Masterstudiengang "Drug Regulatory Affairs"

Master-Thesis

Current experience with PIP approvals ***

Viktoria Behse (Abschlußjahr: 2010)

Summary
Language: English
Today, approximately 70-90% of the medicinal products are used for the diagnostic, prevention or treatment in the paediatric sector without development and evaluated data on efficacy as well as safety although without a registration (off-label).
In 2006 the European Paediatric Regulation (EC) No. 1901/2006 was implemented to enhance the paediatric medical care situation as well as to improve the standard requirements with the benefit from more effective and suitable treatments for children. The basic instrument is the Paediatric Investigation Plan (PIP), an official adapted development plan, including requests for waivers and deferrals.
This master thesis gives an overview on the current experience on PIP applications (e.g. generation, submission and approval) with the main focus on the publicly available PIP decisions and their extractable information (period: 07-2007 until 12-2009). It covers 303 PIP applications (+/- deferral/waiver applications), for which the EMA granted a decision. For complementation of the overview publicly available industry experiences and problems with applications were supplemented.
As result, a couple of problems on preparation, validation and compliance check of the PIP applications (including waiver and deferrals) can be separated. The two main aspects of criticism are the time and resulting delays as well as the high number of requested studies.
For the future, an improvement of the procedure is recommended to avoid delays in the overall medicinal product development plan and to decrease the number of modified/new PIP submissions.
Pages: 50 (thereof 13 administrative, e.g. List of Tables);
Annexes: 4 pages: 38