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Biomarkers: To Be or Not To Be Significant?

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As more and more companies attempt to sell diagnostics assays based on the presence or absence of particular biomarkers, it’s not surprising that eventually the regulatory and investment community would sit up and take notice.

In fact, at its annual CEO & Investor Conference in February, BIO catered to the interest (or, taking a more cynical view, actively promoted interest) in biomarkers by devoting a focus session to the topic. The interaction of biotech businesspeople, regulators, and investors gave me a chance to take stock of the situation.

First and foremost, it’s clear that the FDA wants to do more to encourage researchers and drug developers to make use of biomarkers to diagnose disease, and particularly as an accoutrement to the drug discovery process. Scott Gottlieb, a physician and former editor of the Gilder Biotech Report who has now crossed over to the FDA as senior advisor for medical technology to Commissioner Mark McClellan, discussed the agency’s nascent attempts to build a framework for evaluating the significance of biomarker data.

Through an effort entitled the “Critical Path Initiative,” Gottlieb says the FDA is studying the validity of new types of clinical trials, new toxicity assays, and new methods for assessing how data from “secondary endpoints” — otherwise known as biomarkers — could support or even replace more established metrics for gauging the effectiveness of new therapeutics. Gottlieb envisions a possible scenario where the NIH or FDA would fund intra-agency or academic research to establish guidelines for how the regulatory agency should treat biomarker data. Giving the effort a flashy new name like “Critical Path,” he says, slightly tongue-in-cheek, is also useful as a ploy to shake the piggy-bank.

Of major concern to drug developers is whether the companies will have any recourse in the event that ancillary biomarker data is singled out by the FDA as reflecting poorly on a potential drug’s approval prospects. “Companies are concerned that FDA would say, ‘Hey, this particular gene is over-expressed — what does that mean?’ and it would hold up the approval process,” Gottlieb says. To counter these concerns, he says that his agency plans to create a review panel to arbitrate between the FDA and drug developers when there’s a dispute concerning the significance of data derived from pharmacogenomics or proteomics.

Unfortunately for the companies trying to commercialize biomarker-based diagnostics, having the FDA pay more attention to biomarkers doesn’t necessarily translate to a boost in investor interest. For one thing, investors are even more confused than the FDA about how to value biomarker data.

José Haresco, a computational chemist-turned-investment banker with CE Unterberg, Towbin, began the Q&A session by asking: “Depending on whether you’re a physician, venture capitalist, or investment professional, how does one value a marker?” Needless to say, there wasn’t much of a consensus. In fact, one investor asked whether math played a large role in evaluating the significance of a biomarker. As it turned out, by “math” he meant fractals, or chaos theory.

But more relevant to companies looking to increase their standing in the eyes of investors is the resilient faith in the finance community that only putting approved drugs on the market can guarantee a biotech commercial success. Nor is this idea only on the minds of investors.

After all, for many companies, the primary motivation for trying to commercialize biomarkers was to find a shortcut to generating cash to support longer-term efforts at developing drugs.

“Anything that can shorten the time required for drug development is good,” says Alden Philbrick, CEO of Oxford Finance, a commercial lender to life science businesses. “Biomarkers are essentially a supporting tool.”

John S. MacNeil, a senior editor at Genome Technology, can be reached at [email protected] web.com. His Sense/ Antisense column, which covers government research policy and regulatory issues, appears bi-monthly.

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