Traditional medicine applies the same treatment approach to all patients affected by a disease (‘one size fits all’). However, we are all unique. Our health is determined by our inherited genetic differences combined with our lifestyles and other environmental factors. Personalised medicines are medicines that are targeted to individual patients based on their genetic make-up.

Variants in our genetic code can also be used to predict the potential for adverse drug reactions. For example, the hyper-sensitivity experienced by certain patients to the HIV drug Abacavir has been found to be linked to a particular genetic variant, allele HLA-B5701. The requirement that patients take a test to ensure this allele is absent before being given Abacavir has greatly reduced the incidence of hyper-sensitivity.

Since 2011, personalised medicine has been on the agenda of the European Commission, which has committed two billion Euros of health research funding to the cause. Personalised medicine has been defined by the European Council as a “Medical model using characterisation of individuals’ phenotypes and genotypes or tailoring the right therapeutic strategy for the right person at the right time, and to determine the predisposition to disease and/or deliver timely and targeted prevention, and it relates to the broader concept of patient-centred care, which takes into account that, in general, healthcare systems need to better respond to patient needs.”

In March of this year, the European Medicines Agency held a workshop to explore how clinical practice and public participation can support personalised medicine in the context of EU regulatory activities and to identify areas requiring attention from EU regulators. It was identified, inter alia, that:

  • the success of personalised medicines depends on the development of accurate and reliable diagnostics and on the identification of predictive biomarkers;
  • genomic data submission is needed early on in product development;
  • a significant challenge is the setting of the diagnostic cut-off values that represent significant results; and
  • different clinical trial designs from the classical randomised controlled trial will be required such as, “basket studies” which recruit patients based on their genetic characteristics irrespective of the diseased organ, or “umbrella studies” which screen patients with the same type of disease for a series of hypothesised predictive biomarkers and then allocate them to appropriate therapies, or “platform trials” where multiple treatments are evaluated simultaneously.

It was agreed that next steps would involve: (1) to consider establishing a joint subgroup of working parties that represent patients, consumers, and healthcare professionals to create a common platform to pursue this subject; (2) the pursuit of links between the EMA and the European Reference Networks and European Research Infrastructures; and (3) that a follow-up workshop including industry stakeholders would be organised at a date yet to be determined.