Appeal type: Medical necessity
Decision: Upheld

Diagnosis:  Cardiac/Circulatory Problems/Other (Hyperlipidemia).
Treatment:  Pharmacy/Prescription Drugs (Nexletol).

The health plan denied the requested prescription drug.  The health plan's determination is upheld.

The patient is a man with a history of hypertension, hyperlipidemia, obstructive sleep apnea and morbid obesity with a body mass index (BMI) of 56.  The medical records provided indicate that he has had myalgias, fatigue and rash with statins; however, the agents tried, and dosages were not documented.  There is a copy of an intra-office communication indicating that the patient has heterozygous familial hypercholesterolemia (HeFH).  This is not documented in the visit notes; no low-density lipoprotein (LDL) cholesterol level was provided, and there is no documentation of how HeFH was diagnosed.  Specifically, there is no mention of Simon Broome criteria, the patient's Dutch Lipid Clinic Network score, a family history of HeFH, genetic testing for HeFH, or the presence or absence of tendon xanthomata or arcus senilis.  Medical necessity for bempedoic acid (Nexletol) is at issue.

The health plan's determination of medical necessity is upheld, in whole.

Based on the limited documentation provided, it cannot be determined that bempedoic acid (Nexletol) is medically necessary in this instance.

Bempedoic acid (Nexletol) is a relatively new nonstatin lipid lowering agent.  In a large randomized controlled trial involving 2,230 patients with atherosclerotic cardiovascular disease, bempedoic acid, when added to statin therapy, reduced LDL cholesterol levels by 16.5%.  It is Food and Drug Administration (FDA) approved for use as an adjunct to diet and maximally tolerated statin therapy for the treatment of adults with heterozygous familial hypercholesterolemia (HeFH) or established atherosclerotic cardiovascular disease, who require additional lowering of their LDL cholesterol level.  While the information provided contains an intra-office communication indicating that this patient has a diagnosis of HeFH, this is not documented in the visit notes; no LDL cholesterol level was provided, and there is no documentation of how HeFH was diagnosed.  Specifically, there is no mention of Simon Broome criteria, the patient's Dutch Lipid Clinic Network score, a family history of HeFH, genetic testing for HeFH, or the presence or absence of tendon xanthomata or arcus senilis.  Thus, this diagnosis cannot be confirmed based on the records available for review.  Moreover, since no LDL cholesterol level was provided, it cannot be determined that this patient is in need of a reduction in his level.  Finally, it cannot be determined that the patient is on maximally tolerated statin therapy.  If he has complete statin intolerance, then his maximally tolerated statin dose would be no statin.  However, while the records indicate that he had myalgias, fatigue and rash with statins in the past, the agents tried, and their dosages was not documented.  This information is needed in order to diagnose complete intolerance.  Per the recent Focused Update of the American College of Cardiology (ACC) Expert Consensus Decision Pathway on the Role of Non-Statin Therapies for LDL-Cholesterol Lowering in the Management of Atherosclerotic Cardiovascular Disease Risk, ''A systematic approach to evaluation of possible statin-related adverse effects is critically important to encourage adherence to evidence-based statin treatment....  The approach to statin intolerance should include discontinuation of statin therapy until resolution of symptoms and subsequent rechallenge to verify recurrence of muscle-related symptoms.  Whereas there is not a universally accepted definition of statin intolerance, most experts recommend that patients are documented to have unacceptable muscle-related symptoms that resolve with discontinuation of therapy and occur with rechallenge on at least 2 to 3 statins, preferably ones that use different metabolic pathways and have different lipophilicity, and 1 of which is prescribed at the lowest approved dose....  Non-statin therapies are not considered to be an alternative to evidence-based statin therapy unless statin intolerance has been systematically and rigorously evaluated and documented.''

In summary, based on the information provided, it cannot be determined that this patient meets accepted indications for treatment with bempedoic acid.  The diagnosis of HeFH cannot be confirmed, no LDL cholesterol level was provided, and it cannot be determined that he has complete statin intolerance and that his maximally tolerated statin dose would be no statin.