罕见病患者的权利保障：论争与辨析

充分保障罕见病患者是国家文明程度的重要体现。尽管各国通过法律和制度，力促罕见病患者平等医疗权的实现，但由于发病率低导致治疗成本高，患者权利仍难获保障。学界围绕是否要保障及如何保障展开了激烈论争。当代西方学者的研究集中于：如何优先保障罕见病患者的平等医疗权，让社会接受罕用药低成本效益的正当性。他们提出以平等原则、营救原则取代成本效益原则进行资源分配，或跨越实体公正原则，遵循程序公正原则。这些辩护与尝试虽未让各国各界达成共识，但为罕见病患者权利保障研究提供了伦理指引与道德基础。

Protection of the Rights of Patients with Rare Diseases: Debate and Analysis

Adequate protection of patients with rare diseases reflects the civilization of a country. Although countries have adopted laws and systems to promote the equal medical rights for patients with rare diseases, it is still difficult to protect patients' rights due to low morbidity and high treatment costs. There has been a fierce debate in academic circles about whether and how to protect it. The research of contemporary Western scholars focuses on how to give priority to ensuring the equal medical rights of patients with rare diseases, so that society can accept the legitimacy of low cost-effectiveness of rare drugs. They put forward the principle of equality and rescue to replace the principle of cost-effectiveness in resource allocation, or to transcend the principle of substantive justice and follow the principle of procedural justice. Although these defenses and attempts did not reach a consensus from all walks of life in various countries, they provided ethical guidance and moral foundation for the research on the protection of the rights of patients with rare diseases.