A randomized trial to assess effectiveness and cost in clinical practice: rationale and design of the Cholesterol...

To compare the effectiveness and costs of two alternative approaches to the treatment of hypercholesterolemia, a prospective randomized trial is being undertaken at Southern California Kaiser Permanente, a large health maintenance organization. Six hundred and twelve patients with postdiet LDL cholesterol (LDL-C) levels in the range of 190-230 mg/dl (or 160-230 mg/dl for those with coronary heart disease or two or more coronary risk factors) were randomized to a stepped-care regimen (initial treatment with niacin followed by other agents if needed) or to initial use of lovastatin, an HMG-CoA reductase inhibitor. All patients are being followed for 1 year. The study seeks to approximate conditions of typical clinical practice: provider compliance with these plans of treatment is encouraged but not enforced and patients pay for medication as they customarily would. Principal outcomes of interest include the proportion of participants who achieve goal LDL-C at one year, the mean change in total cholesterol and LDL-C levels between baseline and the end of follow-up, and the costs of cholesterol-lowering therapy.