Does Denying Experimental Drugs to Terminally Ill Patients Really Protect Them?

by Dr. Nileema Conlon Vaswani in


To ensure that a drug is safe before it becomes available for public use seems reasonable and morally obligatory. Drug regulators have a duty to protect the public and to license drugs for use only when they are considered safe. But herein lies a moral paradox. How are we to ensure that the drugs in question are safe?

To determine the safety of a drug for humans, it must be tested on humans. Once enough people have been tested and the drug is cleared for use by the regulators, it is available for public use. But this does not detract from the fact that a handful of people, sometimes more, did have the drug without the benefit of previous testing. They fall outside of the protection of drug regulation because without them, we would not be able to formulate the regulations. They risk their safety by taking untested drugs so that we can know which drugs are safe. They are the participants of the clinical trials.

This week, the US Court of Appeals for the District of Columbia circuit ruled that terminally ill patients should not be given experimental drugs on grounds that they are not safe to use. For many of these patients, these drugs are their only hope. Over the past few years, many patients have died who might otherwise have lived had they been given these experimental drugs. The irony was that all the drugs that were experimental when the patients were alive were approved after their death. If they had been given the drugs when alive, they might have lived to see them be approved. They might even have helped contribute to medical research while helping themselves. Still, in the interests of their "protection" they were not allowed these drugs.

Competent, consenting adults ought to have the right to choose their treatment but this is not the only moral problem with this case. That it compromises individual choice is clear but what is also worrying is that the very protection that denies patients the treatment they want is not given to those in a clinical trial. If the regulations about safety, i.e., protecting the public are to be taken seriously, then one could argue that they ought to be implemented consistently in all cases.

Clearly, the very nature of experimentation is such that such consistency cannot be achieved. The very reason for testing a drug's safety is that we do not know whether or not it is safe. It is, therefore, not possible for the participants of clinical trials to be afforded the same protection as are those outside of a trial. This is not to suggest that the participants of clinical trials are given no protection. Clinical trials are subject to stringent regulations and informed consent, among other factors, does protect the patient's choice to participate. Other protections such as stopping the trial if there are adverse side effects and so on also exist. However, a trial that is intended to determine safety carries the risk of being unsafe and it is this type of lack of "protection" to which I am referring.

It seems odd that although there are people who, within a trial, can undergo certain risks to test the safety of a drug, outside of the trial, patients are not entitled to taking the same risks if they wish to do so. The very protection that disallows patients outside of a trial from taking the drug also denies them the opportunity to benefit from it.

So is protection a right that one can choose to have or something that one is obliged to accept? In other words, can one choose not to be protected? We already know that this choice is available to those in a clinical trial but ought those outside of a clinical trial be entitled to undergo the risks of taking a drug that is still being tested or ought we to insist that they be protected?

If competent adults who are outside of a clinical trial wish to take an experimental drug, this decision ought to be theirs and theirs alone. It is up to them to weigh up the risks and benefits of taking the drug and to compare its potential effectiveness with its potentially being unsafe. And if they are terminally ill, and face the inevitability of a premature death because of their medical condition, then any risk that they wish to take in order to avoid death ought not to be denied to them. For the drug regulators to argue that the drug may in fact hasten death is morally irrelevant. Patients ought to be allowed to choose whether they wish to take drugs that may hasten their death if found to be unsafe or that may prolong their lives if found to be effective. Their choices outside of a clinical trial ought to be no different from those of someone who is faced with these very questions within the context of a clinical trial.

By denying terminally ill patients drugs that might save their lives, we might in fact cause them to die earlier than they would have had they been given the drug. In other medical contexts, outside of a drug trial, individuals regularly make these difficult choices about themselves. An individual who is faced with the decision to undergo a high risk surgery might be aware that the "high risk" refers to death or a serious side effect. But the choice to undergo the high risk surgery and be cured of a particular medical condition if the surgery is successful, or die if the surgery is unsuccessful, is the individual's to make. It is ironic that when discussions surrounding the protection of patients within or outside of a clinical trial often refer to preventing death the decision of the US courts is such that protection from death may in fact just lead to it.