Current landscape of clinical development and approval of advanced therapies

Carolina Iglesias-Lopez, Antonia Agustí, Antoni Vallano*, Merce Obach

*Corresponding author for this work

Research output: Contribution to journalReview articleResearchpeer-review

1 Citation (Scopus)


Advanced therapy medicinal products (ATMPs) are innovative therapies that mainly target orphan diseases and high unmet medical needs. The uncertainty about the product's benefit-risk balance at the time of approval, the limitations of nonclinical development, and the complex quality aspects of those highly individualized advanced therapies are playing a key role in the clinical development, approval, and post-marketing setting for these therapies. This article reviews the current landscape of clinical development of advanced therapies, its challenges, and some of the efforts several stakeholders are conducting to move forward within this field. Progressive iteration of the science, methodologically sound clinical developments, establishing new standards for ATMPs development with the aim to ensure consistency in clinical development, and the reproducibility of knowledge is required, not only to increase the evidence generation for approval but to set principles to achieve translational success in this field.

Original languageEnglish
Pages (from-to)606-618
Number of pages13
JournalMolecular Therapy - Methods and Clinical Development
Publication statusPublished - 10 Dec 2021


  • cell- and tissue-based therapy
  • clinical trials advanced therapies
  • drug approval
  • drug development
  • genetic therapy
  • jurisprudence
  • methods
  • research design


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