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“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 to treat a medical condition 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 main responsible authority in Germany is the Federal Institute for Drugs and Medical Devices (Bundesinstitut für Arzneimittel und Medizinprodukte, BfArM). The European licensing authority is called the European Medicines Agency (EMA). You will find detailed information about all of the situations for which the use of a medication has been approved in the "Indications and Usage" section of the accompanying package insert.

This includes both the specific medical problem to be treated and the group of people who can use it. Some medications have only been approved for certain forms of a disease and only in certain groups of people, such as women, men, children or adults.

One medication can sometimes be used for more than one medical condition or in different groups of people, for example in both children and adults. The manufacturer can apply for separate licenses for each combination of a medical condition and group of people (in other words, for each indication).

What does "off-label" mean?

Doctors can sometimes use a medication in a way that hasn't yet been officially approved. For instance, for a different medical problem. Or in a child, although it has only been approved for use in adults. When a treatment is used in a way that has not been officially approved, it is known as . Off-label use is generally possible.

But doctors' associations still recommend only prescribing a medication for if there's good scientific proof that it is likely to be effective when used in that way. Prescribing a medicine off-label is associated with particular risks for doctors. They might face liability problems if there are serious side effects.

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.

When are the costs of off-label use covered?

The statutory health insurance funds in Germany only rarely cover the costs of medicines that are used off-label. They will only do so after experts have looked into the scientific about using the medication in the way that hasn't been approved. If the experts conclude that in this area is likely to be effective and safe, the health insurer may cover the costs of the treatment. Their conclusion will depend on various factors, such as whether

  • the medical problem is serious
  • no other treatments are available
  • there is good reason to believe that the treatment will be effective

The manufacturer of the medication also has to agree that it can be used off-label. By doing so, they also agree to accept legal responsibility if there are any serious side effects, for instance. Only then will statutory health insurers cover the costs of this of the medication.

Doctors have to inform their patients that this kind of treatment would be off-label, and tell them about the possible consequences. It is then a good idea to find out what is known about the effectiveness and side effects of the treatment, and contact your health insurer to find out whether they will cover the costs. You can find more information about the of medication on the German Federal Joint Committee (Gemeinsamer Bundesausschuss).

Gemeinsamer Bundesausschuss (G-BA). Richtlinie über die Verordnung von Arzneimitteln in der vertragsärztlichen Versorgung – AM-RL. July 5, 2019.

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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“Off-label use”: What to be aware of
Updated on February 4, 2021
Next planned update: 2024

Publisher:

Institute for Quality and Efficiency in Health Care (IQWiG, Germany)

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