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Regulatory News: EMA Publishes Pharmacogenetics Guideline for Evaluating Medicines

2nd March 2012

The European Medicines Agency (EMA) has published guidance advising pharmaceutical companies how to take genetic variability between patients into account during the development of medicines.

Understanding how genetic factors influence the way in which an individual may respond to medicines is referred to as pharmacogenetics.  The guideline on the use of pharmacogenetic methodologies in the pharmacokinetic evaluation of medicinal products is a result of an increase in the knowledge of gene influence on differences between individuals in drug performance.  Genetics may affect drug absorption, distribution, metabolism and excretion (ADME).  These variables may affect the risk-benefit profile of pharmaceutical products in individuals.

The guideline was adopted by the EMA’s Committee for Medicinal Products for Human Use (CHMP) in January 2012 following public consultation.  Pharmaceutical companies applying for marketing authorisations should follow the guideline when it comes into effect 1 August 2012. 

The guideline recognises that pharmacogenetics may not be equally important for every drug.  It is suggested that studies examining the pharmacogenetic impact on drug pharmacokinetics should be performed where variability between individual patients is high enough to influence the safety and/or efficacy of the drug.  This possibility may be apparent from earlier studies (either in vivo or in vitro) that show either that genes subject to significant variation are involved in processing of the drug by the body, or that there is unexplained (non-pharmacogenetic) variability in patient response to the drug.

The guideline outlines several circumstances where pharmacogenetic studies are recommended, rather than required, and these include instances where major differences in pharmacokinetics are observed in different ethnic groups and cannot be explained by other factors.
The guideline also features requirements and recommendations on:

• when pharmacogenetic studies should be performed
• how these studies should be designed and carried out
• how the clinical impact of genetic differences between patients should be evaluated
• how dosing or treatment recommendations for genetic subpopulations should be studied
• consequences for treatment recommendations and labelling
• the impact of interactions between medicines and of impaired or immature organ function

The EMA explains that the guideline applies predominantly to small-molecule drugs as genetic effects on the pharmacokinetics of biological drugs today are much less understood.

Reviewed by Claire Pomeroy, Senior Regulatory Affairs Executive