Tafuri Giovanni, Bracco Andrea, Grueger Jens
Value, Access & Policy, Apellis International GmbH, Zug, Switzerland.
Market Access, Madrigal Pharmaceuticals, Zug, Switzerland.
Expert Rev Pharmacoecon Outcomes Res. 2025 Jun;25(5):639-645. doi: 10.1080/14737167.2025.2479131. Epub 2025 Mar 19.
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.
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.
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.
