Compassionate use program
In some cases an unauthorized (investigational) medicine can become available for treatment of patients, outside of a clinical trial. For this a special program is in place, known as compassionate use. A compassionate use program is only granted for serious or immediate life-threatening diseases, for which no comparable or satisfactory authorized medicine is available. It is intended for groups of patients, not for individual cases. Whenever possible, unauthorized medicines should be used in a clinical trial. Only if this is not possible (for instance when there are no ongoing trials, a patient is not eligible for a trial or logistic reasons prevent a patient from taking part), a compassionate use program can offer a solution.
Because clinical trials have not been completed yet, the known safety information about the medicine may be limited. In general, toxicology studies and early clinical studies will have been completed, however optimal dosing and a full pharmacovigilance profile might still need to be established.
In the EU, compassionate use programs are coordinated and implemented by the National Competent Authorities (NCA). Drug product manufacturers can submit a request to a NCA for enrollment of a medicine in a compassionate use program. The NCA can ask the EMA for an opinion and advice.
If you are interested to learn more about this program and the possibilities, please reach out to us.