Orphan Indications and Clinical Trials - Why Rare Diseases Warrant Special Treatment

By Dr. Stephan de la Motte, Chief Medical Advisor, Synteract

Rare diseases and orphan indications are gaining prominence, with funding starting to open up for clinical trials. Public incentives and facilitations are now making drug development for these health issues more financially viable.

Rare diseases are those for which effective treatments may be available but that affect a relatively small segment of the population (defined as roughly .07% in the U.S. and .05% in the European Union). Orphan indications, on the other hand, have no definitive and convincing treatment. A frequent disease can be orphan, if there is no treatment.

The overlapping of these two terms occurs when the cost of normal drug development is in conflict with the frequency (market size). An “orphan drug,” then, is one for a rare disease for which there are no adequate drugs available, according to the USA Orphan Drug Act definition.