FDA rules aim to speed drug tests and trim costs
Changes will boost scientists at colleges and small companies
By Diedtra Henderson, Globe Staff | January 13, 2006
The FDA’s new standards create an earlier stage of clinical trials that allow researchers to test very small doses of experimental drugs in fewer patients, paring expenses and, drug manufacturers hope, providing a quicker way to flag potential failures.
Right now, the FDA says 9 of 10 experimental drugs fail in clinical testing that can cost companies millions. And many experimental drugs go untested in humans because the current system requires massive investments before the make-or-break stage of drug development.
”This will allow us to test a broader variety of compounds in people early and then pick the best one for taking forward," said Dr. Janet Woodcock, the FDA’s deputy commissioner for operations.
The FDA’s declaration came as drug companies were still digesting the agency’s sobering news for 2005: The FDA approved a mere 20 new drugs last year, compared with 36 approvals the previous year. From 2002 to 2004, newly approved drugs took an average of 8.5 years to work their way through clinical trials to earn FDA approval.
If the length of clinical trials were shaved by 25 percent, drug manufacturers would save $129 million per new drug approved, according to recent research by Joseph A. DiMasi of the Tufts Center for the Study of Drug Development, which put the cost of developing a new drug at $802 million. Halving the time spent on clinical trials would lower drug development costs by 29 percent, saving drug companies $235 million per new drug approved, DiMasi found.
Dr. Andrew von Eschenbach, FDA acting commissioner, said the agency plans a variety of actions to clear barriers that impede drug development. A sweeping FDA effort to examine the ”critical path" to approval for new medications surveyed promising tools and techniques developed by the industry and academia. The agency is beginning to leverage those innovations to speed drug, vaccine, and device development. One example: BG Medicine in Waltham will work with the FDA’s National Center for Toxicological Research to help spot earlier signs that a drug is toxic to the liver.
Meanwhile, the National Cancer Institute plans to take advantage of the new standards within months by asking the FDA to approve two trials, including a test of a new imaging technology that homes in on certain tumors, according to Dr. James Doroshow, director of the NCI’s division of cancer treatment and diagnosis.
Companies in the Boston area offered a mixed response to the FDA’s guidelines. An official at Infinity Pharmaceuticals in Cambridge said the new rules could speed development of their experimental cancer drug that attacks solid tumors, uncontrolled new tissue growth that can signal cancer.
By testing one-100th of the likely dose in a small number of healthy volunteers for about a week, the company could quickly determine how long the drug lingers in the body, how it’s flushed out, and whether it could produce potentially toxic by-products.” If I have two or three drugs in the same class, you can sort of do a beauty contest and see which will be the best-behaved," said Julian Adams, Infinity’s chief scientific officer. Quickly weeding out a weaker drug that could fail the FDA’s safety challenge could save the company up to $4 million, Adams said.
At first glance, the FDA guidance will have little impact at Therion Biologics Corp., in Cambridge. Testing their therapeutic vaccines at a super low dose might not trigger the immune response they seek to attack tumors. ”We might be sort of getting a false negative," said Tom Schuetz, the company’s chief medical officer.
A new nonprofit is already working with six of the nation’s largest drug manufacturers to streamline drug development. One of the first challenges tackled by the Critical Path Institute, established six months ago in Tucson, will be validating proprietary tools that drug companies use to weed out troublesome experimental drugs earlier without forcing companies to share that intellectual property with rivals.
Each of the drug companies will test materials known to be toxic to ensure the proprietary screen yields the correct result.
The method is cheaper and faster than testing the compound in rodents and rabbits. If successful, the screening tests could substitute for the small mammal phase of safety testing. ”If they get the same kind of results, the FDA will say all of these methods are good tests of safety," said Dr. Raymond Woosley, president of the Critical Path Institute. ”Most of this stuff is simple. It’s not sexy. That’s why no one is doing it."
Diedtra Henderson can be reached at email@example.com.
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