Evaluation of the extend of off-label use among the most common prescribed medicinal products based on data of the Statutory health insurance system in Germany – Design of the Evaluation

The prescription of medicinal products outside of their marketing authorisation conditions as specified in the summary of product characteristics (SmPC) is defined as “off-label use”. The off-label use is not prohibited and very common in clinical situations where patients have exhausted all other approved treatment options. Nevertheless the reimbursement of off-label use prescriptions is usually not covered by the statutory health insurance system and therefore often subject of legal actions. A landmark judgement issued by the Federal Social Court of Germany in 2002 claimed three criteria which have to be met to permit reimbursement of off-label use prescription. Among others the court demanded that the off-label use prescription is based on scientific evidence. The evidence level should be comparable to that which has to be provided to support marketing authorisation applications. As a consequence the Federal Ministry for Health implemented scientific expert committees from different medical faculties at the German Federal Institute for Drugs and Medical Devices. The German Federal Joint Committee (G-BA) mandates the expert committees to evaluate the scientific evidence level for distinct off-label use indications. Based on the assessment reports of the expert committee the G-BA bodies finally decide on reimbursed of a particular off-label use. Although the expert committees evaluated various off-label use indications there is ambiguity on the relevance of the selected medical conditions. Therefore, we started an evaluation of statutory health insurance data in accordance to Section 303 a–e of the German Social Code V. The data used for this evaluation include all medical diagnoses documented for the financial settlement by the statutory health insurance system. First, it is intend to examine a sample set of the 178 most frequently prescribed active ingredients of pharmaceuticals identified by use of the German “Arzneimittelindex”. For each of these 178 medicinal products we will then determine all of the listed diagnoses. These diagnoses will be then compared with the approved medical conditions according to the respective marketing authorisations to determine the extent of off-label use in the 178 medicinal products.