Orphan drugs (ODs) for rare diseases (RDs) present pharma companies with the opportunity to address a substantial unmet medical need, with approximately 7,000 RDs, and only about 5% having effective treatments. A large number of patients in the US are affected with these RDs (around 25-30 million people). The majority of RDs affect children. Also, RDs often translate into chronic and deteriorating conditions for patients, the majority being affected and starting in childhood, and often resulting in early death. Significant economic and social burdens exist for patients, caregivers, and the healthcare system to treat RDs.