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.
What does "off-label" mean?
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 if current guidelines, recommendations or recognized scientific literature provide 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.
When will the cost of the treatment be covered?
Whether or not a statutory health insurance fund will cover the costs usually depends on things like whether:
it is a serious medical condition
no other treatments are available
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 on the website of the German Federal Joint Committee (Gemeinsamer Bundesausschuss).
IQWiG health information is written with the aim of helping people understand the advantages and disadvantages of the main treatment options and health care services.
Because IQWiG is a German institute, some of the information provided here is specific to the German health care system. The suitability of any of the described options in an individual case can be determined by talking to a doctor. informedhealth.org can provide support for talks with doctors and other medical professionals, but cannot replace them. We do not offer individual consultations.
Our information is based on the results of good-quality studies. It is written by a team of health care professionals, scientists and editors, and reviewed by external experts. You can find a detailed description of how our health information is produced and updated in our methods.
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