THE ORPHAN DRUG ACT: INCENTIVES, CHALLENGES, AND MARKET TRENDSAbstract
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.
Maleeha Masood, Trapti Saxena * and Maariyah Tahseen,
Department of Pharmaceutics, G. Pulla Reddy College of Pharmacy, Mehdipatnam, Hyderabad, Telangana, India.
03 March 2023
22 May 2023
31 May 2023
01 November 2023