Editorial

In the short time between the FDA's letter and going to
press for this issue, and with the ongoing preparations and
people leaving for the IX International Conference on AIDS next
week in Berlin, we could only begin talking with representatives
of buyer's clubs and others about issues relevant to the FDA's
letter. This editorial outlines some of the concerns we have
heard, and some of our own. We are publishing this preliminary
outline because it is important that persons who are interested
in treatment access move quickly to decide what we want, explore
areas of consensus, and explain any concerns about current policy
directions of the FDA.

(1) While no figures are available, everything we know
suggests that the great majority of persons who obtain medicines
from abroad with the help of AIDS buyers' clubs are using them in
consultation with a physician. In fact, in the historical
examples of ddC and probably of clarithromycin, both obtained
through buyers' clubs before their U. S. approval, the common
pattern, in San Francisco at least, was for patients to go to the
buyers' clubs at the suggestion of their physician. Few patients
make such decisions on their own without medical assistance, and
the AIDS treatment community strongly discourages people from
doing so.

But for physicians, monitoring the use of an unapproved drug
when no other therapy is satisfactory, or even suggesting its use
to patients, is different from allowing themselves to be listed
on an official form used to grant their patients access to drugs
not approved in the U. S. The perceived problem is not with the
FDA, and probably not with state medical authorities, but with
malpractice insurance. Requiring the name on the form could put
physicians in the difficult position of having to balance
professional risks to themselves against the welfare of their
patients.

There is also a burden of paperwork, including (in practice)
extra trips to pick up and deliver forms, which comes on top of
the fact that the patients and physicians involved are trying to
balance many things in their lives already.

This turn toward greater enforcement of the documentation
requirement (including the physician's name) does not seem to
have resulted from any problem which actually occurred in the
context of the AIDS buyers' clubs. It seems more likely that the
real target is elsewhere -- especially offshore operations which
make a business of selling unapproved drugs to Americans (usually
not for AIDS). The FDA strongly opposes these businesses, which
often cite the personal-import policy as legal grounds to protect
what they are doing. The FDA has been accused of uneven
enforcement of the law, giving special privileges to people with
AIDS. In the continuing atmosphere of substantial public
hostility against AIDS, such charges may carry weight in some
circles, even though the FDA clearly knows the difference between
a fast-buck operation on the borderline of the laws, and a
nonprofit self-help group with no other purpose than providing
access to rational and legitimate treatment options for persons
with life-threatening diseases. The FDA has the enforcement
discretion to make this distinction, and we suspect that were it
not for the stigma around AIDS, it would not be embarrassed about
using it.

What happens in the future will depend on what the FDA
intends. In the worst case, if they want to shut down much of
the personal-use import policy, they have positioned themselves
well to do so. For in the ongoing emergencies and stress of
major illness, with hundreds of patients receiving drugs, there
will certainly be cases where documentation is not in order. As
a result, the FDA could successfully raise the issue at any time,
presenting itself to the press and public as protecting patients
from unscrupulous organizations which sold them unapproved drugs
without their physicians' involvement.

We do not believe that the FDA's intent is to make the
import policy unworkable. But we do remember that back in the
clarithromycin days, the ongoing battle was with bureaucrats who
wanted to require a separate shipment from abroad for each
patient, despite the fact that the drug was often needed
urgently. The recent letter lays the groundwork for similar
problems in the future, by expressing a policy which, if fully
enforced, is likely to be unworkable for the reasons cited above.
This is why the community is concerned.

(2) The "drugs from unknown sources" warning is also a cause
for concern, in that most new treatments are likely to start that
way.

This issue was set to arise next with the "tat drug," the
only tat inhibitor in human trials, which has been developed
slowly by Hoffmann-La Roche. As of this writing, the word in the
community is that the Roche tat drug is not working well. If the
current rumors are confirmed, there will be little or no market
for the drug, so it will not be a potential source of dispute
between the FDA and the AIDS community. But the issue will
likely arise with other drugs.

(3) The area addressed by the FDA's letter where there is
least concern in the AIDS community is commercialization or
promotion. There is a sense that if something is truly important,
people will learn about it without being prompted -- and that it
is wrong to create markets for unapproved products for serious
illnesses. People want the freedom of access after they have
made their own decisions; they don't want exploitation of the
desperate, nor additional confusion introduced by commercial
campaigns.

(4) The basic thrust of the FDA's approach is to substitute
legally recognized means of access to potentially useful
unapproved treatments -- such as the expanded access programs for
drugs in development -- for the AIDS treatment underground that
has now existed for several years. No one disputes that the
recognized means are better, in that anyone would choose to use a
drug manufactured to FDA-approved standards of quality control
over a version of the drug that was not. But at the same time,
we cannot find anybody who believes that the currently available
means of making experimental treatments available will always
meet the need. Because of fundamental problems in the U. S.
medical and drug- development systems, there will always be
legitimate, significant cases where a necessary treatment is not
available through any of the legally recognized means, but is
obtainable otherwise. And when these cases do arise, it is the
community's consensus that patients working with their physicians
should be able to make rational medical decisions and then obtain
the treatments they choose.

Any organization has two kinds of motivations which guide
its policies and actions. One relates to its stated fundamental
mission -- in the FDA's case, protecting the public health. The
other relates to institutional self-interest and sometimes to
unstated missions. These can include: maintaining the
appearance of consistency; protecting itself against criticism;
positioning itself for favorable publicity; maintaining the
medical monopoly of large, well-financed interests, which has
done so much to limit the options for practical treatment
development and to increase the cost of care; and preventing the
development of institutions fundamentally at variance with the
existing system (such as offshore businesses to supply unapproved
drugs). We do not know the relative strengths of these two basic
motivations in this case -- and the FDA itself may not know,
either. But both must exist, even though only the former is
publicly reported in the FDA's statements above.

The reason motivation is important is that, to the extent
that public health is primary, the FDA can work together with
everyone else involved to increase access to important treatments
through legally recognized means, thus automatically shrinking
the role of the treatment underground without denying access to
patients. But to the extent that the FDA basically wants to get
rid of alternative means of treatment access, and develops
public-health rationales as justifications or other tools toward
that end, there will be conflict, because the interests of the
regulators will diverge from those of the patients ostensibly
being protected.

The ultimate danger of fighting over access to unapproved
treatments is that it would distract from the real issue --
improving the drug-development system and working together to
find treatments that do work and are approved, which would end
the debate over unapproved treatments. But meanwhile we also
face an immediate danger. If the communities interested in
treatments for AIDS and other diseases are not assertive enough
in negotiating to protect their interests, policy directions may
be set in a vacuum, in the absence of real examples. Then when
important but unrecognized drugs or research opportunities do
come into view, the regulatory system will be an obstacle rather
than an ally.

We must plan for the unexpected, since that is where so many
of the most important advances occur. We need expanded access to
known promising drugs, and will work with the FDA on this. But
we also need regulatory flexibility -- the slack or breathing
room that allows people to make the dysfunctional drug-
development and access systems work. We have had flexibility in
the past; the current unease reflects a fear that there could be
less of it in the future.