Current landscape of clinical development and approval of advanced therapies

Autor: Antoni Vallano, Carolina Iglesias-Lopez, Antònia Agustí, Mercè Obach
Přispěvatelé: Institut Català de la Salut, [Iglesias-Lopez C] Departament de Farmacologia, Terapèutica i Toxicologia, Universitat Autònoma de Barcelona, Bellaterra, Spain. [Agustí A] Departament de Farmacologia, Terapèutica i Toxicologia, Universitat Autònoma de Barcelona, Bellaterra, Spain. Servei de Farmacologia Clínica, Vall d’Hebron Hospital Universitari, Barcelona, Spain. [Vallano A] Departament de Farmacologia, Terapèutica i Toxicologia, Universitat Autònoma de Barcelona, Bellaterra, Spain. Medicines Department, Catalan Healthcare Service, Barcelona, Spain. [Obach M] Medicines Department, Catalan Healthcare Service, Barcelona, Spain, Vall d'Hebron Barcelona Hospital Campus
Jazyk: angličtina
Rok vydání: 2021
Předmět:
Therapeutics::Therapies
Investigational [ANALYTICAL
DIAGNOSTIC AND THERAPEUTIC TECHNIQUES
AND EQUIPMENT]
Genetic therapy
terapéutica::tratamientos en investigación [TÉCNICAS Y EQUIPOS ANALÍTICOS
DIAGNÓSTICOS Y TERAPÉUTICOS]
Cell- and tissue-based therapy
media_common.quotation_subject
Drug development
Review
Therapeutics::Biological Therapy::Cell- and Tissue-Based Therapy [ANALYTICAL
DIAGNOSTIC AND THERAPEUTIC TECHNIQUES
AND EQUIPMENT]
QH426-470
Orphan diseases
methods
cell- and tissue-based therapy
Clinical trials advanced therapies
drug approval
Drug approval
Methods
Genetics
Quality (business)
Molecular Biology
media_common
Medicaments - Desenvolupament
Jurisprudence
técnicas de investigación::desarrollo de medicamentos::autorización de medicamentos [TÉCNICAS Y EQUIPOS ANALÍTICOS
DIAGNÓSTICOS Y TERAPÉUTICOS]
jurisprudence
QH573-671
Teràpia cel·lular
Terapèutica
research design
drug development
Innovative Therapies
Research design
Molecular Medicine
Engineering ethics
Business
clinical trials advanced therapies
terapéutica::terapia biológica::tratamientos basados en células y tejidos [TÉCNICAS Y EQUIPOS ANALÍTICOS
DIAGNÓSTICOS Y TERAPÉUTICOS]
Cytology
genetic therapy
Investigative Techniques::Drug Development::Drug Approval [ANALYTICAL
DIAGNOSTIC AND THERAPEUTIC TECHNIQUES
AND EQUIPMENT]
Zdroj: Molecular Therapy: Methods & Clinical Development, Vol 23, Iss, Pp 606-618 (2021)
Scientia
Molecular Therapy. Methods & Clinical Development
Dipòsit Digital de Documents de la UAB
Universitat Autònoma de Barcelona
ISSN: 2329-0501
Popis: 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.
Graphical abstract
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.
Databáze: OpenAIRE
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