EU decision-making for marketing authorization of advanced therapy medicinal products: a case study

Sofieke de Wilde; Delphi G.M. Coppens; Jarno Hoekman; Marie L. de Bruin; Hubert G.M. Leufkens; Henk Jan Guchelaar; Pauline Meij

doi:10.1016/j.drudis.2018.03.008

EU decision-making for marketing authorization of advanced therapy medicinal products: a case study

Sofieke de Wilde, Delphi G.M. Coppens, Jarno Hoekman, Marie L. de Bruin, Hubert G.M. Leufkens, Henk Jan Guchelaar, Pauline Meij^*

^*Corresponding author af dette arbejde

13 Citationer (Scopus)

Abstract

A comparative analysis of assessment procedures for authorization of all European Union (EU) applications for advanced therapy medicinal products (ATMPs) shows that negative opinions were associated with a lack of clinical efficacy and identified severe safety risks. Unmet medical need was often considered in positive opinions and outweighed scientific uncertainties. Numerous quality issues illustrate the difficulties in this domain for ATMP development. Altogether, it suggests that setting appropriate standards for ATMP authorization in Europe, similar to elsewhere, is a learning experience. The experimental characteristics of authorized ATMPs urge regulators, industry, and clinical practice to pay accurate attention to post-marketing risk management to limit patient risk. Methodologies for ATMP development and regulatory evaluations need to be continuously evaluated for the field to flourish.

Originalsprog	Engelsk
Tidsskrift	Drug Discovery Today
Vol/bind	23
Udgave nummer	7
Sider (fra-til)	1328-1333
Antal sider	6
ISSN	1359-6446
DOI	https://doi.org/10.1016/j.drudis.2018.03.008
Status	Udgivet - 1 jul. 2018

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AU - de Bruin, Marie L.

AU - Leufkens, Hubert G.M.

AU - Guchelaar, Henk Jan

AU - Meij, Pauline

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EU decision-making for marketing authorization of advanced therapy medicinal products: a case study

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