qualification opinions and more advices. Experience from recent qualifications and drug development programs submitted to the Agency point to the fact that there are additional areas where modelling and simulation could have an impact. The suitability of qualification procedure for M&S, and the readiness of the modelling platforms, sponsors and regulators to engage to a strategic partnership towards qualifications will be discussed.
Conclusions: M&S is a powerful tool but in order to reach its full potential it would be essential to initiate discussions with industry, academia and regulators on its qualification and use. The qualification procedure provides the forum for such discussions.
 New pathway for qualification of novel methodologies in the European Medicines Agency (2011) Efthymios E Manolis, Spiros S Vamvakas, and Maria M Isaac. Proteomics Clin Appl 5(5-6):248-55, PMID 21538915
 Qualification of novel methodologies for medicine development http://www.ema.europa.eu/ema/index.jsp?curl=pages/regulation/document_listing/document_listing_000319.jsp. Accessed 17 Jan. 14