Healthcare systems face challenges due to budget constraints, complex therapies, and new treatments for rare diseases. One of the most successful patient advocacy campaigns of all times was initiated by people living with HIV and AIDS in African countries. Facing industry giants, they won court cases allowing governments to ignore intellectual property rights when the price of a medicine was abnormally high. This led to the approval of the international ‘Agreement on Trade Related aspects of Intellectual Property Rights’, which contributed to improving the availability of AIDS treatments for millions of people. Beyond this successful patient advocacy campaign, patient organisations have an important role to play in national discussions on prices of treatments and pharmaceuticals, especially for rare diseases. This article first discusses the specificity of the pricing of orphan medicinal products, and then provides an overview of some of the important actions that can be carried out by patients’ organisations.

由于预算限制、复杂的疗法和罕见疾病的新疗法，医疗保健系统面临挑战。有史以来最成功的患者倡导运动之一是由非洲国家的艾滋病毒感染者和艾滋病患者发起的。面对行业巨头，他们打赢了官司，允许政府在药品价格异常高的情况下无视知识产权。这导致批准了国际“与贸易相关的知识产权协议”，这有助于提高艾滋病的可用性治疗数百万人。除了这一成功的患者倡导活动之外，患者组织在全国性的治疗和药物价格讨论中也可以发挥重要作用，尤其是对于罕见疾病。本文首先讨论了孤儿药定价的特殊性，然后概述了患者组织可以采取的一些重要行动。