Creating new drugs is a process fraught with risk.   The risk involved with discovery and early development is obvious, but increasingly the greatest risk lies at the very end of the process: winning a decent market for your approved product.

But history teaches us there is a sure-fire trick to eliminate a large portion of both the technical and commercial risk from drug development.  A trick that has arguably yielded more blockbusters than any other approach: medicalizing biomarkers.

For sure, only a handful of biomarkers have undergone this transition – from something diagnostic to a target for intervention – but those that have, have yielded eye-watering drug sales.  Drugs to treat elevated LDL-cholesterol (such as Lipitor™ atorvastatin), elevated blood pressure (such as Diovan™ valsartan and other angiotensin receptor antagonists) or elevated fasting glucose (such as insulin and more recently GLP-1 agonists) have garnered countless billions in sales over the last two decades.

The industry will always push the boundaries to justify adoption of new biomarkers as therapeutic targets in their own right

The reason for their success is obvious: unlike complex disease phenotypes, its trivial (and usually very cheap) to identify the significant portion of the population with the elevated biomarker.  Its also straightforward to demonstrate, during clinical development, the impact of the treatment on the biomarker.  So the development risks are much lower than for drugs where unambiguous demonstration of impact on a complex clinical phenotype is required.

And the commercial risk is lower too.  Provided the link between the biomarker and disease is well-accepted, medical practioners, payers and patients alike are keen to embrace prevention rather than treatment when the disease itself manifests (perhaps much) later.

To the chagrin of the pharmaceutical industry, there are but a handful of such mother lodes to mine.  Hypercholesterolemia, hypertension and hyperglycemia are the only diagnostic measures that are universally accepted to justify intervention in the absence of any clinical symptoms.

Not surprisingly, therefore, we see a drive from the industry to expand the list – with elevated triglycerides and low testosterone in the vanguard.  After all, once regulators, doctors or even patients can be persuaded that a new biomarker requires treatment in the absence of symptoms, it would open up a brand new treasure chest.

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RxCelerate Ltd is an outsourced drug development platform based near Cambridge, UK. We specialize in delivering an entire road map of drug development services from discovery and medicinal chemistry through to formal preclinical development and clinical up to Phase IIa. In the last five years, we have witnessed dramatic changes in the drug development …