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Personalized Medicine Pow-wow

Pioneers in genetics-based therapies discuss the evidence for industry's evolution

by Rick Mullin
December 3, 2007 | A version of this story appeared in Volume 85, Issue 49

Innovation Shift:
Credit: Rick Mullin
Burrill sees biology at the helm in a merger between diagnostics and therapeutics.
Credit: Rick Mullin
Burrill sees biology at the helm in a merger between diagnostics and therapeutics.

The 3rd Annual Burrill Personalized Medicine Meeting, hosted in San Francisco last month by the life sciences investment firm Burrill & Co., brought together biopharmaceutical researchers for a two-day discussion of the future of health care—a future that is already beginning to manifest itself in the way that drugs are discovered, developed, and delivered to patients.

Though somewhat nebulous, the term "personalized medicine" is generally understood to describe the practice of employing genetics to understand the nature of disease and then to identify therapies for patients most likely to respond.

This future is already popping up in drugs such as Genentech's Herceptin, a breast cancer treatment specifically targeting the HER-2 receptor, a growth factor protein overexpressed in about 25% of breast cancer patients. It is also evident in the influx of start-up firms developing biomarkers that can determine whether drugs will be effective in individual patients. It popped up again during the week of the conference when deCode Genetics announced deCODEme, a personal genetic scanning service charging $985 per scan.

David R. Parkinson, chief executive officer of Nodality, a biotech firm developing technologies for profiling signaling networks in cells, said he abandoned the one-size-fits-all approach to drug development employed by big pharmaceutical companies like his former employers Novartis, Amgen, and Biogen Idec, in order to join the evolution toward therapies targeted to individual patients.

"I look at the world from a natural selection perspective," Parkinson said during a panel discussion entitled Targeted Treatments vs. The Blockbuster—Pharmacogenomics Comes of Age. "If you don't think natural selection is operative in our world, you are not paying attention."

His copanelists, Eiry W. Roberts, vice president of project and program medical at Eli Lilly & Co., and Andy Williams, head of molecular medicine and antiangiogenesis programs at Pfizer, tended to agree, noting that the big drug companies are reassessing their research approaches. "We are now in an era where technology and our molecular understanding of disease are exploding," Roberts said. "But we need to go back to basics on what technology means to patients and what relevant outcomes are."

According to G. Steven Burrill, the conference's host, the focus of drug discovery and development is shifting from chemistry to biology as therapeutics and diagnostics merge. And this shift is accruing to the benefit of biotechnology companies.

The biopharmaceutical industry's $500 billion in market capitalization, Burrill said, is a reward for innovation. "We are at an all-time high in market value," he said. "The biotech industry market cap is on an upswing, and big pharma companies are on a downswing."

But Ralph Snyderman, CEO of Proventys, which develops diagnostic tests and biomarkers for patient risk assessments, noted that the current health care system is not aligned with advances in personalized medicine and preventive care. "Two things are wrong with the system: how health care is delivered; how it's paid for. And these things are related.

"What is missing in health care today is the focus on the consumer," Snyderman said, adding that 16-17% of U.S. gross domestic product is spent on health care. "No other industry is as bad at serving the customer except for airlines."

Regulators also will need to catch up, and according to Steven I. Gutman, director of the Office of In Vitro Diagnostic Device Evaluation & Safety at the Food & Drug Administration's Center for Devices & Radiological Health, the process has already begun. "FDA is in a listening mode," he said.

Gutman referenced a recent report by the Secretary's Advisory Committee on Genetics Health & Society at the National Institutes of Health, which suggests there are gaps in oversight of genetic testing and calls for public/private partnerships to assess and fill them.

One of the biggest problems for regulators is a lack of analytical specificity and standards. Kathryn A. Phillips, professor of health economics and health services research at the University of California, San Francisco, said the lack of hard scientific evidence for the effectiveness of biomarkers and other personalized medicine tools is creating confusion on the financial front as well.

"There is a variation in the perception between the payer community, industry, government, and academia," she said. "The key challenge is the lack of evidence—the lack of clinical outcomes, the lack of data on economics, the lack of guidelines for patient decisions, and the lack of industry incentives to provide adequate information for coverage decisions."

Bruce Quinn, California medical director for National Heritage Insurance Co., a Medicare management contractor in California, agreed. "Medicare places emphasis on health outcomes," he said. "But they don't know what kind of data are needed to hit that end point." Quinn said that insurance payers may be the least up to speed on trends in health care. "Venture capitalists and Wall Street understand the thought capital, people know a lot about marketing, but then you hit the payers," he said. "You get to a tiny bottleneck at a desk at Aetna, Blue Cross, or Medicare."

Meanwhile, patients are beginning to demand the kind of targeted therapies they read about.

Joe W. Gray, director of life sciences at Lawrence Berkeley National Laboratory, argued that the worlds of drug research, health care delivery, and insurance coverage need to align in response to this demand. "The Internet has a wealth of information," he said, and there is a growing sense that drug development needs to catch up with developments in diagnostic tools. "I sense a strong pent-up frustration with the process," he said.



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