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Journal of Clinical Oncology, Vol 25, No 6 (February 20), 2007: pp. 614-616 © 2007 American Society of Clinical Oncology. DOI: 10.1200/JCO.2006.10.1659
Economic Evaluation in the Journal of Clinical Oncology: Past, Present, and Future1 Departments of Clinical Epidemiology & Biostatistics and Oncology, McMaster University, Hamilton, Ontario, Canada
2 UCLA Schools of Public Health and Medicine, and Jonsson Comprehensive Cancer Center, Los Angeles, CA
3 University Pennsylvania and University of Pennsylvania Cancer Centre, Philadelphia, PA There is much excitement in the oncology community with the recent emergence of molecular targeted therapies, such as rituxumab, trastuzumab, and bevacizumab. Such therapy, however, comes at considerable cost, and we know that there are many more new agents in the pipeline. All countries, irrespective of their health care system, are struggling with how to cope with the existing and further increasing financial burden caused by cancer and its treatment. Oncologists find themselves in a difficult position. On the one hand, as prescribers they can be put in a position of gatekeepers; on the other, they are advocates for their patients. Given that these competing roles occur on a daily basis, it is important that the Journal of Clinical Oncology publish studies on economic evaluation for the readership. Since 2000, the JCO has published approximately five articles per year on economic-related issues. As editors, we have considered—and sometimes struggled with—a number of issues when assessing economic articles. We would briefly like to review some of these issues, to provide some guidance to researchers on the type of manuscript in which we are interested. First, the question should be important and relevant. From time-to-time, we receive an economic analysis for a treatment that has been used for a long time and is based on high quality evidence, such as radiation after breast-conservation surgery. The results of the economic analysis are used to justify the use of the therapy (ie, it's cheap so it is worth giving). This type of economic research is of low priority for the JCO because the treatment will be used anyways. In contrast, a research article examining resource allocation if such therapy was discontinued, thus freeing up resources for other treatments would be of interest. Second, the design of the study needs to be considered. The least rigorous in the design hierarchy is an article that merely describes the cost of an intervention without a comparator. This type of study is also of low priority for the JCO. As we move up the hierarchy we go from cost minimization, to cost effectiveness (C/E), and to cost utility analysis. These three designs involve a comparison between two interventions—usually something new compared with a standard that provides some form of benchmark. We sometimes receive articles where decision analysis is used to compare different therapeutic strategies. We would prefer to see data from a randomized trial that addresses the question, but there are situations where a randomized trial will not be done and a decision analysis using the best available evidence can be helpful.1 A decision analysis can also be used to describe in a very analytic fashion the comparison of alternative therapies where there is a trade-off between efficacy and toxicity and the answer is not clear cut.2 Although a decision analysis can help one think through a complex problem, it might be argued that an experienced clinician presents the information on benefits and risks of treatment to a patient in every day practice to elicit a patient preference. The third issue to consider is the quality of the data used in the economic analysis. The best outcome data come from randomized trials.3 Usually the publications of randomized trials provide data on efficacy (mortality and recurrence) and toxicity. However, economic data (costs and consequences as measured depending on the method used; for example, utilities) are seldom collected prospectively in clinical trials because of the added cost. We would encourage that researchers try and collect economic data prospectively in trials. However, often costs and consequences are obtained from other sources. Although this may be a necessity, it is not optimal. Assumptions are often made to convert toxicity and quality of life into outcome measures such as utilities. This process needs to be data driven, but is still a best guess.2,4,5 For example, the limitations of utility values based on the opinions of volunteers or experts who have actually not experienced the intervention need to be recognized. Economic evaluation that uses efficacy data from nonrandomized studies can be problematic because of the potential for bias. The reader needs to be aware that the economic analysis output, such as C/E ratio, is only as good as the data that is put into the analysis. If the JCO has already published the main results on efficacy of a trial and a good companion economic study is done, we would be interested in publishing it. We also receive what we call "What if?" articles. The data on efficacy and toxicity entered into the economic analysis are based on a randomized trial with relatively short follow-up.6 However, the base case analysis is done by letting the model (often a Markov model) run into the future 10 or 15 years, assuming the benefit of the new therapy continues into the future. If the base case analysis was performed with the actual short follow-up, the C/E ratio is often very high and not acceptable. If the initial, very promising results hold, then with longer follow-up the C/E ratio is favorable. However, if the data do not hold, then the early report of the model is spurious. We would ask that authors make it clear when their base case is based on short follow-up and state the corresponding C/E ratio for that data. They can then present the result when they let the model run into the future. This will be part of the sensitivity analyses that can present other scenarios as well.5 The fourth issue is the