Wall Street Journal
March 23, 2005   p. D7

U.S. Regulators Want to See
Genetic-Test Data for New Drugs


    In new guidelines, federal regulators announced how and when companies should submit genetic-testing information gathered in developing new drugs.
    The Food and Drug Administration said it wants to see such data when they are likely to affect regulatory decision-making – if a drug maker wants to mention a genetic test in the label of its medicine, for example, or if the test was used to decide who would receive the drug in clinical trials. In other cases, the agency said, it will encourage voluntary submissions that wouldn't be part of the drug-approval process, through a new procedure it may eventually apply to other cutting-edge technologies.
    The move is an effort to pave the way toward emerging technologies that are expected to someday transform how medications are used, by personalizing medicine so that individuals get the therapies that work best for them. Eventually, the goal is for doctors to understand which patients are likely to have side effects or respond well to a drug, based on their genes.
    "We hope that industry begins to utilize the technology to a greater extent," after the FDA clarifies how it will review it, said Larry Lesko, who heads the FDA office that will oversee the genetic-data initiative.
    So far, only a few drugs have been approved with concurrent genetic tests, and some drug makers have been leery of integrating genetic testing with their development of new drugs. For one thing, genetic tests risk narrowing the use of blockbuster drugs, if they show that they only work in limited populations.
    "There has been a lot of reluctance by big pharma to play here," said Richard S. Judson, senior vice president and chief scientific officer at Genaissance Pharmaceuticals Inc. The FDA guidance has "clarified the regulatory landscape and made it easier for companies to submit data without it being used for regulatory decision making," he said.
    The impact of the emerging technology can be seen in a licensing deal Genaissance reached with Merck KGaA last fall. The New Haven, Conn., company acquired the rights to develop and commercialize a new antidepressant that showed some benefits in an early trial, but not enough to spend the millions of dollars needed to conduct a full phase III trial, said Dr. Judson. Genaissance is trying to find genetic markers for patients who will respond best to the drug.
    Albert J. Allen, the medical director for the neuroscience platform at Eli Lilly & Co., said the benefits of genetic tests will vary from drug to drug. There may be a substantial difference in the safety and efficacy for some drugs, but not for others.
    "We don't have the answer or biomarker for everything," he said. But he said the guidelines are an "attempt by the FDA to start moving forward to better ascertain the risks and benefits for patients."
    In its guidance, the FDA laid out details of when it would demand to see genetic-testing information that was done as part of a new-drug application. If the tests are "essential to achieving the dosing, safety or effectiveness described in the drug labeling," for instance, they should be included, the agency wrote. The agency also would require genetic results that supported the maker's arguments about dosing or assessing safety.
    But more general information, submitted voluntarily, would be evaluated for "scientific and informational purposes – not for regulatory decision making," the FDA guidance said. But if the data contained information that might be relevant for a regulatory matter, the agency would prod the company to resubmit it through that pathway.