By Steve Usdin
Contributing Editor

WASHINGTON - Although President Clinton's $1.35 billion budget request for FDA for fiscal 2000 would reverse several years of flat or declining budgets, several more years of budget increases are needed to allow the agency to perform all of the tasks it has been assigned.

Congress and the White House have given FDA significant new responsibilities in recent years, the number and scientific complexity of product applications are increasing, and the agency is finding it difficult to attract and retain talented workers.

In fact, the White House budget proposal last week only starts a long process of congressional review that will provide opportunities to haggle over the actual appropriation, which is unlikely to be enacted before the fall. The Biotechnology Industry Organization (BIO) and some patient advocacy groups intend to push for Congress to provide more generous funding than the administration has requested.

Strategies for persuading Congress to increase FDA's budget, and for helping it offer compensation that is competitive with that offered by the biotech and pharmaceutical industries, will be on the agenda when FDA Commissioner Jane Henney meets separately with BIO and the Pharmaceutical Research and Manufacturers of America later this month.

Clinton's budget request submitted to Congress last week would boost FDA funding $216 million, up 18 percent over the amount actually appropriated by Congress for 1999. The total FDA request is $1.35 billion, plus authority to charge $195 million in user fees. In contrast to last year, Clinton has not built an FDA spending plan around large quantities of user fees that Congress is unlikely to approve.

If enacted, the increase would be the largest in FDA's history. But the agency has not yet released detailed documents describing how the proposed funding would be allocated. FDA stated that a portion of the increase is intended for upgrading of the agency's scientific base.

The proposal also would allocate $15.3 million to improve FDA's system for reporting on adverse side effects from drugs and medical devices. FDA also plans to beef up its product safety assurance activities, including setting a goal for inspections of each manufacturer of human and animal drugs, biologics and medical devices every two years.

In addition to the FDA budget request, the agency is likely to receive a large portion of a separate White House request for $13 million for bioterrorism-related activities, including identifying drugs and biological products that could ameliorate or prevent the toxicity of chemical, biological, radiological or nuclear substances. The agency said it will use the funds to expedite development and licensure of new vaccines for anthrax, botulinum and other toxins (see Online Links, A13).

Niggardly NIH request

Congressional champions of biomedical research chastised the White House for requesting a modest increase in funding for NIH last week. Numerous members of Congress from both parties vowed to enact a substantial boost in funding for fiscal 2000, which begins next October.

In a letter sent to President Clinton, Senators Arlen Specter (R-Penn.), Connie Mack (R-Fla.), Tom Harkin (D-Iowa) and Dianne Feinstein (D-Calif.) urged the president to provide NIH with more than the 2 percent increase included in his budget proposal.

Last year, the lawmakers said, "we worked to build on your proposal and were able to provide an increase of $2 billion in the fiscal year 1999 Omnibus Appropriations Act you signed into law last October - the largest in NIH history. This increase set us on the course for meeting our goal of doubling support for medical research in five years."

The senators called Clinton's fiscal 2000 request "a substantial retreat" from last year's consensus. "Your proposal for a 2.1 percent increase is far short of the 15 percent jump necessary to keep on track for doubling funding over 5 years. In fact, it is our understanding that your proposal would result in 1,500 fewer grants in fiscal year 2000 than in fiscal year 1999. In addition, your budget would likely result in researchers with existing NIH grants being denied inflationary adjustments."

In fact, given the politics of budget balancing, the White House may be counting on Congress to provide the additional funds for the politically popular NIH programs, allowing the president to cater to other of his constituencies in the budget proposal.

FDA push to expand cancer labels

The FDA released guidance last week describing steps it will take to facilitate and encourage the approval of supplemental indications for approved drug and biological treatments for cancer.

The guidance lays out the agency's plans for implementing provisions of the FDA Modernization Act (see Online Links, A13). It states that in some cases, the quality and quantity of evidence required to demonstrate safety and efficacy of supplemental uses will be significantly less than that required for an NDA. For example, in the refractory cancer setting, FDA may provide accelerated approval for supplemental uses on the basis of "nonrandomized studies showing that a new treatment provides a significant objective response rate with tolerable treatment toxicity."

If safety and efficacy of a product have been demonstrated in one type of cancer, the agency said a single, adequate and well-controlled, multicenter study would be capable of demonstrating acceptable safety and effectiveness in another form of cancer.

The guidance states that pediatric indications usually will be approved on the basis of a single safety study.

FDA reiterated its intention to aggressively pursue the submission of supplemental applications, and will attempt to review 10 percent to 20 percent of marketed cancer drugs every year to determine if new uses or dosing regimens should be added to the labels. The agency will solicit data and cooperation from product sponsors, government and academic investigators and patient advocacy groups to identify new indications.

In some cases, FDA said it may collect data from investigators and file a supplemental application on its own.

Paying the price for cloning

Rep. Ron Paul (R-Texas), a practicing obstetrician, has introduced the first anti-cloning legislation in the 106th Congress, the Human Cloning Prevention Act of 1999.

The bill, H.R. 571, makes any organization that engages in human cloning or cloning research ineligible to receive federal money for any activities. Under the bill, if a university or research institution performed any cloning research, it would be barred from receiving any federal grants or contracts. In a letter soliciting support for the bill from other lawmakers, Paul stated that "if a state university's biology department began experimenting with human cloning, the entire school would lose all access to federal funds until such experiments and research have ended."

H.R. 571 defines human cloning as "making an identical, or substantially identical, copy of the genetic material of an individual human being, living or deceased, so as to cultivate one or more new human cells which could, if not otherwise engineered, develop into a new individual human being."

Biotechnology Industry Organization President Carl Feldbaum said the broad definition of cloning in H.R. 571 "is unacceptable. This is something the biomedical community cannot tolerate."

While at least one other representative, Vernon Ehlers (R-Mich.), has stated an intention to introduce anti-cloning legislation, the issue is unlikely to gain traction in the Senate. According to congressional staff, Senators Bill Frist (R-Tenn.) and Christopher Bond (R-Miss.), the main backers of a failed effort to enact anti-cloning legislation last year, do not intend to introduce a bill this year.