Paving the Path for Sustainable and Responsible Off-Label Use of Pharmaceutical Products in Europe

• Published in: European journal of health law Vol. 31; no. 2; p. 129
• Main Authors: Díaz Hernández, Miquel, Roslin, Waltter, Mansnérus, Juli
• Format: Journal Article
• Language: English
• Published: Netherlands 13.03.2024
• ISSN: 1571-8093
• DOI: 10.1163/15718093-bja10123

Off-label use of pharmaceuticals involves a wide array of aspects ranging from legal and regulatory ones to clinical to safety considerations. Access to off-label therapies is particularly relevant question for patients in areas of unmet medical need. Simultaneously, off-label use also triggers wider considerations relating to social and economic sustainability of health care systems and access to health. National authorities have adapted different regulatory approaches to off-label use of pharmaceuticals, ranging from (1) "regulatory silence"; to (2) allowing off-label use at the discretion of the treating physician; and to (3) a more stringent approach in which off-label use is subject to third party approval. This article provides a brief overview of these different regulatory approaches from a helicopter perspective, and it discusses benefits and shortcomings these approaches. Finally, it presents ideas for preconditions for sustainable and responsible off-label use of pharmaceutical products to ensure patient safety whilst ensuring their timely access to health.