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Tumor Immunology and Cancer Vaccines pp 351–368Cite as

Clinical Trial Design and Regulatory Issues for Therapeutic Cancer Vaccines

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Part of the book series: Cancer Treatment and Research ((CTAR,volume 123))

Abstract

What is the appropriate end point for a therapeutic vaccine? Althoughwe have begun to see objective tumor responses in some patients, response rates of 10 to 20% are not generally adequate to use as end points in phase 2 trials. In order to see reliable differences in survival in a phase 2 trial, a very striking treatment effect in a wellcontrolled setting is probably required. As a practical matter, to show benefit, most phase 3 studies of therapeutic vaccines have used overall survival in advanced disease or time to recurrence either in the adjuvant setting or following complete resection with a high risk of recurrence. It is important to understand some of the reasons phase 3 vaccine trials have consistently failed to meet expectations following promising phase 2 trials. Phase 2 trial results may be strongly influenced by patient selection and retrospective analysis of subgroups, which may contribute to inadequate phase 3 trials even when based on positive phase 2 data. However, even with well designed trials, rational vaccine development beyond the empiric evaluation of individual products will require a deeper understanding of human tumor immunobiology.

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Authors and Affiliations

  1. Cancer Therapy Evaluation Program, Division of Cancer Treatment and Diagnosis, National Cancer Institute, National Institutes of Health, Bethesda, MD

    Jan Casadei PhD., Howard Z. Streicher M.D. & Jay J. Greenblatt PhD.

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  1. Vaccine Branch, National Cancer Institute, Bethesda Naval Hospital Bethesda, MD, USA

    Samir N. Khleif

  2. King Hussein Cancer Center, Amman, Jordan

    Samir N. Khleif

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Casadei, J., Streicher, H.Z., Greenblatt, J.J. (2005). Clinical Trial Design and Regulatory Issues for Therapeutic Cancer Vaccines. In: Khleif, S.N. (eds) Tumor Immunology and Cancer Vaccines. Cancer Treatment and Research, vol 123. Springer, Boston, MA. https://doi.org/10.1007/0-387-27545-2_15

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  • DOI: https://doi.org/10.1007/0-387-27545-2_15

  • Publisher Name: Springer, Boston, MA

  • Print ISBN: 978-1-4020-8119-4

  • Online ISBN: 978-0-387-27545-1

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