PHP94 The US Orphan Drug Landscape: Before and After EU Orphan Drug Legislation of 2000

Abstract

To encourage the development of orphan drug (OD) products, the European Union (EU) approved OD legislation in 2000, mirroring the US legislation passed in 1983. For the first time in history, these products were recognized as different from other drug products on a global level. Given this new global incentive for OD development, an increased focus on developing these products was expected. The objective of this research is to quantify a change in the rate of global OD development as measured by the rate of US Food & Drugs Administration (FDA) OD designations. All history within the OD database from the FDA website was included. The OD status, designation date, and contact company fields were analyzed to evaluate the OD designation rate and location of developers (US or non-US categories). There was a sharp increase in the number of FDA OD designations after the year 2000, with the rate of OD designations increasing by 475% compared to the prior period. From 1983 until 2000, 516 products received an FDA OD designation and the average increase in OD designation was 2.5 per year. From 2000 through 2011, the number of designations was 1266 and average increase in OD designation was 14.7 per year. After 2000, the proportion of OD designations from non-US countries increased until 2009 when nearly one-third of products with a new FDA OD designation were from non-US companies. The implementation of EU OD legislation may have contributed to a change in the number of FDA OD designations per year and the types of companies developing ODs for the US. With developing markets preparing OD legislation, these policy changes may further stimulate the growth of the OD industry around the world.