Commercialisation of advanced therapies: a study of the EU regulation on advanced therapy medical products

Tutkimustuotos: OpinnäyteVäitöskirjaArtikkelikokoelma


Advanced therapy medicinal products (ATMPs), is a heterogeneous class of modern biotechnology medicines encompassing products based on genes, cells and tissues. ATMPs provide new therapeutic opportunities for many diseases and debilitating injuries to the human body, particularly in the disease areas where conventional treatments have proved insufficient. The ATMP Regulation was set up as a lex specialis to ensure the free movement of ATMPs within the EU in order to facilitate their access to the internal market, and therefore to foster the competitiveness of European pharmaceutical companies while guaranteeing the highest level of protection of public health. Since adoption of the Advanced Therapy Medical Product Regulation (EC) No. 1394/2007 (the ATMP Regulation) only 6 ATMPs have been granted marketing authorisations and 4 of them are still on the market.

The primary objective of this study is to analyse the benefits and limitations of the ATMP Regulation from the perspective of SMEs, academia and non-profit organisations that develop ATMPs. Secondly, it discusses the kind of amendment to the ATMP Regulation and related regulatory instruments and processes required to accelerate translation of research into advanced therapies and to facilitate commercialisation of ATMPs whilst ensuring the safety of patients. In addition, it analyses implications of the EU s limited mandate in the field of public health for developers of ATMPs. This study also investigates whether barriers to commercialisation relate to ATMPs as such or whether something else in the innovation system is impeding their market entry. As an example of potential ATMPs undergoing development, it also considers some specific, regulatory and moral patenting obstacles that impede the market entry of human embryonic stem cell-based products.

To foster research on ATMPs, regulators must take measures to create a facilitative regulatory environment that encourages innovation, protects public health and, finally, enables timely patient access to innovative therapies. Risk-proportionate approaches to clinical trials and GMP manufacture along with the European Medicine Agency s early access incentives and initiatives are presented as potential facilitators of market entry. The main regulatory measures suggested to foster innovation, improve safety and access to advanced therapies include: facilitating R and D by adaptive, risk-proportionate approaches to clinical trials and GMP manufacture, streamlining the ATMP Regulation (classifications and rules on so-called hospital exemption, in particular) and simplifying regulatory processes for ATMPs.
Painoksen ISBN978-951-51-2343-5
Sähköinen ISBN978-951-51-2343-5
TilaJulkaistu - 2016
OKM-julkaisutyyppiG5 Tohtorinväitöskirja (artikkeli)


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