Clinical characteristics and prevalence of cardiovascular disease risk factors in subjects screened for familial hypercholesterolemia – A cross sectional study in The Netherlands

Limitations of traditional health technology assessment methods and implications for the evaluation of novel therapies

“…Among patients with an elevated baseline risk of morbidity and mortality the same relative treatment benefit translates to a greater reduction in absolute risk, implying a greater costs-effectiveness in the high-risk patient population than that in the lower-risk or general patient population. Examples include recommendations for proprotein convertase subtilisin/kexin type 9 inhibitors (PCSK9is) in patients with familial hypercholesterolemia, or evidence of clinical atherosclerotic cardiovascular disease (ASCVD) [5][6][7][8][9] , biologic disease modifying anti-rheumatic drugs (DMARDs) for RA patients with moderate-or high-activity disease who have inadequate response to conventional DMARD (cDMARD) therapy 10 , and newer therapies when metformin monotherapy fails to provide adequate glycemic control to patients with type 2 diabetes after 3 months 11,12 . One recent HTA of biologic DMARDs for RA did not properly account for the relationship between HAQ scores and mortality risk, or account for patient heterogeneity 13,14 .…”