National Cancer Institute
A linchpin of any effort to improve drug development must be improvements in the way science tests new therapies. A large part of today’s clinical trials system is an antiquated one, built around the study of one agent, one hypothesis at a time. It has become clear that a future with multi-agent therapies will demand a better testing system. In 2005, NCI received the final report from its Clinical Trials Working Group, and has been hard at work implementing many of its recommendations. This task requires the collaboration of the FDA, the Centers for Medicare and Medicaid Services, industry, and academia. Yet, despite the hurdles, NCI is the ideal leader of this effort, as a facilitator and honest broker between all parties.
In its own studies, NCI has been a pioneer in the development of so-called Phase 0 (zero) clinical trials, which help researchers and industry make smarter decisions about which experimental agents should move into more extensive human testing. “This new approach has already demonstrated its ability to significantly shorten the drug discovery process,” said Jerry Collins, M.D., director of NCI’s Developmental Therapeutics Program.
Phase 0 clinical trials — the first of which was conducted at the NIH Clinical Center in 2007, along with Abbott laboratories — use very low doses of an investigational drug in just a few patients, to determine whether the drug is likely to be biologically effective and a good candidate to advance to later phase human clinical trials, or if development should be halted before the expenditure of hundreds of millions of dollars and years of work.
Earlier testing of promising compounds in Phase 0 trials should lead to a better understanding of their molecular mechanisms of action, provide a closer approximation of what a safe but potentially effective starting dose will be, and help decrease the failure rates in late-stage oncology drug development. NCI estimates that such studies can reduce drug development times by as much as one year.
“The data you generate in Phase 0 testing allows you to design a Phase I study that is more well-informed,” said Dr. Collins. “Now you can select doses based on data that show you can achieve levels in patients that have an impact on the molecular target and the tumor. You can more rationally design your later clinical trials based on Phase 0 results.”
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