Although individual occurrence is rare, collectively rare diseases is a problem affecting 6–8% of the world population [7]. The Rare Diseases Clinical Research Network was established by The National Institutes of Health (NIH) to address these unique challenges. Among the 7,000 rare diseases, treatments are available only for 5%. Altogether across the globe, there are 350 million people diagnosed with rare diseases and half of them are children, a third of which are below 5-years old [22]. There is a great disparity in access to orphan medicinal products (OMPs) and healthcare services among world countries despite mounting international initiatives [23].