The National Institutes of Health and the Food and Drug Administration are crafting coordinated initiatives aimed at increasing the availability of gene-therapy drugs, according to an article appearing online in the New England Journal of Medicine.
In the article “The Path to Personalized Medicine,” FDA Commissioner Margaret Hamburg and NIH Director Francis Collins write that the initiatives will support the advancement of personalized medicine through rapid development, review and approval of safe and effective genetics-based disease therapies. “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,” they wrote.
The NIH initiative calls for the agency to invest in research that will support scientists' efforts to translate genetic-marker discoveries into effective diagnostic tests and disease therapies. The FDA's program will work to develop an effective and timely pathway for the review and approval of new gene-therapy drugs. FDA and NIH officials said the coordinated efforts should help spur innovation among drugmakers, many of which currently don't see an immediate pathway for turning basic genetic research into marketable high-tech therapies.
“To fill that void, NIH and the FDA will 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,” Hamburg and Collins wrote.