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Cost-Effectiveness Analyses of Targeted Oral Anti-Cancer Drugs: A Systematic Review

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Abstract

Background

Over the last 15 years, a paradigm shift in oncology has led to the approval of dozens of targeted oral anti-cancer medications (OAMs), which have become the standard of care for certain cancers. While more convenient for patients than infused drugs, the possibility of non-adherence and the frequently high costs of targeted OAMs have proven controversial.

Objective

Our objective was to perform the first comprehensive review of cost-effectiveness analyses (CEAs) of targeted OAMs.

Methods

A literature search in PubMed, The Cochrane Library, and the Health Technology Assessment (HTA) reports published by the National Institute for Health Research HTA Programme in the UK was performed, covering articles published in the 5 years prior to 30 September 2013. Our inclusion criteria were peer-reviewed English-language full-text original research articles with a primary focus on CEA related to targeted OAMs. We categorized these articles by treatment setting (i.e. cancer site/type, line of therapy, and treatment and comparator) and synthesized information from the articles into summary tables.

Results

We identified 41 CEAs covering nine of the 18 targeted OAMs approved by the US FDA as of December 2012. These medications were studied in seven cancers, most often as second-line therapy for advanced-stage patients. In over half of treatment settings where a targeted OAM was compared with treatment that was not a targeted OAM, targeted OAMs were considered cost effective. Limitations in interpreting these findings include the risk of bias due to author conflicts of interest, cross-country variation, and difficulties in generalizing clinical trial evidence to community practice.

Conclusions

Several types of cost-effectiveness studies remain under-represented in the literature on targeted OAMs, including those for follow-on indications approved after the initial indication for a drug and for off-label indications, head-to-head comparisons of targeted OAMs with other targeted OAMs and targeted intravenous therapies, and studies that adopt a perspective other than the payer’s. Keeping up with the increasing number of approved targeted OAMs will also prove an important challenge for economic evaluation.

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Acknowledgments

Drs. Shih and Smieliauskas are supported by a grant from the Agency for Healthcare Research and Quality (R01 HS018535), and Dr. Shih is also supported by The University of Chicago Cancer Research Foundation Women’s Board. Dr. Chien is supported by a grant from the Ministry of Health and Welfare (MOHW 103-TD-B-111-03). Dr. Geynisman acknowledges receiving honoraria for speaking from Pfizer. No other potential conflict of interest relevant to this article was reported.

Author Contribution

Drs. Smieliauskas and Chien drafted the manuscript, with the assistance of Dr. Geynisman on the paragraphs that provided the clinical background of cancer drugs. Drs. Smieliauskas, Chien, and Shen drafted the tables. Drs. Chien, Shih, and Geynisman determined search strategies, Dr. Chien conducted the literature search, and Drs. Chien, Shen, and Shih identified the articles included in the review. Dr. Shih supervised the overall progress and provided final revision, review, and comments.

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Authors and Affiliations

  1. Department of Health Studies, The University of Chicago, Chicago, IL, USA

    Fabrice Smieliauskas

  2. Department of Radiation Oncology, China Medical University Hospital, Taichung, Taiwan

    Chun-Ru Chien

  3. School of Medicine, College of Medicine, China Medical University, Taichung, Taiwan

    Chun-Ru Chien

  4. Department of Health Services Research, The University of Texas MD Anderson Cancer Center, Houston, TX, USA

    Chan Shen

  5. Department of Biostatistics, The University of Texas MD Anderson Cancer Center, Houston, TX, USA

    Chan Shen

  6. Department of Medical Oncology, Fox Chase Cancer Center, Temple Health, Philadelphia, PA, USA

    Daniel M. Geynisman

  7. Section of Hospital Medicine, Department of Medicine, Program in the Economics of Cancer, The University of Chicago, 5841 S Maryland Avenue, MC 5000, Chicago, IL, 60637, USA

    Ya-Chen Tina Shih

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  1. Fabrice Smieliauskas
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Correspondence to Ya-Chen Tina Shih.

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F. Smieliauskas and C.-R. Chien are joint first authors.

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Smieliauskas, F., Chien, CR., Shen, C. et al. Cost-Effectiveness Analyses of Targeted Oral Anti-Cancer Drugs: A Systematic Review. PharmacoEconomics 32, 651–680 (2014). https://doi.org/10.1007/s40273-014-0160-z

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