Orphan drugs and paediatric medicinal products

Sprecher, Franziska (2013). Orphan drugs and paediatric medicinal products. Pharmaceutical Policy and Law, 15(3-4), pp. 173-194. IOS Press

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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.

Item Type:

Journal Article (Original Article)


02 Faculty of Law > Department of Public Law > Institute of Public Law

UniBE Contributor:

Sprecher, Franziska


300 Social sciences, sociology & anthropology > 340 Law




IOS Press




Franziska Sprecher Daum

Date Deposited:

20 Nov 2014 11:28

Last Modified:

20 Nov 2014 11:28

Uncontrolled 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, Seltene Krankheiten, Medizinrecht, Heilmittelrecht, Heilmittelgesetz, HMG, Kinderarzneimittel, Arzneimittelrecht, orphan drugs



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