Drug labels: A flawed source of data for studying orphan drug approvals

摘要

To the Editor: We write to bring attention to the important methodological flaws in the study "Raising Orphans: How Clinical Development Programs of Drugs for Rare and Common Diseases Are Different," published in Clinical Pharmacology & Therapeutics by Orfali et al. Although the authors claim to be extending our "approach" in their analysis, this is not at all the case. Orfali et al.1