By Lisa Seachrist

Washington Editor

With FDA reform legislation beginning to crop up on Capitol Hill, the Biotechnology Industry Organization (BIO) and the Pharmaceutical Researchers and Manufacturers of America (PhRMA) have included a fast-track drug program as part of their FDA modernization proposal.

The program would establish an expedited drug development and review procedure in order to get drugs designed to treat life-threatening and serious diseases to patients more quickly. The program would foster close communication between the sponsor and the agency very early in the drug development process.

"We truly think that this can make a difference in the lives of patients," said Alan Goldhammer, BIO's director of technical affairs.

Under the original Prescription Drug and User Fee Act (PDUFA-I), which is set to expire at the end of September, when a company submitted a drug for review to the agency, it would request that the drug receive a priority -- or 6-month -- review because it was designed to treat a life-threatening disease or addressed an unmet medical need. The problem with this approach is that it identified a priority drug at the time of a company's submission of a new drug application (NDA) or a biological license application (BLA).

The fast-track proposal would allow sponsors to apply for admission into the program at the time they submit a Phase I investigational new drug application or anytime thereafter. From the moment drugs are accepted into the program, they would receive special attention from the agency with prompt agency-sponsor meetings and protocol collaboration with the agency if the sponsor chooses.

Like the PDUFA-II proposal that BIO and PhRMA negotiated with the FDA, the fast-track proposal includes clinical trial design agreements that could not be unilaterally altered by the agency absent public health concerns. The proposal also encourages the development and use of appropriate surrogate endpoints.

"This proposal encourages a tighter dialogue between the agency and the sponsor," Goldhammer said. "And the agency will give more guidance to what is appropriate to get approval."

In addition, the proposal suggests that the agency perform "rolling reviews" on these priority drugs. In other words, sponsors would submit sections of their application and the agency would review these sections as they were completed. This would allow the sponsors to address any questions from the agency as they arise, rather than waiting for the NDA/BLA to be submitted, performing the entire review and then providing any questions to the sponsor.

"The sponsor would pay their user fees with their initial submission and the PDUFA time clock wouldn't begin for the agency until the submission was complete," Goldhammer said. "But this could really save time because it would allow questions to come up as they arise, and that may obviate the need to recycle an application because many questions can be resolved within a 6-month time frame."

In order to keep fast-track status focused on innovative products, the proposal prohibits "me-too" submissions from being considered under the program.

Goldhammer said BIO and PhRMA have presented their proposal to the FDA, which is reviewing it and submitting questions and concerns. However, the industry organizations have not received a full response.

"People in the agency, industry and staff on the Hill are really trying to get beyond the rhetorical and see what changes will make a difference in getting safe and effective drugs to market," Goldhammer said. "With this proposal, even if we shave only a month off the clock, we still have gotten the drug to patients faster." *