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Regulation of immunotherapeutic products for cancer and FDA’s role in product development and clinical evaluation

Ramjay S Vatsan1, Peter F Bross1, Ke Liu1, Marc Theoret2, Angelo R De Claro2, Jinhua Lu1, Whitney Helms2, Brian Niland1, Syed R Husain1 and Raj K Puri1*

Author Affiliations

1 Office of Cellular, Tissue, and Gene Therapies (OCTGT), Center for Biologics Evaluation and Research (CBER), Rockville, MD, USA

2 Office of Hematology and Oncology Products (OHOP), Center for Drug Evaluation and Research (CDER), Food and Drug Administration (FDA), Rockville, MD, USA

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Journal for ImmunoTherapy of Cancer 2013, 1:5  doi:10.1186/2051-1426-1-5

Published: 29 May 2013


Immunotherapeutics include drugs and biologics that render therapeutic benefit by harnessing the power of the immune system. The promise of immune-mediated therapies is target specificity with a consequent reduction in off-target side effects. Recent scientific advances have led to clinical trials of both active and passive immunotherapeutic products that have the potential to convert life-ending diseases into chronic but manageable conditions. Clinical trials investigating immunotherapeutics are ongoing with some trials at advanced stages of development. However, as with many products involving novel mechanisms of action, major regulatory and scientific issues arising with clinical use of immunotherapeutic products remain to be addressed. In this review, we address issues related to different immunotherapeutics and provide recommendations for the characterization and evaluation of these products during various stages of product and clinical development.