In the last 20 years, orphan drug legislation (ODL) has been adopted in several countries
around the world (USA, Japan, Australia, and the European Union) and has successfully
promoted R&D investments to develop new pharmaceutical products for the treatment of rare
diseases. Without these incentives, many life-saving new drugs would have not been
developed and produced.
For economic reasons, the development of medicines for the treatment of diseases prevalent
in the developing world (or tropical diseases) is lagging behind. Among several factors, the
low average per-capita income makes pharmaceutical markets in developing countries appear
relatively unprofitable and therefore unattractive for R&D-oriented companies.
The case of ODL may offer some useful insights and perspectives for the fight against
neglected tropical diseases. First, the measures used in ODL may also be effective in boosting
R&D for neglected tropical diseases, if appropriately adapted to this market. Second,
small-sized companies, which have played a successful role in the development of orphan
drugs for rare diseases, may also represent a good business strategy for the case of tropical
diseases.