Off-label use means “non-approved use.” In other words, it is when a medicine is used in a group of people or for a certain medical condition although this type of use hasn’t been approved by the country's regulatory authorities. For instance, a doctor may prescribe a certain medicine for a child although that medicine has only been approved for use in adults.
Every medicine that you can buy from a pharmacy in Europe needs to have been assessed and licensed by a regulatory authority. The European licensing authority is called the European Medicines Agency (EMA). The responsible authorities in Germany are the Federal Institute for Drugs and Medical Devices (Bundesinstitut für Arzneimittel und Medizinprodukte, BfArM) and – for some medications, like vaccines – the Paul Ehrlich Institute (PEI).
What does “indication” mean here?
The package insert of a medication has an “Indications and usage” section with information about who the medication has been approved for (who it is “indicated” for). An indication is usually defined based on both the specific medical problem and the group of people who used the medication in research. 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 more than one group of people, for example in both children and adults. New medications are often only approved for specific forms of a disease and a certain group of people at first. After that, the manufacturer can apply for separate licenses for other forms of the disease and other groups of people (in other words, for other indications).
When doctors prescribe medication to be used in officially approved indications, it is sometimes referred to as “on-label use.”
When is off-label use an option?
When a treatment is used in a way that has not been officially approved by the authorities, it is known as off-label use. Off-label use is generally possible. But doctors' associations still recommend only prescribing a medication for off-label use if there's scientific proof that it’s likely to be effective when used in that way.
If a doctor recommends using a medicine off-label, they have to tell the patient that this type of treatment hasn’t been officially approved. Before starting the treatment, the patient needs to be informed that the use is off-label, and told about the possible consequences. For instance, if there isn’t enough research on the benefits and harms of the medicine when used for that off-label purpose. Prescribing a medicine off-label is associated with particular risks for doctors. They might face liability problems if there are serious side effects.
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 at the scientific evidence about using the medication in the unapproved way. If the experts conclude that off-label use 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 off-label use of the medication. It is a good idea to contact your health insurer early enough and find out whether they will cover the costs.
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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