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*Author for correspondence:

E-mail Address: patcharaphun_k@yahoo.com

Department of Community Pharmacy, Faculty of Pharmacy, Silpakorn University, Sanam Chandra Palace Campus, Nakhon Pathom, Thailand

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Warit Ruanglertboon

https://orcid.org/0000-0003-0540-7427

Discipline of Pharmacology, Division of Health and Applied Sciences, Faculty of Science, Prince of Songkla University, Songkhla, Thailand

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Rapeepun Chalongsuk

Department of Community Pharmacy, Faculty of Pharmacy, Silpakorn University, Sanam Chandra Palace Campus, Nakhon Pathom, Thailand

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Published Online:16 Mar 2022https://doi.org/10.2217/pme-2021-0111

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Abstract

Advanced therapy medicinal products (ATMPs) constitute therapeutic agents based on obtained cells, tissues or genes representing a novel treatment opportunity in medicine. In addition, ATMPs are administered into the cells or tissues of humans from the patient's own cells, donors, or genetically modified cells. Recently, the field of developing ATMPs has become a point of attention due to the clinical efficacy expected in defeating incurable diseases such as cancers and neurodegenerative disorders. Currently, there are two modes regarding the distribution of ATMPs. First, ATMPs that might be legally authorized for marketing. Second, the patients are able to access unapproved ATMPs through the hospital exemption (HE) or clinical practice program or through the compassionate use and expanded access program. The aim of this review is to discuss state-of-the-art knowledge on the regulation of ATMPs and provide regulatory recommendations.

Keywords:

Papers of special note have been highlighted as: • of interest

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Vol. 19, No. 3

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Received 19 August 2021

Accepted 18 January 2022

Published online 16 March 2022

Published in print May 2022

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Acknowledgments

The authors would like to thank the Faculty of Pharmacy, Silpakorn University, Thailand, for their assistance in editing the manuscript.

Financial & competing interests disclosure

The authors have no relevant affiliations or financial involvement with any organization or entity with a financial interest in or financial conflict with the subject matter or materials discussed in the manuscript. This includes employment, consultancies, honoraria, stock ownership or options, expert testimony, grants, or patents received or pending, or royalties.

No writing assistance was utilized in the production of this manuscript.

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State-of-the-art knowledge on the regulation of advanced therapy medicinal products

Abstract

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