The Lancet · 2008 · 77 citations · 15 references
MedicinePharmacologyClinical PharmacologyClinical Trial DesignClinical TrialsDrug DevelopmentEssential MedicinesOrphan Drug DesignationOrphan Drug ApplicationsOrphan Drug Designation StrategyOrphan Drug DevelopmentOrphan Drug StrategyOrphan Medicinal Product Designation
15
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John Whitehead · Biometrics · 1997 · 520 citations
Clinical trials and rare diseases: a way out of a conundrum
Richard Lilford, Jim Thornton, David Braunholtz · BMJ · 1995 · 235 citations