Masterstudiengang "Drug Regulatory Affairs"

Master-Thesis

A regulatory guidance on achieving and maintaining proof of compliance with an agreed PIP ***

Dr. Corinna Weidt (Abschlußjahr: 2009)

Language: Englisch

Since the enforcement of the Paediatric Regulation in 2007, pharmaceutical companies and regulators are obliged to accept the responsibilities and challenges in making medicinal products available for the paediatric population. With the development of a new medicinal product, industry must provide a plan covering details of paediatric development to the Paediatric Committee. This requisite is garlanded by opportunities and challenges for industry, regulators and paediatricians. For industry these challenges include the redaction of a plan for development at an early stage. Regulators on the other hand must provide both guidance on development and ensure that information is gathered on a product to allow the practicing paediatrician safe use of the drug and thereby ensuring that the “spirit” of the Regulation is enforced.

Nevertheless for both industry and regulators, the difficulties become apparent when marrying both the expectations of the Regulation with the demands of drug development. The expectations of industry lie in the pragmatic handling of the demands of the Regulation. At the same time regulators see a strong urge for off-label use of drugs by paediatricians and thus strongly back the collection of clinical data in a controlled setting.

Subjects of debate on the enforcement of the Regulation today are however to be found in the demands of clinical development in the paediatric population outside of adult indications, the extent of clinical development as demanded by the Paediatric Committee (PDCO), the difficulties in obtaining equally distributed incentives for all products and product types as well as potential issues regarding possible delays in the authorisation of products for the adult population. But most of all the PIP must be seen as a legally binding document, representing a commitment to the EMEA and any changes require PDCO approval as otherwise a certificate of compliance may be refused.

For Regulators, difficulties are assumed to lie in the assessment of paediatric development plans at an early stage of product development, distribution of responsibilities between the PDCO and national authorities, workload for the Committee members and intense involvement of the PDCO in the life-cycle of a product including appropriate formulation, pre-clinical and clinical development.

This work is aimed to look at the roots of the Regulation and how interpretation of the legislation is currently and how this takes influence on what the expectations and reality of drug development since its establishment. The main focus will lie on making the reader aware of the demands of the Regulation for drug development for a new medicinal product liable for authorisation under the centralised procedure including the main cornerstone achievements which have to be fulfilled to allow PIP approval, marketing authorisation application validation and liability for incentives.