Affiliations: Institute for European and International Law WU,
Vienna University of Economics and Business, Vienna, Austria | Private University in the Principaly of Liechtenstein,
Vienna, Austria. E-mail: [email protected]
Abstract: While the safety and availability of medicinal products for the
majority of adult patients has steadily improved in recent decades, for
children and people suffering from rare diseases (orphan diseases) there is a
lack of approved medicinal products for these patient populations. Since the
research and development of medicinal products is associated with high costs,
the costs for paediatric medicinal products and medicines for rare diseases
(orphan drugs) may barely be covered under normal market conditions due to the
small patient populations. In order to prevent the continued exclusion of
children and persons suffering from rare diseases from medical progress and to
eliminate the deficits in the research and development of medicinal products
for these patient groups, the European Union created, along the lines of the
U.S. model, a system of incentives and constraints. Since 2000, under
Regulation (EC) No. 141/2000 (Orphan Drug Regulation) there has been an
incentive system to encourage the research and development of orphan drugs.
With the goal of improving the health of children in Europe, Regulation (EC)
No. 1901/2006 (Paediatric Regulation) combines economic incentives with the
requirement to conduct paediatric studies. This article explains and comments
on the specific regulatory framework for orphan drugs and paediatric medicinal
products in the European Union.
Keywords: European law, medical law, orphan drugs, orphan disease, paediatric medicinal products, Regulation (EC) No. 141/2000, EU orphan drug legislation, Regulation (EC) No. 1901/2006, EU paediatric legislation, EU pharmaceutical law, Swiss pharmaceutical law
