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Real-World Evidence: A Comparison of the Australian Herceptin Program and Clinical Trials of Trastuzumab for HER2-Positive Metastatic Breast Cancer


  • Bonny Parkinson

    (Macquarie University
    University of Technology Sydney)

  • Rosalie Viney

    (University of Technology Sydney)

  • Marion Haas

    (University of Technology Sydney)

  • Stephen Goodall

    (University of Technology Sydney)

  • Preeyaporn Srasuebkul

    (University of New South Wales)

  • Sallie-Anne Pearson

    (University of New South Wales
    The University of Sydney)


Introduction Estimating the real-world cost-effectiveness of a new drug relies on understanding the differences between clinical trial data (pre-reimbursement) and clinical practice (post-reimbursement). This is important for decision makers when reviewing reimbursement decisions, prices, and considering other drugs for the same condition. Differences can arise from differences in patient characteristics, but also from the availability of new evidence and evolving treatment practices. This paper examines these issues using a case study. Methods In 2001, the Australian Government funded trastuzumab for the treatment of HER2+ metastatic breast cancer through the Herceptin Program. The administrative arrangements of the Program resulted in rich observational data that captured information about patients treated with trastuzumab between 2001 and 2010 (n = 3830). The dataset included patient characteristics, dispensed medicines, medical service use and date of death. Results Compared to participants in the clinical trials, patients were older, received more prior chemotherapies and a broader range of co-administered chemotherapies. Treatment practices differed from the clinical trials, but also changed over time. For example, in situ hybridization testing, rather than immunohistochemistry testing, and a three weekly administration schedule, rather than one weekly, were increasingly used. Compared to the clinical trials, patients administered trastuzumab with a concomitant chemotherapy generally experienced longer overall survival (151.3 weeks, 95 % CI: 142.6, 163.4), while those who received trastuzumab as a monotherapy experienced shorter overall survival (94.4 weeks, 95%CI: 86.4, 102.9). These findings may be due to a differing relative treatment effect in clinical practice, but may also be due to a range of other factors. Conclusion This analysis demonstrates the challenges for decision makers that arise because new evidence and evolving treatment practices create a gap between clinical trial data and real-world clinical practice and outcomes.

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  • Bonny Parkinson & Rosalie Viney & Marion Haas & Stephen Goodall & Preeyaporn Srasuebkul & Sallie-Anne Pearson, 2016. "Real-World Evidence: A Comparison of the Australian Herceptin Program and Clinical Trials of Trastuzumab for HER2-Positive Metastatic Breast Cancer," PharmacoEconomics, Springer, vol. 34(10), pages 1039-1050, October.
  • Handle: RePEc:spr:pharme:v:34:y:2016:i:10:d:10.1007_s40273-016-0411-2
    DOI: 10.1007/s40273-016-0411-2

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    References listed on IDEAS

    1. Carlson, Josh J. & Sullivan, Sean D. & Garrison, Louis P. & Neumann, Peter J. & Veenstra, David L., 2010. "Linking payment to health outcomes: A taxonomy and examination of performance-based reimbursement schemes between healthcare payers and manufacturers," Health Policy, Elsevier, vol. 96(3), pages 179-190, August.
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    1. Peter Ghijben & Dennis Petrie & Silva Zavarsek & Gang Chen & Emily Lancsar, 2021. "Healthcare Funding Decisions and Real-World Benefits: Reducing Bias by Matching Untreated Patients," PharmacoEconomics, Springer, vol. 39(7), pages 741-756, July.

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