1. Transitioning From Preclinical Evidence to Advanced Therapy Medicinal Product: A Spanish Experience
- Author
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Paloma Gastelurrutia, Cristina Prat-Vidal, Joaquim Vives, Ruth Coll, Antoni Bayes-Genis, Carolina Gálvez-Montón, Institut Català de la Salut, [Gastelurrutia P, Prat-Vidal C] Institut d'Investigació Biomèdica de Bellvitge-IDIBELL, L'Hospitalet de Llobregat, Spain. Insuficiencia Cardíaca y Regeneración Cardíaca Research Program, Fundació Institut d'Investigació en Ciències de la Salut Germans Trias i Pujol (IGTP), Badalona, Spain. Centro de Investigación Biomédica en Red Cardiovascular, Instituto de Salud Carlos III, Madrid, Spain. [Vives J] Servei de Teràpia Cel·lular, Banc de Sang i Teixits, Barcelona, Spain. Grup d’Enginyeria tissular musculoesquelètica, Vall d'Hebron Institut de Recerca (VHIR), Barcelona, Spain. Universitat Autònoma de Barcelona, Bellaterra, Spain. Departament de Medicina, Universitat Autònoma de Barcelona, Bellaterra, Spain. [Coll R] Servei de Teràpia Cel·lular, Banc de Sang i Teixits, Barcelona, Spain. [Bayes-Genis A] Insuficiencia Cardíaca y Regeneración Cardíaca Research Program, Fundació Institut d'Investigació en Ciències de la Salut Germans Trias i Pujol (IGTP), Badalona, Spain. Centro de Investigación Biomédica en Red Cardiovascular, Instituto de Salud Carlos III, Madrid, Spain. Hospital Universitari Germans Trias i Pujol, Badalona, Spain. Departament de Medicina, Universitat Autònoma de Barcelona, Bellaterra, Spain. [Gálvez-Montón C] Insuficiencia Cardíaca y Regeneración Cardíaca Research Program, Fundació Institut d'Investigació en Ciències de la Salut Germans Trias i Pujol (IGTP), Badalona, Spain. Centro de Investigación Biomédica en Red Cardiovascular, Instituto de Salud Carlos III, Madrid, Spain, and Vall d'Hebron Barcelona Hospital Campus
- Subjects
Therapeutics::Therapies, Investigational [ANALYTICAL, DIAGNOSTIC AND THERAPEUTIC TECHNIQUES, AND EQUIPMENT] ,0301 basic medicine ,regulatory agency ,lcsh:Diseases of the circulatory (Cardiovascular) system ,medicine.medical_specialty ,terapéutica::tratamientos en investigación [TÉCNICAS Y EQUIPOS ANALÍTICOS, DIAGNÓSTICOS Y TERAPÉUTICOS] ,media_common.quotation_subject ,Cellular therapy ,Investigació mèdica ,Cardiovascular Medicine ,030204 cardiovascular system & hematology ,03 medical and health sciences ,chemistry.chemical_compound ,0302 clinical medicine ,Policy and Practice Reviews ,Medicine research ,medicine ,Quality (business) ,Medical physics ,Product (category theory) ,media_common ,Medicaments - Desenvolupament ,investigational medicinal product dossier (IMPD) ,localizaciones geográficas::Europa (continente)::España [DENOMINACIONES GEOGRÁFICAS] ,translational (regulation) ,Medicina basada en l'evidència ,business.industry ,Teràpia cel·lular ,Medicina - Investigació - Espanya ,Geographic Locations::Europe::Spain [GEOGRAPHICALS] ,030104 developmental biology ,investigational medicinal product ,chemistry ,Natural Science Disciplines::Science::Research::Biomedical Research::Translational Medical Research [DISCIPLINES AND OCCUPATIONS] ,lcsh:RC666-701 ,New product development ,disciplinas de las ciencias naturales::ciencia::investigación::investigación biomédica::investigación médica traslacional [DISCIPLINAS Y OCUPACIONES] ,advanced therapy medicinal product ,Regulatory agency ,ATMP ,Cardiology and Cardiovascular Medicine ,business - Abstract
Medicament de teràpia avançada; Medicament en investigació; Translacional (regulació) Medicamento de terapia avanzada; Medicamento en investigación; Traslacional (regulación) Advanced therapy medicinal product; Investigational medicinal product; Translational (regulation) A systematic and ordered product development program, in compliance with current quality and regulatory standards, increases the likelihood of yielding a successful advanced therapy medicinal product (ATMP) for clinical use as safe and effective therapy. As this is a novel field, little accurate information is available regarding the steps to be followed, and the information to be produced to support the development and use of an ATMP. Notably, successful clinical translation can be somewhat cumbersome for academic researchers. In this article, we have provided a summary of the available information, supported by our experience in Spain throughout the development of an ATMP for myocardial infarction, from the pre-clinical stage to phase I clinical trial approval. This work was supported by the Spanish Cell Therapy Network (TerCel) [RD16/0011/0006 and RD16/0011/0028]; and CIBER Cardiovascular [CB16/11/00403] projects, as a part of the National R&D&I Plan, and was co-funded by the ISCIII-Sub-Directorate General for Research Assessment and Promotion and the European Regional Development Fund (ERDF); the Government of Catalonia: Agency for Management of University and Research Grants (AGAUR) [2017-SGR-483 and 2017-SGR-719], PERIS Program (Health Department) [SLT002/16/00234 and SLT002/16/00209] and CERCA Program; La Caixa Banking Foundation; grants from the Spanish Ministry of Science, Innovation and Universities (MICINN) [SAF2017-84324-C2-1-R]; Institute of Health Carlos III (ISCIII) [PI18/00256, PIC18/00014, ICI19/00039, and PI19/01788], and cofunded by European Union (ERDF/ESF) - A way to build Europe. This work has also been funded by the Sociedad Española de Cardiología and by the Call for Expression of Interest (EoI) for Collaborative Projects on Regenerative Medicine 2019 of Center for Regenerative Medicine of Barcelona (CMR[B]). The funders of the study had no role in the study design, data collection, data analysis, data interpretation, or writing of the report.
- Published
- 2021