FDA Issues Guidelines for Submission of Data From Genetic Tests
FDA on Tuesday released new guidelines "intended to encourage" pharmaceutical companies to collect and submit data from genetic tests with applications for new medications, a practice that could represent a "significant step ... toward the development of so-called personalized medicine," the New York Times reports. The guidelines, similar to draft recommendations released in November 2003, indicate that FDA will not use most data from genetic tests in approval decisions but will use the information to study "the new science of tying genetic variations to drug response," the Times reports (Pollack, New York Times, 3/23).
According to the guidelines, pharmaceutical companies should include data from genetic tests in applications for new medications when the information is "essential to achieving the dosing, safety or effectiveness described in the drug labeling." Under the guidelines, pharmaceutical companies must submit data from genetic tests to FDA in cases in which medications are tested in clinical trials on a limited population with a certain genetic marker (Wilde Mathews/Davies, Wall Street Journal, 3/23).
In addition, the guidelines require pharmaceutical companies to submit data on valid biomarkers -- genetic variations proven to relate to response to certain medications. The guidelines do not require submission of most other data from genetic tests (New York Times, 3/23). FDA would use voluntary submissions of data from genetic tests for "scientific and informational purposes" (Wall Street Journal, 3/23).
According to the Times, pharmaceutical companies appear "somewhat reluctant" to provide FDA with data from genetic tests because of concerns about the effect on approval decisions and the potential for restricted sales to a limited population. Richard Weinshilboum of the Mayo Clinic said, "When I have consulted with pharmaceutical companies, there's a very strong resistance to the development of a test to be marketed with their drug."
However, the Times reports, the pharmaceutical industry "attitude now seems to be changing" because of recent safety concerns related to some popular medications and because cancer treatments appear more effective when they use genetic markers to help target tumors (New York Times, 3/23). "There has been a lot of reluctance by big pharma to play here," Richard Judson, chief scientific officer at Genaissance Pharmaceuticals, said (Wall Street Journal, 3/23).
However, he added that the new guidelines "will make a lot more companies comfortable about going out and doing this in this space" (New York Times, 3/23). Albert Allen, medical director for the neuroscience platform at Eli Lilly, said that the guidelines are an "attempt by the FDA to start moving forward to better ascertain the risks and benefits for patients" (Wall Street Journal, 3/23).
Edward Abrahams -- executive director of the Personalized Medicine Coalition, which includes companies, academic institutions and others that promote the field -- praised the guidelines, adding, "The government is now the advocate for personalized medicine in an official way."
Janet Woodcock, acting deputy commissioner for operations at FDA, said, "Companies either weren't doing that work because they weren't understanding its regulatory status or they weren't submitting it to us. I can't tell you how important it is for medicine that we move into this paradigm" (New York Times, 3/23).
Larry Lesko, who heads the FDA office that will review the data from genetic tests submitted by pharmaceutical companies, said, "We hope that industry begins to utilize the technology to a greater extent" (Wall Street Journal, 3/23).