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.