Using Cost-Effectiveness Analysis To Target Cholesterol Reduction

  1. Alan M. Garber, MD, PhD
  1. Veterans Affairs Palo Alto Health Care System; Stanford University; Palo Alto, CA (Garber)

    The introduction of 3-hydroxy-3-methylglutaryl coenzyme A reductase inhibitors (statins) in the United States coincided with the massive “know your number” cholesterol awareness campaign. For both physicians and patients, these drugs offered a powerful solution to a problem that was being deeply impressed on the public consciousness. Besides dramatically decreasing levels of low-density lipoprotein (LDL) cholesterol, statins were more convenient and better tolerated than older therapies, such as bile acid–binding resins and niacin. Their popularity was immediate, and they soon accounted for billions of dollars in revenues. Atorvastatin, for example, surpassed $1 billion in sales during the year after its introduction, even though it was approved for use in the United States many years after other statins had become established therapy.

    Although statins represented a breakthrough in lipid management, questions about their role emerged. The importance of LDL cholesterol reduction in high-risk populations had already been widely accepted when the first clinical trials reported that these drugs were effective for secondary prevention of coronary heart disease (1) and for reduction of the risk for coronary events in high-risk primary prevention populations (2). More randomized trials were to come, yet gaps in the clinical evidence supporting widespread use of statins persisted. Notably absent from most clinical trials was adequate representation of elderly patients, in whom coronary disease is highly prevalent. Furthermore, the role of statins in treating patients at low short-term risk for coronary heart disease was controversial. With little disease to prevent, the benefits of risk reduction were necessarily limited and could be negated by even infrequent long-term adverse effects of treatment (3, 4). For the physician and the patient, the question was not whether statins were valuable overall, but whether and how they should be used in populations excluded …

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