FDA INDEPENDENCE BILL WILL BE CONSIDERED BY SEN. KENNEDY; SEN. HATCH SEEKS HELP FOR PREPARING EXPEDITED APPROVALS BILL -- SENATE HEARING ON EDWARDS REPORT

Sen. Kennedy (D--Mass.) will consider legislation to make FDA a free-standing agency within HHS, he said at a May 15 Senate Labor & Human Resources Committee hearing on the final report of the HHS Advisory Committee on FDA (Edwards Committee). "It sounds like [HHS Secretary Louis] Sullivan isn't going to give the commissioner direct reporting [authority,] so it would appear we'll probably have to fashion some legislation here to achieve that," the Senate committee chairman said. Sullivan disagreed with an Edwards Committee recommendation that FDA should be separated from the Public Health Service. Sullivan said he has made himself directly accessible to Commissioner Kessler and that only 10 of more than 700 regulations implemented by FDA last year were reviewed by the secretary's office. Before the hearing, the Edwards Committee held its last meeting at HHS after a year-and-a-half of deliberations to present the secretary with the final report, which contains 29 recommendations on how to improve FDA. Rep. Waxman (D-Calif.) reportedly is considering holding another hearing on the Edwards Committee final report in his House Energy & Commerce/Health Subcommittee. The secretary contended that it would be a "serious mistake" to separate FDA from other health agencies within the PHS whose scientific activities must be coordinated. "It's important to understand that FDA is not just a regulatory agency; it acts on a scientific base," Sullivan noted. "It therefore requires the best degree of coordination with our scientists throughout the PHS, specifically the National Institutes of Health and the Centers for Disease Control." Sullivan maintained that dissociation of FDA from other PHS agencies would prove to be an "encumbrance" to scientific coordination. Asked to respond to Sullivan's defense of including FDA within the PHS, advisory committee Chairman Charles Edwards, MD, (Scripps Clinic president) echoed the committee's conclusion that the PHS represents an unnecessary layer of regulatory review. Acknowledging that not all FDA regulations are re-reviewed by the office of the HHS secretary, Edwards maintained that "most of the regulations do go through the office of the assistant secretary" for health, which heads the PHS. Furthermore, Edwards continued, FDA should be free to make independent policy decisions as well as regulatory ones. "We're talking about the ability of the commissioner to make judgments as it relates to personnel, as it relates to facilities, as it relates to other policies within the agency which are immediate problems," he said. "One of the reasons the agency is in a crisis [now] is because there is a delay in the decisionmaking process." Regarding HHS' desire to coordinate FDA's scientific activities with other PHS agencies, Edwards contended that independence does not preclude coordination. Independent agencies like the Environmental Protection Agency "also coordinate their science with other appropriate government and non-government organizations," he said. In addition, FDA differs from other PHS agencies in that it is "a regulatory agency," Edwards noted. "As such, its mission, its goals, its priorities are totally different from other health agencies within the department, and I think you cannot let this agency any longer get burdened down by the bureaucracy of the PHS." The Edwards Committee's final report states that independence would "strengthen" FDA. Coupled with restoration of FDA authority to issue regulations, the organizational independence of the agency "would strengthen FDA's capacity to manage its own affairs and to deal more effectively with agencies outside HHS." Independence would accord the FDA commissioner "a status commensurate with the heads of the sister regulatory agencies with which the FDA regularly deals, including the Environmental Protection Agency, the Occupational Safety and Health Administration and the Federal Trade Commission," the committee said. The change "also might improve its ability to compete for senior scientists and reduce the time for approval of SES appointments." Sen. Hatch (R-Utah), the Senate committee's ranking Republican, indicated Congress will consider separate legislation to authorize conditional approvals of drugs for life-threatening diseases. Hatch said he supports the Edwards Committee's recommendation for expedited approvals, and asked for help "to formulate the type of legislation needed to accomplish that." Hatch said the Senate committee wants the proposal "to go forward" because "there are some critical drugs out there right now that probably should be moved along in the interest of some of these very difficult populations that have these sort of life- threatening diseases." One experimental drug that appears to meet Hatch's description is Warner-Lambert's Cognex for Alzheimer's disease. Warner-Lambert Chairman and CEO Joseph Williams is a member of the Edwards Committee. Sullivan testified that he also supports expedited review procedures for drugs to treat serious illnesses. Asked by Hatch whether HHS has "any plans to streamline the drug approval process," Sullivan replied that Commissioner "Kessler is already at work with reorganization plans within the agency" to facilitate such procedures. "I expect to have those coming forward to me in the very near future." An internal FDA task force has been working on the "conditional approval" idea since January ("The Pink Sheet" Jan. 21, T&G-1). Edwards Committee member Lawrence Horowitz, MD, said "the critical question" for FDA in addressing the recommendation for expedited approvals is how to strike a "balance" in the effort to "prevent harm by not letting unsafe medicines on the market." Horowitz noted that the Edwards Committee did not recommend "conditional" approvals, just "alternative pathways" to approval. "No additional resources are necessary to do it," he maintained. "This is all" a matter of "internal management"; it is currently "within the power of the agency." The former Kennedy staffer asserted that "it costs lives by taking six months longer to get a needed cancer drug on the market." Noting that "this [Senate] committee in the 1970s recognized" that drugs for certain diseases require different review procedures, Horowitz recalled that the Senate "unanimously" passed "legislation that provided for conditional approvals" but was not passed by the House. "It took AIDS many, many years later to recognize [the] fundamental fact" that different diseases can require different drug reviews, he asserted. The Administration and the Congress should "follow through on policies that we know are necessary," he said. " Biotechnology did not exist" when most FDA regulations were written, and "the regulatory methods that were put into effect then are not appropriate today for all cases," Horowitz asserted. The standards of safety and efficacy remain appropriate, "but the way you meet them are very different; they have to be tailored to particular circumstances." FDA drug reviews must consider the disease, any available therapies and "what degree of risk patients want to take," Horowitz continued. "There ought to be a regulatory process" in which new drugs can be made available to patients with terminal diseases for which adequate therapies do not exist, all information "we know and don't know" is provided, and "postmarketing surveillance to determine exactly what did happen" is required.