Orphan drugs: expensive yet necessary

Abstract

Whether the prices of certain orphan treatments are justified is highly controversial. One argument is that such therapies should not be funded through the public purse or private health plans because a patient with a rare disease requires more than their 'fair share' of a limited health care budget. Orphan medications can also be denied because they fare poorly in the cost-effectiveness assessments of drugs. This paper takes the unusual line that life-saving treatments should be provided regardless of their cost. This contention is based on the Harvard philosopher John Rawls' theory of justice. We offer three rules to limit the use of cost-effectiveness approaches: efficiency assessments should not be deployed (i) when the choice is between an only treatment and no treatment, or to (ii) prioritise between different patients and patient groups. However a well considered cost efficiency calculation may have its place (iii) where a patient has a choice between two or more equally safe and effective treatments. We rebut potential objections to this analysis, and conclude that there has been a tendency to classify appeals for orphan treatments as a minority interest and in conflict with the aims of public health and society at large. Rawls' concept of societal justice shows that a distinction between the individual and society in this context is bogus. The funding of orphan therapies is as much a matter for public health as the funding of treatments for other conditions. Treatment must not be withheld on economic grounds.

A systematic review of moral reasons on orphan drug reimbursement

“…Opposing views in this category concluded, for instance, that special status for reimbursement of OMPs was incompatible with the egalitarian principle to treat each individual equally [ 35 , 36 , 39 , 42 , 47 , 52 , 62 , 70 , 75 , 78 , 81 , 85 – 88 , 98 , 100 , 107 , 110 , 114 , 132 , 137 , 142 , 145 , 149 – 156 ]. On the other hand, an extensive body of literature argued that a special status was justified for reasons of equity [ 8 , 35 , 39 , 46 , 54 , 66 , 68 , 73 , 81 , 85 , 94 , 107 , 116 , 117 , 121 , 122 , 128 , 136 , 137 , 152 , 153 , 157 – 164 ]. Reasons related to this equity principle included: First, that rare disease patients should have the same access to treatments as all other patients should and that this was only possible by providing OMPs even if they were expensive [ 5 , 25 , 37 , 38 , 40 , 42 – 44 , 51 , …”

“…It was also stated that there was a general societal preference for equal access and opportunity [ 40 , 61 , 63 , 79 , 80 , 106 , 142 , 151 , 161 , 175 , 176 , 182 ]. Second, some authors stated that all people should have equal opportunities for good health [ 27 , 35 , 47 , 52 , 56 , 66 , 68 , 69 , 73 , 81 , 88 , 90 , 92 , 93 , 95 , 104 , 105 , 109 , 124 , 128 , 137 , 142 , 146 , 152 , 156 , 173 , 175 , 190 , 195 , 196 ], including a “fair chance to live an autonomous and fulfilling life” [ 124 ], which would particularly prioritize young patients, as often this is the case in patients with rare diseases [ 90 , 124 , 195 , 196 ]. Counter-arguments included that it was not feasible to reach full equality of opportunity [ 69 ], that the focus on a fulfilling life would be unfair towards the elderly or severely disabled [ 47 ], and that this proposal would be best solved in a health care lottery, which was unacceptable for Juth [ 105 ] but proposed by others as being part of a potential solution [ 68 ].…”

Heterogeneity in a large pedigree with Danon disease: Implications for pathogenesis and management

“…Health care systems should - and in the UK are required to - tailor services to ensure that disabilities do not preclude access to treatment and that individual circumstances of patients are taken into account [26]. We discuss elsewhere the ethical and legal arguments to support resource allocation for patients with rare orphan diseases [27] but in brief, ethically, the Harvard philosopher John Rawls argues that the more a disease impairs a person's capacity to pursue their goals, the more urgent it is that their health needs are addressed [28]. A patient group with a reduced cognitive function as compared with other patients eligible for the same treatment should arguably therefore have a stronger, not weaker, claim to the available treatment [26].…”

Compassionate use of orphan drugs

“…We contend elsewhere that public and private health providers should offer life-saving treatments once they are thus available in the market [ 4 , 5 ]. However, if a patient has no alternative treatment available in the meantime, they will be deprived effective treatment of their disease.…”

“…The so-called principles of “beneficence” and “rule of rescue” are not satisfactory expressions of this sentiment since they are arbitrary and place an undue burden on patients who may lack the resources to render themselves identifiable on a sufficient scale. A more objective and generally applicable alternative can be found in John Rawls’ Theory of Justice, which provides a cogent basis for the argument that individual countries (or their private health plans) should pay for expensive orphan drugs [ 5 , 22 ]. Rawls (1921–2002) does not specifically address whether a for-profit drug company has a responsibility to patients, and perhaps extending to patients in other countries where it may not even operate.…”