New clinical trial designs in the era of precision medicine
Garralda, Elena (Universitat Autònoma de Barcelona)
Dienstmann, Rodrigo (Universitat Autònoma de Barcelona)
Piris-Giménez, Alejandro (Universitat Autònoma de Barcelona)
Braña, Irene (Universitat Autònoma de Barcelona)
Rodon Ahnert, Jordi (Universitat Autònoma de Barcelona)
Tabernero, Josep (Universitat Autònoma de Barcelona)
Universitat Autònoma de Barcelona
Vall d'Hebron Institut d'Oncologia

Date:	2019
Abstract:	Cancer treatment has made significant strides towards the promise of personalized medicine. Recent scientific advances have shown that there are numerous genetic deregulations that are common in multiple cancer types, raising the possibility of developing drugs targeting those deregulations irrespective of the tumour type. Precision Cancer Medicine (PCM) was born out of accumulated evidence matching targeted agents with these tumour molecular deregulations. At the same time, the therapeutic armamentarium is rapidly increasing and the number of new drugs (including immune-oncology agents) entering drug development continues to rise. These factors, added to strong collaboration with regulatory agencies, which have approved novel agents based on data obtained from phase 1/2 trials, have led to unprecedented evolution in the design of early-stage clinical trials. Currently, we have seen rapid phase 1 dose-escalation trials followed by remarkably large expansion cohorts, and are witnessing the emergence of new trials, such as adaptive studies with basket and umbrella designs aimed at optimizing the biomarker-drug co-development process. Alongside the growing complexity of these clinical trials, new frameworks for stronger and faster collaboration between all stakeholders in drug development, including academic institutions and frameworks, clinicians, pharma companies and regulatory agencies, have been established. In this review article, we describe the main challenges and opportunities that these new trial designs may provide for a more efficient drug development process, which may ultimately help ensure that PCM becomes a reality for patients.
Rights:	Aquest document està subjecte a una llicència d'ús Creative Commons. Es permet la reproducció total o parcial, la distribució, la comunicació pública de l'obra i la creació d'obres derivades, fins i tot amb finalitats comercials, sempre i quan es reconegui l'autoria de l'obra original.
Language:	Anglès
Document:	Article ; recerca ; Versió publicada
Subject:	Adaptive designs ; Basket of Baskets ; Basket protocols ; Cancer Core Europe ; Umbrella protocols
Published in:	Molecular Oncology, Vol. 13 (february 2019) , p. 549-557, ISSN 1878-0261

DOI: 10.1002/1878-0261.12465
PMID: 30698321

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