Twenty years after the introduction of the European Orphan Regulation, the debate surrounding orphan medicines has become increasingly polarised. While scientific advances have transformed outcomes for many patients with rare diseases, questions continue to be raised about affordability, pricing, sustainability and whether the current incentive framework remains fit for purpose.

This white paper offers a broader perspective on these issues by examining the economic principles that underpin orphan drug policy. Rather than focusing on individual products or legislative proposals, it explores how innovation, incentives, pricing, patient access and sustainability interact to shape the rare disease ecosystem. 

Drawing on years of experience in orphan drug economics, the paper considers some of the most common criticisms levelled at the orphan innovation model, including concerns around pricing, unmet need, access and market incentives, and examines them through the lens of economics rather than rhetoric. It argues that many of today’s policy challenges stem from the complex relationship between scientific progress, commercial investment and public policy, rather than from any single aspect of the orphan medicine model. 

At a time when Europe is reassessing the future of pharmaceutical incentives, the report encourages readers to step back from individual policy proposals and consider the wider system that enables innovation to occur in the first place. It provides valuable context for policymakers, payers, manufacturers and anyone interested in the future of rare disease research.

Whether you are seeking to understand the evolution of orphan drug policy or looking for a balanced perspective on the debates shaping its future, this paper offers an accessible and thought-provoking overview.

Download the paper to explore the economic foundations of orphan medicine policy and what they could mean for the next generation of innovation.