The European Medicines Agency (EMA) has released for public consultation a concept paper on the development and lifecycle of personalised medicines and companion diagnostics that measure predictive biomarkers which help to assess the most likely response to a particular treatment. The concept paper is the first step in the preparation of a guideline that will address the development challenges of personalised medicines with companion diagnostics.
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 devices1 foresees 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 EMA’s proposed concept paper on predictive biomarker-based assay development in the context of drug development and lifecycle is intended to be developed into a guideline which will replace the reflection paper on co-development of pharmacogenomic biomarkers and assays in the context of drug development. The guideline will provide recommendations on the interface between predictive biomarker-based assays including companion diagnostic and the development and lifecycle of a medicine.
2CE marking is the medical device manufacturer's claim that a product meets the essential requirements of all relevant European Medical Device Directives and is a legal requirement to place a device on the EU market.