Paediatric Orphan Drugs

Abstract

Rare diseases are frequently genetically determined and caused by an inborn metabolic error, thus occurring early in life and affecting normal growth, and sexual and CNS maturation. The development of a paediatric orphan medicine presents a real hurdle for pharmaceutical companies because of the nature of the patient population where little knowledge is available. In addition, clinical trials are more difficult to run, take longer and cost more. An overview of the current situation in the paediatric orphan medicines field in Europe is provided below through the identification of unmet therapeutic needs still existing in the field (TEDDY NoE work), which represents a description of the orphan drugs so far authorised in Europe and of the paediatric drugs under development (Paediatric Investigation Plan – PIP).