New rare diseases are being identified and reported regularly, making the field of rare diseases a complex and heterogeneous field. Over the last several decades, the focus on rare diseases is attributed to the intentional incentivizing of the pharmacy and biotechnology industries to develop drugs that would otherwise not be profitable. The 1983 Orphan Drug Act in the United States made available over 420 Food and Drug Administration (FDA) – approved orphan drugs for rare diseases.
Similarly, since 2000, 84 authorized orphan drugs for rare diseases have been approved. This progress is also attributable to the improved genetic, molecular, and biochemical understanding of rare diseases owing to the advanced technologies in the current era. Definition A rare disease is an often-debilitating lifelong disease or disorder with a prevalence of 1 or less per 1000 population (World Health…