Author + information
- Marloe Prince,
- Charles Glueck,
- Parth Shah,
- Kevin Lee,
- Vybhav Jetty and
- Ping Wang
Lowering of LDL cholesterol (LDLC) has been revolutionized by the recent release of the PCSK9 inhibitors, Alirocumab (Praluent) and Evolocumab (Repatha). PCSK9 inhibitors have approved indications as an adjunct to diet and maximally tolerated statin therapy for patients with heterozygous familial hypercholesterolemia (HeFH), homozygous familial hypercholesterolemia (HoFH), or clinical atherosclerotic cardiovascular disease (CVD)where LDLC lowering is insufficient despite maximal tolerated cholesterol lowering therapy.
We have applied FDA approved and commercial insurance eligibility criteria for PCSK9 inhibitor use in 734 patients serially referred to our Cholesterol Diagnosis and Treatment center (within 3 years) and receiving ≥ 2 months maximally tolerated LDLC lowering diet-drug therapy with follow up LDLC ≥70 mg/dl. We obtained estimates of the nature of cohorts with high LDLC who meet FDA and commercial insurance eligibility for PCSK9 inhibitor treatment using LDLC goal-based guidelines.
We included 734 patients consecutively referred patients with LDLC ≥ 70 mg/dl after ≥2 months of maximally tolerated LDLC lowering therapy, and characterized them by FDA indications and commercial insurance eligibility for PCSK9 inhibitor use.
Of 734 patients with LDLC ≥ 70 mg/dl after ≥2 months maximally tolerated LDLC lowering therapy, 220 (30%) had HeFH and/or CVD events with LDLC >100 mg/dl, meeting both FDA and commercial insurance criteria for PCSK9 inhibitor therapy. Sixty-six patients (9%) were statin intolerant only without HeFH or CVD events.
Of 734 patients referred for diagnosis and treatment of high LDLC, with LDLC ≥ 70 mg/dl after ≥2 months on maximally tolerated cholesterol lowering therapy, 220 (30%) had HeFH and/or CVD with LDLC >100 mg/dl, meeting both FDA and commercial insurance criteria for PCSK9 inhibitor therapy. If high LDLC treatment cohorts include up to 30% of eligible HeFH-CVD patients, then specialty pharmaceutical pricing models ($14,000-14,600/year for PCSK9 inhibitors) previously reserved for drugs which benefitted limited patient populations, will collide with prospective treatment cohorts in the tens of millions of patients. Whether the health care savings arising from the anticipated reduction of CVD events by PCSK9 inhibitors justify the extraordinary costs of broad population use of these agents remains to be determined.