Since the introduction of the EU Regulation on Orphan Medicinal Products (OMPs), over 260 OMPs to treat rare disease have been authorised in the EU. Naturally, this has led to an increase in spending on OMPs, which has prompted policy discussions about its sustainability and impact on healthcare budgets. However, a closer examination reveals that such concerns may not be warranted.
Today, European countries spend on average 6.6% of their pharmaceutical budgets – or just 1.2% of total public healthcare spending – on OMPs. These levels have been achieved gradually over 25 years and are the result of deliberate policy choices, including targeted regulatory incentives, scientific advances, and a shared commitment to addressing unmet medical needs of rare disease patients.
Our analysis shows that OMP spending is unlikely to threaten the sustainability of pharmaceutical budgets. As more OMPs lose exclusivity, generic and biosimilar competition will ease pressure on budgets. In parallel, the pace of new orphan designations has slowed in recent years, and future developments are expected to focus increasingly on ultra-rare diseases with lower overall budget impact.
Importantly, the differences in how much countries spend on OMPs appear to be driven less by economic capacity and more by political prioritisation and national policy choices, such as early access programmes and support for European Reference Networks.
The study was commissioned and funded by Alexion, AstraZeneca Rare Disease.
The results of this study will be presented and discussed on 16 September at the Symposium on Sustainable Funding for Rare Disease Innovation. For more information and to register, visit this link.
Download
