Terminally ill patients and the right to try new untested drugs

In the United States nearly half of the states have passed a “right to try” law, which attempt to give terminally ill patients access to experimental drugs. Some scientists and health policy experts believe such laws can be harmful by causing false hopes and even suffering.  Rebecca Dresser argues that states should not implement such laws due to dashed hopes, misery, and lost opportunities which can follow from resorting to unproven measures, see  hastings centre report . For instance, someone might lose the opportunity to spend his last days with his family in a futile attempt to extend his life. In this posting I want to examine the right of terminally ill patients to try experimental drugs which have not been fully tested.My comments here will only apply to experimental drugs but I would suggest that they could equally apply to all experimental treatments such as the use of Crispr gene editing tools. In what follows experimental drugs will refer to new drugs which have not yet been fully tested. Of course pharmaceutical companies must be willing to supply these drugs. I am only examining the right of patients to try experimental drugs which pharmaceutical companies are willing to supply and not patient’s rights to demand these drugs. In practice pharmaceutical companies might be unwilling to supply such drugs because of a fear of litigation, I will return to this point at the end of my posting.

I fully accept Dresser is correct when she asserts that experimental drugs might cause dashed hopes, misery, and lost opportunities. Untested drugs can cause harm. It is this harm that forms the basis for not allowing terminally ill patients access to these drugs. I now want to examine in more detail the harm that access to experimental drugs might cause to the patients who take them. I will then examine how access to these drugs might harm future patients by distorting drug trials.

How might access to experimental drugs harm the patients who take them? Firstly they might further limit a patient’s already limited lifespan. Secondly they might cause a patient greater physical suffering. Lastly they might cause him psychological suffering by falsely raising hopes and then dashing these hopes if they fail. I will now examine each of these three possible harms in turn. Previously I have argued that terminally ill patients, those suffering from Alzheimer’s disease and other degenerative conditions should have a right to assisted suicide, see alzheimers and suicide  . If terminally ill patients have a right to end their lives it seems to follow that the fact that experimental drugs might possibly shorten someone’s life does not give us a reason to prohibit the taking of such drugs. It might be objected that someone taking a drug to end his life and someone taking an experimental drug to extend his life have diametrically opposed ends. However, even if this is true a patient taking a drug to try and extend his life should be aware that it might do the opposite. Provided a patient is reasonably competent and aware that such a drug might shorten his life it should be up to him to decide if he is prepared to accept the risk of shortening his life in order to have the possibility of extending it. It might now be objected that by providing experimental drugs to someone we are not acting in a caring way, we are not acting beneficently. In response I would argue the opposite holds and that if we prohibit the use of these drugs we are caring for patients rather than caring about them. Caring for differs from caring about. If I care for a dog I must care about what is in its best interests. If I care about a person I must care about what is in his best interests and what he thinks are in his best interests. Failure to do so is a failure to see him as the sort of creature who can decide about his own future and displays moral arrogance. I have argued elsewhere that If I care about someone in a truly empathic way I must care about what he cares about, rather than simply what I think might be in his best interests, see woolerscottus . It appears to follow that competent patients should not be prohibited from taking experimental drugs which might shorten their lives provided they are aware of this fact. After all smoking shortens many smokers’ lives but because we respect autonomy smoking is permissible.

It might be objected that the above argument is unsound as often terminally ill patients are not the sort of creatures who can really make decisions about their own future. The above objection as it stands is unsound as the terminally can make some decisions about their treatment. For instance, it is perfectly acceptable for a patient to choose forgoing some life extending treatment in order to have better quality of life with his family. The above argument can be modified. It might be argued that terminally ill patients are not good at making decisions about their future or lack of it. This might be caused by stress, a disposition for false or exaggerated optimism and an inability to understand probabilities. In response I would point it is not only the terminally ill but the public and some doctors who are not very good at understanding probability, see Helping doctors and patients make sense of health statistics . Nonetheless false optimism remains and this false optimism might distort a terminally ill patient’s decision making capacity. What exactly is meant by false optimism? Is it just a failure to understand probability or is it someone assigning different values, weights, to the things he finds to be important? No decisions are made without reference to these weights, our values, and it follows changing our values might change the decisions we make without any alteration to the probability of certain events occurring. What might appear to us as false optimism might be someone giving different weights to what he finds to be important. I would argue we must accept that the terminally ill have a right to determine their own values and assign their own weights to things they find pertinent to their decision-making for two reasons. Firstly, we should recognise that the terminally ill remain the sort of creatures who can and should make decisions about their own future. Secondly, most of us are in a state of epistemic ignorance about what it means to experience terminal illness and if we criticise the values of the terminally ill we are guilty of epistemic arrogance. It would appear if we accept that the terminally ill are the kind of creatures who can make decisions about their own future the fact that experimental drugs might shorten their lives does not give us reason to prohibit them from using such drugs.

