Reality Check | November 30, 2009 Issue - Vol. 87 Issue 48 | Chemical & Engineering News
Volume 87 Issue 48 | p. 22
Issue Date: November 30, 2009

Reality Check

Meeting looks at science, regulatory, and health care hurdles to personalized medicine
Department: Business
Keywords: genetics, genome, information technology, healthcare, finance
Burrill (left) grills FDA Commissioner Hamburg on the regulatory challenges to developing targeted therapies.
Credit: Rick Mullin/C&EN
Burrill (left) grills FDA Commissioner Hamburg on the regulatory challenges to developing targeted therapies.
Credit: Rick Mullin/C&EN

Attendees at Burrill & Co.’s fifth annual conference on personalized medicine in San Francisco this month took a step back from the canvas. Venture capitalists, researchers, and health care professionals discussed ways to arrange an abstract collage of scientific and health care activity into a unified vision of genetics-based medicine tailored to the individual. Speakers addressed the scientific and regulatory obstacles, as well as the formidable problem of bringing doctors into the picture.

The contemplative pause at what has become a leading personalized medicine gathering for researchers and investors may have been prompted by the collapse of capital markets that are vital to many of the companies involved in developing therapies and diagnostics. It became evident during the two-day meeting, however, that other problems—such as the lack of standards in research and patient care and the inadequate level of collaboration between the researchers, physicians, patients, and regulators—may be of greater concern.

In his opening presentation, G. Steven Burrill, head of the eponymous venture capital and merchant banking firm, provided an overview of the biotech sector. Despite the downturn, he said, biotech firms have fared well compared with large pharmaceutical companies. Noting early signs of an economic recovery, he said financing is available, some of it from nontraditional sources: “There is a lot of capital out there. Your job is to go out and get it.”

Getting it, he said, will require companies to have a clear assessment of the value created by their ventures—an understanding that many start-ups lack. “You need to understand value in personalized medicine is not a derivative of sales and earnings,” Burrill said. More nebulous variables, such as potential market size and intellectual property, make for a complicated equation. “Value has become a negotiable event, and entrepreneurs are poorly prepared for dialogue on the value equation,” he said.

In a panel discussion on getting doctors to adopt personalized medicine techniques, Geoffrey Ginsburg, director of the Center for Genomic Medicine at Duke University, bemoaned the lack of uniformity in collecting and archiving patient histories and other data essential to personalized medicine. But he cited a more fundamental shortcoming in health care: “We have a perverse system that is volume- rather than value-based.”

Ginsburg and panelist Joseph D. McInerney, executive director of the National Coalition for Health Professional Education in Genetics, agreed that physicians are only one part of the health care communit that needs to come up to speed. McInerney noted that physician assistants, nurse practitioners, and nurses have made more headway than doctors in incorporating personalized approaches to patient care.

In a presentation titled “The $1,000 genome: When will it arrive and how will it transform medicine?” Leroy Hood, president of the Institute for Systems Biology, also lamented the lack of standards in personalized medicine. While genetic advances are certain to bring the cost of sequencing a human genome below $1,000 in the next five years, an infrastructure for dealing with “billions of data points from hundreds of millions of patients” isn’t there. Hood also noted significant ethical and social hurdles to deploying that information.

Regulatory concerns were addressed in a presentation by newly appointed Food & Drug Administration Commissioner Margaret Hamburg, who stayed onstage for an interview with Burrill. Hamburg told attendees that FDA needs to adapt the regulatory process to new science. “FDA is a science-based and science-led organization, and it must respond to new science that challenges our capacity for R&D and for policy. Billions of dollars in biomedical research will not result in cures without an investment in regulatory science.” Funding that investment is a top priority, Hamburg stated. “The agency has been underappreciated and underfunded for too long.”

The agency is working with the National Institutes of Health on the connection between diagnostic technologies and therapeutic outcomes, Hamburg said, predicting that draft guidance on biomarker qualifications will come by the end of the year.

Asked by Burrill about the agency’s flexibility in dealing with scientific innovators in the biotech sector, Hamburg acknowledged a need for improvement, especially in communications.

In comments at the close of the meeting, Burrill noted that the medical community is known for its resistance to change, but that change is under way. He praised Hamburg’s commitment to developing a regulatory framework for personalized medicine but noted that she faces other pressing issues, such as ensuring the global drug supply chain. “And she will be sidetracked by the ‘F’ part of FDA,” he said. “We will only get part of her attention, but we’ve had an interesting discussion with a rookie who cares a lot about us.”

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