Abstract

Orphan drugs are medications that are created to treat rare diseases, often with limited therapeutic options for specific groups of people. This article reviews orphan drugs, highlighting their significance in addressing treatment gaps for rare diseases, and the supportive policies that promote their development. It includes a list of key orphan drugs and discusses rare diseases that lack sufficient treatments, focusing on leishmaniasis, trypanosomiasis, lymphatic filariasis, phenylketonuria, and cystic fibrosis. For each of these diseases, a variety of approved or investigational orphan drugs are showcased, along with descriptions of their clinical applications and existing constraints. The article delves into the regulatory frameworks created to promote the advancement of orphan drugs. It explores how health authorities, such as the FDA, foster innovation in the industry while guaranteeing drug safety and efficacy by offering incentives such as expedited approvals and financial assistance. Despite these initiatives, challenges such as high costs, limited market incentives, and access barriers continue to persist. To tackle these concerns, the article proposes measures to enhance affordability, accessibility, and international collaboration. The conclusion highlights the importance of ongoing endeavors to address treatment disparities in rare diseases. Future perspectives are further explored in the article, with emphasis on how advances in research and technology, as well as policy, can expedite the creation of orphan drugs while enhancing regulatory mechanisms and making these therapies more accessible to patients.