Introduction
The idea of experiments involving human beings may invoke memories of Nazi Germany’s brutal and sadistic experiments on concentration camp prisoners; or the infamous, decades-long Tuskegee “study in nature” of untreated syphilis among Macon County’s African-American citizens; or our government’s experiments studying the effects of radiation on unsuspecting citizens, to name only some of the notorious abuses of medicine.
Moreover, recent reports about gene-therapy clinical trials have not exactly been positive. There have been some unexplained deaths, and some questions about whether investigators reported these “adverse events” to the proper authorities.
Consequently, the whole concept of a clinical trial might give a person pause. Indeed, no one wants to be used as a human guinea pig, to be poked, prodded and medicated all in the name of science. Having said that, let me say to you that:
Clinical Trials May Present You With A Valuable Opportunity
Imagine yourself in any one of the following situations:
You have a serious illness. Your doctor has tried everything; nothing seems to work, and you’re getting worse. One day, she suggests you enroll in an experiment, a clinical trial in which investigators hope to prove that some drug yet to be approved by the FDA is effective in treating the very illness you have. You are being offered an opportunity to access a potentially beneficial treatment that would otherwise be unavailable to you.
Health
But here’s the catch. If you enroll in the study, you have a fifty-fifty chance of receiving a placebo (a sugar pill), because the investigators need to have a control group in order to determine for sure whether the experimental drug is significantly more effective than a dummy pill. This can only be justified if there are no known alternative treatments for the given condition, or if the study has already tried all the alternative treatments and found them to be ineffective. In some studies, the control in not a placebo but an active drug; such experiments are intended to compare the efficacy of the experimental drug to that of the known therapy.
In such a study, known as a double-blind, placebo-controlled clinical trial, neither physician nor patient knows beforehand who is getting the study drug and who isn’t. Moreover, even if you are randomized to the study drug, there is no guarantee that the drug will help you; indeed, it could even make you worse.
Be that as it may, at this point you are willing to try anything, and if your doctor, whom you trust, is recommending it, then it must be a good idea. Right? Besides, the possibility of helping medical science achieve a more effective treatment for your illness appeals to your sense of altruism.
Or consider this: You’ve been feeling blue for some time now and you’re having trouble sleeping. You’re becoming more discouraged by the day, but you don’t have insurance, or your insurance is woefully inadequate for mental conditions. Then, one day, you see an advertisement posted on your school bulletin board: “Down in the dumps? Having trouble sleeping? You may be suffering from depression! If so you may be eligible for a new exciting study in the treatment of depression. Call this number for a free checkup and diagnosis.”
The investigators are offering free study medication for those who meet their inclusion criteria. What do you have to lose?
Finally, consider this: You are seriously ill with breast cancer. The cancer is spreading despite surgery and chemotherapy. As a treatment of last resort, your physician recommends a clinical trial intended to measure the efficacy of a bone marrow transplant. Although there is no solid evidence suggesting that such a drastic and harrowing treatment is any more effective than standard chemotherapy, you are desperate to try anything at this point. Then you learn that there are places where such treatment is offered – at a steep cost, but offered nonetheless. Why enter a clinical trial and risk being randomized to the placebo arm? And why are physicians able to offer you this costly, yet unproven, treatment? After all, they can’t offer you a drug until and unless the FDA approves it, and that only happens after its efficacy and safety has been demonstrated by hard evidence borne of rigorous clinical trials.
Protecting Your Rights As A Research Subject
Federal law requires that every medical center that receives funding from the government have a committee, known in medical circles as an Institutional Review Board (IRB) or a Committee on Human Rights in Research. The primary purpose of such a committee is to ensure that research subjects’ rights are being adequately protected. This means reviewing both the protocol – which sets forth specifically what it is that the investigators propose to do in the study – and the informed consent document that will be given to all the subjects before they are enrolled. In other words, the committee’s job is to protect you.
When all is said and done, however, it is you who must protect yourself. IRB’s are notoriously overworked and are as fallible as any group of people can be. Most of their members are physicians who, although conscientious in looking out for your interests, may at times, being researchers themselves, identify more with the investigators than with the potential study subjects.
How Informed is Informed Consent?
All an IRB can do is insure that the informed consent that is given to you includes all necessary information. In truth though, study after study demonstrates that people don’t fully comprehend experiments in which they enroll, even after having the protocols explained to them, and even after they have had ample time to review the informed consent documents. Informed consent documents are loaded up with information, because that is what the law requires. It is not that the information is unimportant, but rather, it is a lot to take in. Add to that the fact that you are probably feeling anxious anyway, and you may well just go along because your physician suggests it.
But that may not always be wise. Beware of what is known in the medical ethics literature as the “therapeutic fallacy”, the belief of potential research subjects that, because a member of the medical profession is offering you this option, it must be to your benefit.
What Can You Do To Ensure That You Are Making A Truly Informed Decision?
Whatever you do, don’t rush into a decision. Take the form home with you. Show it to other people whom you trust and discuss it with them. In sifting through the mountain of information about a clinical research trial, be sure to get the answer to the following questions:
- How is this experiment different from the type of treatment that is already available to me?
- Do the potential benefits to be gained from participating in the study outweigh the potential risks?
- Is the study drug already FDA approved for other uses? If it is, you may be able to get a prescription for it "off label", which would obviate the need for your enrolling in a clinical trial in which you may be randomized to the placebo rather than the study drug.
Beware Of Conflicts Of Interest
Just as in any profession, physicians may find themselves in conflicts of interest. For example, if your doctor is involved in the study, he has an interest in enrolling as many ostensibly suitable subjects as possible. He may be so invested in the success of the study that he loses perspective as to whether he is doing his best by you as his patient. What to do? Before you decide whether or not to enroll, seek another opinion from a physician not involved in the study.
Often, a major drug company is the sponsor of the study. It has become somewhat common for pharmaceutical companies to offer financial incentives to physicians, who may or may not be involved in the studies themselves, for referring patients as research subjects. Don’t count on the informed consent document to reveal that fact. As delicate as it may be, ask your physician whether he is receiving any financial benefits from referring you to the study.
Sometimes, a physician who is conducting a study has a financial interest in the drug or biotech company itself! And finally, medical research is, for all its benefits, big business,which includes awarding significant grants to the investigators.
What Else Do You Need To Know?
All the important questions about a study are supposed to be addressed in the informed consent document offered to subjects to review and sign before they can enroll. Here are a few points to look for:
- Just what exactly is expected of you as a research subject? How many visits will you have to make to the study sites? How many and what type of tests will be performed? How long will the study continue? And so forth.
- Will all costs be borne by the study sponsors? What if there are complications? Will your insurance be expected to pick up any unforeseen costs that may arise? If a hospital stay is required, who will pay for it?
- If it is for psychiatric reasons and your insurance company must cover it, will that deplete your allotted annual time for such coverage?
- Will you be informed of any new findings?
Summary
In summary, if the possibility of participation in a clinical trial is presented to you, keep an open mind, ask a lot of questions, and don’t rush into any decisions. It may end up being of great benefit to you, but do not let the wish that it be so overwhelm your ability to consider all the pros and cons dispassionately.
