The aim of this project, which was commissioned by NICE through the NIHR HTA Programme as part of the NICE’s Single Technology Appraisal process, was to review the evidence for the clinical and cost-effectiveness of Ezetimibe for treating primary heterozygous-familial and non-familial hypercholesterolaemia. The clinical evidence submitted by the company (Merck Sharp & Dohme Ltd) responsible for manufacturing Ezetimibe (Ezetrol) consisted of 25 RCTs of either Ezetimibe versus placebo, or Ezetimibe plus a statin versus a matching statin dose. The Aberdeen HTA Group acted as the Evidence Review Group (ERG) for the project and provided an independent critique of the clinical and cost-effectiveness evidence submitted by the company.

After taking into consideration the company submission and the findings of the ERG’s critique, NICE released clinical guidance (TA385, February 2016). Ezetimibe monotherapy is currently recommended as an option for treating primary (heterozygous familial or non familial) hypercholesterolaemia in adults who cannot tolerate statin therapy or for whom initial statin therapy is contraindicated. In adults who have started statin therapy, Ezetimibe is currently recommended when (i) serum total or low-density lipoprotein cholesterol concentration is not appropriately controlled either after appropriate dose titration of initial statin therapy or because dose titration is limited by intolerance to the initial statin therapy, and (ii) a change from initial statin therapy to an alternative statin is being considered.


Miriam Brazzelli:




Scotland, G, Javanbakht, M, Scott, N, Cruickshank, M, Sharma, P, Fraser, C, Simpson, W, Brazzelli, M. Ezetimibe for treating primary (heterozygous-familial and non-familial) hypercholesterolaemia. . Evidence Review Group report in support of the NICE STA Programme. University of Aberdeen: Health Services Research Unit; 2015. URL: