There is no common definition of personalised medicine, but in the context of this guidance the term refers to the targeted use of a treatment in a patient on the basis of the individual’s characteristics and genetic makeup and the understanding of how the treatment works.
The choice of a personalised medicine relies on the use of a companion diagnostic, a diagnostic medical device which allows identifying patients who are most likely to benefit from a specific medicine. The device can also identify patients who are likely to be at increased risk of serious adverse reactions as a result of treatment with the associated medicine. It is very important to clarify how evidence to support the validation of a companion diagnostic can be generated during the development of a medicine.
While EMA does not issue recommendations on companion diagnostics, the recently revised European Union (EU) legislation on in vitro diagnostic medical devices1foresees cooperation between medicines regulators and EU notified bodies, which conduct the conformity assessment of medical devices in the EU, in the evaluation of new companion diagnostics to obtain the CE label2.
The future guideline will replace the existing reflection paper on co-development of pharmacogenomics markers and assays in the context of drug development.