U.S. Moves to Improve Drug-Approval Process

FDA initiatives designed to aid scientists in early stages of research

THURSDAY, Jan. 12, 2006 (HealthDay News) -- The U.S. government is introducing measures to alleviate barriers to drug development, with an eye toward speeding the process of getting medicines from the lab to the people who need them.

The changes affect the earliest stages of clinical research, officials from the U.S. Food and Drug Administration announced Thursday.

The current process of drug, biologic and device approval and testing is "long, complex and expensive," Dr. Andrew von Eschenbach, acting commissioner of the FDA, said at a news conference. "Nine out of 10 compounds in the lab fail in human studies and in large part because they behave differently in people than in animals or in laboratory tests."

The new initiatives are intended to help researchers identify earlier whether a particular compound really has promise for humans.

New drug approvals in the United States are slowing, according to a recent report in The New York Times. The FDA approved only 20 new drugs in 2005, down from 36 in 2004. The downturn comes even as pharmaceutical industry spending soars, suggesting that therapies are stalling in the lab.

The first measure announced Thursday is called Exploratory IND (Investigational New Drug) Studies guidance. It's designed to serve as a sort of adjunct to traditional Phase 1 drug trials.

Conventional Phase I testing involves giving increasing doses of a drug to see how much humans can tolerate. The new trials, known as "Phase 0" or "Pre-phase 1," will involve giving microdoses of less than 1/100th of a therapeutic dose to eight or 10 subjects to see if the compound actually has any biological effect.

"This is particularly important for academic researchers who are seeking to find out if discoveries in the laboratory actually have promise in people," said Dr. Janet Woodcock, the FDA deputy commissioner for operations. "Now we can learn tremendous amounts of information without exposing people to very high doses."

The new tests would still come after extensive animal and laboratory testing, and stringent informed-consent procedures for human subjects will remain in place, officials said.

The second initiative concerns good manufacturing practices (GMP) compliance for Phase I trials. To date, regulations have taken a one-size-fits-all approach. "You had the same requirements for huge plants making millions of doses and for the production of tiny doses for initial human use," Woodcock said.

The new measures will allow the FDA "for the first time to give direction and advice to researchers on how to safely prepare and produce small quantities of compounds in the laboratory that can then be used in people," Woodcock said. "This will be especially valuable for [the] National Institutes of Health, academic and medical schools that have many discoveries but no large facilities at their disposal."

Dr. Steven Rosenberg, chief of surgery at the National Cancer Institute, said, "We have been at the mercy of large biotech and pharmaceutical companies that have the resources to fulfill very stringent regulations. These new guidances and regulations are going to facilitate greatly our ability to take new ideas to small numbers of patients with desperate disease and test those agents. This will eliminate those that don't work from those that are likely to work."

The initiatives are likely to be especially helpful in cancer, one of the biggest health problems the country faces, officials said.

Anna D. Barker, deputy director of advanced technologies and strategic partnerships at the National Cancer Institute, said, "We have unparalleled progress in molecular genetics and molecular diagnostics and immunotherapy. We have a perfect storm of opportunity here to really make progress against cancer, but one of the areas we need to focus on is streamlining the drug-development process."

"These new guidance documents really address areas of critical importance to thousands of investigators working on new cancer drugs," Barker added. "We will be able to encourage our investigators to begin to look at a larger number of agents in microdoses, and hopefully turn these into a larger number of drugs in later clinical development for cancer."

The documents released Thursday are part of the FDA's Critical Path Initiative, launched in March 2004 to streamline and modernize the drug-development process.

"There's a bottleneck getting drugs to people and we may now be able to test thousands of compounds that would not have been evaluated under the prior method," Woodcock said.

More information

Visit the FDA for more on the new initiatives.

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