Avoiding the most common pitfalls with orphan applications

摘要

The emotive term 'orphan' was first coined in the United States in the late 1970's by patient organisations, many of whom had sponsored academic research in their disease area but then found that companies were unwilling to develop the product because the small target population made their commercial development non-viable. However, intensive lobbying meant that by 1983 the Orphan Drug Act was introduced, which set out a number of incentives to encourage pharmaceutical companies to develop orphan drugs, as outlined in Table V. Europe was somewhat behind the USA in introducing orphan legislation and it was not until 2000 that the concept of an 'orphan medicinal product' was enshrined in EU law. The incentives for EU orphan medicines are outlined in Table 2 and these seem to have encouraged the development of orphan products. In 2000 the Committee for Orphan Medicinal Products (COMP) gave 26 positive opinions but by 2010 there were 123 positive opinions.