Access to Nonstatin Lipid-Lowering Therapies in Patients at High Risk of Atherosclerotic Cardiovascular Disease
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High-intensity statins are recommended for all patients with familial hypercholesterolemia (FH), and nonstatin lipid-lowering therapies (LLTs) are indicated when there is an inadequate response to statins.1,2 In the pre-PCSK9 inhibitor (PCSK9i) era, only ≈40% of FH patients achieved an low-density lipoprotein cholesterol (LDL-C) level <100.3 In part, on the basis of the need for additional therapeutic options in high-risk FH patients, PCSK9 inhibitors were approved for treatment of heterozygous and homozygous FH in 2015. Nevertheless, emerging anecdotal data suggest that access to nonstatin LLTs has been a challenge for FH patients, although this has not been systematically evaluated. The FOCUS study (FH Optimal Care of the US) was designed by The FH Foundation to assess current treatment patterns of FH patients, and allowed us to assess rejection rates of PCSK9 inhibitors in those with FH or atherosclerotic cardiovascular disease (ASCVD).
The FOCUS data set couples diagnostic information and pharmacy claims adjudication data from QuintilesIMS Inc. for >140 million unique individuals including diagnoses, procedures, laboratory tests, and prescriptions. Of these, 1.12 million individuals had a claim for either a PCSK9i or ezetimibe, forming the basis for our analyses (Figure). These data span the period March 1, 2012, to June 30, 2016, with the exception of pharmacy claim adjudication histories (August 1, 2014, to July 31, 2016) and laboratory data (January 1, 2012, to May 31, 2015). Patients were assigned to 2 cohorts (presumed FH or ASCVD) on the basis of medical histories, laboratory tests, therapies, diagnoses, procedures, and the value of their maximum and latest LDL-C. The final disposition …