ABSTRACT

Orphan medicinal products, or “orphan drugs,” constitute a class of drugs that have been developed specifically to treat a rare medical condition generally referred to as “orphan disease.” The label “homeless or orphan drugs” was first used in the United States by G.P. Provost in 1968 to qualify all categories of medications in which the pharmaceutical industry seemed to have very little interest.1 As the name suggests, rare diseases occur in a very small population.