On the 16th of December 2021, the United Nations General Assembly adopted the first-ever Resolution on ‘Addressing the challenges of persons living with a rare disease and their families.’ This milestone is a manifestation of the genuine wish of the 300 million people living with a rare disease globally to be recognised, and a formal commitment of the 193 UN Member States, including the 27 European Union Member States, to support rare disease policies to promote and protect the rights of everyone living with a rare disease and their families.