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Experience with voluntary and required genomic data submissions to the FDA: summary report from track 1 of the third FDA-DIA-PWG-PhRMA-BIO pharmacogenomics workshop

The Pharmacogenomics Journal volume 6, pages 296–300 (2006)Cite this article

In May of 2002, the first of a series of FDA – industry workshops on pharmacogenomics was held with the intent to assess the use and status of pharmacogenomic research in drug development.1 To facilitate communication between FDA and industry regarding appropriate applications of pharmacogenomic data, the FDA proposed that industry submit pharmacogenomic data to the Agency under a concept that was termed ‘Safe Harbor’. As the title suggests, the idea was to create a submission process that would provide (1) an exempt status for exploratory pharmacogenomic information so that it would not compromise regular IND, NDA, or BLA submissions, and (2) a platform for an open, scientifically driven information exchange between the Agency and industry. Under this proposal, industry and FDA could explore the use and value of this type of data in drug development outside the usual formal regulatory environment. The need for both was acknowledged at this workshop.

The result of the meeting was a clear directive for FDA to create a guidance document that would help to clarify what type of information would fall within this ‘Safe Harbor’ process and to clarify how the Agency would review such data outside the usual regulatory process. An important caveat was the recognition that this voluntary process supplemented but did not replace standard regulatory requirements currently in place for new drug submissions. Proceedings of this workshop have been published2 and the workbook is available online.1

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References

  1. http://www.fda.gov/cder/calendar/meeting/phrma52002/default.htm.

  2. Lesko LJ, Salerno RA, Spear BB, Anderson DC, Anderson T, Brazell C et al. Pharmacogenetics and pharmacogenomics in drug development and regulatory decision making: Report of the First FDA-PWG-PhRMA-DruSafe Workshop. J Clin Pharmacol 2003; 43 (4): 342–358.

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Authors and Affiliations

  1. Office of Clinical Pharmacology and Biopharmaceutics, Center for Drug Evaluation and Research, US Food and Drug Administration, Silver Spring, MD, USA

    F W Frueh & A Rudman

  2. Office of In Vitro Diagnostic Device Evaluation and Safety, Center for Devices and Radiological Health, US Food and Drug Administration, Rockville, MD, USA

    K Simon & S Gutman

  3. Genaissance Pharmaceuticals, New Haven, CT, USA

    C Reed

  4. Wyeth Research, Cambridge, MA, USA

    A J Dorner

Authors
  1. F W Frueh
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  2. A Rudman
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  6. A J Dorner
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Correspondence to F W Frueh.

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Frueh, F., Rudman, A., Simon, K. et al. Experience with voluntary and required genomic data submissions to the FDA: summary report from track 1 of the third FDA-DIA-PWG-PhRMA-BIO pharmacogenomics workshop. Pharmacogenomics J 6, 296–300 (2006). https://doi.org/10.1038/sj.tpj.6500380

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