CurrentMass General Brigham Health PlanPolicy N/A

Daybue (trofinetide) coverage for Rett syndrome

Coverage, prior authorization, and quantity/benefit constraints for Daybue (trofinetide) under Mass General Brigham Health Plan pharmacy benefit (specialty drug). Applies to commercial/exchange and MassHealth UPPL plan members.

Policy Summary

PayerMass General Brigham Health Plan

PolicyDaybue (trofinetide) coverage for Rett syndrome

Policy CodePolicy N/A

Change TypeNo material changes

Effective DateSep 1, 2023

Next Review DateN/A

Key ActionObtain prior authorization and submit medical records documenting MECP2 mutation, clinical manifestations of Rett syndrome, and evidence of benefit for continuation; ensure dispensing through a contracted specialty pharmacy.

SourceLink

POLICY UPDATE CHANGES

No material clinical or coverage changes in this revision.

12Initial and reauthorization approval duration

>=2Minimum patient age for coverage (years)

SpecialtyDispensing designation

Authorization Criteria for Daybue (trofinetide)

Initial Authorization — New members not currently receiving Daybue

Trek Health ingests and normalizes Transparency in Coverage data and payer policy updates to give provider organizations a clear view of how commercial reimbursement behaves across markets, payers, and services. Our platform transforms raw payer disclosures into structured intelligence that supports contract evaluation, payer negotiations, and service line strategy. By combining market benchmarks with ongoing policy visibility, Trek helps teams identify variability, risk, and opportunity in commercial reimbursement. The result is faster insight, stronger negotiating positions, and more informed financial decisions.

Continuation of Therapy / Reauthorization

Authorization (12 months) may be granted for continued treatment when ALL of the following are met:

ALL of the following

Member is currently receiving Daybue
Policy allows continuation for members who are currently receiving treatment (excludes product obtained as samples or via manufacturer patient assistance programs)
Member is experiencing benefit from therapy (e.g., stabilization or improvement in repetitive movements, mood dysfunction/disruptive behavior, vocalization, ambulation)

Exceptions / Transition-in Therapy

Authorization may be granted for members new to Mass General Brigham Health Plan who are currently receiving treatment with Daybue, except when medication was obtained as samples or through manufacturer patient assistance programs.

ALL of the following

Member is new to the plan and currently receiving Daybue
Daybue was NOT obtained as samples or via manufacturer patient assistance programs

Authorization, Documentation, and Dispensing Requirements

Prior Authorization

Prior authorization required

Prior authorization is required for Daybue (trofinetide). Authorization criteria require a diagnosis of Rett syndrome confirmed by a mutation in the MECP2 gene, documentation of clinical manifestations of Rett syndrome, and the member to be 2 years of age or older. Continuation/reauthorization requires evidence of clinical benefit.

Diagnosis of Rett syndrome
Medical records confirming MECP2 gene mutation
Medical records showing clinical manifestations (e.g., hand wringing, apraxia, developmental delays, gait abnormalities)
Member is ≥ 2 years of age
Continuation requires documented benefit (e.g., stabilization or improvement in repetitive movements, mood dysfunction/disruptive behavior, vocalization, ambulation)

Documentation Required

Required documentation

Submit medical records that confirm the diagnosis by MECP2 mutation and that document the member’s clinical manifestations of Rett syndrome. For reauthorization, include documentation demonstrating clinical benefit from Daybue. Do not submit requests for members who obtained the medication only as samples or via manufacturer patient assistance programs.

Medical records confirming MECP2 mutation
Documentation of clinical manifestations (examples listed in policy)
For reauthorization, evidence of benefit (stabilization or improvement in listed symptoms)
Exclude requests for members who received product only as samples or through manufacturer patient assistance programs

Billing Rule

Specialty pharmacy requirement

Daybue has been designated a specialty medication and must be dispensed through a contracted specialty pharmacy per the plan’s pharmacy benefit requirements.

Medication is designated specialty and must be filled at a contracted specialty pharmacy
Contact specialty pharmacy phone: 877-519-1908; fax: 855-540-3693

Clinical and Administrative Background

Daybue (trofinetide) is indicated for the treatment of Rett syndrome in adults and pediatric patients 2 years of age and older. The medication is designated as a specialty drug and must be filled at a contracted specialty pharmacy under the Mass General Brigham Health Plan pharmacy benefit.

Coverage and prior authorization criteria require documentation confirming a diagnosis of Rett syndrome by a mutation in the MECP2 gene, medical records demonstrating clinical manifestations of the disease (for example, hand wringing, apraxia, developmental delays, gait abnormalities), and that the member is at least 2 years old.

Continuation (reauthorization) requires that the member is currently receiving Daybue and is demonstrating clinical benefit from therapy (for example, stabilization or improvement in repetitive movements, mood dysfunction/disruptive behavior, vocalization, or ambulation). Initial approvals and reauthorizations are granted for 12 months.