Orphan Diseases
- The orphan drug designation trends of the past five years is an illustration of modern science and the remarkable progress American medicine has made in 35 years since the 1983 Orphan Drug Act was first enacted
- Today, drug development and medical care is more personalized, genetically targeted, and more likely to address rare diseases
- The Orphan Drug Act represented a promising advance in medical care for the nearly 30 million1 Americans that have a rare disease2 -- defined by the Orphan Drug Act as a disease that affects fewer than 200,000 people in the United States3
- While there are thousands of rare diseases, it is estimated that 85-90 percent are serious or life threatening with only a few hundred that may be treated with currently available treatments3
- Developing rare disease treatments remains enormously challenging and can be extremely costly3
- Many rare conditions lack a clear scientific understanding of the mechanistic basis and some are so rare that studying them present numerous challenges to researchers3
- Haffner, ME, Whitley, J and Moses, M. Two decades of orphan product development Nature Reviews Drug Discovery 1, 821-825 (October 2002) http://www.nature.com/nrd/journal/v1/n10/full/nrd919.html
- Rao, Gayatri. The Rise in Orphan Drug Designations: Meeting the Growing Demand. FDA Voice. July 18, 2016. https://blogs.fda.gov/fdavoice/index.php/2016/07/the-rise-in-orphan-drug-designationsmeeting-the-growing-demand/# Accessed December 14, 2017.
- Boat TF, Adamson PC, Asbury C, et al. Rare Diseases and Orphan Products, Accelerating Research and Development Institute of Medicine (US) Committee on Accelerating Rare Disease Research and Orphan Product Development. Washington DC: National Academies Press; 2010.