chenodiol — Blue Cross Blue Shield of Oklahoma

Initial criteria

• 1. 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 ULN AND B. Urine positive for bile alcohols AND C. Clinical findings consistent for CTX [e.g., xanthomas (lungs, tendons, bone or CNS), infantile-onset diarrhea, childhood-onset cataract(s), adult-onset progressive neurologic dysfunction (dementia, psychiatric disturbances, pyramidal and/or cerebellar signs, dystonia, atypical parkinsonism, peripheral neuropathy, seizures] OR B. The patient has another FDA labeled indication for the requested agent and route of administration AND 2. 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 for the requested agent OR B. There is support for using the requested agent for the patient’s age for the requested indication
• 2. The patient has had a baseline liver transaminase (ALT and AST) and total bilirubin level prior to initiating the requested agent
• 3. The prescriber is a specialist in the area of the patient’s diagnosis (e.g., gastroenterologist, geneticist, hepatologist, endocrinologist, neurologist), or has consulted with such specialist
• 4. The patient does NOT have any FDA labeled contraindications to the requested agent
• Alternate approval pathway: The member resides in Ohio AND 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 supported in compendia for the requested agent and route of administration OR 3. The prescriber submitted TWO peer-reviewed professional medical journal articles supporting the proposed use as generally safe and effective (acceptable non-oncology compendia: DrugDex 1,2A,2B; AHFS-DI supportive; oncology: NCCN 1 or 2A, AHFS-DI supportive; DrugDex 1,2A,2B; Clinical Pharmacology supportive; LexiDrugs A; or peer-reviewed literature)

Reauthorization criteria

• 1. The patient has been previously approved for the requested agent through the plan’s Prior Authorization process
• 2. The patient has had clinical benefit with the requested agent
• 3. 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
• 4. The prescriber is a specialist in the area of the patient’s diagnosis or has consulted with such specialist
• 5. The patient does NOT have any FDA labeled contraindications to the requested agent

Approval duration

12 months