1. Off-label use

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In some regions, for example in the US, the Summary of Product Characteristics (SmPC) is called ‘label’, therefore the term ‘off-label’. There is no definition on the term ‘off-label use’ of medicines in the EU legislation, but off-label use is commonly understood as the use of a medicine outside the terms of its marketing authorisation (MA), i.e., not in accordance with the approved Summary of Product Characteristics (SmPC).

The reference terms for off-label use thus are the terms of marketing authorisation in the country where the product is used.

Examples for off-label use are:

  • using a different dosage regimen (a different dosage, frequency, route or method of administration),
  • use for an indication (in terms of medical condition) other than that listed in the SmPC,
  • use in a group of patients for which it has not been authorised, such as a certain age group like children (paediatric use),
  • use despite a contraindication, i.e. a specific situation where the medicine should not be given for safety reasons.

The so-called ‘irrational prescription and use of medicines’ could also to a certain degree be considered off-label use. Examples are:

  • the use of too many medicines per patient (polypharmacy), not considering possible interactions between different medicines as per SmPC
  • the inappropriate use of antibiotics for non-bacterial infections,
  • the failure to prescribe in accordance with the best evidence-based clinical guidelines, and
  • inappropriate self-medication

While not optimal, off-label prescribing may remain essential to address unmet medical needs of patients. However, the manner in which countries deal with the off-label use of medicines is not harmonised across the EU as shown in the next section.