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Why advanced therapy medicinal products struggle in clinical translation: an in-depth analysis of developmental challenges in the EU
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DOI：10.3389/fmed.2026.1839531.png)
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Abstract
En 中文
Advanced Therapy Medicinal Products (ATMPs) can offer unprecedented therapeutic benefit for patients with limited treatment options. However; their clinical translation and early-stage clinical development remain highly complex; particularly for academic developers and small- and medium-sized enterprises (SMEs). This study presents a comprehensive analysis of the different types of challenges that ATMP developers face in initiating clinical trials; based on a scoping literature review; conducted according to the JBI methodology for scoping reviews and reported according to PRISMA-ScR. PubMed; Embase and Scopus were searched for peer-reviewed literature covering the last decade; focusing on publications discussing regulatory challenges for ATMP developers at the stage of clinical trial initiation. Our findings show that key challenges exist in four areas; comprising (a) preclinical evidence and product characteristics; (b) cell sourcing and manufacturing; (c) clinical trials and (d) regulatory landscape. Early engagement with regulators and the availability of up-to-date guidance documents emerged as key strategies to align developer efforts with regulatory expectations; while the review also identifies practical recommendations to support more efficient and harmonized clinical translation. In line with the objectives of JOIN4ATMP; these insights are valuable for developers and informative for regulators to foster more efficient and aligned ATMP development pathways.
Keywords:
scoping review
clinical translation
advanced therapy medicinal product (ATMP)
cell and gene therapy (CGT)
regulatory challenges
Journal
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Citations:
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