Introduction: Access Challenges in Rare Diseases Rare diseases are conditions affecting fewer than 1 in 2,000 persons, yet over 7,000 mostly genetic rare diseases impact 3.5%-5.9% of the global population, which makes up about 263 to 446 million individuals. 1 Despite the progress made over the past decades, there is still no effective and safe treatment available for many of the rare diseases. Various barriers related to the development of medicinal products intended for the diagnosis, prevention or treatment of rare disorders (or orphan drugs) can be identified . 2 These include the following: 2 The small number of patients and therefore a small market , The high costs associated with drug development , Lack of knowledge about the natural course of the disease and so on . Rare diseases are often serious, chronically debilitating, life-threatening or life-limiting. 3 Availability and access to medicines are important to reduce morbid...