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.
Hulton
Sally-Anne
SA
Birmingham Children's Hospital NHS Trust, Birmingham.
Greener
Mark
M
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*