CurrentNeighborhood Health Plan of Rhode IslandPolicy N/A

Symdeko (tezacaftor/ivacaftor) for cystic fibrosis

Defines coverage and prior authorization criteria for Symdeko (tezacaftor/ivacaftor) for treatment of cystic fibrosis in members age 6 years and older with specific CFTR mutations, including required documentation, prescriber specialty, and continuation criteria.

Policy Summary

PayerNeighborhood Health Plan of Rhode Island

PolicySymdeko (tezacaftor/ivacaftor) for cystic fibrosis

Policy CodePolicy N/A

Change TypeNo material change

Effective Date

Next Review Date

Key ActionSubmit genetic testing report confirming appropriate CFTR mutation with initial prior authorization requests; authorization may be granted for 12 months if criteria met.

POLICY UPDATE CHANGES

No material clinical/coverage changes

1Covered indication (cystic fibrosis)

>=6Minimum age (years)

12 monthsAuthorization duration

Coverage Summary

Defines coverage and prior authorization criteria for Symdeko (tezacaftor/ivacaftor) for treatment of cystic fibrosis in members age 6 years and older with specific CFTR mutations. Coverage is covered_with_criteria when the member is either homozygous for F508del or has at least one CFTR mutation responsive to tezacaftor/ivacaftor based on in vitro data and/or clinical evidence, and required documentation and prescriber specialty criteria are met.

Initial Therapy Criteria

Initial therapy - Cystic fibrosis

Authorization of 12 months may be granted for treatment of cystic fibrosis when all of the following criteria are met:

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Provider Actions — Initial Authorization

Documentation requirements (for initial requests)

Submission of the following information is necessary to initiate the prior authorization review:

ALL of the following

Genetic testing report confirming the presence of the appropriate CFTR gene mutation must be submitted.

Prior Authorization

Prior authorization required with genetic testing report

Submit a genetic testing report confirming the presence of the appropriate CFTR gene mutation with initial prior authorization requests. If the authorization criteria are met, an authorization may be granted for up to 12 months.

Documentation Required

Prescriber specialty documentation

Medication must be prescribed by or in consultation with a pulmonologist. Document the consultation as part of the prior authorization submission.

Denial Risk

Combination with other ivacaftor products

Symdeko must not be used in combination with other medications that contain ivacaftor. Claims will not meet criteria if Symdeko is used with other ivacaftor-containing products.

Continuation Therapy Criteria (Reauthorization)

Continuation of therapy (re-authorization)

Authorization of 12 months may be granted for continued treatment in members requesting reauthorization for an indication listed in Initial Therapy who are experiencing benefit from therapy as evidenced by:

ANY of the following

Disease stability
Disease improvement (e.g., improvement in FEV1 from baseline)

Documentation Required

Continuation evidence

For reauthorization, provide evidence of clinical benefit such as disease stability or disease improvement (for example, improvement in FEV1 from baseline) to support continued therapy. Authorization of up to 12 months may be granted when benefit is demonstrated.

Clinical Evidence

Evidence supporting FDA approval includes the product package insert and key clinical trials: the Symdeko package insert (Vertex Pharmaceuticals; August 2023) and the pivotal trials by Taylor-Cousar JL et al., N Engl J Med. 2017 and Rowe SM et al., N Engl J Med. 2017. The background notes that FDA approval is based on these sources.

Background

Symdeko (tezacaftor/ivacaftor) is FDA-approved for treatment of cystic fibrosis in patients age 6 years and older who are homozygous for the F508del mutation or who have at least one CFTR mutation that is responsive to tezacaftor/ivacaftor based on in vitro data and/or clinical evidence. If the patient’s genotype is unknown, an FDA-cleared CF mutation test should be used to detect the presence of a CFTR mutation, followed by verification with bi-directional sequencing when recommended by the mutation test instructions for use. Submission of a genetic testing report confirming the presence of the appropriate CFTR gene mutation is required with initial prior authorization requests. The medication must be prescribed by or in consultation with a pulmonologist, and authorization of up to 12 months may be granted when criteria are met. Symdeko must not be used in combination with other medications containing ivacaftor. References include the Symdeko package insert (Vertex Pharmaceuticals; August 2023) and the key NEJM trials by Taylor-Cousar et al. and Rowe et al.

F508del is a common CFTR gene mutation defined as the phenylalanine deletion at position 508 and is referenced as F508del in this policy.