The Emperor Has No Clothes: Notes on AIDS Drug Testing and Access

The following comments were edited from an August 5 telephone conversation with Nathaniel Pier, M.D., a New York
physician treating several hundred patients with AIDS and related conditions:

"We have lost something in the struggle to find therapies for AIDS. The individual patient with the counsel and guidance of his or her physician must have the right to make choices, to have options. People with AIDS and at risk for AIDS have abdicated their responsibility far too much to the medical establishment and the research establishment to make this decision for them.
"The primary role of a clinician such as myself is to synthesize a course of action with a patient that makes sense. That means every patient is offered the opportunity of choosing from the options that are available, under the careful guidance of their clinician. This should not be a major issue.
"The current system of doing drug studies has failed. It has bureaucratized to the point where it takes two to three
years to test a drug that should be tested in two to four months. There is no proof that this system works. So let's go
back to the old system where individual clinicians are given the opportunity of synthesizing a course of action.
"Under supervised protocols from a national group, we should get access to drugs that are in phase II trials, including drugs that have not had efficacy demonstrated, and clinicians should be allowed to authorize the option of trying these drugs if patients want to. (Editor's note: drugs in "phase II" testing have already passed the "phase I" test for dosage, toxicity, and safety.) It should be up to the patient with the clinician's guidance to make such decisions. The fact that we have to wait two to four years for a drug like lentinan or dideoxycytidine to come down as a potentially useful drug is not fair to the patients who have no other options, who are simply going to die.
"The system is essentially telling patients with immune deficiency disease, 'We don't care about you, go home and die. We will cure this disease in our own time with our designer drugs.' Every patient should demand that anybody with HIV disease should have access to a number of experimental options through their clinician, and these options should be offered to them as supervised protocols.
"A good example is the multi-drug protocols for cancer. If you're diagnosed with colo-rectal cancer, for example, your
oncologist ties in to a national computer where your statistics are kept confidentially; out the other end comes a protocol.
Not only are you allowed to participate in a potentially lifesaving drug regimen, but the data that's collected from you goes back to a center that very quickly will collect information as to whether this particular combinations of drugs works.
"We should be doing this in AIDS. There is no reason that after 18 months of AZT, we shouldn't be allowed to combine other drugs for both antiviral and immunomodulatory effect. We could do this on thousands of patients overnight if there were a national registry and a system for doing it.
"We wouldn't need to do complex virological or immunological tests. The studies requiring such tests should be done in smaller protocols at major medical centers. Otherwise, once a month the physician would do the blood work specified in the protocol, and send the data to the NIH, and the physician will fill in clinical data. Probably within three or four months we could find out which treatment combinations are best. People with AIDS should be demanding that the system be reformed to allow anybody at risk access to these drugs under supervised protocols.
"It seems that groups advocating for people with AIDS have lost this primary goal, that it should be the patient's option, to use experimental drugs under guidance, supervision, and monitoring.
"The system now in place for developing and testing and distributing AIDS drugs is not working, it is not producing new therapeutic options. In the last two years all we've had is AZT. AIDS advocates need to start saying that the emperor has no clothes. So let's start a system that is going to work--one where people with AIDS-related problems get rapid access to experimental drugs in protocol, on a routine basis through their clinicians, whether private physicians or at a clinic.
"Let's stop wasting time. I have done my rounds of researchers, and there is nothing, there are no new drugs close to approval. It's a scandal that our system with its funding still isn't producing.
"The system claims to be working, but isn't. Why aren't more people demanding access to these drugs which are potentially effective and are safe to use? We must stop this idea that testing drugs on a small number of patients, then on a slightly larger number of patients is a reasonable approach when people are dying. We want to make experimental therapies available to people as a matter of course, as a matter of therapy, so that clinicians caring for AIDS patients can get access to these drugs under supervised protocols. Not only would patients have more options, we also would learn quickly what works.
"It is completely unreasonable that two years after AZT there is no new drug, and combination therapies using
antivirals and immunomodulators are not available. It is unreasonable that Frank Young of the FDA says that very few
drugs are likely to be approved between now and 1991, when we're losing 48 people a day to this disease.
"It's not working, so let's come up with a new system. Let's use experimental treatments as potential therapy for ailing patients, and do it in a manner that's going to provide useful information. Set up a national registry, check out protocols of combination therapies, and let people use them. Similar systems have been used for cancer and heart disease.
"For example:
"At the recent Stockholm conference, information from NIH demonstrated that an AZT and dideoxycytidine on alternating weeks may be an effective alternative for people who can no longer take AZT continuously. Why isn't this drug combination available to these people, who have few other choices?
"NIH made dextran sulfate a top priority drug in January 1988. It has taken eight months since then to get ready to
start its first study, a multicenter trial with all of 60 patients. When thousands of people have no other choice, it's inhuman not to make such a safe drug available to patients through their doctors, as part of a study.
"Where are new antivirals such as DDA or DDI? NIH has been recruiting for clinical trials for about six months.
"My problem with the treatment underground is that for the last two and a half years we have tried naltrexone, AL 721,
antabuse, dextran sulfate. Something has come along every few months that has offered people hope, and access to therapy that they couldn't get other places. That's very important. But I as a clinician am tired of my patients not knowing which of these therapies is the best to choose, tired of patients being bamboozled by people into taking this or that or the other thing, tired of not being able to assess whether or not an approach or combination is really benefitting the patients. We need a system so that when a therapy comes along, we canevaluate the efficacy very quickly.
"What I'm proposing is very clear. We need a national registry, and if a patient has an AIDS-related problem at any
stage of the illness, and if that patient elects to try to intervene, there should be protocols available to that patient's clinician. There should be multiple protocols for every stage of the disease, so that we can quickly assess which intervention might in fact make a difference.
"These protocols should be available not just through a few selected centers, not just to the lucky few who can afford
to go to doctors who can get them into these studies. They should be available to every physician who treats AIDS patients, not just in the major cities. We know enough about this disease to develop a logical approach involving antiviral, immunomodulatory, prophylactic, and anti-inflammatory therapies.
"There are enough drugs to be tried so that we could develop these protocols in a few weeks and make them available to clinicians through a national computer. The data from these combination trials could start pouring in, and we would know within a few months which of the available combinations are working best, rather than the years and years that it's currently taking.
"This approach will give patients hope, and access to humane, well-supervised medical treatments, instead of allowing them to live in fear or desperation, or search for the few trials available. At the same time it will allow them to
participate in a larger effort, to find what therapies work best for everybody, making their situation meaningful not only
for themselves but for the world.
"We want to participate, to know what is happening with the research and what the future plans are. We want to reduce the secrecy, the sense that scientific data is proprietary or exclusive in the face of this epidemic.
"The current system simply has not produced the goods. And if Dr. Frank Young's prediction (of few new drugs approved by 1991) is any indication, it will not produce the goods for a long time to come. This consigns large numbers of people to death without giving them a dignified chance to fight back. This is not an acceptable human or reasonable approach to doing research in this epidemic.
"After five years of being on the front lines, my heartfelt feeling is that the top priority for people with AIDS and people who care about AIDS is to demand access to experimental therapies to try to save their lives.
"I appeal to people to organize this effort immediately, to bring it forward in their local groups, then present the case to their political people, and to the people who are running the present medical system of testing drugs. It's time we told them that the emperor has no clothes, that the current system is not working. It's time to insist on wider access to promising therapies, and rapid testing of existing drugs to develop better treatment options."
**************************************************