Orphan Indications and Clinical Trials - Recruiting

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

Rare diseases and orphan indications are gaining prominence, because 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.

Both of them have some unique characteristics. Today we look at ways to proactively recruit so as to overcome some of these challenges.