Clinical Outcomes Alone

This type of study is concerned solely with clinical efficacy such as improved response rates, survival, and symptom management; it ignores treatment costs entirely. This is the easiest method by which to make decisions for treatments that are clearly superior to any others available. A good example of a medication that fits into this category is imatinab mesylate (gleevec) in the treatment of chronic myelogenous leukemia (CML). It clearly has better response rates, time to disease progression, and toxicity profile than the traditional treatment of interferon and cytarabine and has become the standard of care as first-line treatment for CML.7 The decision to use imatinib mesylate is quite easy for physicians given the lack of any other similarly effective agents. But at $24,000/year (www.drugstore.com), an obvious economic consequence of this method is that some patients will not be able obtain a medication due to prohibitive costs, for instance, a 20% co-payment or lack of insurance.

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