FDA Issues Final Rules On 'Fast Track' Drugs

WASHINGTON—The Food and Drug Administration has given final approval to a set of regulations that will give some patients with immediate life-threatening diseases quicker access to experimental drugs. The rules have been revised in an attempt to allay scientists' fears that such a "fast track" would abandon traditional safety requirements and jeopardize clinical trials, and drug companies' concerns that it might prolong the process of gaining final approval. Yet the final rules may not have put those fears to rest. Charles Moertel, a cancer researcher at the Mayo Clinic, said the final version doesn't please him any better than the initial draft. "It fundamentally doesn't do anything but put a verbal smoke screen around the same reproposed rules," he said in a telephone interview. "In sum, it's a totally irresponsible regulatory act." The new rules governing investigational new drugs (IND) will make it possible for AIDS victims and others to receive experimental drugs when there is no comparable or alternative drug or other therapy available. Under the rules, manufacturers can make experimental drugs available to physicians with less testing than in the past. Administration officials acknowledge that the impetus behind the rule change—a proposal that has languished since it was first suggested in 1983—comes from a growing demand to help those with AIDS. The FDA received more than 300 comments to its March 19 draft from scientists, consumer groups and others. Most of the comments recognized the need for a mechanism to provide compassionate use of experimental drugs for the seriously ill, but few thought the original proposal was the answer. "Release of any drug prior to demonstration of clear clinical benefit would make it impossible to conduct properly controlled trials of drug safety and efficacy," wrote AIDS researcher Martin Hirsch of Harvard Medical School. If such drugs were made available, he and others noted, no one would be willing to serve as a control to determine the drug's efficacy. The Kefauver-Harris Act of 1962, triggered by the thalidomide tragedy in which pregnant women who had taken the sedative gave birth to severely deformed children, was evoked by several witnesses at a congressional hearing on the proposal. Moertel testified that the proposal, in effect, repeals the 1962 law. He predicted it "will have a seriously detrimental effect on drug research." Loss of Data Concern from agency scientists also surfaced at the hearing. A memo from Robert Temple, director of FDA's Office of Drug Research and Review, declared that "we don't object to treatment INDs. We invented them." But, Temple wrote in a note to his boss, Paul Parkman, "data needed to determine whether a drug is unsafe or fraudulent will never become available if the drug is not extensively studied." Temple was also troubled by the apparent disregard for the value of collecting information on efficacy. "There seems to be a lack of any requirement for a reason to believe the drug works," he wrote. In response, the agency modified the rules so that in cases of serious disease—including Alzheimer's disease or advanced multiple sclerosis—it can deny release of a drug if there is "insufficient evidence of safety and effectiveness." In cases of life-threatening disease, the agency may deny release if scientific evidence fails to provide a "reasonable basis" for concluding that the drug may be effective and would not expose patients to "unreasonable and significant" risk. The initial proposal also allowed manufacturers to charge for the drugs, a provision seen as an incentive to the pharmaceutical industry, and welcomed by many fledgling biotechnology firms as a way of recouping some of the millions of dollars spent on developing such drugs prior to FDA approval. The sale of certain drugs might lead to a two-tiered system of treatment, warned several patient groups, in which the wealthy can afford to purchase the experimental therapy and the poor are forced into clinical trials. Such an outcome would violate present regulations involving human subjects, observed Charles McCarthy, who heads NIH's Office for Protection from Research Risks, because of the requirement that the selection of. the human subjects for the research be equitable. The final rules were tightened to require manufacturers to show why they needed to charge for a drug and to limit the price of the drug to the actual costs of manufacture, research, development and handling. Published in the May 22 Federal Register, the rules go into effect June 22. Marwick writes for the Journal of the American Medical Association.

FDA Issues Final Rules On 'Fast Track' Drugs

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