Compassionate use is defined as an optional exceptional measure in Article 83 of Regulation 726/2004/EC. It was translated into National Law by amending the Austrian Medicinal Products Act in 2009 and incorporated as §8a.
Compassionate use facilitates the provision of unauthorised medcinal products to patients for whom no adequate treatment modalities exist and who cannot be included in clinical trials. Scientific data on Safety and Efficacy of the medicinal product have to be provided, and the applicant commits to the submission of a marketing authorization in due course. These requirements and the temporary limitation of the compassionate use programm were instated to assure that only promising medicinal products qualify for compassionate use programmes.
Compassionate use is granted as a temporary exemption for a group of (unnamed) patients if the medicinal product in question meets the following criteria:
- the medicinal product is used to treat, prevent or diagnose a chronic or serious disease leading to disability or a life-threatening disease,
- the disease cannot be treated satisfactorily with approved and available authorised medicinal products,
- based on the scientific data provided, there is a realistic probability of therapeutic benefit,
- the medicinal product is either already in a centralised marketing authorisation procedure in accordance with Article 6 of Regulation 726/2004/EC or it is in clinical trials in the European Union or a third country.
In Austria, Compassionate Use, as a programme approved by the authorities for a defined group of patients, can be clearly distinguished from other terminologies.
In contrast to the Compassionate Use, the Named Patient Use always only refers to a specific patient. In Austria, the Named Patient Use is not subject to authorisation by or notification to the authorities, but is the so responsibility of the treating physician.
Other terms such as "Early Access Programme" are also used internationally, but do not appear in Austrian law.
Medicinal products that are shipped to or imported into Austria in the context of an approved CUP are exempted from the requirements of the Medicinal Products Importation Act (Arzneiwareneinfuhrgesetz 2010, BGBl. I No 79/2010), according to article 11 para 1.3. Therefore no shipment notification or import application to the BASG is required.
For imports in the context of a named patient use ("Heilversuch"), please refer to the FAQ on importing medicinal products.