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 for this work

13 Citations (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.

Original language	English
Journal	Drug Discovery Today
Volume	23
Issue number	7
Pages (from-to)	1328-1333
Number of pages	6
ISSN	1359-6446
DOIs	https://doi.org/10.1016/j.drudis.2018.03.008
Publication status	Published - 1 Jul 2018

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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.",

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

Abstract

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