Current landscape of clinical development and approval of advanced therapies
Iglesias-Lopez, Carolina (Universitat Autònoma de Barcelona. Departament de Farmacologia, de Terapèutica i de Toxicologia)
Agustí Escasany, M. Antònia (Hospital Universitari Vall d'Hebron)
Vallano, Antoni (Servei Català de la Salut)
Obach, Mercè (Servei Català de la Salut)

Fecha:	2021
Resumen:	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. Most authorized ATMPs are based on small, open-label, uncontrolled, and single-arm pivotal trials using single and intermediate variables to evaluate outcomes. Health authorities are demanding methodologically sound clinical development to guide higher-quality evidence generation and decision-making. New standards for ATMP development are required to ensure consistency in clinical development.
Derechos:	Aquest document està subjecte a una llicència d'ús Creative Commons. Es permet la reproducció total o parcial, la distribució, i la comunicació pública de l'obra, sempre que no sigui amb finalitats comercials, i sempre que es reconegui l'autoria de l'obra original. No es permet la creació d'obres derivades.
Lengua:	Anglès
Documento:	Article ; recerca ; Versió publicada
Materia:	Drug development ; Drug approval ; Research design ; Methods ; Clinical trials advanced therapies ; Cell- and tissue-based therapy ; Genetic therapy ; Jurisprudence
Publicado en:	Molecular Therapy. Methods & Clinical Development, vol.23 (november 2021) p. 606-618, ISSN 2329-0501

DOI: 10.1016/j.omtm.2021.11.003
PMID: 34901306

13 p, 705.8 KB

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