as part of an agency-wide initiative to speed development of new medical products through the science of pharmacogenomics, the fda on march 22 issued a final guidance, titled "pharmacogenomic data submissions."
pharmacogenomics allows healthcare providers to identify sources in an individual's profile of drug responses and predict the best possible treatment options. for example, genomic tests are helping to identify cancers that have a good chance of responding to a particular medication or regimen, and has enabled the development of targeted therapies, such as herceptin for metastatic breast cancer, gleevec for chronic myeloid leukemia and erbitux for metastatic colorectal cancer.
"fda's efforts will bring us one step closer to 'personalizing' medical treatment," explained dr. janet woodcock, md, acting deputy commissioner for operations, fda. "this new technology will allow medicines to be uniquely crafted to maximize their therapeutic benefits and minimize their potential risks for each patient."
instead of the standard hit-or-miss approach to treating patients, where it can take multiple attempts to find the right drug and the right dose, fda said doctors will eventually be able to analyze a patient's genetic profile and prescribe the best available drug therapy and dose from the start.
both the guidance and a new web page are part of a broad effort underway at fda to foster pharmacogenomics during drug development. fda's new pharmacogenomics web page is available at http://www.fda.gov/cder/genomics/default.htm
the web page, dubbed genomics at fda
, includes detailed information on submitting genomic data, including a decision tree to simplify data submissions, relevant regulatory information and fda contact information.
fda also recently 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.
"we hope ultimately to bring pharmacogenomics, a way in which to foster the personalizing of medicine, to every healthcare professional's prescription pad for the benefit of their patients and u.s. consumers," dr. woodcock said.
the guidance, titled "pharmacogenomic data submissions," clarifies how pharmacogenomic data will be evaluated. 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, which will be reviewed by an internal, agency-wide group and will not be used for regulatory decision making, will help fda and industry gain valuable experience as this new field continues to evolve.
fda believes this approach will save time and resources and eliminate possible delays in the application review process because parties will be able to familiarize themselves with novel pharmacogenomic approaches as they evolve.
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 on this exciting emerging technology.