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European regulatory experience with advanced therapy medicinal products

Nature Reviews Drug Discovery volume 18, pages 8–9 (2019)Cite this article

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During the initial evaluation of the first 20 MAAs for ATMPs (listed in Supplementary Table 1), a number of common issues were identified as 'major objections', that is, issues that would prohibit licensing of the product if not resolved. These issues include suitable quality and clinical data demonstrating the efficacy and safe use of the ATMP (Fig. 1b; Supplementary Box 1 and Table 2). To identify 'hot spots' in ATMP development, we analysed the 20 MAAs and all SA given for ATMPs by the EMA so far, which provide the basis for the general points highlighted in this article and the more detailed recommendations provided in Supplementary Box 1. The EMA also has several tools in addition to PRIME designation that can help ATMP developers in building a robust data package for their MAA, such as the opportunity for small and medium-sized enterprises developing ATMPs to receive an opinion from the EMA's Committee for Advanced Therapies on the approvability of their quality and non-clinical data (see Supplementary Box 2).

Overall, for ATMPs, it is particularly important to carefully map the product definition and initial development strategy at the beginning to avoid the need to unexpectedly have to generate new data and/or conduct additional studies at a later stage. Early characterization of the product in terms of identity, purity, stability and strength is necessary to identify the critical parameters that will be used for in-process, release and stability testing. This has particularly been a challenge for cell therapies owing to their complexity and the multitude of parameters from which to choose.

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Figure 1: Experience in the evaluation of advanced therapy medicinal products in Europe.

Acknowledgements

We thank R. Hemmings, S. Ruiz, H.-G. Eichler, P. Celis, S. Vamvakas and J. Llinares for their contributions to the review of the article.

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Author notes

  1. Lisbeth Barkholt and Caroline Voltz-Girolt: These authors contributed equally: Lisbeth Barkholt, Caroline Voltz-Girolt

Authors and Affiliations

  1. Medicinal Products Agency, Uppsala, Sweden

    Lisbeth Barkholt

  2. European Medicines Agency, London, UK

    Caroline Voltz-Girolt

  3. Medicines and Healthcare Products Regulatory Agency, London, UK

    June Raine

  4. Medicinal Products Agency, Uppsala, Sweden

    Tomas Salmonson

  5. Paul-Ehrlich-Institut, Langen, Germany

    Martina Schüssler-Lenz

Authors
  1. Lisbeth Barkholt
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  2. Caroline Voltz-Girolt
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  3. June Raine
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  4. Tomas Salmonson
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  5. Martina Schüssler-Lenz
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Corresponding author

Correspondence to Caroline Voltz-Girolt.

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Competing interests

The authors declare no competing financial interests.

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Supplementary information

Supplementary information S1 (box)

Analysis of scientific advice and major objections for ATMPs (PDF 543 kb)

Supplementary information S2 (box)

EMA support to ATMP developers (PDF 137 kb)

Supplementary information S1 (table)

Status of the 20 marketing authorisation applications as of June 2018 (PDF 101 kb)

Supplementary information S2 (table)

Analysis of the major objections raised during the evaluation of the marketing authorisation applications (cut-off June 2018) (PDF 183 kb)

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Barkholt, L., Voltz-Girolt, C., Raine, J. et al. European regulatory experience with advanced therapy medicinal products. Nat Rev Drug Discov 18, 8–9 (2019). https://doi.org/10.1038/nrd.2018.200

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