Despite Saudi Arabia's significant healthcare advances in recent decades, rare diseases have remained under-recognized within national healthcare priorities — partly due to the absence of a nationally accepted definition and the complexity of diagnosing and treating patients with orphan drugs. A multi-stakeholder workshop has called for expedited approval pathways for high-priority therapies, fast-track access committees, and alignment of local regulations with international standards to reduce delays and improve availability of orphan drugs while maintaining financial sustainability.
The SFDA has launched an Orphan Drug Program offering pre-submission meetings and priority review for treatments targeting rare diseases affecting fewer than 5 individuals per 10,000 people in Saudi Arabia.