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.
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Hence, I will not consider the interesting and closely related question on whether or not to accept worse evidence for the effect of orphan drugs than treatments for common diseases .
In these systems, the practical question is rather: ‘what should private insurance cover?’ .
Although different areas of legislation have different definitions, this makes negligible difference for the discussions . For example, all the examples used would qualify as rare diseases according to legislations also in the US, Japan, or Australia, all of which have slightly different definitions.
I have suggested elsewhere that it should .
“[A]pproximately five new are described in the medical literature every week” .
The estimation of cost varies depending on what factors are included: it would be lower if one discounted, for instance, marketing costs. However, even if estimated differently, the expected cost for developing drugs for rare diseases need not be lower than for common diseases. However, bringing orphan drugs to the market is generally somewhat cheaper for two reasons: typically, regulation allows a lesser degree of evidence and therapies that have previously been in use off label for a particular rare condition can sometimes be relabelled as orphan treatments, and enjoy extended protection (I would like to thank an anonymous reviewer for bringing this to my attention).
Since Tony Hope’s seminal discussion of these topics .
Since 2007, the patent for statins has expired, likely making the cost effectiveness ratio even better. I would like to thank an anonymous reviewer for bringing this to my attention.
In this instance, non-smoking males 55 years or older with 170 mm Hg (SBU , p. 478). Although the level of evidence in both the statin-study and the report on hypertension must be deemed as high (SBU , p. 451), it must be remembered that the calculation of QALY rests on a number of assumptions, e.g. on what to measure and how to measure it, differences in which may affect results.
All translations in the text are mine.
It is unclear to what extent the authors themselves accept the validity of this argument , p. 854.
It is coherent to hold that cost has no relevance at all: it could even be held as a laudable expression of unwillingness to aggregate on the moral basis of separateness of persons (I would like to thank an anonymous reviewer for pointing this out to me). Although I find it implausible to hold that cost does not matter whatsoever, for instance since it would lead to an unacceptable degree of levelling down , pp. 900–902, there is no room for a comprehensive argument to this effect in this article. However, denying the relevance of cost does not imply prioritizing rare diseases.
The term prioritization is here used in a broad sense, not only referring to the ranking of individual patients, but also including the distribution of health care resources, e.g. by allowing a higher cost per QALY for rare diseases than non-rare diseases. However, as is the case with this sort of distribution, distribution has consequences for which patients that actually get treatments.
On a luck egalitarian conception of justice, we, perhaps, should consider all these indirect factors as relevant and, hence, try to even them out (I would like to thank an anonymous reviewer for pointing this out to me). However, luck egalitarianism would consider the difference between rare and common diseases as irrelevant in itself, since the rarity of disease does not determine whether it is the result of option luck or brute luck.
In order to avoid any misunderstandings, I am talking about principles of need in health care, i.e., substantial ideas on how health care ought to be prioritized and not the concept of health care need, i.e., what it means to say that someone(s) have a need for health care (although the former, of course, always presuppose or imply some view on the latter) [14, 25].
Moreover, this interpretation likely captures the core idea of principles of need most straightforwardly .
The second of the statements in this paragraph is sometimes called the positive thesis (of sufficientarianism) and the third one the negative thesis .
For instance, below that level we have special entitlements to publicly funded health care .
For instance, on how to set a non-arbitrary level of sufficiency .
What I have previously called the minimise insufficiency version of the principle of need .
At least if one excludes versions of prioritarianism that give absolute priority to the worst off. However, these versions are not very plausible .
This would be analogous to moving left on Temkin’s famous sequence .
For an elaborate formulation and defence of positive egalitarianism, see .
I will not enter the question about what could be meant by “good” health since it will not matter to the argument, but only presuppose that it means absence of disease.
As the state of medical art actually is, i.e., fancy philosophical thought experiments apart.
I presuppose that all these examples would constitute clear deviations from full health, since they are included in standard classifications of diseases like the International Classification of Diseases and Related Health Problems (World Health Organization .
Unlike outcome egalitarianism, where some levelling down would perhaps be acceptable to achieve more equal health, it is hard to see any upside with worsening someone’s probability of achieving health.
I suppose it could be claimed (although it never actually has) that those with rare diseases are less responsible than those with common ones to a degree that would make it justifiable to treat them differently in general, perhaps on the basis of the fact that a great many rare diseases are monogenetic and hardly any common diseases are. However, quite a few rare diseases, like certain kinds of melanoma, are not monogenetic and many common diseases, like diabetes, Alzheimer’s, and cardiovascular diseases, are at least partly due to genetic factors, so anyone trying to draw on such differences have a considerable amount of arguing to do.
The authors seem to suggest that this should be understood in accordance with the first interpretation above , p. 897, which is, again, a non-starter.
If patient groups should be individuated according to diseases. Another way to individuate them would be according to ‘rare diseases’ and ‘non-rare’, but then one would present the thesis rather than argue for it.
However, some patients with common diseases are identifiable in the sense defined below and, most probably, some patients with rare diseases are statistical. Nonetheless, there is sufficient overlapping between the groups, not least between patients with rare diseases and identifiable individuals, to make the argument interesting to scrutinize.
It has been argued that the rule of rescue is especially problematic within public policy areas where agent-neutral reasons presumably play a larger role , such as the one we are dealing with here. However, the rule has also been questioned as a sound principle in more concrete clinical situations .
Besides the already mentioned articles on the rule of rescue, there is a good overview of many of the contributions in .
The disease and its treatment have several similarities to the initial example of Hunter’s, but the treatment also has life prolonging effects .
In fact, we often have more reliable evidence for the beneficial effects of treatments for common diseases, like the ones mentioned, than for rare ones, simply since it is easier to get sufficient patient material regarding common disorders—lack of evidence is a greater problem for orphan drugs .
So James does not claim that identifiability matters in itself, but that it is sometimes wrong to benefit the statistical rather than the identifiable person because it would unfair (which matters morally in itself) and the only difference between these persons is the very fact that the latter, unlike the former, is identifiable .
I must say that my intuitive response to the case was that we should flip a coin, since the outcomes in terms of lost lives are identical on both options and flipping a coin would give passenger 1,000 a fair chance of being saved.
In the described (metaphysical) sense, for the described reasons (of fairness), in the described situations (of many statistical persons)—this will be presupposed in the following.
Actually, if we compare individuals pairwise, this problem arises also if identifiability is seen merely as a tie-breaker.
See  for an insightful discussion on what to consider more precisely when determining whether to pay for these kinds of treatments.
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Juth, N. For the Sake of Justice: Should We Prioritize Rare Diseases?. Health Care Anal 25, 1–20 (2017). https://doi.org/10.1007/s10728-014-0284-5
- Cost effectiveness
- Orphan drugs
- Rare diseases