Balancing Scientific Rigor and Humanitarian Concerns in Medical Trials (1989)

That is no treatment at all. When Robert Schmidt found out he was receiving a placebo in a clinical trial last year, he broke the rules and started treating himself with drugs he got from a doctor outside the study. Once a willing subject for medical research, Schmidt today takes whatever drugs he can lay his hands on and is finished with science. When researchers figured out from blood test he was taking AZT, instead of the placebo they'd given him, they terminated Schmidt from their study. I was pissed. I wasn't going to take placebos anymore. And I'm not so sure that by giving me a placebo that there is any research advantage anyway. I mean, all they're going to do is follow me to quote what they call the end point. And when I said, what is the end point? And they said, well, Bob, it's a nice way of saying, when you croak, well, I'm not willing to keep the research, quote, pure. By deceiving the doctors, Schmidt was working at cross-purposes not only with scientific research,

but with the many AIDS patients who depend on it. People who break the rules and experimental drug trials can lead scientists to misinterpret data and draw wrong conclusions. It doesn't help us if we're, for example, testing DDI and people on the side are taking AZT instead. If we see a side effect, then we might make the mistake of assuming that it's from DDI. In fact, it's from some other drug. Dr. Volberdine did once, say, at a lecture I went to. Research is research and treatment is treatment. And frankly, I want treatment. I don't want research. I'm not thrilled about being a guinea pig. Research is why we have AZT. It's why we have aerosol pentaminine. It's why we have BDI. We wouldn't have any of the drugs we have if we weren't doing research. The fact that physicians in the community are now prescribing AZT, which was developed through a research process involving a placebo control.

The fact that aerosol pentaminine was a subject of both laboratory and human experimentation. It was an example of what research does. But frustration with officially sanctioned research is so high that people are tempted not just to break the rules, but to make up their own. Last year, Martin Delaney spearheaded underground trials of a drug called compound Q. The trials gave medical supervision to people who are already experimenting on themselves with the drug. How can you blame people for acting in their own self-interest in a system which has perhaps not addressed their needs adequately? I think you have to ask why are people forced into situations in which they feel the need to act in that manner? And that's where the answer is going to lie, you know, in the design of studies that meet people's needs rather than force them to fight for themselves. The compound Q trials were criticized as unethical for lacking accepted scientific safeguards. But activists aren't the only ones who have been the rules.

I think if someone is failing a protocol or the failing the study and not achieving what they need to achieve, and I have some means of intervening there, I would rather sacrifice the protocol than sacrifice the patient. Dr. Rick Wollets gave one of his patients two experimental drugs at the same time, even though he knew it was against their rules. I justify because the patient's still with us. Patients want access to promising new drugs, scientists want controlled studies and clean data to prove the new drugs work. Activists say there's an alternative, change the way science is done. We also want to learn as quickly as possible and it's been a theme in what you've heard already, what the side effects of a new drug are. When can we expect to see them? Researchers recently announced details of a new drug study designed to take patients' concerns into account. The drug to be tested is DDI, said to be as effective against the AIDS virus as AZT and less toxic.

Shown in the left for those that are aficionados of this is the chemical structure of DDI. The new study won't use placebos and people who don't qualify can still receive DDI with their doctors' approval to a process called open label. I think it's entirely possible to create ethical studies that meet the needs of the individual while also getting the answers science needs. And I think there have been a number of studies that have done that. I think the current studies of DDI are designed in a way that will do that. But when you're dealing with human beings in clinical research, the first hat that the researcher has to wear is as physician.