Center for Population-Level Bioethics, Rutgers University, New Brunswick, New Jersey, USA
J Med Ethics. 2022 Feb;48(2):86-92. doi: 10.1136/medethics-2021-107691. Epub 2021 Nov 23.
Orphan drug policy often gives 'special treatment' to rare diseases, by giving additional priority or making exceptions to specific drugs, based on the rarity of the conditions they aim to treat. This essay argues that the goal of orphan drug policy should be to make prevalence irrelevant to funding decisions. It aims to demonstrate that it is severity, not prevalence, which drives our judgments that important claims are being overlooked when treatments for severe rare diseases are not funded. It shows that prioritising severity avoids problems caused by prioritising rarity, and that it is compatible with a range of normative frameworks. The implications of a severity-based view for drug development are then derived. The severity-based view also accounts for what is wrong with how the current system of drug development unfairly neglects common diseases that burden the developing world. Lastly, the implications of a severity-based view for current orphan drug policies are discussed.
孤儿药政策通常通过对特定药物给予额外的优先权或例外对待，来为罕见疾病提供“特殊待遇”，这取决于它们所针对的疾病的罕见程度。本文认为，孤儿药政策的目标应该是使疾病的流行率与资金决策无关。本文旨在表明，正是疾病的严重程度而不是流行率，导致我们认为在没有为严重罕见疾病提供治疗时，重要的诉求被忽视了。本文表明，优先考虑严重程度可以避免因优先考虑罕见性而导致的问题，并且与一系列规范框架兼容。然后得出基于严重程度的观点对药物开发的影响。基于严重程度的观点还解释了当前药物开发系统如何不公平地忽视了发展中国家负担沉重的常见疾病所存在的问题。最后，讨论了基于严重程度的观点对当前孤儿药政策的影响。
