Increasing the use of orphan drugs in clinical practice.
Orphan drugs allow people with rare disease to access well-tolerated, safely formulated and effective drugs. Over the next few years, orphan drugs will probably be increasingly adopted into everyday clinical practice, partly because of genomic stratification.
Birmingham Children's Hospital NHS Trust, Birmingham.
Sally-Anne
SA
Hulton
Mark
M
Greener
D002626:Chemistry, Pharmaceutical; D002986:Clinical Trials as Topic; D004345:Drug Industry; D004992:Ethics, Medical; D006801:Humans; D007880:Legislation, Drug; D009965:Orphan Drug Production / Q000331:legislation & jurisprudence / Q000706:statistics & numerical data*; D035583:Rare Diseases / Q000188:drug therapy*