In an online commentary in the New England Journal of Medicine, US Food and Drug Administration Commissioner Margaret Hamburg and the National Institutes of Health (NIH) Director Francis Collins provided their perspectives on the challenges and opportunities in personalized medicine and the ways in which FDA and NIH are seeking to facilitate scientific and commercial efforts in this still nascent field.

“…We have a shared vision of personalized medicine and the scientific and regulatory structure needed to support its growth,” said Hamburg and Collins. “Together, we have been focusing on the best ways to develop new therapies and optimize prescribing by steering patients to the right drug at the right dose at the right time. We recognize that myriad obstacles must be overcome to achieve these goals.”

To that end, Hamburg and Collins said in their commentary that the NIH and FDA will invest in advancing translational and regulatory science, better define regulatory pathways for coordinated approval of codeveloped diagnostics and therapeutics, develop risk-based approaches in reviewing diagnostics to assess their validity and clinical value, and make information about those tests more readily available.

They cited data that showed that only about 10% of labels for FDA-approved drugs contain pharmacogenomic information, underscoring the deficiencies in information for advancing personalized medicine “There has been an explosion in the number of validated markers but relatively little independent analysis of the validity of the test used to identify them in biologic specimens,” they said.

To fill this information void, NIH, with advice from FDA, other Department of Health and Human Services agencies, and other stakeholders, is creating a voluntary genetic testing registry as a single public source of information on the more than 2000 genetic tests that are available through clinical laboratories. The registry will include information such as whether a test has been approved by FDA and data on genetic variants.

NIH and FDA also pointed to other efforts, such as a recent NIH-FDA collaboration on regulatory and translational science, announced in February 2010, which includes joint funding for regulatory sciences. They also pointed to NIH-supported research centers, the NIH Therapeutics for Rare and Neglected Diseases Program, the NIH Clinical and Translational Sciences Award Program, FDA’s efforts under its Critical Path Initiative to improve evaluation tools such as biomarkers and assays, and FDA’s Voluntary Genomic Data Submission Program (a forum under which companies can discuss genomic information with FDA separate from the product-review process) as ways in which policymakers are facilitating the advancement of personalized medicine.

Such collaboration is important, but the key question going forward is whether it will be enough to advance a promising but still unknown field. In their concluding comments, Collins and Hamburg likened the government’s efforts to building “a national highway system for personalized medicine.” Let’s hope that we are going down the right road.