Author + information
- Kevin Lee, MD,
- Sarah Min, MD,
- Marloe Prince, MD,
- Charles Glueck, MD,
- Ping Wang, PhD and
- Kenneth Lee
Lowering of LDL cholesterol (LDLC) has been revolutionized by PCSK9 inhibitors, Alirocumab (Praluent) and Evolocumab (Repatha). PCSK9 inhibitors have approved indications as adjunct to diet and maximally tolerated lipid lowering therapy (MTLLT) for patients with heterozygous familial hypercholesterolemia (HeFH), homozygous familial hypercholesterolemia, or clinical atherosclerotic cardiovascular disease (CVD) where LDLC lowering is insufficient despite MTLLT.
We have applied FDA approved and commercial insurance eligibility criteria for PCSK9 inhibitor use in 1090 patients serially referred to our Cholesterol Diagnosis and Treatment center (within the last 3 years), who, after ≥ 2 months of MTLLT, retained follow up LDLC ≥ 70mg/dl. We documented the percentage of patients with HeFH and/or CVD who met FDA insurance criteria for PCSK9 inhibitor therapy using LDLC goal-based guidelines.
We included 1090 consecutively referred patients with LDLC ≥ 70mg/dl after ≥ 2 months of MTLLT, and characterized them by FDA indications and commercial insurance eligibility for PCSK9 inhibitor use.
Of the 1090 patients with LDLC ≥ 70 mg/dl after ≥ 2 months of maximally tolerated MTLLT, 353 (32%) had HeFH and/or CVD events. Of these 353 patients, 140 (13% of the cohort of 1090) had HeFH and/or CVD, with LDLC > 100 mg/dl on MTLLT, meeting both FDA and commercial insurance criteria for PCSK9 inhibitor therapy. Fifty-one patients (5%) were statin intolerant only without HeFH or CVD events.
Of 1090 patients referred for diagnosis and treatment of high LDLC, with LDLC ≥ 70 mg/dl after ≥ 2 months on MTLLT, 140 (13%) had HeFH and/or CVD, with LDLC > 100 mg/dl, meeting both FDA and commercial insurance criteria for PCSK9 inhibitor therapy. Extrapolating from our referral cohort where 13% of hypercholesterolemic patients would be eligible by FDA-commercial insurance criteria for PCSK9 inhibitors, it is possible that at least 6 million Americans would be candidates for PCSK9 inhibitor therapy, where specialty-priced drugs would need to be used for treatment of a common public-health problem.