Biomarkers: (Much) Faster Please

Alex Tabarrok's vision of a "Consumer Reports" FDA depends on the widespread use of biomarkers and diagnostics - not just in the labs of pharmaceutical and biotech companies, but in your doctor's office or local pharmacy. Widespread consumer access to those tests - and confidence on what the tests are telling us - is still several years away.

What's slowing the field down?

On the industry side companies are investing heavily in using biomarkers to help guide new drug development, and are clearly shifting to an R&D strategy built around personalized medicines, but so far (outside of cancer treatments) those investments haven't found their way into many drug labels or companion diagnostics.

A 2010 report from the Tufts Center for the Study of Drug Discovery noted that while many companies are submitting data for the FDA's program for Voluntary Exploratory/Genomic Data Submissions, they also said that "they cannot use pharmacogenomic data in an approval package due to a variety of regulatory concerns."

I spoke with Christopher-Paul Milne, the author of the Tufts report, to get more clarity on what concerns companies are expressing about regulatory challenges. He told me that

[Companies] noted the difficulties of establishing validated biomarkers, uncertainty whether certification of lab diagnostics will be done by CMS under the Clinical Laboratory Improvement Amendments (CLIA) or FDA, [and] an overall lack of harmonization among the major regulatory agencies, and a lack of guidance and standards in the EU and US on how the process works.

This is a bit of a chicken and egg problem, because until the pathways are better defined, companies won't submit the data to the FDA in an approval package - which is how biomarker data would make its way into a drug's labeling and drive development of a companion diagnostic (if it wasn't co-developed with the drug).

One bright spot, he added, was that successful biomarkers were often seen by companies as "tipping the benefit/risk ratio in favor of product approval."

"In fact," Milne reported, "two big pharma companies of the eight we interviewed (along with five biotechs) are taking FDA at its word that companion diagnostics are the preferred model from a regulatory standpoint, while a third has recently announced plans to establish its own in-house diagnostic capabilities."

Clearly, personalized medicines and companion diagnostics the wave of the future - a postive sign for Tabarrok's vision for regulatory reform.

What will slow the progress of personalized medicines is the tension between how quickly companies can develop novel diagnostics tests and the FDA's comfort level in confirming their clinical value before approving them for marketing.

Right now, the FDA seems to be taking a cautious approach to biomarker driven diagnostics. "Under certain circumstances, according to the FDA's Dr. Woodcock, almost everyone can accept lesser evidence," Milne says, "and so the real challenge is where to draw the line in a specific case. That's not a question FDA wants to answer in a vacuum, so they are busy collecting data through their voluntary exploratory data submissions program (as is EMA in a similar effort) to feel more comfortable with the science."

How long will it take for the agency to become "comfortable with the science" isn't clear. At the end of the day, Milne believes that the agency will have to embrace a "spirit of experimentation" since the clinic - the real world - may be the only place where these questions can be answered quickly, perhaps through a progressive or rolling approval mechanism for biomarker-based drug approval that some experts have suggested.

More on this in a future post.

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