FDA's Office of Orphan Products Development: providing incentives to promote the development of products for rare diseases

Abstract

There are nearly 30 million Americans that suffer from at least one of the more than 7000 rare diseases identified to date. Therapies for treating, preventing, or diagnosing rare diseases have been limited due to various reasons. Incentives are provided to sponsors in an effort to promote the development of therapies for rare diseases and to encourage the availability of therapeutically superior drugs or biologics. This paper will discuss the mission of the Office of Orphan Products Development within the Food and Drug Administration (FDA), the specific programs within the office and the relation to incentives provided, achievements of the programs, and continued challenges in rare disease product development.

Keywords: Incentives; Orphan Drug Act; Orphan grants; Orphan-drug designation; Rare disease.