For the purpose of the concept paper, the term “personalised medicine” 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. To that end, it is necessary to measure the predictive biomarkers (BM) and this is made by means of a device called companion diagnostic (CDx). CDx is defined for the first time in the new in vitro diagnostic medical devices regulation (IVDR) as “a device which is essential for the safe and effective use of a corresponding medicinal product to: (i) identify, before and/or during treatment, patients who are most likely to benefit from the corresponding medicinal product; or (ii) identify, before and/or during treatment, patients likely to be at increased risk for serious adverse reactions as a result of treatment with the corresponding medicinal product”.
If it is recommended in the labelling that a medicinal product should be used in conjunction with a predictive BM, any commercial assay used for this purpose will be considered a CDx and will require an appropriate conformity certificate (CE mark).
In Europe, the legislations covering the marketing of medicinal products and IVD medical devices are not directly linked. The new IVDR envisages cooperation between notified bodies and medicines regulators in the evaluation of new CDx for obtaining a CE mark, although this will not lead to approval by medicines regulators of one or more specific CDx for use in conjunction with a given drug.
The fact that developments of medicinal products and IVDs are often independent, coming together only superficially towards the end causes gaps in evidence and validations. The proposed guideline intends to help to optimise the co-development of medicinal products and companion diagnostics, including their combined use in clinical trials to generate evidence required to support validation of the diagnostic.
Comments on the concept paper may be submitted by 15 November 2017. Then, the process will continue with the issuance of a draft guideline, that will be available 9-12 months after the end of the public consultation of the concept paper and will be released for 6 months external consultation.