COMPREHENSIVE REVIEW ON ORPHAN DRUG REGULATIONS IN THE USA, EU, JAPAN, AND AUSTRALIA: POSSIBLE RECOMMENDATIONS TO SPECIFIC CHALLENGESAbstract
Orphan drugs are medicinal products intended to diagnose, prevent, or treat life-threatening or debilitating rare diseases. In some parts of the world, a rare disease is also addressed as an orphan disease due to its poor diagnosis mechanism, low prevalence, and limited treatment options. The United States, Japan, Australia, and the European Union have adopted policies promoting orphan drugs development and commercialization. The United States was the first nation to adopt a law on orphan drugs in 1983, followed by Japan (1993), Australia (1998), and the European Union (2000). Orphan drug development also possesses challenges like limited scientific knowledge, low number of patients available, high pricing of these drugs & other regulatory challenges. This review compared the orphan drug regulations in the USA, the EU, Japan, and Australia and addressed the challenges involved in orphan drug development along with possible recommendations.
Manasa Tadepalli *, Sri Sowmya Veeravalli and Ravi Kumar Reddy Juturi
Department of Pharmaceutical Regulatory Affairs, Shri Vishnu College of Pharmacy, Vishnupur, Bhimavaram, Andhra Pradesh, India.
27 March 2021
26 June 2021
30 June 2021
01 February 2022