Over the past few years, it has become increasingly clear that promising ideas for orphan medicines often stall before they reach patients. Sometimes this is due to scientific uncertainty, but more often it reflects the way the subsequent development and access pathway is organised.

All steps in medicine development are closely interconnected, yet they are still too often approached as sequential, separate phases, including within academia. This increases the risk that development trajectories come to a halt, or that unequal access emerges between countries and patient groups.

In this invited review, Carla Hollak and colleagues systematically examine the full development and access pathway, drawing on their shared experiences, as well as the role that academics can play within it. The article discusses where continuity is lacking, where collaboration can be improved, and how early choices around governance and data can shape patient access later in the process.

Especially in light of the ongoing debate about a future-proof medicines system, such an integrated approach is essential. By better aligning development, pricing, and appropriate use of new products, it becomes possible to work towards a system that stimulates innovation while safeguarding sustainable and socially acceptable access for patients.

Read the full article here: Medicine Development and Access for Rare Diseases: Can We Do Better? – Hollak – 2026 – Journal of Inherited Metabolic Disease – Wiley Online Library