triheptanoin — Blue Cross Blue Shield of Texas

Initial criteria

• 1. The patient has ONE of the following: A. The patient has a diagnosis of long-chain fatty acid oxidation disorder (LCFAOD) AND ALL of the following: 1. The diagnosis has been confirmed by at least TWO of the following: A. Disease-specific elevations of acylcarnitines on a newborn blood spot or in plasma; B. Enzyme activity assay (in cultured fibroblasts or lymphocytes) demonstrating deficiency of an enzyme associated with LCFAODs; C. Genetic testing demonstrating pathogenic mutation in a gene associated with LCFAODs AND 2. The patient had symptomatic LCFAOD prior to therapy with the requested agent AND 3. The patient will not be concurrently using another medium chain triglyceride product AND 4. The patient will not be using the requested agent for more than 35% of the patient’s total prescribed daily caloric intake OR B. The patient has another FDA labeled indication for the requested agent and route of administration OR C. The patient has another indication that is supported in compendia for the requested agent and route of administration
• 2. The prescriber is a specialist in the area of the patient’s diagnosis (e.g., endocrinologist), or the prescriber has consulted with a specialist in the area of the patient’s diagnosis
• 3. The patient does NOT have any FDA labeled contraindications to the requested agent
• Additional approvals when ONE of the following is met: 1. Request is for a BCBS NM Fully Insured or NM HIM member AND ALL of the following: A. The patient does NOT have any FDA labeled contraindications to the requested agent AND B. The requested indication is a rare disease AND C. 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 agent and route of administration OR 2. ALL of the following: A. The member resides in Ohio AND B. The plan is Fully Insured or HIM Shop (SG) AND C. The patient does NOT have any FDA labeled contraindications to the requested agent AND D. 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 articles from major peer-reviewed professional medical journals (e.g., JAMA, NEJM, Lancet) supporting the proposed use as generally safe and effective (case studies not acceptable)

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. If the patient has LCFAOD, BOTH of the following: A. The patient will not be concurrently using another medium chain triglyceride product AND B. The patient will not be using the requested agent for more than 35% of total prescribed daily caloric intake
• 4. The prescriber is a specialist in the area of the patient’s diagnosis (e.g., endocrinologist) or has consulted with a specialist in the area of the patient’s diagnosis
• 5. The patient does NOT have any FDA labeled contraindications to the requested agent

Approval duration

12 months