threshold value. Typically, an economic article on C/E or cost utility analysis reports an incremental C/E ratio and then justifies that the therapy is C/E because the ratio is less than a threshold number (for example, $50,000 and/or comparable to other treatments commonly in use). There is a major flaw in this assumption.7 It assumes that the pot of money available to fund cancer treatment is unlimited. This clearly is not the case. In order to make room for this new therapy from available resources it should displace something of less value. Even if new resources are available presumably there are other new treatments that will compete for funding. We would ask authors to discuss the implications of funding a therapy based on their analysis. It is not good enough to provide a C/E ratio in a league table. We wonder whether threshold values ever influence whether payers actually provide additional funding for a new treatment. Finally, the JCO receives economic studies from the manufacturer of a drug or a contract research organization engaged by the sponsor. The reader has to be concerned about the motivation for the submission. We would prefer that economic articles be submitted by academics and any support of the research by the sponsor must be declared. In this issue of the JCO, we are pleased to publish three original economic research articles and accompanying editorials, through which we hope to give the reader and researchers an idea of the type of economic research articles we would like to see submitted. The two articles on the C/E of trastuzumab in early breast cancer sparked much discussion at the monthly editors' conference call. When these articles were submitted, trastuzumab had already been funded in most countries and was being used widely. It was unclear how an economic evaluation analysis would have any impact. Even though the reports in the JCO by Kurian et al8 and Liberato et al9 show similar results (which is reassuring), they were based on different assumptions and were conducted on different continents. However, these articles provided an opportunity for Hillner and Smith10 to teach us the importance of assumptions in the model on the benefit of adding trastuzumab to chemotherapy, and the sensitivity of the C/E ratio to decreases in magnitude and duration of benefit. They also provide a clarion call that society cannot sustain the current mode of adding new expensive therapies without foregoing other programs in the envelope of health care expenditures. The article by Woo et al11 is the first in the JCO that deals with resource allocation (ie, how to maximize the delivery of a screening program within a fixed budget). They describe different combinations of cancer screening programs in Hong Kong and how they can be best delivered within different funding envelopes. In the accompanying editorial, Schecter12 discusses the weakness of a league table threshold C/E ratio. He reminds us of the sobering principle that guides economic theory, "economists define costs in terms of lost opportunities: the opportunity cost of choosing A is the value of the next most valuable alternative, B, which must be foregone to have A." Based on these considerations, we are interested in articles which deal with resource allocation. In conclusion, we recognize that economic analysis is complex and often difficult to do well. However, it is important given the challenges that our health care systems face. We have recognized the importance of this topic by having an entire special issue devoted to economics in cancer in early 2007. We encourage submission of economic articles to the JCO. In Table 1 we describe criteria we would like to see satisfied as a guide to authors.
AUTHORS' DISCLOSURES OF POTENTIAL CONFLICTS OF INTEREST The authors indicated no potential conflicts of interest. AUTHOR CONTRIBUTIONS Manuscript writing: Mark Levine Final approval of manuscript: Patricia A. Ganz, Daniel G. Haller
REFERENCES
1. Hershman D, Sundararajan V, Jacobson JS, et al: Outcomes of tamoxifen chemoprevention for breast cancer in very high-risk women: A cost-effectiveness analysis. J Clin Oncol 20:9-16, 2002 2. Lee JJ, Bekele BN, Zhou X, et al: Decision analysis for prophylactic cranial irradiation for patients with small-cell lung cancer. J Clin Oncol 24:3597-3603, 2006 3. van den Brink M, van den Hout WB, Stiggelbout AM, et al: Cost-utility analysis of preoperative radiotherapy in patients with rectal cancer undergoing total mesorectal excision: A study of the Dutch Colorectal Cancer Group. J Clin Oncol 22:244-253, 2004 4. Mehta MP: Models support prophylactic cranial irradiation. J Clin Oncol 24:3524-3526, 2006 5. Simunovic M, Gafni A, Levine M: Economics of preoperative radiotherapy with total mesorectal excision: What can we learn from the Dutch experience? J Clin Oncol 22:217-219, 2004 6. Punglia RS, Kuntz KM, Winer EP, et al: Optimizing adjuvant endocrine therapy in postmenopausal women with early-stage breast cancer: A decision analysis. J Clin Oncol 23:5178-5187, 2005 7. Birch S, Gafni A: Information created to evade reality (ICER). Pharmacoeconomics 24:1121-1131, 2006[CrossRef][Medline] 8. Kurian AW, Thompson RN, Gaw AF, et al: Cost effectiveness analysis of adjuvant trastuzumab regimens in early HER-2/neu-positive breast cancer. J Clin Oncol 25:634-641, 2007 9. Liberato NL, Marchetti M, Barosi G: Cost effectiveness of adjuvant trastuzumab in HER2-positive breast cancer. J Clin Oncol 25:625-633, 2007 10. Hillner BE, Smith TJ: Do the large benefits justify the large costs of adjuvant breast cancer trastuzumab? J Clin Oncol 25:611-613, 2007 11. Woo PPS, Kim JJ, Leung GM: What is the most cost effective population-based cancer screening package for Chinese women? J Clin Oncol 25:617-624, 2007 12. Schecter CB: Cost effectiveness in context. J Clin Oncol 25:609-610, 2007
Related Correspondence
This article has been cited by other articles:
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Copyright © 2007 by the American Society of Clinical Oncology, Online ISSN: 1527-7755. Print ISSN: 0732-183X
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