Masterarbeit

Impact of scientific advice on the outcome of marketing authorisation applications submitted to the CHMP between 2004 and 2007 ***

Dr. Tatiana Reimer (2008)

Language: English

Scientific Advice can be requested during any stage of the clinical development of a medicinal product but before the submission of a marketing authorisation application. The advice should be understood as a form of communication between the applicant/company/sponsor and the regulatory authority. Advice may be requested on different matters including the manufacture of medicinal products, non-clinical or clinical development or on matters concerning certain regulatory issues like significant benefit for orphan drugs. The aim of this project was to analyse whether compliance to scientific advice may affect the success of marketing authorisation applications submitted to the CHMP. The analysis was performed retrospectively using only marketing authorisation applications which received scientific advice and were submitted to the CHMP between 2004 and 2007. One hundred ninety four applications were submitted to the CHMP within this period. Out of them, 72applications received either scientific advice or protocol assistance, depending on the orphan designation status. Out of these, there were 35 applications for NCEs, 28 for biopharmaceuticals and 9for known active substances. The majority of submitted applications were antineoplastic and immunomodulating agents followed by anti-infectives and alimentary tract and metabolism agents. There were a total of 51 applications with orphan designation of which 21 received SA/PA. Within 41 applicants/products that received SA/PA and were compliant to the given advice, 98 % received a marketing authorisation whereas only 36 % applicants/products were successful without being in compliance with scientific advice. This finding was statistically significant and confirmed the importance of scientific advice.

In conclusion, scientific advice with its supportive role for the pharmaceutical industry has a positive impact on pharmaceutical development. Following scientific advice increases the chances for a positive outcome of marketing authorisation application.

Pages: 53, Annexes, pages: 2