The document, whose approval has been delayed numerous times since it was released as a draft in November 2003, may encourage drug makers to use pharmacogenomics technologies in their drug-discovery efforts. This in turn may spur greater investment in SNP-genotyping, gene expression, RNAi, and gene sequencing technologies and services.
The FDA hopes the guidance, which can be found here, will bring drug research "one step closer to 'personalizing' medical treatment," said Janet Woodcock, deputy director for operations at the FDA, and a chief architect of the document. "We hope ultimately to bring pharmacogenomics ... to every healthcare professional's prescription pad. ..."
The 28-page guidance aims at clarifying how the FDA plans to evaluate pharmacogenomic data during the drug-discovery and drug-development process. The document also "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 FDA said in a statement.
As expected, the guidance also describes a way for industry to voluntarily submit certain research data to "further the scientific exchange of information as we move into more advanced areas of pharmacogenomic research," the agency said.
These will be reviewed by an internal, agency-wide group and "will not be used for regulatory decision making," the FDA stressed. Rather, they "will help FDA and industry gain valuable experience as this new field continues to evolve."
In its statement, the FDA said it 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."
Some industry-advocacy groups and companies welcomed the release. "The FDA's guidance is a significant step forward for the pharmacogenomics industry, and provides a clear path for how to gain marketing approval of drugs targeted by an individual's genetics," Kevin Rakin, president and CEO of Genaissance, said in a statement. "It represents an endorsement of pharmacogenomics as a promising path for developing after and more effective medicines."
Rakin said that he expects the FDA guidance to "stimulate wider use" of the company's clinical trial support services, and "to streamline its efforts in developing a portfolio of pharmacogenomically guided drugs."
"The FDA's guidance removes much of the regulatory uncertainty surrounding pharmacogenomic data submission, bringing us one step closer to realizing our goal of commercializing high-value, genetically-targeted medicines," he said.
Dennis Gilbert, chief scientific officer at Applied Biosystems, said: "These guidelines provide the industry with much-needed guidance on how to employ [pharmacogenomics] technology to advance targeted medicine."
Meantime, the Personalized Medicine Coalition, which counts as members drug and diagnostic companies, academic medical centers, patient-advocacy groups, payors, and providers, said the guidance is noteworthy because it enables drug makers "to have broad technical discussions with [FDA] experts about research data without undue concern that the submitted data will be used for regulatory decision-making.
"Such a concept is extremely important for progress in this highly complex technology, and is of broad applicability to other emergent and related areas of regulation, such as biomarkers," the PMC said in a statement.