Renewal sought for drug approval user fee law

Washington -- A growing chorus of former FDA commissioners, medical journal editors, researchers, clinicians and consumer groups are voicing concern about the reauthorization of a 15-year-old law that allows pharmaceutical companies to contribute a large portion of the Food and Drug Administration's drug approval budget in exchange for quick review of their products.

Their message is that agency funds should come solely from federal coffers and not in the form of user fees from the same industry the FDA regulates. These experts are urging Congress via letters and journal articles to eliminate the Prescription Drug User Fee Act or substantially alter it.

The user fee, they charge, is a funding mechanism that threatens the integrity of the drug approval system and even could affect the agency's already shaky safety procedures by pushing for fast approval of possibly dangerous drugs.

But Congress is intent upon reaffirming the measure, calling it a "must-do piece of legislation," according to a spokesman for Sen. Mike Enzi (R, Wyo.), ranking member of the Senate Health, Education, Labor and Pensions Committee. It is currently being considered as part of a larger bill . Without congressional action, the law will expire Sept. 30.

PDUFA supporters -- who include the FDA commissioner and pharmaceutical companies --contend that the fees paid by the firms are as necessary now as they were when the law was first enacted in 1992 to help speed drug approvals. They would like to hang onto those funds, which would amount to nearly $400 million next year, $87 million more than last year when the fees accounted for about 40% of the FDA's total human drug program.

Under the current law, user fee amounts are renegotiated every five years by the Pharmaceutical Research and Manufacturers of America, which acts as the drug companies' representative, and the FDA. Although the majority of the fees pay for quick drug approval, the new agreement will, for the first time, direct about $29 million to postmarket drug safety.

This year's burgeoning user-fee amount even prompted a supporter, Billy Tauzin, president and CEO of PhRMA, to remark that Congress should "think about whether it needs to put more skin in this game."

In a February meeting in Washington, D.C., Jane Henney, MD, FDA commissioner from 1989 to 2001, said she worried when the law was first passed that the drug review process would be perceived as being too heavily influenced by the drug industry. She urged Congress to pay close attention to the balance between user fees and appropriations when considering this year's funding levels.

What the money pays for

Although costly, PDUFA has delivered, its supporters say. For example, Tauzin said, 82 additional medical officers were hired to review new drug applications.

In urging the Senate HELP Committee to renew the measure, FDA Commissioner Andrew von Eschenbach, MD, said that during the PDUFA era, FDA reviewers approved 76 medicines for cancer, 178 anti-infective medications, 111 medicines for metabolic and endocrine disorders, and 80 for cardiovascular and renal diseases.

The amount of time needed to bring a drug to market was reduced from a median of 27 months in 1993 to 10.5 months in 2004, says a report by the George Washington University School of Public Health and Health Service. But speedy approval may come at the price of safety, the report warned. The agency's reputation has suffered over the last several years as popular drugs were withdrawn because they posed too great a health risk.

"The major concern is that the pharmaceutical industry has gotten too close to the approval process at the FDA," said Jerome Kassirer, MD, distinguished professor of medicine at Tufts University School of Medicine in Boston, who signed one of the letters to Congress. Dr. Kassirer is a former editor of the New England Journal of Medicine.

He worried that the industry had injected itself into the process of drug approval. "It's hard to ignore somebody who is paying a good part of your salary."

Another signer, Jerry Avorn, MD, professor of medicine at Harvard Medical School in Boston, called Congress' seeming willingness to reauthorize PDUFA shortsighted in the April 26 New England Journal of Medicine. "Colleagues at the FDA have told me of a worrisome side effect of PDUFA: The growing sense that the organization is accountable to the industry it regulates," Dr. Avorn added.

Others recommended that if the law had to be reauthorized to ensure that the FDA remains adequately funded, that it be continued for only one year and that several changes be instituted. Among additional suggested changes: FDA leadership be allowed to determine how the agency allocates fees collected, deadlines for quick approval be removed, and adequate resources be made available for agency activities such as scientific research and staff training.

Curt Furberg, MD, PhD, professor of Public Health Sciences at Wake Forest University School of Medicine in Winston-Salem, N.C., who also signed a letter, said he would like to see PDUFA user fees handled the same way fees are handled in other programs -- that is, by leaving it to the receiving agency to decide how to spend the money.

Dr. Furberg, who also serves on an FDA advisory committee, added that he would like the crafting of PDUFA agreements to occur in a public venue rather than "behind closed doors" between the pharmaceutical industry and the FDA.