Pharmaceutical Research

, 35:165 | Cite as

Preclinical Development of Cell-Based Products: a European Regulatory Science Perspective

  • James W. McBlane
  • Parvinder Phul
  • Michaela Sharpe
Research Paper
Part of the following topical collections:
  1. Living Cell Medicines: Perspectives and Challenges



This article describes preclinical development of cell-based medicinal products for European markets and discusses European regulatory mechanisms open to developers to aid successful product development. Cell-based medicinal products are diverse, including cells that are autologous or allogeneic, have been genetically modified, or not, or expanded ex vivo, and applied systemically or to an anatomical site different to that of their origin; comments applicable to one product may not be applicable to others, so bespoke development is needed, for all elements - quality, preclinical and clinical.


After establishing how the product is produced, proof of potential for therapeutic efficacy, and then safety, of the product need to be determined. This includes understanding biodistribution, persistence and toxicity, including potential for malignant transformation. These elements need to be considered in the context of the intended clinical development.


This article describes regulatory mechanisms available to developers to support product development that aim to resolve scientific issues prior to marketing authorization application, to enable patients to have faster access to the product than would otherwise be the case.


Developers are encouraged to be aware of both the scientific issues and regulatory mechanisms to ensure patients can be supplied with these products.


biodistribution cell therapy regulatory science toxicity tumorigenicity 



Committee for Advanced Therapies


cell based medicinal product


Committee for Human Medicinal Products


Cell Products Working Party


deoxyribonucleic acid


European Commission


European Medicines Agency


Food and Drug Administration


Good Laboratory Practice


human pluripotent stem cells


hematopoietic stem cells


International Conference on Harmonisation


mesenchymal stromal cells


Scientific Advice Working Party


Safety Working Party


Acknowledgments and Disclosures

The authors did not receive any funding from any agency in the public, commercial or not-for-profit sectors to write this article. JW McBlane is an alternate member of the Committee for Advanced Therapies (CAT) and of the Scientific Advice Working Party (SAWP) of the Committee for Human Medicinal products (CHMP), both operated by the European Medicines Agency. However, views expressed in this article represent only those of the authors and not those of these committees and working party.


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Copyright information

© Springer Science+Business Media, LLC, part of Springer Nature 2018

Authors and Affiliations

  • James W. McBlane
    • 1
  • Parvinder Phul
    • 1
  • Michaela Sharpe
    • 2
  1. 1.Licensing DivisionMedicines & Healthcare Products Regulatory AgencyLondonUK
  2. 2.Nonclinical Safety, Cell and Gene Therapy CatapultGuy’s HospitalLondonUK

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