Off-label use - Who pays for patient healing?

Fri, 2019 / 09 / 06
Doctors have a lot of freedom in prescribing the so-called off-label use of pharmaceutical drugs outside the scope of their approval. Though the off-label use is increasingly common, there are fundamentals questions that need to be answered, why there is an increase in the off-label use? And who will be responsible for covering the treatment cost when a drug is prescribed outside its approved indication?

Off-label use is defined as the use of pharmaceutical drugs beyond their approved indication, dosage and age group as a result of a physician assessment based on published scientific evidence. Off-label use is commonly adapted in the treatment of children and rare disease. As, in these special cases the patient's needs are different, hence a customized therapy is required for a successful treatment while tending to the patient's needs.

A remarkable case occurred at the University Hospital in Dresden, Germany. A patient suffering from the rare autoimmune disease systemic lupus erythematosus (SLE) did not benefit from the use of the standard cyclophosphamide therapy and at the same time had the desire to have children, a desire that would not be fulfilled with the cyclophosphamide due to its damaging side effects on the ovaries and the kidneys. As a result, the treating physicians adapted the therapy to the patient's needs and decided to prescribe Rituximab as a treatment although, Rituximab is prescribed as a treatment for cancer and autoimmune diseases and is not approved as a treatment of SLE. Today, the patient's condition is improved with no side effects on her kidneys and she gave birth to a child few years back.

Despite the improvement in the patient conditions and the fulfillment of her desire to mother a child, the patient's health insurance company refused to pay the treatment costs and won the legal battle over this matter in court. As a result, the University Hospital in Dresden, Germany had to cover the treatment costs. According to the Dresden Social Court, the treating physician should have reattempted a second try with cyclophosphamide and that the prescription of Rituximab was „medically unnecessary" as the studies did not clearly validate it as a successful treatment for SLE.

Currently there is no clear legal regulation of the off-label use of drugs. Moreover, health insurance and pharmaceutical companies are accused of underinvesting in supporting individuals with personal, case-specific needs and difficulties, as the costs in these areas of application would not be profitable. Kordula Schulz-Asche, a politician from the German Green Party, pointed out the fundamental problem that pharmaceutical companies carry out research where they expect to achieve high profits further down the line. This leads to overlooking the off-label use and the potential it has for the treatment of children and rare diseases.

At SKC, we support the idea of a health system that puts the welfare of the human being at the heart of the community. Therefore, we support companies in accessing the market of drugs for rare diseases, commonly referred to as orphan drugs. The publication "Orphan Drugs nach GSAV - New basic conditions for approval and reimbursement" by Prof. Matthias P. Schönermark summarizes the key points of the current new legal regulations.

By Janina Spottke, B.B.A. Hons., Ezzaldin Alfar and Jonas Feldkamp, B.Sc.

G-BA: Definition Off-Label-Use
BDI: Rechtliche Aspekte beim Off-Label-Use
FAZ Artikel "Die Grenzen des Beipackzettels"
Krankheitsbilder Systemische Lupus Erythematodes
Patienteninformation Cyclophosphamid

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