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Abstract

Orphan drugs are used to treat rare diseases that affect fewer than 200,000 people in the United States. Development and approval of orphan drugs are challenging due to the limited patient population and substantial development costs; the U.S. Food and Drug Administration (FDA) has established several programs, such as the Orphan Drug Designation program and Orphan Product Grants program to encourage the development of medications that treat rare diseases, the rare pediatric disease voucher program supports research and growth of rare diseases that affect pediatric populations, the humanitarian use device program regulates medical devices associated with rare diseases, the main aim of these incentives is to provide financial and regulatory support to sponsors. Overall, the regulatory framework in United States offers a supportive environment for drug manufacturers in developing and approving orphan products; these initiatives ensure that patients with rare diseases have access to safe and effective treatments. United States is one of the top leaders in developing orphan drugs and continues to play a critical role in the global effort to bring new therapies to patients with this rare disease. INTRODUCTION:Orphan drugs treat rare diseases or disorders that occur in less than 200,000 people in the U.S.; orphan drugs are drugs used in the prevention, treatment, and diagnosis of rare diseases (diseases with less available information) or diseases that do not commonly occur 1 . The drugs eligible for orphan drug designation are new molecular entities (drugs newly developed for treating certain rare diseases), secondary indications (expansion of already existing symptoms, and any newly developed formulations 2 .