• Martina Perini, Beatrice Castiglioni, Elisabetta Galai, Dario Trapani, Armando A Genazzani, and Gianluca Miglio.
• Department of Pharmaceutical Sciences, Università del Piemonte Orientale Amedeo Avogadro, Novara, Italy.
• Lancet Oncol. 2025 Feb 1; 26 (2): e103e110e103-e110.

AbstractThe definition of a therapeutic indication formulated by regulatory agencies is a decisive element for the marketing authorisation of medicinal products and for patient access. Trotta and colleagues found that oncological indications granted by the European Medicines Agency (EMA) and the US Food and Drug Administration (FDA) in the period between 1999 and 2008 were clinically different in about 10% of cases. In this Policy Review, we compared the 162 therapeutic indications of 80 medicinal products for solid tumours and blood cancers authorised by the EMA between January, 2015, and September, 2022, with the corresponding labels approved by the FDA. Clinically relevant discrepancies in the EMA summary of product characteristics and FDA labels were identified for 51·9% of the evaluated indications. Differences arise in the place in therapy, in the need for patients to be refractory to previous therapies, in biomarker requirement for eligibility, in concomitant treatments, or in patient characteristics. These differences lead to a different population for which drugs can be prescribed on label, with the FDA granting broader indications more often than the EMA. Therapeutic indications are a legal basis that define the eligible population to specific treatments. The fact that about half of the indications are different between the USA and Europe affect patient access to medications and should stimulate a debate on the weight that uncertainty plays in regulatory decisions.Copyright © 2025 Elsevier Ltd. All rights reserved, including those for text and data mining, AI training, and similar technologies.

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