Abstract
Aim: Off-label prescription is not regulated on the European Union (EU) level and therefore not harmonised in the EU Member States (MS). Despite this, the use of medicines outside of the drug label occurs in clinical practice, and it can be included in treatment guidelines and/or reimbursed in some cases. It is, however, currently not clear to what extent off-label use can be included in regulatory discussions at a European level at the different committees at the European Medicines Agency. In this article, we provide an overview of the current legislation on MS level regarding off-label prescription in order to support EU regulatory discussions. Methods: Relevant national legislation regarding off-label prescription from MS was identified by distributing a questionnaire to EMACOLEX. Case law was excluded. The identified categorical elements and prerequisites in the national legislation were then categorised. Subsequently, a comparison was made to the five Good Off-Label Use Practice (GOLUP) principles. Results: Based on the obtained responses from 10 MS, we observed a large heterogeneity in the legislation of MS regarding off-label prescription. Five (out of 10) MS regulate off-label prescription explicitly and seven (out of 10) MS have prerequisites. One or more prerequisites per MS were reflected in the GOLUP principles as formulated in 2017. Conclusion: The main contribution of this work is to flag that off-label prescription actually needs to be well defined and understood before it can be appropriately taken into consideration in regulatory discussions. There is a heterogeneity in legislation regarding off-label prescription in the investigated MS, which may lead to different perspectives. A common understanding of the concept and more alignment in off-label prescription practices and their regulation at MS level may contribute to further regulatory discussions.
Highlights
Off-label use and prescription are often referred to in the context of different discussions related to medicine development, approval and use
The main contribution of this work is to flag that off-label prescription needs to be well defined and understood before it can be appropriately taken into consideration in regulatory discussions
There is a heterogeneity in legislation regarding off-label prescription in the investigated Member States (MS), which may lead to different perspectives
Summary
Off-label use and prescription are often referred to in the context of different discussions related to medicine development, approval and use. Recent judgements of the Court of Justice of the European Union have triggered a discussion about off-label use of medicinal products and the extent to which these practices and data generated may be considered in the context of assessment of dossiers for approval of medicinal products[3]. Several categories of medicines, including orphan medicinal products, medicines for paediatric use and medicines in oncology and the central nervous system, should be approved via the centralised procedure. This centralised authorisation procedure is covered by European Union (EU) legislation[4]. Such a procedure can be started when an active substance of a medicinal product has been used for more than 10 years and its efficacy and safety have been well established, often based on results from the scientific literature[9]
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