On 10th-12th May 2018, EURORDIS hosted the ninth European Conference on Rare Diseases & Orphan Products (ECRD) in Vienna, welcoming almost 1000 participants from more than 50 countries. In between the various panel discussions, Professor Marc Dooms, author of the Declaration on Good Off-Label Use Practices, known as the GOLUP (Good Off-Label Use Practice) was there to introduce participants to a poster, drawing attention to the widespread use of rare disease medical products off-label, driven by a lack of authorized medicines for specific indications. A European Commission study in this area, published February 2017, identified the large amount of rare disease products being used off-label and the difficulty and high cost of carrying out clinical trials, as the main reasons for the lack of authorized drugs.
A wide number of participants showed an interest in the campaign, developed in reaction to the national developments promoting off-label use when alternatives exist, thus creating unnecessary risks for patients. Moreover, the off-label use of medicinal products in the rare diseases area was referred to on numerous occasions during the lectures and discussions that took place throughout the conference.
Under the leadership of the European Brain Council a number of organizations have endorsed the GOLUP. The coalition of signatories have worked together to raise awareness about this important issue by organizing events and meetings at European and national level, aiming to provide a clear framework on when and how the off-label use of medicinal products can safely take place.