Using four decades of FDA orphan drug designations to describe trends in rare disease drug development: substantial growth seen in development of drugs for rare oncologic, neurolog...

by Kathleen L. Miller, Lewis J. Fermaglich, Janet Maynard
Published 2021-06-01

FDA orphan products clinical trial grants: assessment of outcomes and impact on rare disease product development

by Kathleen L. Miller, Christine Mueller, Gumei Liu, Katherine I. Miller Needleman, Janet Maynard
Published 2020-09-01