Masterstudiengang "Drug Regulatory Affairs"

Language: English

Before medicinal products for human use are placed on the market, extensive studies, including pre-clinical tests and clinical trials have to be carried out to ensure that the medicinal product is safe, of high a quality and effective for use in the target population. In the European Union, 50 % or more of medicines used in children have never been studied in this demographic group.
The use of unlicensed and off-label medicine in children is widespread and it has been an increasing concern in recent years.

The scope of this thesis is to demonstrate the opportunities of the new Regulation (EC) No 1901/2006 on medicinal products for paediatric use for off-patent drug substances.

The first part of this thesis deals with the historical development of this new Paediatric Regulation and short definitions of some main pillars of the new Paediatric Regulation are drawn up.

The main focus of this thesis is the new type of marketing authorisation, the so called Paediatric Use Marketing Authorisation (short PUMA), which is laid down in the new Regulation. The incentives and obligations as well as the issue of the reimbursement of this type of marketing authorisation are examined.

The application for a PUMA should include the submission of data concerning use of the product in the paediatric population. This required data may be derived from published literature or from new studies, because unnecessary studies in the paediatric population have to be avoided.

Within this thesis, a proposal for the general conditions of a development programme is examined. The basic idea in this development strategy is that all available and published data of the off-patent substance have to be compiled. Based on this data and in consideration of the relevant guidelines, a preliminary estimation regarding which study will additionally be necessary and which could be the significant one which bridges the adult data with the proposed paediatric indication.

Furthermore, a proposal for the structure and the principal content of the later PUMAPIP, an expected time-table for application activities as well as an application strategy are examined in this document.

In conclusion, all open questions about the new Regulation (EC) No 1901/2006 on medicinal products for paediatric have not been answered yet, but all in all it can be said that the Regulation offers opportunities for off-patent substances.

Pages: 38