AIDS TREATMENT NEWS No. 061 - July 29, 1988
John S. James

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 policy.

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.

Potential Benefits

* 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 made available.

* 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.

Potential Risks

* 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 abroad.

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.

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