An Evolving Regulatory Environment for Rare and Orphan Diseases
Rare diseases affect fewer than 200,000 people each, totaling around 30 million in the U.S. Orphan drugs face R&D barriers, but FDA support has increased since 1983.
orphan drugs
Rare diseases affect fewer than 200,000 people each, totaling around 30 million in the U.S. Orphan drugs face R&D barriers, but FDA support has increased since 1983.