It’s asking what you would do so I would pick the first on and that would say “ Imagine you have a terminal illness. Your physicians informs you that in attempting to treat your illness, all known interventions or treatments have been tried, but have all shown to be ineffective. This effectively leaves you without viable treatment options. This situation is a reality for a significant amount of patients who then, ideally, receive palliative care to attempt to provide the best possible end of life. However, imagine your physician informs you that she is aware of a new drug that is currently in development. This drug has had promising results in laboratory and animal testing, and has also undergone phase I and phase IIa testing on human participants. Currently, a phase IIb randomised clinical trial (RCT) is being set up. Imagine you set your hopes on this new drug and would like to get access to it, what are your options? One option is to wait for the drug to get market approval and reach the market. However, such testing takes time. Available research suggests that the average time from the first clinical tests on humans (phase 0 or I) to market approval is more than 7 years [1]. Terminally ill patients are thus likely to pass away before this medical intervention reaches the market, making this an unviable option.
Another alternative is to attempt to get enrolled in randomised controlled trial (RCT) for the drug which gives you a decent chance of receiving the experimental new agent. It is not assured you receive the drug since there is likely to be a control group receiving a placebo or the best currently available treatment and you could be (randomly) assigned to this group. Such clinical trials have a strong allure for terminally ill patients, and it is known that they will often ‘try anything to stay alive’
However, such a strong willingness of terminally ill patients potentially poses problems. First, it might make the offer or option to participate in an RCT a coercive one that some patients are unable to refuse. This might invalidate free and informed consent [3], although others have argued that this is not necessarily the case [4]. Second, it has been argued that it might even compromise the clinical trial system. Schuklenk (2014), for example, uses the example of AIDS trials in the past and argues that: ‘[Patients] lied and cheated to get into trials and left trials in such large numbers as to threaten the viability of the AIDS clinical trials system.’ ([5]: p.21).
As such, there is increasing attention for other ways to provide terminally ill patients with access to experimental and innovative drugs.
