3. Off-label use


1. Off-label use

(This section is organised in the form of a book, please follow the blue arrows to navigate through the book or by following the navigation panel on the right side of the page.)

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.