Challenges in the Research and Development of Orphan Drugs: A Review

Pharmaceutical agents which treat rare medical conditions like orphan or rare diseases are called “orphan drugs”. The name ‘orphan’ itself indicates that the pharmaceutical industries are showing less interest in the development and marketing of drugs intended only for a small number of patients. Difficulty in the diagnosis and therapeutic management of rare disease is the major challenge in the development of orphan drug.

In recent years, progress has been made in the development of orphan drugs by pharma industries due to enactment of different regulations, administration authorities, tax benefits, marketing rights and public awareness by different countries. This review provides an overview of incentives, marketing rights and administrative authorities of different countries like U.S, European Union, South Korea, Japan, Australia and Taiwan.