E-DRUG: Orphan cancers and drug development (3)
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Indisputably, orphan drug status was beneficial to rare diseases and have
met with some success. The US orphan drug act (ODA) accelerated drug
development and marketing for rare diseases : 837 products designated
between 1983 and 1997 compared to 34 in the same period preceding the
passing of the Act.
However this scheme is not so perfect as described by Blaine Carmichael.
1.
- The Act is often viewed as a mechanism to obtain seven years of exclusive
marketing rights, especially for companies which do not hold patents for
the drug ;
- the ODA became a mechanism to obtain a patent-like protection ;
- various parties have questioned the unregulated pricing policy that may
penalize patients (Love, 1992).
2.
Dispite of good intentions, its effectiveness for tropical diseases has
been disappointing even useless.
3 of 837 products benefitted directly from orphan drug status
(eflornithine, halofantrine, mefloquine). The 2 antimalarials had already
been developed by the Walter Reed Army Institute of Research as part of the
US military infectious disease research program and the production of the
trypanocide eflornithine has been discontinued due to lack of commercial
value.
(Trouiller P et al. Is orphan drug status beneficial to tropical disease
control ? Comparison of the American and future European orphan drug acts.
Tropical Medicine and International Health 1999 (4),6: 412-420).
The unexpected success of the US ODA demonstrates that pharmaceutical
companies do not invest in R-D simply because they have the cash on hand
and with the aim of a social welfare mission but because the prospects for
future returns look promising.
Patrice Trouiller
Hospital pharmacist
- M�decins sans Fronti�res Foundation, Paris
- Centre Hospitalier Universitaire de Grenoble, France
Fax 33 476 76 51 09
E-mail pat.trouiller@wanadoo.fr
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