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trofinetide oral soln 200 MG/ML — Blue Cross Blue Shield of Oklahoma

other FDA labeled indications

Initial criteria

  • ALL of the following:
  • 1. Diagnosis of classic/typical Rett syndrome (RTT)
  • 2. Disease-causing mutation in the MECP2 gene [chart notes required]
  • 3. If the patient has an FDA labeled indication, then ONE of the following:
  • a. Patient’s age is within FDA labeling for the requested indication, OR
  • b. There is support for using the requested agent for the patient’s age for the requested indication
  • 4. Patient’s weight ≥ 9 kg
  • 5. Prescriber has assessed baseline status of RTT symptoms (e.g., speech patterns, hand movements, gait, growth, muscle tone, seizures, breathing patterns, quality of sleep)
  • 6. Prescriber is a specialist in the area of the diagnosis (e.g., geneticist, neurologist, pediatrician) or has consulted with such a specialist
  • 7. Patient does NOT have any FDA labeled contraindications to the requested agent
  • OR The following conditions apply:
  • a. Member resides in Ohio AND the plan is Fully Insured or HIM Shop (SG)
  • b. Patient does NOT have any FDA labeled contraindications to the requested agent AND ONE of the following:
  • i. Patient has another FDA labeled indication for the requested agent and route, OR
  • ii. Patient has another indication supported in compendia (DrugDex level 1, 2A, 2B; AHFS-DI supportive narrative; NCCN 1 or 2A; Clinical Pharmacology supportive narrative; LexiDrugs evidence level A), OR
  • iii. Prescriber has submitted TWO articles from major peer-reviewed professional medical journals (e.g., JAMA, NEJM, Lancet) showing the use as safe and effective; case studies not acceptable

Reauthorization criteria

  • ALL of the following:
  • 1. Patient previously approved through plan’s Prior Authorization process
  • 2. Patient has had clinical benefit with the requested agent
  • 3. Prescriber is a specialist in the area of the diagnosis or has consulted with such a specialist
  • 4. Patient does NOT have any FDA labeled contraindications to the requested agent

Approval duration

BCBSIL: 12 months; others: initial 3 months, renewal 12 months