The Voice of Rare Disease
Patients in Europe
Improve 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).
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.
François Houÿez, Treatment Information and Access Director, Health Policy Advisor at EURORDIS, commented, “Compassionate Use Programmes can save lives by providing early access to promising new medicines for rare disease patients. Patients with chronic, seriously debilitating or life-threatening diseases should not have to wait to access a new medicine.”
He added, “The new proposals included within this position set out why there is a need for CPU programmes in all countries and how first steps could be taken to implement or improve such systems. We need to aim to reduce inequalities between countries in patients’ access to CPU programmes and new medicines.”
Recommendations to patient organisations, industry, Member States & European authorities
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.
What is compassionate use?
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’. Read more.
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
- CUPs are not clinical trials, however in Member States that do not have a regulatory scheme for compassionate use, open label trials can serve to provide a product available on a compassionate basis. Use keywords such as “compassionate” or “open label” to find them via www.clinicaltrialsregister.eu.
Eva Bearryman, Communications Manager, EURORDIS