For the Sake of Justice: Should We Prioritize Rare Diseases?

Abstract

This article is about the justifiability of accepting worse cost effectiveness for orphan drugs, that is, treatments for rare diseases, in a publicly financed health care system. Recently, three arguments have been presented that may be used in favour of exceptionally advantageous economic terms for orphan drugs. These arguments share the common feature of all referring to considerations of justice or fairness: the argument of the irrelevance of group size, the argument from the principle of need, and the argument of identifiability. It is argued that all of these arguments fail to support the conclusion that orphan drugs should be subsidized to a larger extent than treatments for common diseases. The argument of the irrelevance fails to distinguish between directly and indirectly relevant considerations of fairness or justice. The recent attempt to revive the moral relevance of identifiability has provided no novel reasons to think that identifiability is morally relevant in itself or due to considerations of fairness and justice. The argument from the principle of need does not fail due to any inherent flaw in the principle as such. Rather, this principle can be interpreted in different ways, and none of these interpretations support exceptionally advantageous terms economically for treating rare diseases specifically. It is concluded that we are awaiting justice based reasons for the preferential treatment of orphan drugs.

Principles of Need and the Aggregation Thesis

“…[12,13,27,41,44,45]. some common ground: to give some kind of special normative weight to people who are worse off [24,25]. The claim that need principles share a special concern for the worse off should not be confused with Absolute Priority, the implausible view that one should always give absolute priority to the worst off.…”

“…Fourth, we assume a conception of health care need according to which need is a graded rather than binary concept [cf. 12,22,24], i.e. that it makes perfect sense to talk about degrees of needs.…”

“…It has been suggested that a principle of need is best understood as a priority or a sufficiency principle, or some combination of the two [8,24,25,32]. In this section we shall sketch the basis for these distributive principles.…”

“…Although need principles may be interpreted in various ways, they all ascribe some special normative importance to being badly off [24,25]. However, such a common ground has no clear implications for how a plausible principle of need should relate to the aggregation of benefits across individuals.…”

Healthcare Priority Setting and Rare Diseases: What Matters When Reimbursing Orphan Drugs

“…If one general cost-effectiveness threshold was strictly applied as a decision criterion, many orphan drugs would not be made available to patients. Whether or not this is acceptable from an ethical perspective can be questioned, based on competing theories of what constitutes distributive justice (Gericke et al, 2005, Juth, 2014, Sandman and Gustavsson, 2017. In addition, maximising health gains, no matter the distributional consequences, is not always in line with public preferences for allocating resources (Nord et al, 1995a).…”