“Off-label use”: What to be aware of

Off-label use means “non-approved use.” In other words, it is the use of a medicine in an indication for which it has not been licensed by the country's regulatory authorities.

Every medicine that you can buy from a pharmacy or drugstore in Europe needs to have been assessed and licensed by a regulatory authority. The responsible authority in Germany is the Federal Institute for Drugs and Medical Devices (Bundesinstitut für Arzneimittel und Medizinprodukte, BfArM). The European licensing authority is based in London and is called the European Medicines Agency (EMA), and its counterpart in the United States is called the Food and Drug Administration (FDA).

Please note that the information below reflects the situation in Germany and may not apply to other countries.

A single medicine can sometimes be used for different conditions or in different groups of people, for example in both children and adults. The use of that medicine first has to be approved separately for each indication and each group of people, though. Pharmaceutical companies apply to the regulatory authorities for such approval of use. But they are under no obligation to apply for the same kind of approval for a product in all countries. This means that a particular medicine might be licensed for a particular indication in one country (country A), but not in another (country B). If a person in country B is prescribed that medicine for that purpose anyway, then they are using the medicine off-label.

Prescribing a medicine off-label is associated with particular risks for doctors. They might face liability problems if there are serious side effects. For this reason, doctors' associations recommend only considering off-label use if current guidelines, recommendations or recognized scientific literature provide evidence supporting such use.

If a patient is receiving a medicine off-label, she or he needs to be informed that the use is off-label, and told about the possible consequences. It is important for patients to know that there may not be enough research on the benefits and harms of the medicine when used for that off-label purpose. Also, the statutory health insurance funds in Germany only rarely cover the costs of medicines that are used off-label.

Whether or not a statutory health insurance fund will cover the costs usually depends on things like whether:

  1. it is a serious medical condition
  2. no other treatments are available
  3. there is good reason to believe that the treatment will be effective (according to BfArM)

If you, a family member or a friend are considering using a drug off-label, be sure to find out exactly what is known about the potential side effects, and whether the health insurance fund will cover the cost of treatment. You can find more information on off-label use on the website of the German Federal Joint Committee (Gemeinsamer Bundesausschuss).