Studies That Have Changedor Could Change Practice

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There are a number of studies that would seem appropriate for economic evaluation because of high costs, small benefit, large societal impact, or some combination of these (Table 11.7). Cost or cost-minimization analysis showed that positron emission tomography (PET) scans prevented 21% of futile thoracotomies for resectable small cell lung cancer26; one could argue that PET scans would be used anyway, but they appear to lower costs overall by preventing more expensive surgery. The nonplatinum combination of gemcitabine-paclitaxel was no better or less toxic than other regimens in a large randomized trial and cost 25% more.27 Our own analysis of inpatient palliative care units showed a 60% cost savings in a matched case-control set and prospective clinical-financial analysis.28 Many other studies were done, but in general, they would not be expected to have either a clinical or economic impact, so are not included.

Cost-effectiveness studies showed that some commonly used treatments were within the range of accepted cost-effectiveness ratios but some were not. Postmastectomy radiation in premenopausal women improved survival at a cost of $24,900/LY. Capecitabine/docetaxel in metastatic breast cancer, compared to docetaxel alone, improved survival by 3 months29 at a cost-effectiveness ratio30 of approximately $3,700/LY (Canadian), well within accepted standards of treatment. In this study, utility was not included but the magnitude of survival benefit would likely offset the negative effects of toxicity associated with capecitabine. Of note, the alternative strategies of sequential therapies were not tested, so no conclusion can be drawn. A more expensive initial strategy, autologous stem cell transplant for myeloma instead of melphalan and prednisone, had longer survival that offset the cost, so the incremental cost-effectiveness was acceptable. One recent study31 looked at cost-effectiveness through a spreadsheet-based model comparing three strategies for treating pain caused by cancer: guideline-based care, oncology-based care, and usual care. Treatment strategies included medications and procedure-based interventions. The effectiveness unit used was "additional patient relieved of cancer pain," rather than the typical additional year of life gained. Guideline-based care (GBC) was more effective at relieving cancer pain compared to oncology-based care (OBC) or usual care (UC): 80% versus 55% and 30%, respectively. The incremental cost-effectiveness for GBC compared to OBC was $452 per additional patient relieved of cancer pain, whereas the cost-effectiveness of OBC compared to UC was $601 per additional patient relieved of cancer pain.

Several cost-utility studies produced noteworthy results. Single fraction radiation for painful bone metastases instead of the usual six treatments was as effective and cost substantially less.32 Gordois and colleagues analyzed the impact of imatinib mesylate (gleevec) in the treatment of accelerated-phase CML and blast crisis CML compared to conventional chemotherapy and palliative care in hospital or at home.33 Imitinab mesylate improved QALYs in accelerated-phase CML of 2.09 years at $45,000/QALY and in blast crisis CML of approximately 0.58 months at $63,000/ QALY. An analysis of imitinab mesylate in chronic-phase CML, a much more common and costly treatment, compared with interferon and cytarabine, or bone marrow transplantation, has not yet been published in the English literature. Testing for HER-2 positivity by various methods, and treatment with trastuzamab if HER-2 positive, gave better survival by a few months; however, the cost was always over $100,000/LY.34 The more common and economically important question, at what cost does trastuzamab gain a year of life, especially in second- or third-line treatment, was not addressed.

Many important advances of the past years have not been studied, including the cost-effectiveness of adjuvant therapy for non-small cell lung cancer, dose-dense therapy of breast cancer, and the effectiveness and cost-effectiveness of most palliative chemotherapy regimens.

TABLE 11.7. Evidence-based new data.



No. of Year patients

Randomized Stage


Median follow-up

Outcome of interest


Cost or cost minimization

PET scan in resectable NSCLC Cisplatin-paclitaxel, P-gemcitabine, vs. PG Palliative care inpatient units

Cost-effectiveness Post-mastectomy radiation therapy in premenopausal women Capecitabine + docetaxol vs. docetaxol alone

Autologous stem cell transplant vs. chemotherapy in myeloma Pain control

Cost-utility Single vs. 6 fractions radiation for painful bone metastases Testing for HER-2 positivity






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