AIDS TREATMENT NEWS No. 061 - July 29, 1988
In a surprise announcement on July 23 to the Lesbian and Gay
Health Conference and AIDS Forum in Boston, U. S. Food and Drug
Administration Commissioner Frank E. Young told an audience of
several hundred people that the FDA would allow patients to
import small quantities of unapproved medicines for personal
use. The policy applies to AIDS and other diseases as well.
For some time the FDA has quietly not objected to patients
bringing small quantities of medicines from abroad, if they
brought them in personally when returning from a trip. However,
most patients cannot afford to fly periodically to Europe,
Japan, or elsewhere if they need medicines unavailable in the
United States. The controversy about dextran sulfate as a
possible AIDS treatment led to negotiations between the FDA and
AIDS treatment organizations, leading to a compromise allowing
patients to receive dextran sulfate by mail, under certain
conditions which both sides could live with. This compromise
has now become formalized as a written "pilot guidelines for
release of mail importations" -- to the surprise and approval
of AIDS treatment activists, who had expected the agency to
implement the compromise quietly, and only for dextran
sulfate or for other drugs for which great pressure developed.
(News of the policy announcement appeared on page 1 of the West
Coast edition of Monday's New York Times; it may have appeared
in late Sunday editions elsewhere.)
The last element of the compromise concerned what happens in
the minority of cases where packages get stopped by customs. In
those cases the patient will have to sign a form to receive the
package, acknowledging that he or she knows that the drug has
not been proved safe and effective. The FDA wanted a
physician's prescription or at least a physician's signature on
a letter stating that the patient was being monitored. The
AIDS organizations said that such an arrangement was
unworkable, as physicians would not sign due to fear of
potential lawsuits, and reluctance to put their names on an FDA
list. The compromise was that the patient would have to name
his or her physician to receive the package, if it was stopped.
The FDA had been reluctant to allow individuals to receive the
drugs by mail at all, because it feared the development of
offshore businesses set up to sell unapproved drugs to
Americans. The agency has made it clear that it will not
tolerate commercialization of unproven drugs under its new
Analysis and Comment
The new policy reflects the spirit of the "third moral appeal"
of the ARC/AIDS Vigil in San Francisco: "We appeal to the FDA
to immediately allow American physicians to prescribe medicines
and treatments for ARC and AIDS that are available to their
colleagues in other countries." (The ARC/AIDS Vigil has held a
continuous vigil and AIDS information center outside the Old
Federal Building in San Francisco, with people there 24 hours a
day every day for over two years.)
The recent FDA announcement could mark a major improvement. On
the other hand, it might prove unworkable or ineffectual; or it
might be abused, leading to pressures for its cancellation. At
the time of this writing -- two days after the announcement --
we believe that the FDA deserves credit for a good-faith effort
to develop cooperation with the AIDS community.
This policy does not address all of the research and access
problems, however. For example, it will probably do nothing to
make drugs like ampligen more quickly available.
* If it proves workable, the new policy could let us move
attention away from the underground mindset and operation, in
order to focus on the more important issue of what drugs do
work, and how should the drug trials be organized. While the
question of a person's freedom to use a treatment whether or
not it works is indeed an important issue -- for empowerment
and for other reasons -- the more important question is what
treatments do in fact work, and how can the evidence be
collected, evaluated, and applied quickly and effectively.
If the FDA's new announcement proves effective in practice,
it will let everyone focus less on fights over access, and
more on how we can work together to get treatments tested and
* Second, the new policy could remove major red-tape obsta-
cles hindering community-based trials of some of the drugs
most interesting to scientists and physicians. We have heard
that some of the most highly regarded physicians are
delighted that now they can set up community-based clinical
trials for the benefit of their patients, using the most
appropriate experimental treatments that otherwise were in
practice prohibitively difficult to get. If the patient
can order a personal supply of the drug from France, Japan,
or elsewhere, certain well-justified trials which otherwise
would not happen can now take place, leading to faster AIDS
research for everyone's benefit.
* Researchers hope that the new policy will reduce the
incidents of patients in drug trials secretly violating the
trial conditions by using other treatments and not telling
the investigators. Such secret treatment while one is in a
trial degrades the data and harms everyone, by casting a pall
over any trial results. But as long as patients are put in
"lie or die" situations by poorly designed clinical trials,
which compare medically unjustified treatment options such as
one drug alone vs a placebo, without proper management of
opportunistic infections in either study arm, no amount of
preaching will be effective.
The new FDA announcement moves away from the policy of forc-
ing patients into trials by denying other means of access to
treatments. If those who only want dextran sulfate, for
example, can get it on their own and not only through a trial,
then the motives for volunteering for a trial can shift more
toward altruism, rather than drug access.
Much work remains to be done in reconciling the needs of
researchers with those of patients and their physicians. For
example, many trials cannot recruit volunteers, often because
the studies have imposed conditions so much against the
volunteers' interests that physicians will not recommend that
their patients participate. We are seeing some hopeful signs
that researchers are moving away from forcing patients into
trials, toward making the trials more attractive -- for
example, by using blood-test "markers" such as poor blood
counts as study endpoints, instead of severe illness or death.
* And legal access to drugs not yet approved in this country
could end the festering scandal of drugs used by thousands of
people in Europe, Japan, or elsewhere being unavailable to
Americans, even when they are unquestionably the only
appropriate treatment for the patient. In the past these
drugs have been accessible only to those Americans who could
afford to travel to pick them up -- a loophole for the rich
and influential which allowed the system to continue, because
if enforcement had been tightly applied to everyone,
political opposition would long ago have forced reform.
Allowing patients to receive the drugs by mail will help to
end this shameful situation of one medicine for the rich and
another for everyone else.
It takes six to eight years to get a drug approved in the
United States -- and the clock starts only after the
physicians, scientists, and investors most knowledgeable about
the drug have good reason to be interested in it. The process
costs so much that companies often don't bother with U. S.
approval, even for drugs which unquestionably do work, meaning
that Americans will never have regular access to drugs in
common use elsewhere. The system must be reformed, but reform
takes time; allowing Americans to order drugs not approved
here is a stopgap which can save lives in the meantime.
* The new policy might prove unworkable in practice -- as
happened with the "treatment IND", announced by the FDA over
a year ago to speed access to experimental drugs for serious
or life-threatening conditions, which has had almost no
practical effect since then.
We believe that today's situation is different, however. With
the treatment IND, all the initiative had to come from the top.
Nothing happened unless both the FDA and a drug company agreed
-- and neither one had incentive to act. But allowing patients
to order personal-use quantities of unapproved drugs
decentralizes the initiative to patients and physicians. No
central approval or other action is needed.
And if patients and physicians are frustrated in their attempts
to obtain appropriate treatments, the public will know of the
problem right away. With the treatment IND, it took a year of
hoping that something was happening behind the scenes for the
public to realize that the system wasn't working.
* Another danger is that fast-buck artists will abuse and
discredit the system. They could, for example, use their
First Amendment rights to promote dubious treatments in the
U. S., while secretly benefitting from businesses they set up
There is no quick fix for this problem. The AIDS community
will have to put more effort into warning itself about frauds,
and urging people to talk with their doctors before they part
with their money. In the past we have had to work first and
foremost for access. Now, if the FDA's new policy proves work-
able, patients can focus less on how to get a drug, and more on
whether or not they should do so.