An analysis of common ethical justifications for compassionate use programs for experimental drugs

Abstract

Background: When a new intervention or drug is developed, this has to pass through various phases of clinical testing before it achieves market approval, which can take many years. This raises an issue for drugs which could benefit terminally ill patients. These patients might set their hopes on the experimental drug but are unable to wait since they are likely to pass away before the drug is available. As a means of nevertheless getting access to experimental drug, many seriously ill and terminally ill patients are therefore very willing to participate in randomised controlled trials. However, only very few terminally ill patients are able to actually participate, and those that do participate are at risk of participating solely as a way of getting experimental drugs. Currently, there are, however, ways of getting access to drugs that have not (yet) gained market approval. One such mean is via expanded access or compassionate use programs where terminally ill patients receive experimental new drugs that are not yet market approved. In this paper, I examine some of the common justifications for such programs.

Main body: The most frequently voiced justifications for compassionate use or expanded access programs could be put in one of three categories. First, there are justifications of justice, where compassionate use programs could be seen as a just or fair way to distribute experimental new drugs to patients who are denied access to RCT's through no fault of their own. Second, such programs could be justified by reference to the ethical principle of beneficence where it could be claimed that terminally ill patients stand to benefit greatly at very little risk (as they are already dying). Third, there are considerations of autonomy where, it is claimed, patients should be able to exercise their autonomy and have access to such drugs if that is there free choice and they are fully aware of the risks associated with that choice.

Short conclusion: In this paper, I argue currently all justifications are potentially problematic. If they truly form the basis for justification, compassionate use programs should be designed to maximize justice, beneficence and autonomy.

Keywords: Analysis of common justifications; Compassionate use; Expanded access; Research ethics.