Affiliations: [a] Senior Orphan Drug Pharmacist, University Hospital, Leuven, Belgium
| [b] Partner, White & Case LLP, Brussels, Belgium
Correspondence:
[*]
Address for correspondence: Marc Dooms, Bondgenotenlaan 155, 3000 Leuven, Belgium. Tel.: +32 016 22 35 45; E-mail: dooms@evonet.be.
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, therapeutic freedom, patient safety, patient consent, good practice
