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Hamburg, Collins Chart Course to Personalized Medicine in NEJM

By a GenomeWeb staff reporter

NEW YORK (GenomeWeb News) – US Food and Drug Administration Commissioner Margaret Hamburg and National Institutes of Health Director Francis Collins presented their vision for the future of personalized medicine in a perspectives article appearing online in the New England Journal of Medicine yesterday.

In it they explained that while researchers are inching closer to personalized medicine — identifying genetic variants involved in diagnosing disease and predicting treatment response — there are still a range of significant scientific and policy issues that need to be dealt with before personalized medicine reaches its full potential.

"Real progress will come when clinically beneficial new products and approaches are incorporated into clinical practice," Hamburg and Collins wrote. "As the field advances, we expect to see more efficient clinical trials based on a more thorough understanding of the genetic basis of disease. We also anticipate that some previously failed medications will be recognized as safe and effective and will be approved for subgroups of patients with specific genetic markers."

Consequently, the duo noted, both the FDA and NIH are setting out a series of goals and policies aimed at nudging personalized medicine closer to reality.

Along with highlighting the importance of basic science, translational research, and regulatory studies in achieving personalized medicine, Hamburg and Collins described some of the programs and initiatives that they believe will support these processes.

"[T]he NIH and FDA will invest in advancing translational and regulatory science, better define regulatory pathways for coordinating approval of co-developed diagnostics and therapeutics, develop risk-based approaches for appropriate review of diagnostics to more accurately assess their validity and clinical utility, and make information about tests readily available," they wrote.

Echoing comments that Collins made at the American Association for Cancer Research meeting earlier this year, Hamburg and Collins said the agencies envision an integrated pipeline for helping researchers move from basic research to the development of approved, clinically validated treatments.

In particular, the team pointed to initiatives such as the NIH's Therapeutics for Rare and Neglected Disease program, aimed at supporting the preclinical development of specific compounds, the Clinical and Translational Sciences Award, and the FDA's Critical Path initiative and Voluntary Genomic Data Submission program.

Hamburg and Collins also touched on the need for linking tissue bank samples with clinical data and emphasized the importance of validating genetic and other tests used during treatment and diagnosis.

On the latter front, they explained that the NIH, FDA, and other agencies within the Department of Health and Human Services hope to create a voluntary genetic testing registry, while the FDA intends to tweak its therapeutic and diagnostic test review process to meet some of the specific issues associated with the sorts of genetic tests that are already being used to diagnose subsets of patients and direct treatments.

"The FDA is coordinating and clarifying the process that manufacturers must follow regarding their claims," Hamburg and Collins noted. "The agency will ensure that claims that a test will improve the care of patients are based on solid evidence, and developers will get straightforward, consistent advice about the standards for review and the best way to demonstrate that the combination works as intended."

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