What the Orphan Drug Act Has Done Lately for Children With Rare Diseases: A 10-Year Analysis

What the Orphan Drug Act Has Done Lately for Children With Rare Diseases: A 10-Year Analysis. Chandana Thorat, BTech, Kui Xu, MD, PhD, Scott N. Freeman, PhD, Renan A. Bonnel, PharmD, MPH, Francesca Joseph, MD, M. Ian Phillips, PhD, DSc, Menfo A. Imoisili, MD, MPH. Pediatrics Vol. 129 No. 3 March 1, 2012. pp. 516 -521 (doi: 10.1542/peds.2011-1798)

KEYWORDS: orphan drugs, rare diseases, Orphan Drug Act, pediatrics