2. Early access
3. Compassionate use
3.2. Initiation and oversight of compassionate use programmes
Compassionate use implementation lies in the competence of a EU member state (MS). Article 83 of Regulation (EC) No 726/2004 on compassionate use (‘Legal basis’ below) is complementary to national legislations but includes an option to MS who wish to receive a CHMP opinion regarding the conditions for compassionate use of a specific medicine which falls within the scope of Article 83. This means that CUPs are co-ordinated and implemented by the MS, according to national rules and legislation. Their authorisation is similar to clinical trials, and patients are followed in the same way as patients in a clinical trial. Not all MS have legislation in place to cover compassionate use programmes, and where it exists, it may differ between MSs, for instance with respect to the required documentation to be submitted with an application for a CUP.
Compassionate use programmes are generally requested by manufacturers and marketing-authorisation applicants providing the medicine in question. This is often triggered by a doctor.
CUPs may continue in certain Member States until the medicine becomes available on the market. Legislation requests that, where a compassionate use programme has been set up, the applicant (company) ensures that patients taking part also have access to the new medicine during the period between authorisation and placing on the market.
The national authority keeps a register of the patients treated with the medicine within the CUP, and systems are in place to record any side effects reported by the patients or their doctors.