Is orphan drug status beneficial to tropical disease control ? Comparison of the American and future European orphan drug acts.
OBJECTIVES To quantify past outcomes of tropical pharmacology research and development (R & D) and to assess past benefits of the American orphan drug act and potential benefits of the future European orphan drug regulation on tropical diseases.
METHODS This paper presents two analyses :
A 1983-97 retrospective study of the United States Orphan Drug Act concerning rare diseases and a prospective study of the European Proposal for a Regulation Concerning Orphan Drugs and its possible impact on tropical diseases.
RESULTS Different programmes have in the past tried to stimulate R & D in this area, but results remain limited.
Of 1450 new chemical entities marketed between 1972 and 1997,13 were specifically for tropical diseases and considered as essential drugs.
Between 1983 & 1997, the US Orphan Drug Act approved 837 drugs and marketing of 152 new molecular entities (NMEs).
Three NMEs have been designated for malaria and human African trypanosomiasis.
Seven others, already commonly used in tropical diseases, received either orphan designation or an orphan approval for another indication.
Pharmaceutical companies benefit from the US framework only when the US market exclusivity clause was applicable.
Future European orphan drug regulation appears to be similar to the US Orphan Drug Act.
CONCLUSION The orphan drug programmes relating to rare diseases have met with some success.
Considering tropical diseases rare diseases seems inadequate to boost pharmaceutical R & D. (...)
Mots-clés Pascal : Médicament orphelin, Maladie tropicale, Pharmacologie, Recherche, Homme, Essai clinique, Etude comparative, Statut, Lutte sanitaire
Mots-clés Pascal anglais : Orphan drug, Tropical disease, Pharmacology, Research, Human, Clinical trial, Comparative study, Status, Sanitary control
Notice produite par :
Inist-CNRS - Institut de l'Information Scientifique et Technique
Cote : 99-0359438
Code Inist : 002B30A03B. Création : 14/12/1999.