CurrentNeighborhood Health Plan of Rhode IslandPolicy 1884-A

Ivacaftor (Kalydeco) — Coverage Criteria for Cystic Fibrosis

Covers prior authorization, clinical criteria, and renewal requirements for Kalydeco (ivacaftor) when prescribed for cystic fibrosis patients with responsive CFTR mutations; applies to Neighborhood Health Plan of Rhode Island members covered by this policy.

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 genetic testing report confirming an eligible CFTR mutation with initial prior authorization requests.

SourceLink

POLICY UPDATE CHANGES

No material clinical or coverage changes in this revision.

12 monthsauthorization duration

≥1 monthminimum age

~120 listedeligible mutations

Pulmonologistprescriber

Coverage Criteria for Ivacaftor (Kalydeco)

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CF diagnosis and testing: Member has cystic fibrosis and genetic testing was conducted to detect a mutation in the CFTR gene.genetic test positive for listed mutation

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

Eligible CFTR mutation present: Member has one of the specific CFTR mutations listed in the policy (see mutation list).mutation must match one in list

Presence must be confirmed by genetic testing report submitted with the prior authorization request.

Age requirement: Member is at least 1 month of age.>= 1 month

Prescriber specialty: Medication must be prescribed by or in consultation with a pulmonologist.pulmonologist involvement

Continuation of therapy

Covered when ALL of the following are met

Indication previously approved: Continuation requested for an indication listed in the coverage criteria section.

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

Authorization of 12 months may be granted for continued treatment.

All indications for ivacaftor (Kalydeco) that are not among the FDA‑approved responsive CFTR mutation indications or established compendial uses are considered experimental/investigational and not medically necessary. The policy therefore excludes coverage for uses outside the listed indications and will not cover ivacaftor when prescribed for other, non‑listed conditions.

Use of ivacaftor outside the specified FDA‑approved responsive CFTR mutations is considered investigational and will not be approved. Additionally, ivacaftor must not be used in combination with another CFTR modulator (for example, Orkambi or Symdeko) for the treatment of cystic fibrosis; such combinations are not an approved indication under this policy.

Provider Actions and Documentation Requirements

Prior Authorization

Prior Authorization Required

Prior authorization is required for Kalydeco. Initial requests must include genetic testing confirming an eligible CFTR mutation to initiate review.

Authorization may be granted for up to 12 months when coverage criteria are met.

Documentation Required

Genetic Testing Documentation Required

For initial requests, submit the genetic testing report confirming the presence of the appropriate CFTR gene mutation. Failure to submit this documentation may result in denial of the prior authorization.

Attach genetic testing report to the prior authorization request.

Denial Risk

Do Not Combine with Other CFTR Modulators

Kalydeco must not be used in combination with another CFTR modulator for the treatment of cystic fibrosis (e.g., Orkambi, Symdeko).

Concurrent use with other CFTR modulators is not permitted.

Documentation Required

Reauthorization — Evidence of Benefit Required

For reauthorization requests, provide objective evidence of benefit from therapy (for example, documented disease stability or improvement such as improvement in FEV1 from baseline).

Reauthorizations may be approved for up to 12 months when benefit is demonstrated.

Background

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

Definitions

Responsive CFTR mutation

DefinitionA CFTR gene mutation that is responsive to ivacaftor potentiation based on clinical and/or in vitro assay data; presence must be confirmed by genetic testing.

Confirmation requirementGenetic testing must be conducted to detect the CFTR mutation; if genotype is unknown use an FDA-cleared CF mutation test with verification by bi-directional sequencing when recommended.

Eligible mutationsMember must have one of the CFTR mutations listed in the policy (extensive list provided in the document).

DocumentationSubmit genetic testing report with the prior authorization request to confirm the responsive mutation.