Answers
10/05/1900
FDA Responds to ACT UP Demands
Oct. 5, 1988
FDA RESPONDS TO ACT UP DEMANDS
In recent weeks FDA has begun to receive calls about an Oct. 11 public
demonstration being planned by ACT UP (AIDS Coalition to Unleash Power), and
ACT NOW (AIDS Coalition To Network, Organize and Win), AIDS activist
organizations. They are staging the protest to demonstrate their concern
about the availability of drugs and other therapies to combat AIDS. FDA has
on several occasions met with these and other organizations that are concerned
with these issues. On Oct. 5, the Commissioner met with representatives from
these two groups to exchange views and information on what the agency is doing
to help the development of promising AIDS therapies. The following can be
used to respond to questions.
Since AIDS was observed and recognized by scientists at the Centers for
Disease Control and around the world in 198l, FDA and its sister agencies have
made the fight against this dread disease their top priority. In 1984,
scientists at the National Cancer Institute in Bethesda, Md., and at the
Pasteur Institute in Paris, isolated the virus Human Immunodeficiency Virus
(HIV) and identified it as the source of AIDS. Less than a year after the
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isolation of AIDS, FDA had licensed an ELISA test to detect the presence of
antibody in the blood -- to help protect the American blood supply.
Although a great deal has been done by the government and private industry
on AIDS research, there is as yet no cure for AIDS and no vaccine to prevent
the disease. Thus, medical scientists are concentrating on developing drugs
that can either stop the multiplication of the virus, drugs to boost the
depleted immune system of AIDS patients, and drugs to treat the
AIDS-associated opportunistic infections and other diseases which are
responsible for the lethal effects of AIDS.
A widely held notion -- that FDA actually does the clinical testing of
drugs before they are marketed -- is incorrect. Pharmaceutical manufacturers,
the National Institutes of Health and other research institutions across the
country do the testing. It is FDA's responsibility to review and analyze the
results of testing to see if a drug is safe and effective for commercial use
by the general public.
FDA has given all potential AIDS products the highest possible priority
for review. The agency's expedited review of zidovudine (marketed as Retrovir
but more commonly known as AZT), which became the first approved
treatment for AIDS, has served as the model for a number of steps the agency
has taken to speed the drug review process. FDA reviewed and approved
zidovudine's new drug application (NDA) in less than four months -- 107 days
after its submission. FDA stands ready to repeat this performance whenever a
suitable candidate drug or biological product is brought forward.
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It should be noted that FDA as a regulatory agency can not create
breakthrough drugs where they don't exist, but can and has been removing
certain regulatory requirements to help biomedical scientists capitalize on
breakthroughs when they do occur. The unfortunate fact is that there are no
candidate products to directly treat the AIDS virus pending before the agency
at this time. There are about 150 studies which the agency has approved to
examine more than 80 distinct experimental drug and biological products in
humans. But, until data from clinical trials are developed on these
experimental drugs, their status remains experimental.
FDA is very supportive of ACT UP's concern that drugs be developed as soon
as possible to treat AIDS. FDA also appreciates ACT UP's desire to know as
much as possible about experimental AIDS products. Under federal laws to
protect trade secret and commercial confidential information, FDA is
prohibited from publicly discussing or acknowledging the status of
experimental studies unless the sponsor has released this information. Thus,
it is often difficult for FDA to debunk a number of myths that have circulated
in the media and among people with AIDS about alleged drug availability and
the alleged effectiveness data for certain drugs.
Following is a list of responses to specific issues raised by ACT UP and
ACT NOW:
#1 -- Why can't there be a national registry of AIDS drug trials?
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Response -- Each month FDA does announce new research activities and
developments in AIDS. Each quarter, FDA makes a listing of as much clinical
trial information as is possible under the law, along with telephone numbers
for additional information on each drug. (A complete registry of drugs is
impossible because of federal laws to protect commercial confidential drug
development information.) The Pharmaceutical Manufacturers Association also
publishes a quarterly list with input from FDA. In addition, FDA has asked
the pharmaceutical industry to meet with ACT UP and other AIDS activist
organizations to see what additional information can be made available.
#2 -- Why can't the clinical trial process be decentralized so that AIDS
patients and their doctors can have self-determination on their treatment?
Response -- Here, the right of self-determination and flexibility must be
balanced against the urgency to get AIDS therapies developed quickly. The
fastest way to develop a drug is with carefully controlled clinical trials
such as was done with AZT in a specific patient group. The more open-ended
the design of a clinical trial, the less likely the chance the trial will
provide answers.
#3 -- Why have women of child-bearing age and children been kept from AIDS
clinical trials?
Response -- Since most antiviral drugs being considered for AIDS are
potentially quite toxic, pregnant women have not been enrolled in trials out
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of concern for the fetus. Women of childbearing age who are not pregnant and
practice a reliable method of birth control are eligible for most clinical
trials. Likewise, children were not studied in initial trials of AIDS drugs
because of the potential toxicity of the drugs. Moreover, assessing the
safety and efficacy of potential AIDS therapies in studies involving children
is much more difficult because of the dramatic differences in the ability of
children of different ages to tolerate potentially toxic substances.
Recently, however, with additional information now available about the drugs,
trials have begun in children. Similar trials in pregnant women are expected
to begin in the near future.
#4 -- Why are placebo-controlled trials necessary?
Response -- They often are not required and may be unethical if a good
treatment exists. Placebo controlled trials are not uniformly required by
FDA. Placebo-controlled trials are the fastest way to find out if an
experimental drug is effective against a disease with no known cure. Once a
treatment is found, such as AZT, it can replace a placebo as the control arm
of a clinical trial. FDA considers the design of proposed studies to insure
collection of data in a manner that balances the need for information with
ethical standards.
#5 -- Why are people with AIDS who participate in drug trials denied
access to their medical records and not advised of trial results?
Response -- Clinical trials must be conducted in this manner using codes
so that patients and doctors are not aware of what product is being used in
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each patient. Blinded -- or coded -- studies help avoid errors in determining
results caused by overenthusiasm or other forms of bias among patients and
investigators. Once trials are completed, it is appropriate in most instances
for patients and their doctors to receive trial results. Since these results
are often proprietary, FDA can encourage but not demand that sponsors provide
this information.
#6 -- Why do AIDS drug trials forbid participants from taking preventive
treatments?
Response -- AIDS drug trials do not uniformly forbid patients from taking
preventive treatments. However, there are several factors that make the use
of preventive treatments in some clinical trials counter productive. For
example, no form of prophylaxis for pneumocystis carinii pneumonia has been
established as effective at this time. For earlier stages of the disease,
before the immune system has become seriously compromised, patients are not at
risk for pneumocystis carinii pneumonia and therefore prophylaxis is not known
to be of benefit at this time.
FDA Commissioner Frank E. Young, M.D., Ph.D. said, "FDA regards the ACT
UP/ACT NOW demonstration as another opportunity for information exchange and
outreach. I believe that the FDA is part of the solution to the AIDS problem
and that well-informed activist groups, health organizations and citizens
groups also contribute to the fight against this deadly disease."