CurrentNeighborhood Health Plan of Rhode IslandPolicy N/A

Kalydeco (ivacaftor) — Coverage Criteria for Cystic Fibrosis

Covers medical necessity and authorization criteria for Kalydeco (ivacaftor) for treatment of cystic fibrosis in members, including initiation and continuation; applies to members under Medicaid scope for Neighborhood Health Plan of Rhode Island.

Policy Summary

PayerNeighborhood Health Plan of Rhode Island

PolicyKalydeco (ivacaftor) — Coverage Criteria for Cystic Fibrosis

Policy CodePolicy N/A

Change TypeNo material change

Effective DateJan 1, 2019

Next Review DateN/A

Key ActionObtain prior authorization and document CFTR mutation testing showing at least one ivacaftor‑responsive mutation; initial approvals may be granted for 6 months.

SourceLink

POLICY UPDATE CHANGES

No material clinical or coverage changes in this revision.

1 monthminimum age for covered use

6 monthsinitial authorization duration

12 monthscontinuation authorization duration

2quantity limit (per day)

Coverage Criteria for Kalydeco (ivacaftor)

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Initial Therapy (Cystic Fibrosis)

Covered when ALL of the following are met:

Initial authorization: Member has a diagnosis of cystic fibrosis and documentation that genetic testing was conducted to detect a CFTR mutation; member has at least one CFTR mutation responsive to ivacaftor (examples listed in the policy: A120T, A234D, A349V, R1070W, R1162L, R1283M, S549N, S549R, S589N, S737F, S945L, S977F, S1159F, S1159P); member is at least 1 month of age; member does not have the F508del mutation on both alleles of the CFTR gene; medication is prescribed by or in consultation with a pulmonologist; Kalydeco will not be used in combination with other ivacaftor-containing medications; if requesting granules, member is age 1 month to under 6 years.

Authorization of 6 months may be granted

Continuation Therapy

Covered when ALL of the following are met:

Continuation authorization: Member previously approved for an indication listed in the policy and is experiencing benefit from therapy.

Authorization of 12 months may be granted for continued treatment

All uses of Kalydeco (ivacaftor) that are not FDA‑approved indications or recognized compendial uses are considered experimental/investigational and are not medically necessary.

Use of Kalydeco (ivacaftor) in members who are homozygous for the F508del mutation on both alleles of the CFTR gene is not covered.

Prior Authorization, Documentation, and Prescriber Requirements

Prior Authorization

Prior Authorization Required

Prior authorization is required. Initial approvals may be granted for up to 6 months for treatment of cystic fibrosis when criteria are met. For members requesting reauthorization who are experiencing benefit from therapy, continuation approvals may be granted for up to 12 months.

Initial authorization duration: 6 months
Continuation/reauthorization duration: 12 months when member is benefiting from therapy

Denial Risk

Non‑covered/Not Medically Necessary Indications

Requests for uses that are not FDA‑approved or supported by compendia (i.e., non‑listed CFTR mutations or other indications) are considered experimental/investigational and are not medically necessary; such requests will be denied.

Non‑covered: any indication not FDA‑approved or compendial
Non‑covered: uses lacking appropriate CFTR mutation documentation or for genotypes excluded by policy (e.g., homozygous F508del)

Background

Ivacaftor is a CFTR potentiator that increases the function of the cystic fibrosis transmembrane conductance regulator protein and is indicated to treat cystic fibrosis in patients 1 month of age and older who have at least one CFTR mutation demonstrated to be responsive to ivacaftor based on clinical or in vitro assay data.

Genetic testing to identify a responsive CFTR mutation is required prior to treatment; if the genotype is unknown an FDA‑cleared CF mutation test should be used and, when recommended by the test instructions, verified with bidirectional sequencing. Authorization for initial therapy may be granted for 6 months when the coverage criteria are met.

Definitions

Responsive CFTR mutation (definition)

DefinitionA CFTR gene mutation demonstrated by clinical and/or in vitro assay data to respond to ivacaftor potentiation.

Clinical requirementMember must have one responsive CFTR mutation to be eligible for Kalydeco per FDA-approved indication.

Testing recommendationIf genotype is unknown, use an FDA-cleared CF mutation test and verify with bidirectional sequencing when recommended.

Example responsive mutations listedA120T, A234D, A349V, R1070W, R1162L, R1283M, S549N, S549R, S589N, S737F, S945L, S977F, S1159F, S1159P.