Food and Drug Administration, U.S. Department of Health and
HHS Secretary Otis R. Bowen, M.D., today announced a revision
of the Food and Drug Administration's regulations that will
make promising experimental drugs more readily available to
desperately ill or dying patients.
"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
These safeguards include:
-- That the patient is fully informed of the risks and
-- 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
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.