Ctexli — Blue Cross Blue Shield of Texas

Initial criteria

• ONE of the following:
• A. BOTH of the following:
• 1. ONE of the following:
• A. The patient has a diagnosis of cerebrotendinous xanthomatosis (CTX) as confirmed by ONE of the following:
• 1. Genetic testing confirming variants in the CYP27A1 gene OR
• 2. ALL of the following: A. Elevated plasma cholestanol ≥ 5 to 10 times upper limit of normal (ULN) AND B. Urine positive for bile alcohols AND C. Clinical findings consistent for CTX (e.g., xanthomas in lungs, tendons, bone or CNS; infantile-onset diarrhea; childhood-onset cataracts; adult-onset progressive neurologic dysfunction such as dementia, psychiatric disturbances, pyramidal and/or cerebellar signs, dystonia, atypical parkinsonism, peripheral neuropathy, and seizures) OR
• B. The patient has another FDA labeled indication for the requested agent and route of administration AND
• If the patient has an FDA labeled indication, then ONE of the following: A. The patient’s age is within FDA labeling for the requested indication OR B. There is clinical support for using the requested agent for the patient’s age for the requested indication AND
• The patient has had a baseline liver transaminase (ALT, AST) and total bilirubin level prior to initiating therapy AND
• The prescriber is a specialist in the area of the patient’s diagnosis (e.g., gastroenterologist, geneticist, hepatologist, endocrinologist, neurologist) or has consulted such a specialist AND
• The patient does NOT have any FDA labeled contraindications to the requested agent
• OR The member resides in Ohio AND the plan is Fully Insured or HIM Shop (SG) AND BOTH of the following:
• A. The patient does NOT have any FDA labeled contraindications to the requested agent AND
• B. ONE of the following: 1. The patient has another FDA labeled indication for the requested agent and route of administration OR 2. The patient has another indication that is supported in compendia for the requested agent and route of administration OR 3. The prescriber has submitted two peer-reviewed clinical articles supporting the proposed use as generally safe and effective (acceptable compendia or literature sources as listed)

Reauthorization criteria

• The patient has been previously approved for the requested agent through the plan’s Prior Authorization process AND
• The patient has had clinical benefit with the requested agent AND
• The patient is monitored for changes in liver transaminase (ALT and AST) and total bilirubin level AND BOTH of the following: A. Liver transaminase levels < 3 × ULN AND B. Total bilirubin level < 2 × ULN AND
• The prescriber is a specialist in the area of the patient’s diagnosis (e.g., gastroenterologist, geneticist, hepatologist, endocrinologist, neurologist) or has consulted such a specialist AND
• The patient does NOT have any FDA labeled contraindications to the requested agent

Approval duration

12 months