Masterstudiengang "Drug Regulatory Affairs"

Master-Thesis

Approval of a biological medicinal product within the Mutual Recognition Procedure (RMS: Germany) - regulatory strategies and potential challenges from a consulting company's point of view with regard to current pharmaceutical legislation ***

Kristiane Kempny (Abschlußjahr: 2011)

Language: English
 
Biological medicinal products can be used to treat many diseases and are of a great potential for patients and the pharmaceutical industry. Their definition is given in Part I of Annex I of Directive 2001/83/EC. For biologicals, special legal rules apply due to their complexity and impact on public health. In the future, revolutionary treatments for diseases, such as Alzheimers, may be offered by Advanced Therapy Medicinal Products (ATMPs) using gene therapy, somatic cell therapy or tissue engineering. Biological medicinal products that fall within the Annex of Commission Regulation (EC) No 726/2004 “Medicinal products to be authorised by the Community” (such as developed by biotechnological processes using recombinant DNA) must be authorised via the Centralised Procedure (CP). This approval procedure leads to one Marketing Authorisation that makes the medicinal product available to patients and healthcare professionals all over the European Union. However, biological medicinal products that do not fall under the above-mentioned Annex can also be approved by a purely National Procedure (NP), a Mutual Recognition Procedure (MRP) or a Decentralised Procedure (DCP).

This master thesis provides an overview on how a biological medicinal product can be approved during a Mutual Recognition Procedure with Germany as Reference Member State (RMS) and with the Paul-Ehrlich Institute (PEI) as Competent Authority. In order to provide clarity, a fictional case of approving an allergen product is studied. Furthermore, it is discussed how such a large project can successfully and efficiently be managed from a consulting companys point of view. Particular attention has been paid to the responsibility assignment between the applicant and the consultant required for such a project.

In its first part, this master thesis provides a brief introduction and gives general information about requirements to biological medicinal products, the conduction of a Mutual Recognition Procedure, and the role of a consulting company within an approval procedure. In the second part, the key data for the case study are mentioned. To identify possible regulatory strategies and critical aspects with regard to the case, the main part is subdivided into before, during and after authorisation via MRP. Therefore, the planning prior to submission of the application dossier, as well as the requirements following receipt of the approval is discussed. Within each subdivision focus is laid on important challenges, such as Scientific Advice meetings (to discuss the approval strategy with the authorities), an Article 29 CMDh Referral (to solve a disagreement between the Member States in case of a raised Potential Serious Risk to Public Health) and the batch release procedure (in Germany, biological medicinal products, such as allergen products, that are manufactured in batches require official batch control and batch release by the Paul-Ehrlich Institute). Finally, this thesis concludes with a discussion and an outlook. The outlook deals with a field of increasing importance: the approval of similar biological medicinal products, the so-called biosimilars according to Article 10(4) of Directive 2001/83/EC.

Pages: 65, Annexes: 6 pages