By Lisa Seachrist

Washington Editor

Industry and patient groups aired their views on renewing the Prescription Drug and User Fee Act (PDUFA) and FDA reform at a hearing of the Senate Labor and Human Resources Committee Friday.

Noting that "we and our families will all be patients one day," Chairman Jim Jeffords (R-VT) called for a bipartisan effort to get PDUFA reauthorization and FDA reform legislation on track with the appropriations process at the beginning of May.

The act, which allows the FDA to assess user fees to drug sponsors in return for reviewing the drug under performance standards, is set to expire at the end of September. At a March 19 hearing of the committee, Jeffords stated his intention to link PDUFA with larger FDA reform.

"Getting PDUFA reauthorized should proceed in a non-partisan atmosphere," Sen. Barbara Mikulski (D-MD) said. "I fully support PDUFA and believe it should not have a partisan patina to it."

Representing both the Biotechnology Industry Organization (BIO) and the Pharmaceutical Research and Manufacturers Association (PhRMA), Gordon Binder, Chairman and CEO of Amgen Inc., told the committee that the biotech and pharmaceutical industries support a five-year renewal of the PDUFA, level funding for the agency and FDA reform legislation.

"PDUFA is a success story," Binder said. "It has reduced approval times by nearly half and in 1996 the agency approved 53 new drugs plus nine new biologics. "

Binder noted that as a result of PDUFA, the agency has received $327 million dollars and hired 600 new reviewers to meet the performance goals set forth as part of the act.

In his testimony, Binder maintained that PDUFA is based on four bedrock principles: it represents a long-term commitment by Congress, requires that the fees be added to the FDA's baseline appropriations, dedicates the fees to the drug review process, and provides quantifiable performance standards.

In reference to suggestions by some members of Congress, Binder said that it is not enough to simply renew PDUFA on a year-by-year basis. "Relying on a year-to-year authorization of PDUFA will hamper the agency's ability to plan and implement needed changes," he said.

Negotiations between FDA and industry representatives have resulted in a "PDUFA II" proposal that includes a 21 percent increase in user fees as well as timelines for setting key meetings, a 30-day response deadline for the agency to answer sponsor's inquiries and written protocol agreements.

Mikulski asked Binder if the industry actually supported an increase in fees and Binder responded, "We are practical people. And, we believe these fees will further accelerate approvals."

However, Binder also noted that a failure to fully fund the agency "will scuttle the promise of PDUFA II."

The Clinton Administration budget for fiscal year 1998 calls for an 8 percent reduction in the agency's appropriations to be made up by a variety of user fees. Binder maintained that without level funding of the agency, there will be inevitable slow downs in the drug approval process and that current proposal could result in a large staff reduction at the agency.

In addition, the proposal flies in the face of current PDUFA legislation which earmarks the funds collected in user fees for the drug approval process. The administration's plan appears to have little support in the committee as Mikulski was willing to speak for herself and members of her party saying "the Democrats support full funding for the FDA."

Binder also called attention to efforts between the agency and industry to devise a reform plan. Those talks have resulted in proposals that one clinical trial may be sufficient for a new drug application (NDA), proposals for electronic submissions of data and proposals to streamline the supplemental NDA.

When Jeffords asked whether the industry desired third-party review of NDAs, Binder replied, "Under PDUFA the agency has every right to seek outside review, however, an adequately funded agency should be able to keep review in house."

Mikulski then noted that much work had been done on FDA reform in the last congress by former Sen. Nancy Kassebaum (R-Kan.). Mikulski asked for the industry's perspective on the hammers provision which would automatically send an application to outside reviewers if the agency review took longer than 180 days.

"We believe that the hammers were one of the few provisions in Sen. Kassebaum's bill that was highly controversial and that they were one of the reasons that the bill was not passed," Binder said. "The option is enough, we think it is inappropriate to force the agency to go to outside review."

Jeffords intends to introduce a PDUFA reauthorization and FDA reform bill by the end of May. *