Washington Editor

WASHINGTON - Serious scientific deficiencies at the FDA have put the agency in the precarious position of not being able to meet its current or emerging regulatory responsibilities, a panel of federal advisers said.

The agency's scientific base has eroded and its scientific organizational structure is weak, a subcommittee of the Science Board to the FDA said in a new report, which is scheduled to be discussed at a public meeting Monday in Gaithersburg, Md.

Advisors argued that the FDA cannot fulfill its mission of regulating drugs and vaccines and protecting the nation's food supply because its scientific work force does not have sufficient capacity and capability. While drug discovery and development has undergone revolutionary change - shifting from cellular to molecular and gene-based approaches - the FDA's evaluation methods have remained largely unchanged over the past 50 years, panelists contended.

The FDA's "inability to keep up with scientific advances means that American lives are at risk," the advisers charged. In addition, they said, the agency's information technology infrastructure is woefully inadequate.

Although there has been some progress to improve information sciences and technology at the FDA over the past few years, the advisers said that progress has been slow and significant gaps remain.

The demands on the FDA have soared in recent years due to the advance of scientific discoveries, the complexity of the new products and claims submitted to the agency for premarket review and approval, the emergence of challenging safety problems and the globalization of the industries that the FDA regulates, the advisers noted.

Yet, the agency's resources have not increased in proportion to those demands.

"This imbalance is imposing a significant risk to the integrity of the food, drug, cosmetic and device regulatory system, and hence the safety of the public," the advisers said.

Panelists noted that during the past two decades, Congress has enacted 125 statutes that directly impact the FDA's regulatory responsibilities, in addition to the core provisions of the 1938 Act and its amendments.

Those statutes generally require some type of scientific knowledge or expertise, and in some cases, laboratory research, for the agency to adequately address them, yet "none of these statutes has been accompanied by an appropriation of the new personnel and increased funding necessary to enable adequate implementation," panelists contended.

The advisers noted that the number of appropriated personnel in 2007 was roughly the same as it was 15 years ago.

Currently, each taxpaying American pays about 1.5 cents a day to fund the FDA.

"An increase to 3 cents daily would not, in our view, be a great price to pay for the assurance that our food and drug supply is, indeed, the best and safest in the world," panelists recommended.

While human and financial resources alone will not be enough to fix the FDA's deteriorating scientific structure, the advisers said that "without a substantial increase in resources, the agency is powerless to improve its performance, will fall further behind and will be unable to meet either the mandates of Congress or the expectations of the American public."

A strong FDA is "crucial for the health of our country," panelists maintained. If the FDA's science is at risk, "the nation is at risk," the advisers argued. "The benefits of a robust, progressive agency are enormous; the risks of a debilitated, underperforming organization are incalculable."

Senate Probes Planned Avastin Restrictions

Sen. Herb Kohl (D-Wis.) announced he is leading a Senate committee investigation of Genentech Inc.'s proposed plan to stop making its cancer drug Avastin (bevacizumab) available to certain compounding pharmacies.

Kohl, chairman of the Special Committee on Aging, said the Senate is concerned that Genentech's plan potentially could have a negative impact on what Medicare pays for the drug, which is widely prescribed to beneficiaries for an off-label indication of wet age-related macular degeneration (AMD).

Compounding pharmacies repackage Avastin into syringes that contain a once-monthly dose of the drug for use in the eye at a cost of about $40.

Physicians have charged that Genentech's plan to limit Avastin is a move to boost the firm's sales of Lucentis (ranibizumab), which is chemically similar but far more expensive. A once-monthly dose of Lucentis, which is approved for AMD, costs about $2,000.

The National Institutes of Health (NIH) is conducting a head-to-head study of Avastin and Lucentis in treating eye ailments, including AMD. However, South San Francisco-based Genentech has refused to be part of that comparative trial.

Genentech has contended that the repackaging of Avastin by compounding pharmacies makes the drug potentially unsafe and could put patients at risk.

If availability of Avastin is curbed, Kohl noted, it could cost taxpayers an additional $1 billion to $3 billion.

"I take very seriously the Committee's responsibility to protect and advocate on behalf of our nation's seniors," Kohl said in a letter to Susan Desmond-Hellman, Genentech's president of product development.

"Part of this responsibility is ensuring that seniors are receiving appropriate and cost-effective prescription drugs," he wrote.

Kohl asked Desmond-Hellman to turn over documents and emails to the aging committee related to Genentech's decision not to participate in the NIH study and the firm's plans to limit Avastin.

Journals Promoting Off-Label Use?

Rep. Henry A. Waxman (D-Calif.), chairman of the House Oversight and Government Reform Committee, said a plan by the FDA to allow drug companies to use journal articles to promote off-label uses of products short-circuits the drug approval process.

In a Nov. 30 letter, Waxman urged FDA Commissioner Andrew von Eschenbach to refrain from putting in place a guidance document that the California Democrat called "ill-advised."

Waxman said he recently obtained a copy of the internal document.

"A fundamental tenet of our drug and device laws is that a manufacturer cannot market a drug or device for a therapeutic use without FDA approval," he told von Eschenbach. "The draft guidance would carve a large loophole in the law and create a pathway by which drug and device manufacturers can promote unapproved (off-label) uses of their products without first obtaining FDA approval by passing out journal articles about the off-label use to physicians."

Published reports of company-funded studies "can be biased in favor of the company's product," Waxman charged. "Allowing drug and device companies to freely disseminate these articles can result in doctors using questionable study results to guide their prescribing habits."

In addition, he said, allowing marketing through journal articles "can reduce the incentive for drug and device companies to conduct the rigorous studies needed to win full FDA review and approval, leaving physicians and patients without definitive data on the benefits and risks of medical products."

Such a practice, Waxman asserted, "undercuts the prohibition on marketing of unapproved uses of drugs and devices and puts the public at risk for ineffective and dangerous uses of drugs."

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