Access and pricing of medicines for patients with rare diseases in the European Union: an industry perspective. A 2025 update

Abstract

Introduction: In a previous publication, we provided an overview of the current situation of patient access to orphan drugs in the European Union (EU), of the evolving rare disease policy landscape and recommendations for areas of improvement throughout the lifecycle of orphan drugs. This article aims to provide an update on evolving new policies impacting access to orphan drugs in the European Union from a health technology developer perspective.

Areas covered: We provide an update on the emerging competitiveness gap in the pharmaceutical sector between the EU and the United States. We discuss the latest developments of the European Commission's revision proposal of the EU's pharmaceutical legislation and the threats and opportunities of the new HTA Regulation with a focus on orphan drugs. Additionally, we present the latest policy updates from Germany and the United Kingdom impacting orphan drug developers.

Expert opinion: While our initial set of recommendations remains unchanged, the policy landscape for orphan drugs seems to be deteriorating both at the EU and at the Member State level.

Keywords: Orphan drugs; access; health technology assessment; orphan drug regulation; pharmaceutical legislation; pricing and reimbursement.