The revision of Europe’s Orphan Regulation represents one of the most significant policy changes affecting rare disease innovation in more than two decades. While the proposed reforms seek to improve patient access and affordability, they also raise important questions about how changes to incentives could influence future investment in orphan medicines.

Building on earlier research into the economics of orphan drug development, this report examines the likely impact of the European Commission’s proposals using a risk-adjusted net present value (rNPV) model. By simulating how developers make investment decisions, the analysis estimates how proposed changes to orphan market exclusivity and related incentives may affect future innovation across Europe. 

The findings suggest that even relatively modest changes to the current incentive framework could reduce the number of orphan medicines developed over the coming decade, with more extensive reforms having an even greater impact. Beyond presenting headline estimates, the report explores the assumptions behind the modelling, discusses the limitations of different analytical approaches and considers the broader implications for patients, developers and policymakers. 

As discussions around pharmaceutical legislation continue to evolve, understanding the trade-offs between encouraging innovation and improving access is essential. This report provides an evidence-based contribution to that debate, helping stakeholders better understand how policy choices may influence future research and development in rare diseases.

Ideal for policymakers, industry leaders, health economists and market access professionals, the report offers valuable insight into one of Europe’s most important regulatory reforms.

Download here to explore the analysis and understand what proposed changes to the Orphan Regulation could mean for future innovation.