A proposal to improve the supply of orphan drugs.
Industrial medicinal products are not always available for the treatment of some specific patients or diseases. Appropriate research on orphan drugs, neglected doses or administration routes and patients' sensitivity to excipients, as an example, might lead to interesting results in this respect. In the United States, a specific legislation on orphan drugs dates back to 1983; in the European Union, regulations are currently being studied. The industrial production of orphan drugs might be improved following the adoption of adequate incentives. New regulations should also take care of neglected doses, and stimulate the preparation of galenic products, as an example by including a number of monographs on orphan drugs in Pharmacopoeias and National Formularies. As a matter of fact, magistral and officinal preparations made in community and hospital pharmacies in accordance with a medical prescription or with the prescription of a Pharmacopoeia or an Official Formulary, can represent an effective alternative. In this paper, the regulations enforced in the USA and Japan, the European situation, possible incentives to the industry and the role of galenic products will be discussed.
Minghetti
P M
PM
Department of Pharmaceutical Chemistry, University of Milan, Italy.
Giudici
E M
EM
Montanari
L
L
D004335:Drug and Narcotic Control; D005060:Europe; D006801:Humans; D007564:Japan; D009965:Orphan Drug Production; D014481:United States