alpelisib (PROS) — Blue Cross Blue Shield of Oklahoma

Initial criteria

• Patient has a diagnosis of PIK3CA-Related Overgrowth Spectrum (PROS) confirmed by ALL of the following:
• • Presence of somatic PIK3CA mutation [medical records required]
• • Congenital or early childhood onset
• • Overgrowth is sporadic and mosaic
• • ONE of the following:
• 1. Patient has at least TWO of the following features: Overgrowth; Vascular malformations; Epidermal nevus
• OR 2. Patient has at least ONE of the following features: Large isolated lymphatic malformations; Isolated macrodactyly OR overgrown splayed feet/hands, overgrown limbs; Truncal adipose overgrowth; Hemimegalencephaly (bilateral)/dysplastic megalencephaly/focal cortical dysplasia; Epidermal nevus; Seborrheic keratoses; Benign lichenoid keratoses
• AND Patient has severe manifestations of PROS that require systemic therapy
• AND If the patient has an FDA labeled indication, then ONE of the following applies: Patient’s age is within FDA labeling for the requested indication; OR There is support for using the requested agent for the patient’s age for the requested indication
• AND Prescriber is a specialist experienced in PROS or has consulted with one
• AND Patient does NOT have any FDA labeled contraindications to the requested agent
• For members residing in Ohio with Fully Insured or HIM Shop (SG) plans, approval also requires ALL of the following:
• • Patient does NOT have any FDA labeled contraindications
• • ONE of the following applies: Patient has another FDA labeled indication; OR Indication is supported in compendia for agent and route; OR Prescriber submits TWO peer-reviewed journal articles (e.g., JAMA, NEJM, Lancet) supporting proposed use as generally safe and effective (case studies not acceptable)
• Accepted compendia: DrugDex level 1, 2A, or 2B; AHFS-DI (supportive text required); NCCN 1 or 2A; Clinical Pharmacology (supportive); LexiDrugs evidence level A

Reauthorization criteria

• Patient has been previously approved for the requested agent through plan’s Prior Authorization process
• Patient has had clinical benefit with the requested agent
• Patient has NOT had disease progression (e.g., increased lesion number or volume) with the requested agent [medical records required]
• Prescriber is a specialist experienced in PROS or has consulted with one
• Patient does NOT have any FDA labeled contraindications to the requested agent

Approval duration

6–12 months