Patients Volunteering for Research and the “Therapeutic Misconception”

A patient volunteers to participate in a drug research project which is related to the patient’s own illness. The question is: why would the patient want to participate? An obvious answer might be that the patient is motivated about the possibility that the new drug will benefit recovery or management of the illness better than the drugs which the patient has been prescribed. The patient may believe that he or she will, in the study, will have access to the new drug. This thinking and believing really represents a misconception. It is based on the patient’s conception, which may be fostered by inadequate or misleading information about the study, that sufficient benefit to the patient’s illness will be the result of participation in a clinical trial that will trump any of the risks of the study. This is the “therapeutic misconception”, a term invented by Roth, Appelbaum and Lidz some 25 years ago, regarding patient involvement in clinical research.

What is the misconception? Well, first might be the patient considers him/herself as a patient in the study. Many studies are designed not to treat the volunteer with the appropriate illness as a patient but to consider the volunteer as a experimental subject. Yes, in the United States there are overseeing governmental mechanisms in clinical research attempting to protect the human volunteers as human subjects but not necessarily as patients. Being attended to as a patient demands that the professional keep only the best interests of that patient in the decision making. But, in clinical research, the goal is to attend to the best interests of the research study. For example, toxic effects of drugs on the volunteers are generally monitored and if the risk to the volunteers, through statistical monitoring, becomes greater than initially anticipated, the study is stopped. Well, one would agree with that kind of interest in the study subject. But sometimes, if the beneficial results of the drug study are strongly positive, and this is also monitored, then the study also may be stopped to conserve the costs but perhaps only to the detriment of the patient whose illness was improving with the drug under study. There, unlike in usual medical practice, benefit for the patient is ignored. A complicating issue in this conflict of benefits is when the patient’s physician is also a member of the research team (a topic which I covered on this blog in February 2005 as “Wearing Two Hats: Clinician and Researcher”). How can the physician resolve the conflicting responsibilities?

Another misconception is that the patient will receive a drug in the study which could be to the patient’s benefit. The basis of a true clinical research random controlled study is not to study the known but to study the unknown and undetermined. The usual first approach to study the unknown is to discover if a drug has benefit for a patient as compared with no drug (a placebo pill). If there are already drugs available to treat the illness, then another unknown would be to determine if the new drug’s known benefit is better than the other drugs benefit. This can be tested by a study comparing the beneficial effects of the new drug against the established drug. The studies are performed by, in the first case providing the new drug to one group of randomly selected patients and providing the second group of randomly selected patients the placebo. The second group will not be treated with an active medication. If it turns out that the new drug is ineffective, the first group will get no benefit. The risks include the possible absence of an effective drug in the first group and both groups may not be getting benefit from an established drug for their illness during the study.

In the second case, one randomly selected group will get the new drug and the second randomly selected group will get the established drug. The first group may not be benefitted by the new drug. The second group may not have the benefit of the new drug, if that drug is found better than the established drug. Again, the risks for the first group would be not getting benefit from an established drug if the new drug is less beneficial. For the second group, they would be missing the opportunity, if the new drug is more effective, to take the new drug.

So what does this all mean? It means that volunteers must realize that a clinical study is not a treatment, it is an experiment. The volunteers should be aware that they are not patients, they are experimental subjects. The volunteers should know that they will be randomly selected, will not know to which group they have been selected and they may not be given a drug that is effective treatment for their illness or may be given one that is less or no greater benefit than established drugs for their illness. They should know that during the study, the pill they are taking may be inert or less effective than the drug they had been taking previously. Finally, there is no guarantee for any participant that an effective drug found by the study will be accessible or available for treatment of the patient after the study is finished.

What this all this also means is that all volunteer patients should personally make certain they obtain and understand, as part of the informed consent,the full nature of the study they are considering to enter, the role of their own physician in the study, the risks that they are undertaking and the hoped for later benefits and understand that their participation really should represent their own altruism, to sacrifice themselves for other’s (and hopefully their own) later benefit. This “conceptual clarity” on the part of the patient at the outset should remove the possibility of the therapeutic misconception. ..Maurice.