FDA guidelines to help personalized drug development

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. For example, genomic tests are helping to identify cancers that have a good chance of responding to a particular medication or regimen.

This technology has enabled the development of targeted therapies like Genentech’s Herceptin for metastatic breast cancer, Novartis’ Gleevec for chronic myeloid leukemia and ImClone’s Erbitux for metastatic colorectal cancer.

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, 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 webpage are part of a broad effort underway at the FDA to foster pharmacogenomics during drug development.

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.

The guidance 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 to voluntarily submit research data to further the scientific exchange of information. The voluntary data, which will be reviewed by an internal, agency-wide group and will not be used for regulatory decision making, will help the FDA and industry gain valuable experience as this new field continues to evolve.

The 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’s efforts will bring us one step closer to ‘personalizing’ medical treatment,” explained Dr Janet Woodcock, acting deputy commissioner for operations at the FDA. “This new technology will allow medicines to be uniquely crafted to maximize their therapeutic benefits and minimize their potential risks for each patient.”

The 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 emerging technology.