Compassionate Use

Compassionate Use in Austria

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 non-licensed medcinal products to patients for whom no adequate treatment modalities exist and who cannot be included in clinical trials. Safety and Efficacy of the medicinal product have to be demonstrated and the applicant committs 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 exceptional and temporary measure for a group of patients, when the medicinal product in question meets the following conditions:

• it is intended for the treatment, prevention or diagnosis of a serious or rare disease,
• there is no suitable therapeutic alternative (medicinal product or other) available in Austria,
• and the benefit/risk ratio of the medicinal product is presumed to be positive,
• The medicinal product is either the subject of an application for a centralised marketing authorisation in accordance with Article 6 of Regulation 726/2004/EC or is undergoing clinical trials in the European Union and/or elsewhere.

The guidance for the submission of Compassionate Use Applications is currently under development.