CurrentNeighborhood Health Plan of Rhode IslandPolicy 1884-A

Ivacaftor (Kalydeco) — Coverage Criteria for Cystic Fibrosis

Covers authorization criteria, documentation, prescriber specialty, and duration for Kalydeco (ivacaftor) use in members with cystic fibrosis who have responsive CFTR mutations. Applies to payers' pharmacy benefit management for members receiving this drug.

Policy Summary

PayerNeighborhood Health Plan of Rhode Island

PolicyIvacaftor (Kalydeco) — Coverage Criteria for Cystic Fibrosis

Policy CodePolicy 1884-A

Change TypeNo material changes

Effective Date

Next Review Date

Key ActionSubmit prior authorization with genetic testing report confirming a CFTR mutation responsive to ivacaftor and ensure prescription is by or in consultation with a pulmonologist.

SourceLink

POLICY UPDATE CHANGES

No material clinical or coverage changes in this revision.

≥1 moMinimum eligible member age

12 monthsAuthorization duration

MultipleResponsive mutations listed

PulmonologistPrescriber

Coverage Criteria for Ivacaftor (Kalydeco)

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Initial Therapy

Covered when ALL of the following are met

Age: Member is at least 1 month of age≥ 1 month

Diagnosis of cystic fibrosis

Authorization of 12 months may be granted for treatment of cystic fibrosis

Genotype confirmation: Genetic testing report confirming presence of an appropriate CFTR gene mutation responsive to ivacaftor potentiationdocumented responsive mutation (see list)

If genotype unknown, use an FDA-cleared CF mutation test with verification by bi-directional sequencing when recommended

Prescriber: Medication prescribed by or in consultation with a pulmonologist

Mutation list: Member has one of the enumerated CFTR mutations (A120T, A234D, A349V, R347L, R352Q, R553Q, R668C, R792G, R933G, R1070Q, R1070W, R1162L, R1283M, S549N, 3849+10kbC->T)specific listed mutations

Continuation of Therapy

Covered when ALL of the following are met

Prior indication: Reauthorization requested for an indication listed in the coverage criteria

Clinical benefit: Member is experiencing benefit from therapy as evidenced by disease stability or improvement (e.g., improvement in FEV1 from baseline)demonstrated stability or improvement

Authorization of 12 months may be granted for continued treatment

All indications for use of Kalydeco (ivacaftor) other than the specific cystic fibrosis indications described in this policy are considered experimental/investigational and not medically necessary. Additionally, Kalydeco must not be used in combination with another CFTR modulator for treatment of cystic fibrosis; requests for combination therapy will not meet coverage criteria and are subject to denial.

Use of Kalydeco for indications other than the FDA‑approved or compendial cystic fibrosis indications listed in this policy is considered not medically necessary. If the member’s genotype is unknown, an FDA‑cleared CF mutation test with verification by bi‑directional sequencing when recommended should be performed to establish eligibility for therapy.

Provider Actions and Documentation Requirements

Prior Authorization

Prior Authorization Required

Prior authorization required for Kalydeco — member must have a documented CFTR mutation responsive to ivacaftor, be at least 1 month old, and meet other PA requirements (authorization may be granted for up to 12 months when criteria are met).

Member age: ≥ 1 month
Responsive CFTR mutation required (see genetic testing requirement)
Authorization duration: up to 12 months

Note

Do not combine with other CFTR modulators

Kalydeco must not be used in combination with another CFTR modulator for the treatment of cystic fibrosis.

Documentation Required

Genetic testing report required for initial requests

For initial authorization requests, submit a genetic testing report confirming the presence of a CFTR gene mutation that is responsive to ivacaftor potentiation. If genotype is unknown, an FDA‑cleared CF mutation test should be used and followed by verification with bi‑directional sequencing when recommended by the test instructions for use.

Denial Risk

Denial conditions

Requests will be denied if the requested indication is not FDA‑approved or supported by compendia, or if the member does not have a CFTR mutation known to be responsive to ivacaftor potentiation.

Definitions

Responsive CFTR mutation — definition and genotype confirmation

DefinitionA CFTR gene mutation demonstrated to be responsive to ivacaftor potentiation based on clinical and/or in vitro assay data.

Genotype unknown — testing requirementIf the patient's genotype is unknown, use an FDA‑cleared CF mutation test to detect CFTR mutations with verification by bi‑directional sequencing when recommended by the test instructions.

Documentation requiredFor initial authorization requests, submit a genetic testing report confirming the presence of the appropriate CFTR gene mutation responsive to ivacaftor.

Clinical contextResponsiveness may be determined from clinical trial data or validated in vitro assays demonstrating potentiation by ivacaftor.

Coverage implicationMembers must have a documented responsive CFTR mutation to meet coverage criteria for ivacaftor; all other indications are considered experimental/investigational and not medically necessary.

Background

Ivacaftor (Kalydeco) is a CFTR potentiator indicated for treatment of cystic fibrosis in patients aged 1 month and older who have at least one CFTR gene mutation that is responsive to ivacaftor potentiation based on clinical or in vitro assay data. When genotype is unknown, an FDA‑cleared CF mutation test should be used with verification by bi‑directional sequencing when recommended by the test instructions.