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Methods Mol Biol. 2017;1606:19-36. doi: 10.1007/978-1-4939-6990-6_2.

Innovations in Clinical Trial Design in the Era of Molecular Profiling.

Author information

1
Center for Applied Proteomics and Molecular Medicine, Institute for Advanced Biomedical Research, George Mason University, 10920 George Mason Circle, Manassas, VA, 20110, USA. jwulfkuh@gmu.edu.
2
Virginia Cancer Specialists, 8503 Arlington Blvd, Suite 400, Fairfax, VA, 22031, USA.
3
Department of Surgery, Inova Fairfax Hospital Cancer Center, 3300 Gallows Road, Falls Church, VA, 22042, USA.
4
Center for Applied Proteomics and Molecular Medicine, Institute for Advanced Biomedical Research, George Mason University, 10920 George Mason Circle, Manassas, VA, 20110, USA.

Abstract

Historically, cancer has been studied, and therapeutic agents have been evaluated based on organ site, clinical staging, and histology. The science of molecular profiling has expanded our knowledge of cancer at the cellular and molecular level such that numerous subtypes are being described based on biomarker expression and genetic mutations rather than traditional classifications of the disease. Drug development has experienced a concomitant revolution in response to this knowledge with many new targeted therapeutic agents becoming available, and this has necessitated an evolution in clinical trial design. The traditional, large phase II and phase III adjuvant trial models need to be replaced with smaller, shorter, and more focused trials. These trials need to be more efficient and adaptive in order to quickly assess the efficacy of new agents and develop new companion diagnostics. We are now seeing a substantial shift from the traditional multiphase trial model to an increase in phase II adjuvant and neoadjuvant trials in earlier-stage disease incorporating surrogate endpoints for long-term survival to assess efficacy of therapeutic agents in shorter time frames. New trial designs have emerged with capabilities to assess more efficiently multiple disease types, multiple molecular subtypes, and multiple agents simultaneously, and regulatory agencies have responded by outlining new pathways for accelerated drug approval that can help bring effective targeted therapeutic agents to the clinic more quickly for patients in need.

KEYWORDS:

Biomarkers; Clinical trial; Design; Endpoints; Neoadjuvant therapy; Tumor

PMID:
28501991
DOI:
10.1007/978-1-4939-6990-6_2
[Indexed for MEDLINE]

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