Washington Editor

BETHESDA, Md. - Although most everybody sees the need for user fees these days, safety will play a role in reauthorizing the law that mandates their use by the FDA.

That was at the forefront of a myriad of messages delivered at Monday's public meeting on the Prescription Drug User Fee Act (PDUFA), now in its third incarnation. The gathering, the first of more to come, was held as an initial entrée to passing a fourth version of the bill, as PDUFA III will sunset in less than two years. The legislation originally was written into law in 1992.

"I don't know if it's fair to draw conclusions based on this one meeting," Scott Gottlieb, the FDA's Deputy Commissioner for Medical and Scientific Affairs, told BioWorld Today.

"But I think there was a definite expression of people wanting some re-emphasis on things that we can be doing post-market, to follow drugs after they're approved. And also an expression that there is a lot we need to do to continue to make sure that the development process continues to evolve and remain sophisticated," he said.

Much of the FDA's top brass attended the meeting, all of whom pointed to the law's beneficial impact on its drug development oversight activities, which include management at the investigational phase, during the application process and beyond.

"Clinical drug review is one of the backbones of FDA," said the agency's acting commissioner, Andrew von Eschenbach, later noting that the regulatory process has grown "more rigorous, more transparent and more efficient than ever before" during the PDUFA era. Since the law went into effect, said Janet Woodcock, the agency's deputy commissioner of operations, it has had "a significant increase in the number of scientific staff" from 1,300 people in 1992 to about 2,500 now.

Along those lines, Steven Galson, the director of the FDA's Center for Drug Evaluation and Research, said PDUFA has allowed his division "to dramatically increase our scientific expertise." Jesse Goodman, the director of the agency's Center for Biologics Evaluation and Research, said that the law has supported "an expert and efficient managed review process." He also praised a resulting increase in agency-industry interactions.

However, the FDA leaders also noted that some of their functions are challenged by growing workloads. Notably, during the last fiscal year, fees generated by PDUFA surpassed congressional funding appropriated to the agency for product review - a first since it became law. There are concerns that the government - facing a massive deficit - could use the PDUFA fees to justify further reducing funding.

But since PDUFA passed, review periods have decreased, a fact held up in support of its success. And although some worry that shorter review times threaten drug safety, the numbers show no increase in drugs pulled from market since the legislation was written into law.

But although the length of reviews has declined, clinical development has slowed, a matter "somewhat of concern," said Kenneth Kaitin, the director of the Tufts Center for the Study of Drug Development. He said the FDA should work to "identify and address regulation-related causes of lengthening clinical development times."

To improve drug development slowdowns, new user fees should go toward the so-called "Critical Path Initiative," suggested Raymond Woosley, the president and CEO of the Critical Path Institute at the University of Arizona, adding that the FDA does not have the funding to participate in it. He called for matching congressional appropriations, noting that "shared investment by government and industry" would improve lagging clinical development.

"People are concerned about the pipeline," Gottlieb said, "and the fact that the complexity of drugs are increasing. There are things we need to do to try to rationalize the development process through things like Critical Path."

The Biotechnology Industry Organization supported the reauthorization. Speaking on behalf of the trade association, Mary Lawton, Genzyme Corp.'s senior vice president of global regulatory affairs and corporate quality systems, called the law "a direct contributor" to better patient access to drugs. She added that BIO would push for more consistency on trade name review, noting that it is not presently conducted in a timely manner.

Another suggestion for improvement came from Diane Dorman, the vice president of the National Organization for Rare Disorders, who said the law's fourth reiteration ought to include more waivers for companies developing FDA-designated orphan drugs. Other patient organization representatives said their members want a seat at the reauthorization table.

PDUFA has its naysayers, though, and many at the meeting voiced criticism of a perceived coziness between the FDA and the drug industry. Some charged that the company/FDA relationship built through PDUFA has led to diminished safety oversight.

It "undercuts the independence and objectivity of the agency," said William Vaughan, a senior policy analyst for the Consumers Union. Alison Rein, the assistant director for food and health policy at the National Consumers League, noted that user fees should better address the FDA's ability to review and regulate direct-to-consumer advertising.

PDUFA critics grudgingly acknowledged the fiscal need for user fees, given Vaughan's assertion that "the federal budget is a disaster."

Vaughan added that they should be collected and used without "industry-entangling strings," especially relative to safety matters such as post-approval studies and surveillance. Arthur Levin, the director of the Center for Medical Consumers, stressed that safety should receive a fair share of the money. Other speakers from professional health groups also called for better funding for safety and surveillance.

In contrast, Ellen Sigal, the chairwoman of Friends for Cancer Research, cautioned against getting "consumed with safety." She added that PDUFA "has been a very good thing for the patient community, and certainly the cancer community," and later noted that the FDA should look to disease foundations and patient advocacy groups as "other sources of funding."

Going forward, the FDA will meet with PDUFA stakeholders to frame specifics for passing a fourth version of the law before PDUFA III expires on Sept. 30, 2007.