Karlsruhe rules on medication cost coverage - German Court to Rule on Unapproved Drug for Terminal Duchenne Patient

A young man with Duchenne muscular dystrophy is taking his fight for an unapproved medication to Germany's highest court. The Federal Constitutional Court will decide on Wednesday whether health insurers must cover the cost of Translarna, a drug not fully authorised for his condition. The case centres on whether terminally ill patients should have access to treatments beyond standard approvals.

The plaintiff, born in 2004, lost the ability to walk in 2015 due to Duchenne muscular dystrophy (DMD). His insurer, AOK Rheinland-Pfalz/Saarland, refused to pay for Translarna, which the European Medicines Agency (EMA) approved in 2014—but only for ambulatory patients aged five and older with a specific mutation. Since then, further restrictions have excluded non-walking patients like him.

The Mainz Social Court initially rejected his claim for coverage. However, the Rhineland-Palatinate State Social Court later overruled this decision, ordering the insurer to provide the drug. It argued that potential benefits justified the treatment. The Federal Social Court then overturned that ruling, siding with the insurer. It found no evidence that Translarna could meaningfully improve the disease's progression. The court also noted that the manufacturer's attempt to expand the drug's approval had failed, reinforcing doubts about its effectiveness for non-ambulatory patients. In its judgment, the court emphasised drug safety, even in cases of fatal illness.

The Federal Constitutional Court's ruling on Wednesday will determine whether insurers must fund unapproved treatments in terminal cases. The decision could set a precedent for patients seeking access to medications outside their authorised use. A verdict is expected after the 9:30 AM hearing.