Scientific Publications by FDA Staff

Drug Info J 1994 Apr;28(2):495-503

Applications of the Orphan Drug Act to Special Patient Populations

Haffner ME

On January 4, 1983, the president signed the Orphan Drug Act (P.L. 97¿414), which amended the Food, Drug, and Cosmetic Act; this established for the first time a federal government policy to cooperate and assist in a program to facilitate the development of drugs for rare diseases. The act provided important new incentives designed to encourage sponsors to develop drugs without commercial value. Patient populations eligible for orphan designation have shifted and evolved during the nine years since the enactment of the Orphan Drug Act; the size of populations with orphan diseases keeps changing. In the early days of orphan drug development, prevalence figures were very small ones. In 1983, children's diseases were recognized as potential orphans; however, rare cancers, AIDS, and diseases affecting the elderly, as well as the burgeoning market of biotech products, were not considered. In order to qualify for orphan drug status, an indication must have a prevalence of fewer than 200,000 people in the United States, or produce no profit from sales in the United States for seven years. The operative population is that which is ascertained by the applicant and agreed to by the Food and Drug Administration (FDA) on the date of application. It may not always be easy to obtain figures; if a disease grows into an epidemic such as AIDS, the population will, regrettably, grow. Small numbers and wide distribution of a disease population are disadvantageous in conducting clinical trials, and in acquiring data necessary to obtain product approval. Despite the fact that markets are small, most orphan diseases are lifetime diseases and individuals often require chronic treatment. Criticism of the Orphan Drug Act occurs if more than one firm has an interest in developing the same drug, cost for the drug is high, and competition is discouraged by the exclusivity provisions of the act. There is no question that some orphan drugs have become expensive. The populations are still small, and the markets remain limited and widely dispersed throughout the United States. Clearly, the program has been extraordinarily successful. It has, nevertheless, broad implications not considered in 1983, and continues to evolve.