Patients who take experimental drugs might cause themselves physical harm. The first principle in medical ethics is to do no harm, non-maleficence, so it might be argued that the prescription of such drugs by medical practitioners should be prohibited. The above argument is unsound. Chemotherapy harms patients but this harm is offset by its benefits. Let us accept that an experimental drug might harm a patient but that it might also benefit him. Indeed, such drugs are only tested because it is believed that they might benefit patients. The above argument might be modified. It might now be argued that that the prescription of experimental drugs by medical practitioners should be prohibited until such a time as the benefits of taking these drugs can be shown to offset any harm they cause. However, the above argument is also unsound. Chemotherapy does not always benefit patients and so does not always offset any harm it causes. If we accept the above argument then we should prohibit chemotherapy, such a suggestion is nonsensical. However, the modified argument might be still further modified. It might now be argued that that the prescription of experimental drugs by medical practitioners should be prohibited until such a time as the benefits of taking such drugs can be shown to offset any harm they cause in the majority of cases. This further modified argument is about how much risk patients should be exposed to.

The reason we don’t want to expose patients to excessive risk is because we care about them. However we don’t prohibit paragliding because we care about those who participate. Who should determine what risks are acceptable? I can use the same argument I employed above, showing that patients have the right to risk shortening their lives if there is some limited chance of life extension provided they understand the risk involved, to deal with the risk of patients harming themselves. I would suggest that if we prohibit the use of experimental drugs which might harm patients but also might benefit them that once again we are caring for patients rather than caring about them. I argued above that we should care about people in a different way to the way we care for dogs. Failure to do so is a failure to see patients as the sort of creature who can make decisions about their own future and displays moral arrogance. If patients understand the risks involved it should be up to patients to decide if they are prepared to accept these risks. The last way the use experimental by patients might harm them is by causing psychological suffering by raising false hopes and dashing these hopes if these drugs fail. I believe in this context the above argument can once again be applied and I will not repeat it. To summarise it would seem that possible harm to actual patients is not a reason to prohibit access to experimental drugs provided patients are aware of this possible harm.

Even if we accept the above somewhat tentative conclusion it doesn’t follow that we don’t still have a reason to prohibit access of experimental drugs to terminally ill patients. Future patients might be harmed because the effectiveness of drugs might not be fully tested in the future. Drug trials are expensive and if pharmaceutical companies can rely on data obtained by using a drug on terminally ill patients then they might be reluctant to finance fully fledged trials. Doing so might lead to two problems. Firstly, some drugs which appear not to harm terminally ill patients might harm other patients. The long term effects of a drug, which extends a patient’s life in the short term, might not become apparent. Secondly some drugs which do not appear to have any effect on terminally ill patients might be effective on less seriously ill patients. Such drugs might not become available to future patients. Can these two problems be solved?

Regulation might solve the first problem. Experimental drugs might be used on terminally ill patients if they desire them but their use should not be permitted on other patients until after undergoing a full clinical trial. It might appear that because there are less terminally ill patients compared to other patients that pharmaceutical companies would continue to conduct full clinical trials on experimental drugs. However this appearance might be unsound. Pharmaceutical companies might try to extend the definition of a terminally patient so as to continue using some drugs without them ever having to undergo a full trial. This problem might be overcome by regulatory authorities insisting that experimental drugs are only used on those who are terminally ill. Applied philosophers might aid them in this task by better defining what is meant by terminal illness. The well-known physicist Stephen Hawking has motor neurone disease and it is probable that this disease will kill him but at the present he would not be classed as terminally ill. Terminal illness should be defined by how long someone will probably live rather than the probability that his illness will kill him. Perhaps someone should not be considered to have a terminal illness unless it is probable that he has less than six months to live. Let us consider the second problem. Might some pharmaceutical companies be tempted not to fully trial some drugs which might benefit some patients on the basis of incomplete evidence gathered from their use on terminally ill patients? Once again regulation might solve this problem. I would suggest that provided that terminal illness is defined tightly enough that this problem shouldn’t arise. A tight definition of terminal illness means fewer terminally ill patients for pharmaceutical companies to test drugs on forcing them to conduct full clinical trials. To summarise once again it appears harm to future patients does not give us reason to prohibit access to experimental drugs for the terminally ill provided that terminal illness is tightly defined.

Lastly at the beginning of this post I suggested that in practice pharmaceutical companies might be unwilling to supply experimental drugs due to a fear of litigation. It should be possible to overcome this fear if patients are required to sign a comprehensive consent form making it clear not only that there are risks involved but also that these risks include as yet unknown risks.

The above discussion leads to the rather tentative conclusion that the terminally ill should not be prohibited from trying experimental drugs subject to certain safeguards. These are,

1. Terminal illness must be clearly and tightly defined. Philosophy can play an important part in doing this.
2. No drugs which have not been fully tested should be used on non-terminally ill patients except for the purpose of testing
3. Any terminally ill patient taking an experimental drug must sign a comprehensive consent form in the same way patients taking part in trials do. This form must make it clear that they are prepared to accept as yet unknown risks.