title: Don't Do Different Things…Do Things Differently! Drug Development in Rare Diseases.
abstract: Although definitions of rare disease vary, most acknowledge that there are small numbers of affected patients compared with other conditions. Small numbers of patients, overlapping involvement of investigators as researchers and caregivers, as well as close relationships between researchers and manufacturers require a different pattern of drug development. Regulatory guidances for rare diseases are available, as well as ones for specific rare diseases. Maintaining drug supply for rare diseases also demands innovative approaches.
authors.affiliation: Department of Oncology and Department of Medicine, Division of Clinical Pharmacology, The Johns Hopkins University School of Medicine and The Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins, Baltimore, Maryland, USA.
authors.forename: M A
authors.initials: MA
authors.lastname: Rudek
authors.affiliation: Clinical Pharmacology, Rare Diseases, Global Product Development, Pfizer Inc., Collegeville, Pennsylvania, USA. joan.korth-bradley@pfizer.com.
authors.forename: J M
authors.initials: JM
authors.lastname: Korth-Bradley
mesh_terms: D017277:Drug Approval; D055808:Drug Discovery / Q000379:methods*; D017408:Guidelines as Topic; D006801:Humans; D009965:Orphan Drug Production / Q000592:standards*; D035583:Rare Diseases / Q000188:drug therapy*