Rare diseases (RDs), a heterogeneous group of health conditions, affect a small number of subjects compared to other common diseases in the general population, and represent a major public health issue at the global level.

Under the governance of the Saudi MOH, Key Experts are rallying for a moonshot program pledge focused on rare diseases. The Standalone aims to unite all distinguished experts involved in Rare Diseases management to pool competencies and direct attention toward advancing rare diseases end-to-end care in the Kingdom of Saudi Arabia. It will bring together an ecosystem of rare disease experts and researchers to explore opportunities for collaboration and support a range of public-private partnerships to Enhance the end-to-end rare diseases management ecosystem while optimizing clinical, access and regulatory pathways for very small patient populations to de-risk and enhance management.