The heads of the U.S. Food and Drug Administration and the National Institutes of Health say they want to help bring the benefits of personalized medicine to patients by allowing researchers and drugmakers to use genetic information to ensure patients get the right drug at the right dose. In a commentary published online in the New England Journal of Medicine, the two agency heads say they will invest in the translational and regulatory science needed to advance the co-development and coordinated approval of paired diagnostics and therapeutics.

 

FDA Commissioner Margaret Hamburg and NIH Director Francis Collins say this pathway will include NIH-supported centers high throughput screening of chemical compounds to find potential drug candidates, as well as public–private partnerships to help move drug candidates into commercial development.

 

The editorial comes as several commentators have noted that ten years after the mapping of the first human genome, there is little medical progress to point to that has come as a result. Today, about 10 percent of labels of FDA approved drugs include pharmcogenomic information.

 

The two agency heads say researchers have discovered hundreds of genes variations that are implicated in human illness, identified genetic variability in patients' responses to dozens of treatments, and begun to target the molecular causes of some diseases. Scientists, they also note, are developing and using genetic-based diagnostics to better predict patients' responses to targeted therapy. But they say the challenge is to deliver benefits of the work to patients.

 

While they list scientific and policy challenges ranging from determining which genetic markers have the greatest clinical significance to finding a level of regulation for genetic tests that both protects patients and encourages innovation, they seek to overcome them by working to develop a more integrated pathway that connects all the steps between the identification of a potential therapeutic target by academic researchers and the approval of a therapy for clinical use.

 

The NIH, through such efforts as its Therapeutics for Rare and Neglected Diseases program, will accelerate development of new drug by allowing researchers to take promising compounds through preclinical development. It is also expanding efforts to develop tissue banks containing specimens along with information linking them to clinical outcomes to allow for a better understanding of genetic variation across a range of conditions. And, NIH is harnessing its Clinical and Translational Sciences Award program to translate basic research findings into clinical applications.

 

On the FDA side, the agency is working to establish scientific standards needed to use genetic information in drug and device development and clinical decision making through the development of better evaluation tools, such as biomarkers and new assays. The agency is also coordinating and clarifying the process that diagnostics manufacturers must follow regarding their claims, including defining the times when a companion diagnostic must be approved or cleared before or concurrently with approval of the therapy.

 

“Real progress will come when clinically beneficial new products and approaches are incorporated into clinical practice,” Hamburg and Collins write. “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.”