Masterstudiengang "Drug Regulatory Affairs"


Regulatory Science on Orphan Drug Application within Paediatric Field ***

Dr. Ulrike Domack (Abschlußjahr: 2004)

This thesis describes the imperative of the development of medicinal products for patients with rare or orphan diseases, especially for children. The therapeutic lack of treatment options for children within the orphan filed is pointed out.

The pharmaceutical industry has been in general reluctant in the investigation of specific treatments or the adaptation of existing medicines to meet the special needs of children. The particularities of children result from developmental, physiological and psychological differences in comparison to adults. Ethical concerns, the developmental preclinical and clinical affords for several age groups of children with different developmental status and probably particular formulations of the medicinal product have to be considered within the development of medicines for children. The market for orphan medicinal products as well as for children is small, especially the market for children with orphan diseases. In general drugs for orphan diseases, so called orphan medicinal products are not developed by the pharmaceutical industry for economic reasons but with respond to public health.

The USA acts as precursor in the process of legislative support of the development of pharmaceuticals for orphan diseases and for children. The initiatives are borne by incentives in the case of Orphan drug regulation and obligations and incentives in the event of medicines for children. The European Union follows these examples, whereas the legislation for better medicines for children is at present in the beginning of legislative procedure with a proposal for a regulation on medicinal products for paediatric use from the European Commission of September 2004. The similarities and differences of orphan drug legislation between Europe and USA as well as the obligations and incentives from the current laws in the paediatric field in USA and the proposal of the European Commission for the European Community are compared and the strategies for drug development are deduced by an example.

As example acts an acquired orphan disease,  the acute traumatic spinal cord injury. The regulatory environment of orphan drug development within the paediatric field is outlined. The problems of paraplegia and tetraplegia, resulting from acute spinal cord injury, with the particularities of theses conditions to children are delineated. It is assumed that a fictive company, OrphaMed Ltd., wants to develop an established drug substance, for the treatment of acute spinal cord injury in adults and children. The drug regulatory affairs manager builds up a procedure and regulatory strategy for an orphan medicinal product development for the treatment of acute traumatic spinal cord injury for children, taking account for the legislative background in Europe and USA.

Pages: 69