Marketing Authorisation of New Medicines in the EU: Towards Evidence-based Improvement
(2013; 211 pages)

Thesis Utrecht University -with ref.- with summary in Dutch. The studies presented in this thesis were performed in the context of the Escherproject (T6-202), a project of the Dutch Top Institute Pharma. Parts of this thesis were performed in collaboration with the Dutch Medicines Evaluation Board, Utrecht, The Netherlands Julius Center for Health Sciences and Primary Care, Utrecht, The Netherlands European Medicines Agency, London, United Kingdom.


Marketing approvals of new medicinal products can count on large interests of both patients in need of new medicinal therapies and the pharmaceutical industry. Drug regulatory authorities, such as the European Medicines Agency (EMA) and the US Food and Drug Administration (FDA) act as independent governmental third parties that decide about marketing authorisation. The EU regulation of marketing approval of new medicinal products aims on the one hand to protect public health by preventing that low-quality, unsafe, or inefficacious products enter the market. On the other hand the regulation aims to promote public health by ensuring that patients gain access to medicines without unnecessary delay.

Consequently, regulators have to find the appropriate balance between the need to ensure that decision making is based on scientifically valid data and the need for access to new medicines. They have to balance efficacy and safety evidence (with its inherent uncertainties), while taking into consideration the need for (better) medicines to treat the disease. This system has been very successful in bringing many valuable safe and efficacious medicines to the market and, thus, contributed to improving public health. There are also important challenges that this system has to face in the future, in order to continue to serve both (sometimes conflicting) objectives of protecting and promoting public health.

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