Washington Editor

WASHINGTON - In a white paper that was made public last spring, the FDA said there was a lack of concerted effort to apply new scientific knowledge such as gene expression, analytic methods and bioinformatics, to product development.

Enter the Critical Path Initiative, through which the FDA is urging the development of several solutions to that problem, such as the use of new genetic technologies to create animal models that are more predictive of human responses to drugs. Also, it suggested that information technologies and amassed data on the safety and efficacy of approved and failed products could be employed to predict outcomes, the agency said. It also pointed to a need for new biomarkers and better trial designs.

"There's a huge investment out there in basic research and discovery," said Janet Woodcock, the FDA's acting deputy commissioner for operations, during a public broadcast on the topic in December. "Within the FDA, we're looking at a flat pipeline, despite all the investment in research, despite all the discoveries of the past 30 years and more recent genomics-related discoveries. We are not seeing any surge in new, innovative therapies."

And that's where Pharsight Corp. comes in.

"We've always had a drug development cycle that has been somewhat dependent upon blockbuster success," said Shawn O'Connor, the president and CEO of Pharsight Corp., whose predictive technology was recently used by the FDA. "But the delivery of a blockbuster drug has overshadowed a lot of failed attempts and missteps along the way with other drugs. When those blockbusters become fewer and fewer, some of these blemishes become more apparent."

The agency made use of Pharsight's Trial Simulator program as part of a modeling effort related to an undisclosed anti-HIV drug, in preparation for an end-of-Phase IIa meeting. O'Connor said the agency came to the company for input related to the Critical Path Initiative.

"The Trial Simulator is a very sophisticated tool that allows one to design a clinical trial for a drug," he told BioWorld Today, "and perform a simulation of that clinical trial that allows one to anticipate the likely outcome and data that is going to be gleaned from that clinical trial, and allows one to assess the value of that information."

Regulatory interactions with drug sponsors at a point such as the end of Phase IIa are being encouraged by the FDA. As a result, the agency expects to provide sponsors of clinical trials with input earlier in the drug development cycle, aided by predictive tools such as the simulation software provided by Pharsight, to create optimal clinical trial protocols for late Phase II and Phase III studies.

Pharsight, a 10-year-old company headquartered in Mountain View, Calif., provides modeling and simulation software such as WinNonlin for pharmacokinetic and pharmacodynamic modeling, the Pharsight Knowledge Server (PKS) data repository and the Trial Simulator program. It also employs scientific consultants who apply and deploy those technologies for clients. The company, which has generated seven straight quarters of revenue growth and four consecutive quarters of profitability, boasts more than 900 clients in the biotech and pharmaceutical space. O'Connor said that the same services that have attracted so many drug companies to Pharsight are applicable within the framework of the Critical Path Initiative.

"We've got a situation whose characteristics leave tremendous room for improvement," he said. "So if the objective is to improve, then certainly one of the ways to do that is through the use of modeling and simulation."

On Thursday, Pharsight's shares (OTCBB:PHST) gained 21 cents, or 17.7 percent, to close at $1.40.