Off-label use of medicines: The need for good practice guidelines

Int J Risk Saf Med. 2017;29(1-2):17-23. doi: 10.3233/JRS-170737.

Abstract

Background: 'Off-label use' is the term used for the prescription and dispensing of a medicinal product for any indication, patient group, route of administration, dosage or treatment regimen other than that listed in the Summary of Product Characteristics.

Objective: In this article the authors present a brief overview of current practices of off-label use in Europe and the applicable European law and jurisprudence. They then go on to present a set of guidelines for best practice in off-label use which underlines the need for guidance on prescription to be firmly rooted in the need to ensure patient safety above all other concerns.

Methods: The article was written from desk research and expert engagement, including a presentation and Q&A in the European Parliament.

Results: This article intends to demonstrate that off-label use entails increased risks for patients, especially when it is not underpinned by rigorous clinical studies or the reporting routes for use are not well defined.

Conclusions: Europe is seeing a growing trend the promotion of off-label prescription of medicinal products for reasons other than pure medical need, including motives such as cost-containment. This poses a numer of questions for the ethical and legal framework for medicine prescription and dispensing in Europe.

Keywords: Off-label use; good practice; patient consent; patient safety; therapeutic freedom.

MeSH terms

  • Cost Control
  • Europe
  • Humans
  • Liability, Legal
  • Off-Label Use / economics
  • Off-Label Use / legislation & jurisprudence
  • Off-Label Use / standards*
  • Patient Safety*
  • Practice Guidelines as Topic*
  • Practice Patterns, Physicians'