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 o n tropical diseases. METHODS This paper presents two analyses: a 1983-97 retrospective study of the United Stains Orphan Drug Act concerning rare diseases and a prospectiv e study of the European Proposal for a Regulation Concerning Orphan Drugs a nd its possible impact on tropical diseases. RESULTS Different programmes have in the past tried to stimulate R & D in t his area, but results remain limited. Of 1450 new chemical entities markete d between 1972 and 1997, 13 were specifically for tropical diseases and con sidered as essential drugs. Between 1983 & 1997, the US Orphan Drug Act app roved 837 drugs and marketing of 152 new molecular entities (NMEs). Three N MEs have been designated for malaria and human African trypanosomiasis. Sev en others, already commonly used in tropical diseases, received either orph an designation or an orphan approval for another indication. Pharmaceutical companies benefit from the US framework only when the US marker exclusivit y 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 wi th some success. Considering tropical diseases rare diseases seems inadequa te to boost pharmaceutical R Sc D. However, some provisions of the European text may be: relevant to tropical diseases, admitting the need for a more specific rule for evaluations of this kind of drug and recognizing the exis tence of 'diseases of exception'.