Orphan drug approvals in Europe since 2001: an analysis by indication

Abstract

Introduction: Europe belatedly implemented a European Community-wide orphan drug policy in 2000. Since April 2000, the European Medicines Agency's Committee for Orphan Medicinal Products has considered 1568 applications for orphan drug status at a rate that has increased steadily year by year. By November 2012, 76 of these drugs have gained European marketing approval for their use in treating 84 specified indications.Areas covered: This review analyses these approvals by therapeutic indication and considers how the legislation has impacted on the development of orphan drugs in Europe. The primary source of data was the European Medicines Agency's list of approved orphan drugs and the reports issued by the Committee for Orphan Medicinal Products. Literature searching focused upon reviews of orphan drug approvals and on the usage of orphan drugs in Europe.Expert opinion: The approval of 76 orphan drugs has clearly provided improved treatments for patients suffering from selected genetic diseases and certain rare diseases. However, 76 approvals in 11 years leaves most rare diseases still lacking specific treatments. If significant progress is to be made in providing treatments for a much higher proportion of rare diseases than is currently the case, then consideration will need to be given to additional methods to stimulate the development of orphan drugs.