EURORDIS publishes its position on improving Compassionate Use Programmes to ensure patients’ early access to medicines
EURORDIS-Rare Diseases Europe has published a new position on compassionate use calling for the adoption of measures to revolutionise patients’ access to new medicines through Compassionate Use Programmes (CUP). According to the EU regulation on pharmaceuticals (art. 83.2), compassionate use is ‘making a medicinal product available for compassionate reasons to a group of patients with a chronically or seriously debilitating disease or whose disease is considered to be life threatening, and who cannot be treated satisfactorily by an authorised medicinal product’. Under CUPs, medicines that have not yet been authorised are made available to patients whose disease would otherwise reach a stage that is severe and irreversible or who may pass away before the medicine is brought to market. In the position paper, EURORDIS puts forward several policy proposals as possible solutions to improve compassionate use across Europe. The position paper also sets out recommendations to patient organisations, industry, EU Member States and European authorities on how to advocate for, create and manage CUPs, including:
- A call on patients to engage in early discussion with medicines developers to agree on if and when a CUP could be relevant, and for which patients;
- A call on industry to plan for an adequate supply of the product to be provided through the CUP;
- A call on national authorities to improve transparency of the CUPS they authorise in their Member State, so that clinicians and patients are aware of programmes and how to join them;
- And a call on the European Commission to compare different national schemes for CUPs in Europe.
To find more information on compassionate use and CUPs in specific countries, please visit:
- List of departments responsible for CUPs in Europe (Heads of Medicines Agencies)
- European Medicines Agency website
