Compassionate Use

Campaign for access to medicines for people with rare diseasesThere is nothing worse for a patient, from a psychological and human standpoint, than being severely ill or even dying from a disease, when experimental treatments are  out there, pending final evaluation.

Nowadays, patients are aware of new product developments; research is no longer a secret. In some countries, regulators have responded to this situation, and medicines can be obtained on a Compassionate Use basis prior to their marketing authorisation, to help treat patients who have no other options and who do not have the time to wait for the end of clinical trials and the authorisation process.

These pages inform patients and healthcare professionals about Compassionate Use possibilities. Compassionate Use can save lives.


EURORDIS position on compassionate use

EURORDIS position arguing for compassionate use programmes to be made more widely available to those patients in need of urgent help.


Good to know

This section explains the role of Compassionate Use Programmes: who are they for, and the conditions for accessing  a medicine under compassionate use.


How to proceed

This section explains how your physician can request authorisation to prescribe to you a medicine on a compassionate basis 



Useful services: where you can learn more about Compassionate Use Programmes.

The voice of rare disease patients in EuropeEURORDIS The international voice of people living with rare diseases, Rare Diseases International is a EURORDIS initiativeRare Disease International Bringing together patients, families and experts to share experiences in a moderated multilanguage forum, RareConnect is a EURORDIS initiative RareConnect The Rare Barometer Programme is a EURORDIS initiative that carries out surveys to transform rare disease patients' experiences into figures and facts that can be shared with decision-makers.Rare Barometer An international awareness raising campaign taking place on the last day of February each year, Rare Disease Day is a EURORDIS initiativeRare Disease Day Join the largest gathering of rare disease stakeholders across Europe, at the biennial European Conference on Rare Diseases and Orphan Products. ECRD is a EURORDIS initiativeEuropean Conference on Rare Diseases