Clinical application of companion diagnostics

doi:10.1016/j.molmed.2015.05.003

Trends in Molecular Medicine

Volume 21, Issue 7, July 2015, Pages 405-407

https://doi.org/10.1016/j.molmed.2015.05.003 Get rights and content

Highlights

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The prescription of most drugs is still largely based on ‘trial and error’.
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Companion diagnostics (CDx) hold the promise of more effective and safe pharmacotherapy.
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CDx assays have so far been reserved for oncology.
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With regards the more general use of CDx assays, we still seem to have a long way to go.

Optimally, any prescription should rely on an in-depth understanding of the disease biology and the mechanism of action of the drug. However, despite the hype about precision medicine in recent years, the prescription of most drugs is still, except for a few anticancer drugs, largely based on ‘trial and error’ and not on solid pharmacogenomic biomarker data.

Section snippets

Companion diagnostics (CDx)

A CDx assay, according to the US FDA guidance document issued in August 2014, is defined as an in vitro diagnostic device that provides information that is essential for the safe and effective use of a corresponding therapeutic product [2]. Furthermore, the FDA specifies several areas in which such an assay could be essential, and which overall can be summarized as outcome prediction (efficacy and safety) as well as therapy monitoring. No doubt, the efficacy predictions of CDx assays have

Clinical application of companion diagnostics and drug efficacy

Optimally, any prescription should rely on an in-depth understanding of the disease biology and the mechanism of the action of the drug. This has yet to be realized, despite pharmacogenetics having been on the agenda for the past 50 years. However, within one disease area, advances in molecular medicine and molecular diagnostics seem to have made an entry into clinical practice – and this is within oncology. Here, the new molecular understanding has given sufficient insight to allow us to

Concluding remarks

Despite the hype about precision medicine in recent years, the prescription of most drugs is still largely based on trial and error and not on solid pharmacogenomic biomarker data. Such an approach can have serious medical consequences for the individual patient as well as economic consequences for the healthcare system. For most serious chronic diseases, early diagnosis and early intervention are two elements of key importance, and the intervention needs to be correct. So far, CDx assays have

Disclaimer statement

Jan Trøst Jørgensen is working as a consultant for Dako/Agilent and Euro Diagnostica, and has given lectures at meetings sponsored by AstraZeneca, Merck Sharp & Dohme, and Roche.

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In the drug-diagnostic codevelopment model the assumption is that specific molecular characteristics need to be present in the individual patient for the drug to be effective. In order to identify these molecular characteristics an assay needs to be developed in conjunction with the drug. When such an assay is linked to a specific drug, it is called a companion diagnostic (CDx). During the different development stages, it must be documented that the assay is analytical reliable and clinically able to select the likely responding patients. The drug-diagnostic codevelopment model has shown to improving the predictability of the drug development process, as well as being an important tool for the clinical use of the drugs after regulatory approval. CDx assays have taken up a central role in the development process for a number of targeted anticancer drugs, and the success of these types of drugs largely depends on the performance of these assays.
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For decades, we have known that the response to a pharmacological intervention varies from patient to patient; however, it is often difficult to explain this variation and to predict who might be the responders [1].
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Trends in Molecular Medicine

Science & SocietyClinical application of companion diagnostics

Highlights

Section snippets

Companion diagnostics (CDx)

Clinical application of companion diagnostics and drug efficacy

Concluding remarks

Disclaimer statement

Trends Mol. Med.

Guidance for Industry and Food and Drug Administration Staff. In Vitro Companion Diagnostic Devices

Use of chemotherapy plus a monoclonal antibody against HER2 for metastatic breast cancer that overexpresses HER2

N. Engl. J. Med.

Science & Society
Clinical application of companion diagnostics