Abstract

COMPARATIVE REVIEW OF ORPHAN DRUG DESIGNATION IN THE USA AND EUROPE AND NEW APPROVAL TRENDS FROM 2021 TO 2023

Rahul Ashok Raut*

ABSTRACT

Orphan drugs are developed to treat rare diseases, which affect a small population, often with life-threatening risks, particularly for children. These diseases are frequently genetic, and diagnosing and developing treatments present significant challenges. The U.S. Orphan Drug Act (1983) and the EU Regulation (EC) No. 141/2000 provide legal frameworks for orphan drug designation, with prevalence limits set at fewer than 200,000 people in the U.S. and fewer than 5 in 10,000 in the EU. Both frameworks offer incentives such as tax credits, grants, and market exclusivity (7 years in the U.S. and 10 years in the EU) to encourage development. In the U.S., the FDA's Office of Orphan Products Development (OOPD) oversees the process, while the European Medicines Agency’s Committee for Orphan Medicinal Products (COMP) manages it in Europe, with pre-submission advice available in the EU. Between 2021 and 2023, significant progress was made, with the U.S. approving 28 orphan drugs in 2023 and the EU granting 17 marketing authorizations. Despite similarities, differences in criteria, tax benefits, and exclusivity periods reflect the varied regulatory landscapes between the two regions.

Keywords: Orphan drugs, Rare Disease, orphan drug designation, USFDA, EMA, office of orphan product development, committee for orphan medicinal product.