Advent of New, More Effective and Personalized Medicines

The FDA today issued a final guidance titled "Pharmacogenomic Data Submissions" to speed development of new medical products through the science of pharmacogenomics.

Pharmacogenomics allows health care providers to identify sources of an individual's profile of drug response and predict the best possible treatment option for this individual. This technology has enabled the development of targeted therapies like Herceptin for metastatic breast cancer, Gleevec for chronic myeloid leukemia and Erbitux for metastatic colorectal cancer.

This new approach will allow medicines to be uniquely crafted to maximize their therapeutic benefits and minimize the potential risks for each patient. Doctors will eventually be able to analyze a patient's genetic profile and prescribe the best available drug therapy and dose from the start.

FDA also approved the first laboratory test, the Amplichip Cytochrome P450 Genotyping Test, which will enable physicians to use genetic information to select the right doses of certain medications for cardiac, psychiatric diseases and cancer.

The final guidance describes what data will be needed during the marketing application review process, the format for submissions, and the data that will be used during regulatory decision-making. The guidance also explains a new mechanism for industry to voluntarily submit research data to further the scientific exchange of information as we move into more advanced areas of pharmacogenomic research.

The voluntary data, will be reviewed by an internal, agency-wide group and will not be used for regulatory decision making, but will help FDA and industry gain valuable experience. FDA believes this approach will save time and resources and eliminate possible delays in the application review process.

FDA has already received several pharmacogenomic data submissions through both the regulatory and voluntary processes and will continue to work closely with industry and the healthcare community.

In addition to announcing the availability of its final guidance and the new pharmacogenomic Web page, the agency also reminded the public of the meeting being held on pharmacogenomics issues.

The joint FDA/DIA meeting, "Pharmacogenomics in Drug Development and Regulatory Decision Making” will focus on integrating pharmacogenomics in clinical trials for new drugs, biologics, and associated devices. FDA will also address ways to translate pharmacogenomics into medical product development and clinical practice.