Affordable orphan drugs: a role for not-for-profit organizations

Davies, E. H., Fulton, E., Brook, D., and Hughes, D. A. (2017). Br J Clin Pharmacol, doi: 10.1111/bcp.13240.

Although the regulatory steps required to obtain an MA for an orphan drug are numerous and challenging, they are not insurmountable and can be achieved by not-for-profit organizations that are socially motivated to reduce the costs of orphan drugs to the payers of healthcare. Opportunities for orphan drug development resulting in affordable products lie mainly with repurposed drugs.


 Affordable orphan drugs: a role for not-for-profit organizations