Lisa Seachrist

Washington Editor

Noting the importance of getting new drugs to sick patients quickly, patient organizations are beginning to weigh in on the importance of reauthorizing the Prescription Drug and User Fee Act of 1992 (PDUFA) before it expires at the end of September.

Acting FDA commissioner Michael Friedman and David Blois, vice president of world regulatory affairs at Merck & Co., addressed a patient and research coalition on Capitol Hill highlighting the strides made to reduce drug approval times in order to emphasize the importance of reauthorizing PDUFA.

"In 1986, the average time to approve a drug was 33 months; in 1996 that time had dropped to 14.3 months," Friedman told the FDA council. "It shows you that in a cooperative environment there is a lot the industry and agency can achieve together."

Blois agreed with Friedman, noting that "in 1992, the industry felt that what we did with PDUFA was an experiment. It was a very successful experiment."

That 1992 experiment involved industry paying FDA fees in order to get the drug reviews completed in 12 months for standard drugs and 6 months for priority drugs. Friedman pointed out that at the time there were very substantial questions raised about the performance of the agency.

"A big component in PDUFA was adding resources to allow drug reviews to move more quickly," Friedman said.

Blois noted that the fees did substantially improve approval times, but "the biggest achievement of PDUFA was to lend predictability to the process of drug review."

As successful as PDUFA has been, it was drafted with a sunset clause that causes the legislation to expire at the end of September. FDA and industry representatives have been working on the terms for reauthorizing PDUFA for the past six months.

PDUFA II, as the reauthorization legislation is being referred to, would enhance the predictability gained in PDUFA I, Blois said. For one, the agency would provide companies with questions about their applications as soon as the questions arise within the agency, rather than waiting to dump the entire list on companies at the end of the review. And the agency would respond to requests for a meeting within 30 days. Blois also noted that the agency and industry have agreed to a format for dispute resolution.

Several House and Senate hearings have been held on the issue. Sen. Jim Jeffords (R-Vt.), chair of the Labor and Human Resources Committee, and Rep. Michael Bilirakis (R-Fla.), chair of the Subcommittee on Health and Envi-
ronment, have stated their intention to attach wider FDA reforms to PDUFA reauthorization.

"Our druthers would be for two bills, one addressing PDUFA reauthorization and one addressing FDA modernization," Friedman said. "But we recognize that many in Congress see this as a perfect opportunity for achieving FDA reform."

Blois noted that while FDA reform is a high priority for industry, "PDUFA has a deadline that makes it a vital priority."

As a result of PDUFA, the agency has hired more than 600 full time employees to ensure that reviews occur in a timely manner. The vast majority of those hired were product reviewers. Should Congress fail to reauthorize PDUFA or the President fail to sign it into law, the agency could not afford to keep the additional reviewers.

"There are no resources to cover the [reviewers]," Friedman said. "It would be devastating should PDUFA be allowed to expire, because we have assembled a cadre of extremely skilled reviewers and we don't want to lose that skill. If we do, the public health will suffer."

However, Blois said, "One of the pillars of PDUFA that allowed industry to support it was that the fees would be additive to the appropriations levels." The current Clinton Administration budget calls for an 8 percent cut in funding that would be paid for by a wide variety of user fees. Blois noted that should the cut remain in the FY98 budget, "We would have to go back to the table to renegotiate the terms of PDUFA II."

The National Health Council, a consortium of more than 100 national health organizations, weighed in on support not only for PDUFA, which the organization maintains provides "timely access to innovative and potentially lifesaving therapies," but also urged Congress to provide funding for the agency in order to guarantee reauthorization of PDUFA.

"PDUFA is entirely dependent on funding levels," Martha Keys, senior public policy advisor for the National Multiple Sclerosis (MS) Society, said. "Congress doesn't even know how to write the legislation without knowing the funding situation."

In addition, Keys said, for patients who have diseases for which there are few treatments, like MS, PDUFA is vitally important.

"I can think of little that is more important for these patients," Keys said. "It is only with the timely emergence of new therapies that they have any hope to combat their disease. PDUFA resulted in drugs becoming available 15 months sooner. That is a tremendous amount of time for these patients." *