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A Panel of Industry Experts Eye Impact on FDA

The traditional divide in the way the U.S. Food and Drug Administration regulates the development and use of diagnostic tests and pharmaceuticals is being challenged by rapid advances in the science of personalized medicine, according to participants in the 2012 Wharton Alumni Health Care Conference sponsored by the Wharton Health Care Management Alumni Association (WHCMAA).
"These two spaces are starting to weave together in a way they never did before," said Personalized Medicine panelist Elizabeth
Elizabeth Mansfield

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Panelist Elizabeth Mansfield is Director of Personalized Medicine at the FDA's Office of In Vitro Diagnostic Device Evaluation and Safety.
Mansfield, Director of Personalized Medicine at the FDA's Office of In Vitro Diagnostic Device Evaluation and Safety.

The financial implications of this change driven by advances in genetic science could be significant because, she explained, the majority of medical decisions are motivated by diagnostic tests, yet diagnostic tools are currently very poorly reimbursed.

"Everybody thinks about the drug and not the diagnostic," Mansfield continued. "Are we moving to a point in healthcare and in business where the diagnostic actually takes on much greater significance, is much more highly valued, and if so, how is that all going to all play out in the payer space, so that there's more innovation because a company's actually going to make money at the end of the day?"

"Personalized medicine" is a term generally used to describe the new field of that uses human genome data to inform diagnostic procedures as well as therapies targeted at an individual's unique genetic makeup. All acknowledge that the science of genetic medical treatment is enormously complicated and that the latest research suggests those complexities may be even greater than previously thought.

Genetic diagnostics, on the other hand, can provide information that can be of use to clinicians in understanding how an individual patient may respond to a specific existing genetic or non-genetic drug or intervention.

'A lot of misunderstanding'
Panel moderator Jay Mohr, Managing Director of Locust Walk Partners and president of WHCMAA, told the audience, "there's a lot of misunderstanding about what personalized medicine really means." He pointed out that the conference panel was focusing on in vitro diagnostics rather than the gene-targeted drug treatments.

The FDA regulates the two completely differently, Mansfield said. The two sectors represent completely different points of view in business development, yet because
Intel telemedicine
Rapid advances in genetic science are altering the traditional regulatory relationship between between diagnostics and treatments in the area of personalized medicine.
diagnostic tests are pivotal in determining whether targeted, personalized therapies are safe and effective, the two sectors have started to work together, establishing various types of partnership models.

"We're wondering at what point things are going to start to coalesce, are little guys going to get bigger, are bigger guys going want to work with littler guys?" said Mansfield.

"Who should capture the largest share of the value equation?" asked Mohr. "Historically it's been the pharmaceutical companies, but it may be shifting."

While customized, targeted therapies are already on the market, many questions remain about how personalized medicine will shape patient care and the drug, diagnostics and medical device industries of the near future.

PricewaterhouseCoopers in 2009 estimated the core U.S. personalized medicine market -- diagnostics, therapeutics, lab services and test sales -- at $24 billion, and predicted it would grow 10 percent a year to $42 billion by 2015.

23 personalized medicine drugs approved
The U.S. has 23 approved personalized medicine drugs with $26 billion in annualized sales, the vast majority of them are cancer therapies, according to Mohr. "It is a large and rapidly growing subset of drugs being developed this way," he said.

But the panel acknowledged a number of obstacles confronting the new industry.

For the past four or five years, the reimbursement system's treatment of companion diagnostics has held back personalized medicine, according to panelist Jeffrey Marrazzo, Chief Business Officer for MolecularHealth, a personalized medicine information technology company. This has become a dominant topic at personalized medicine conferences, he said.

With the laboratory testing industry being a small $60 billion piece of the $2.7 trillion spent on healthcare in the United States, payers, such as health insurers, have placed little emphasis on it, he said.

'Hasn't been enough evolution'
"The entire structure around everything from the nitty gritty of coding to how do you actually assess and value these things is really still continuing to mature, and there hasn't been enough evolution, which may come out of some of the reform efforts, to try to pay generally for value in an episodic basis or otherwise," Marrazzo said.

Joseph Leveque, Vice President, U.S. medical-oncology at drugmaker Bristol-Myers Squibb, sees technological challenges, including questions about the biological basis of disease. Researchers don't necessarily know, for instance, whether a tumor cell is emerging because of one mutation or a variety or sequence of mutations, he said. Leveque noted that one of two recently approved therapies for metastatic melanoma targets patients whose cancer has a particular BRAF gene mutation, but that half of the patients with that mutation who get the drug don't respond to it. This creates issues for the prognostic value of the test, he said.

By the time doctors realize a patient isn't responding, the disease has progressed too much and the patient has no other options, creating a dilemma for clinicians, Leveque said.

The answer likely lies in the molecular profile of the patient, he said.

Not about one biomarker
"It's not about one biomarker and it's not even a couple of biomarkers, it's about the heterogeneity of the biology and the biology of the tumor," Leveque said. "Understanding those elements better will solve a lot of the problems both in terms of the clinical outcomes and the economics associated with it."

The FDA's Mansfield, while noting that many products are in the pipeline, agreed that more progress is needed in developing multiple drugs and targeting multiple biomarkers. As it stands, she said, targeted therapies aren't curing cancer, although they are prolonging people's lives.

"It's actually very simplistic right now," said Mansfield. "We're talking about one biomarker and one drug, and that really isn't enough. There's just a whole lot of biology to work with here in combining drugs and combining biomarkers. But I think that is going to happen eventually and will make a huge difference."

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Dinah Wisenberg Brin is a journalist and former staff reporter for Dow Jones Newswires, the Associated Press and Congressional Quarterly.

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