WASHINGTON — In a historic change in the regulation of the nation's pharmaceutical industry, the Food and Drug Administration on Thursday gave its approval to a regulation that will make experimental drugs significantly more available to seriously ill or dying patients.
The controversial new rule will allow people with "immediately life-threatening diseases" to decide for themselves either to take an experimental drug--with all its concomitant risks and side effects--or die untreated.
The rule authorizes the FDA commissioner to make available drugs that appear promising in clinical experiments, on a case-by-case basis. One prerequisite is that there be "no comparable or satisfactory alternative therapy" for the patient or the ailment in question.
The new regulation, to take effect in 30 days, was hailed by the pharmaceutical industry as being long overdue.
But it was harshly criticized by many leading biomedical researchers and physicians, including the president of the American Heart Assn. and the chief medical officer of the American Cancer Society. They said making unproven drugs broadly available unnecessarily endangers the well-being of already very ill people.
The new rule, spurred in part by the AIDS epidemic and the Reagan Administration's commitment to deregulation, was published Thursday in the Federal Register, taking up 53 pages.
It applies only to drugs that are already undergoing traditional tests in humans for safety and efficacy. The regulation also specifies that such clinical experiments must continue even as the test drug is being made available.
Patients who will qualify for such drugs include those for whom there is "a reasonably likelihood that death will occur within a matter of months," according to the regulation. Such patients also must demonstrate that they have exactly the form of the disease for which the test drug is undergoing clinical trials.
A Major Priority
In addition, physicians of such patients must demonstrate that they are qualified to supervise the administration of such experimental drugs.
Dr. Frank Young, in an interview, said that providing "desperately ill" patients with "breakthrough drugs" has been among his major priorities since he became FDA commissioner nearly three years ago.
Young noted that certain experimental drugs for several diseases have been available in the past, particularly in individual cases, and he added:
"The terrible disease AIDS and the desperate need for an effective treatment have focused public attention on this issue as never before."
An FDA spokesman cited the recent experience with azidothymidine, or AZT, an experimental drug against acquired immune deficiency syndrome, as "sort of a pilot" for the new regulation.
AZT was being tested in about 200 AIDS patients when it appeared that its recipients were living longer than AIDS patients in the clinical trial who received only a placebo. At that point, even though the trial was far from over, the FDA allowed the drug's manufacturer to make it available to all 6,000 AIDS patients who had the same kind of pneumonia as those who had responded to AZT in the tests.
Among the critics of the new rule is Alan C. Sartorelli, president of the American Assn. for Cancer Research, an organization composed chiefly of researchers who conduct trials of experimental cancer drugs.
The rule would "create chaos," he predicted. "There will be patients dying from the effects of the drugs because many (cancer) drugs are very toxic," said Sartorelli, who directs the comprehensive cancer center at Yale University.
He said many community physicians who presumably would be administering test drugs may not be prepared to handle the severe side effects, such as kidney and liver failure, that such toxic agents can cause.
"I understand why terminally ill patients want to be able to decide what happens to their body," Sartorelli said. "But in cancer, we are always concerned that patients not be given drugs that will harm more than they help."
Another critic, Dr. Kenneth Shine, president of the American Heart Assn. and dean of the UCLA Medical School, said he is concerned that drug companies will be less motivated to go to the expense of conducting a series of trials to demonstrate a drug's efficacy if, under the new regulation, they might be able to sell the drug to patients who are outside clinical trials.
According to the regulation, a drug company or other sponsors of a drug are allowed to charge patients to recover the costs of the drug, including its manufacture, the firm's research and development costs and handling expenses.
Although these costs are presently covered for experimental medical devices, this is the first time this provision has been applied to experimental drugs.
Distribution of the drugs will be limited to licensed medical practitioners who are qualified by training and experience to use the drugs.