DNAprint Genomics (DNAG)

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Woodcock Again.
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http://www.nature.com/cgi-taf/DynaPage.taf?file=/nrd/journal/v2/n12/full/nrd1274_fs.html

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December 2003 Vol 2 No 12 NEWS ANALYSIS




Nature Reviews Drug Discovery 2, 937-938 (2003); doi:10.1038/nrd1274


[231K]

FDA guidance on pharmacogenomics data submission

Donna R. Savage

Donna R. Savage, Washington


A US FDA pharmacogenomics workshop sought to inform and reassure the industry about submitting data

"Much fear exists, but the reality is that most of the pharmacogenomic data currently available is not suitable for regulatory decision making, because it is not well-enough established scientifically." With these words, Janet Woodcock, Director of the Center for Drug Evaluation and Research (CDER) of the US Food and Drug Administration (FDA), challenged more than 500 pharmaceutical company and government representatives from around the world who gathered in Washington, DC, on 13–14 November to discuss the FDA's draft guidance for industry on pharmacogenomic (PG) data submission.

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Janet Woodcock, Director of the CDER.



The draft guidance (http://www.fda.gov/cder/guidance/5900dft.pdf) provides specific criteria and recommendations for submission of PG data (defined as the use of a pharmacogenomic or pharmacogenetic test), which includes submissions for Investigational New Drug Applications, New Drug Applications and Biologic License Applications. Information is provided on what data are needed and how the FDA will or will not use such data in regulatory decisions.

The workshop provided a forum for discussing the draft guidelines, as well as for defining voluntary submission of PG data, the form and process for presenting such data and the regulatory review procedure.

Because of the need for scientific exchange within this new field, the FDA is asking researchers and sponsors to submit research data voluntarily to help the FDA gain experience as the PG field evolves. But apprehension about what the FDA will do with this voluntarily submitted data constitutes the fear among companies that Woodcock referred to.

However, Woodcock stressed that voluntary submission of PG data is being requested because "we need to become more knowledgeable." The goal is to collect much-needed data to establish a framework so that the PG field can move from being a new science to one that is useable and widely utilized (see the editorial on page 935).

Discussion at the workshop centred on the definition of biomarkers and how to handle intellectual property (IP) issues. Biomarker definition was discussed at length because of the perceived lack of clarity about what constitutes a probable, known or valid biomarker. Woodcock offered descriptions for the different categories of biomarker (see Box), which most people agreed were helpful.

Although acknowledging that pharmacogenomics has the potential to improve the drug development process as well as public health, industry representatives remained concerned about IP issues and the proprietary nature of PG data that will be submitted voluntarily. Larry Lesko, director of the FDA's Office of Clinical Pharmacology and Biopharmaceutics, acknowledged the need for the FDA to resolve IP issues and concomitant concerns about patents on research tools.

Early PG results are expected in drug metabolism, because scientific understanding of pharmacogenomics is most advanced in this field. However, GlaxoSmithKline (GSK) has previously indicated its intent to submit, as a test case to the FDA, data supporting abacavir (Ziagen) and related PG technology that tests for a rare human leukocyte antigen type that is predictive of the risk of developing abacavir hypersensitivity in individuals infected with HIV-1. But, because submission is voluntary, companies have not revealed when they plan to start submitting their data.

"The appearance of guidelines in this emerging field is sincerely welcomed by all practitioners," stated Bill Pennie, director of molecular and investigative toxicology and drug safety evaluation at Pfizer. "As with industry, the regulatory community — not just the FDA — has been on a steep learning curve with these new technologies, and the FDA has kept on top of it extraordinarily competently."

Pennie is also chair of the International Life Sciences Institute's (ILSI) Committee on Application of Genomics and Proteomics to Mechanism-Based Risk Assessment. The ILSI consortium has garnered experience in generating and analysing data sets and has offered to share those with the FDA. Collaboration with the ILSI consortium would give the FDA real and genuinely voluntary data that is not proprietary.

The draft guidance has a comment period that lasts the statutory 90 days, until 2 February 2004, at which time the FDA will analyse and incorporate comments, and craft its final guidance.

Boxes


Biomarker definitions



Valid biomarker. A biomarker that is measured in an analytical test system with well-established performance characteristics and for which there is an established framework or body of evidence that elucidates the physiological, toxicological, pharmacological or clinical significance of the test results. (This definition does not fully describe the situation in drug development, in which data are often rapidly evolving and where some data might be proprietary and in single hands.)

Known valid biomarker. A biomarker that is measured in an analytical test system with well-established performance characteristics, and for which there is widespread agreement in the medical or scientific community about the physiological, toxicological, pharmacological or clinical significance of the results.

Probable valid biomarker. A biomarker that is measured in an analytical test system with well-established performance characteristics, and for which there is a scientific framework or body of evidence that seems to elucidate the physiological, toxicological, pharmacological or clinical significance of the test results.

Source: Draft Guidance for Industry: Pharmacogenomic Data Submissions. US FDA.
© 2003 Nature Publishing Group
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