2. Early access

3.1. Applicable Medicines

Compassionate use programmes (CUP) allow a patient to receive a promising but not yet fully studied or approved medicine and only applies to medicines that are expected to help patients.  The medicine must be undergoing clinical trials or have entered the marketing-authorisation application process. For medicines in clinical trials, at this stage in the development, generally, toxicology studies will have been completed and analysed, and early studies looking at how the medicine is handled by the body will have been completed. However, its safety profile (which side effects it may cause) and dosage guidelines may not be fully established.  

In terms of efficacy, the assumptions for compassionate use may be based on mature randomised phase III trials (e.g., in case of parallel assessment of compassionate use and application for marketing authorisation). However, acceptable assumptions may also rely on promising early data observed in exploratory trials (e.g phase II trials).