A decision of the BSG of 29 June 2023 (Ref.: B 1 KR 35/21 R) concerns the question of whether persons insured under the statutory health insurance system are also entitled to a medicinal product that has been negatively assessed by the European Medicines Agency (EMA) during the marketing authorisation procedure as part of their treatment for a fatal disease.
The plaintiff, born in 2004, suffers from a genetic progressive disease that typically proves fatal in early adulthood. The plaintiff is unable to walk since 2015. The defendant is a statutory health insurance fund to which the plaintiff had applied for coverage of the costs of the drug “Translarna”. The defendant refused to cover the costs on the grounds that the drug had only been approved for the treatment of the condition in patients who were able to walk, but not in those who were unable to walk. The EMA rejected the manufacturer's applications to extend the indication to patients who are no longer able to walk due to negative assessments. The Higher Social Court of Rhineland-Palatinate ruled in favour of the plaintiff, ordering the defendant to provide the plaintiff with the drug “Translarna” in accordance with Section 2 para. 1a of the German Social Code Book Five (SGB V). The court reasoned that treatment with “Translarna” in a patient who was no longer able to walk had a not entirely remote prospect, based on circumstantial evidence, of having a noticeable positive effect on the course of the disease.
However, on 29 June 2023, the BSG (Ref.: B 1 KR 35/21 R) ruled that the plaintiff was not entitled to be supplied with the drug “Translarna” under Section 2 para. 1a SGB V. The court set aside the decision of the Higher Social Court and dismissed the action. Insured persons would be entitled to medical treatment under less stringent conditions if they were in an emergency situation due to a life-threatening or regularly fatal disease. This would require a not entirely remote prospect of a cure or a positive impact on the course of the disease. This includes, in particular, drugs whose efficacy has not yet been fully proven. The necessary prospect of success is lacking, however, if the medicinal product received a negative assessment in the authorisation procedure, regardless of whether the assessment is based on a meaningful study situation or the medicinal product's medical benefit could not be confirmed due to methodological problems in the selection and analysis of the data submitted by the manufacturer. This is the case with the drug “Translarna”, as it has been given a negative assessment by the EMA. According to the case law of the First Senate, a medicinal product that has been assessed negatively in the marketing authorisation procedure has a bar effect under Section 2 para. 1a SGB V. The purpose of the medicinal products authorisation is to protect patients with serious illnesses from incalculable risks. The authorisation process provides a particularly high level of assurance of the scientific and independent nature of the assessment due to the expertise of the regulatory authorities. The Medicinal Products Act has its own structured quality assurance system and allows for simplified approvals and exceptional decisions in cases of hardship. If a case falls outside these regulations, no further exception can be granted.
