Drug development for rare neurological diseases
ORPHAN DESIGNATION IN DRUG DEVELOPMENT
Orphan drug (US) and orphan medicinal product (EU) designations are part of regulatory frameworks designed to provide incentives for the development of drugs to meet important unmet needs in rare indications. Some of the advantages of orphan designation are:
There is today a trend towards investment in orphan development programs in both small and large companies – the search for “niche busters” as an alternative to “block busters”.
Diseases with fewer than 200,000 patients in the US, or with a prevalence of fewer than 50 per 100,000 in the EU, are considered rare by the corresponding health authorities. Japan, Australia and several other countries today have similar regulatory frameworks, supporting broad application of an orphan strategy.