The Food & Drug Administration issued guidance (PDF file) just over a month ago on how it will evaluate pharmacogenomic data, hoping to encourage pharmaceutical companies to conduct studies and generate the data during drug development (C&EN, Nov. 10, page 29). The agency then held a workshop with drug and biotechnology industry representatives to discuss its proposed policy and related issues.

	DIRECTED Genentech's drug Herceptin (trastuzumab) targets a specific form of breast cancer in which gene amplification or protein expression testing finds that tumors are overexpressing the Her2 protein. GENENTECH PHOTO

FDA says scientific understanding of pharmacogenomics--which looks for associations between genetic variations and responses to drugs--is most advanced in the drug metabolism area. Nevertheless, the agency anticipates rapid evolution in other uses, such as testing to help identify cancers with a high probability of responding to particular treatments, such as Genentech's Herceptin. The agency plans to issue further guidance on the codevelopment of drugs and pharmacogenomic tests.

"Pharmacogenomics is a new field, but we intend to do all we can to use it to promote the development of medicines," FDA Commissioner Mark B. McClellan said when releasing the guidelines. It holds great promise, he added, "to shed scientific light on the often risky and costly process of drug development, and to provide greater confidence about the risks and benefits of drugs."

The initial draft guidance "essentially lays out a clear pathway about what type of pharmacogenomic information is reportable and what FDA would encourage a company to report but would not use in making decisions," explains Gerald F. Vovis, executive vice president and chief technology officer at Genaissance Pharmaceuticals. His company already sells a genetic test that reveals if patients are at risk for developing acute toxicity to the thiopurine class of drugs.

Industry representatives seem to welcome the guidelines and discussion process with FDA, having been uneasy about generating pharmacogenomic data in the absence of any regulatory plans (C&EN, July 28, page 34). The draft guidance proposes a mechanism for "comfortably sharing exploratory pharmacogenomic information with FDA without stalling ongoing drug development," says Gillian R. Woollett, the Biotechnology Industry Organization's vice president for science and regulatory affairs.

Woollett, like FDA and others, views the move to integrate pharmacogenomics into R&D and the regulatory process as a significant step toward "personalized medicine," in which genetic analysis will help guide prescribing decisions and tailor drugs to specific patient populations. FDA, she adds, will need the pharmacogenomic information voluntarily submitted under the new guidelines to frame its thinking for evaluating such products.

Industry and regulators anticipate continuing, cooperative work in the emerging area of pharmacogenomics and related regulatory policy. "It's obviously a very important area, and we're taking it very seriously," notes the head of genomics operations at a major drug firm.

Cover Story

THE GENOMICS EVOLUTION Small technology providers and major drug firms become allies to find the causes of disease, to validate targets, and to understand drug response WELL DEFINED Brush Up On Your 'Omics' TECHNOLOGY Proteomics Emerges As The Next Frontier REGULATIONS FDA Offers Guidelines On Pharmacogenomic Data

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