Important note: Information in this article was accurate in 1987. The state of the art may have changed since the publication date.
Food and Drug Administration, U.S. Department of Health and Human Services - May 21, 1987
Bill Grigg; (301) 443-4177
"Under the final procedures announced today," Secretary Bowen said, "drugs that are in controlled clinical trials can be provided outside those trials to treat patients with serious or immediately life-threatening diseases such as AIDS or certain heart conditions for which no comparable or satisfactory alternate therapy exists, assuming certain safeguards are followed."
These safeguards include:
-- That the patient is fully informed of the risks and expressly consents.
-- That the drug is not promoted or otherwise "commercialized", though drug companies can charge patients to recover the cost of the drug's manufacture, research, development and handling, under the same rules that already apply to medical devices.
-- That fully enrolled clinical trials are underway and continue unimpeded, and the sponsor of the drug actively pursues marketing approval of the drug with "due diligence."
In proposing the procedures March 10, FDA Commissioner Frank E. Young, M.D., Ph.D., said that when no alternative therapies exist, "an experimental drug may provide the only hope."
He said that certain heart drugs and some other drugs in research have been made available in the past. Assistant Secretary for Health, Robert E. Windom, M.D., chairperson of the PHS Executive Task Force on AIDS, added, "the terrible disease AIDS and the need for effective treatments and vaccines have focused public attention on this issue as never before."
The drug's manufacturer (or other sponsor) must notify FDA 30 days before broadening the distribution of the drug or charging for it.
To prevent use of the procedures for useless, dangerous or fraudulent products, the new rules stipulate that FDA's commissioner can block the distribution of products when the evidence submitted by the drug's sponsor fails to provide a reasonable basis for concluding that the drug may be effective or would not expose patients to unreasonable risk.
In addition, the sponsor of the drug (generally a drug company, but sometimes a university or government research agency) is obliged, according to the procedures, to distribute the drug only to licensed medical practitioners who are qualified by training and experience to prescribe the drug.
"Life-threatening diseases" are defined as illnesses at a stage at which death is likely to occur within months, or in which premature death is likely without early treatment. Examples of such diseases listed in the preamble of the regulation include advanced congestive heart failure, herpes simplex encephalitis, advanced and incurable cancers, advanced emphysema, bacterial endocarditis (a dangerous infection of the heart) and subarachnoid hemorrhage (a sudden bleeding in the brain) as well as advanced AIDS and other severe immunodeficiency syndromes, and uncontrollable cardiac arrythmias.
"Serious" diseases include, for example, Alzheimer's, advanced multiple sclerosis and advanced Parkinson's disease, transient ischemic attacks (strokes), progressive ankylosing spondylitis (a severe arthritis of the spine), active and advanced lupus and certain forms of epilepsy and diabetes.
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SOURCE: Food and Drug Administration (FDA).
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