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Table 2 Ethical principles that favours price premium of orphan drugs and their criticism

From: Systematic review on the evaluation criteria of orphan medicines in Central and Eastern European countries

Ethical principle Description Critics
“Non-abandonment” • Society should not abandon individuals who are suffering from a serious and rare condition [19, 96].
• Reimbursement of ODs promotes the appearance of social solidarity where vulnerable groups are supported [35].
• Social justice requires treating everybody with dignity and respect as a human being [97].
• Public healthcare should guarantee the best supportive care for everyone. Restrictions made only for drugs that are far from being cost-effective [8, 44].
“Rule of rescue” • Society puts greater value on health gains of individuals who are in immediate peril, and there are a small number of cases where no alternative treatments are available [34].
• Identifiable individuals are an essential part of this principle [98].
• Lifesaving ability should be considered in the reimbursement decision, but more specifically, only for therapies of life-threatening diseases, which have no alternative treatments. In this later case, the drugs should be financed irrespectively of their cost [28, 31, 96].
• Immediate, life-threatening peril also characterized several other diseases, for which treatment can be more cost-effective [19, 90].
• Since every person faces imminent death in certain periods of time, this cannot be a differentiating characteristic of rare diseases [8].
• It is not right to select one orphan drug over another as having particular social value, because it is not equal to value lifesaving drugs more than cosmetic drugs [26].
• “Rule of rescue” cannot be feasible at population level in an era of constrained resources [90].
“Rights based approach” • Social solidarity requires that all members of the society have access to a decent minimum standard of healthcare because it is the right and fair thing to do [92, 97].
• Right of access to high-quality health care is embedded in the legislation of the developed countries [36, 75, 96].
• “Right-based approach” would not necessarily favour the treatment of rare conditions over more prevalent conditions, because these patients also receive the same standard of care [34].
“Equality of opportunity” • Every member of the society should have the same opportunities to receive treatment and this must be true for rare disease patients as well as other patients with more frequently occurring disorders [60, 96].
• Everybody should have a fair chance to receive not only some treatment, but also the best available treatment [47].
• The equality of opportunity should be the paramount consideration in determining social value [65].
• Effectiveness of ODs is not sufficiently proven in several cases (See section Efficacy, effectiveness)