CurrentNeighborhood Health Plan of Rhode IslandPolicy N/A

Kalydeco (ivacaftor)

Defines coverage, clinical criteria, age and genotype requirements, continuation, quantity limits, and billing-relevant rules for Kalydeco (ivacaftor) for Medicaid members.

Policy Summary

PayerNeighborhood Health Plan of Rhode Island

PolicyKalydeco (ivacaftor)

Policy CodePolicy N/A

Change TypeClarified

Effective DateJan 1, 2019

Next Review Date

Key ActionProvide documentation that genetic testing was conducted to detect a CFTR mutation; if genotype unknown use an FDA-cleared CF mutation test and verify with bi-directional sequencing when recommended.

SourceLink

POLICY UPDATE CHANGES

Continuation authorization duration described as 12 months for members experiencing benefit.

>=1 moMinimum age for use

1-<6 yrsGranules allowed age range

2/dayQuantity limit

6 moInitial auth duration

12 moReauth duration

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Coverage stance: covered with criteria for Kalydeco (ivacaftor). Scope: defines coverage, clinical criteria, age and genotype requirements, continuation, quantity limits, and billing-relevant rules for Medicaid members. Initial authorization may be granted for 6 months when criteria are met; continuation (reauthorization) may be granted for 12 months for members experiencing benefit. Policy effective date: 1/1/2019; last review: 5/2025.

Key thresholds & stats

Minimum age>= 1 month

Granules allowed age range1 month to <6 years

Quantity limit2 tablets/packets per day

Initial authorization duration6 months

Reauthorization duration12 months

Initial Therapy Criteria (FDA-Approved Indication)

FDA-Approved Indication / Initial Authorization for Cystic Fibrosis

Authorization of 6 months may be granted when ALL of the following are met:

ALL of the following

Genetic testing: Documentation that genetic testing was conducted to detect a mutation in the CFTR gene.
If genotype unknown, use an FDA-cleared CF mutation test and verify with bi-directional sequencing when recommended.
Prescriber specialty: Medication is prescribed by or in consultation with a pulmonologist.
CFTR mutation list
- mutation: A120T
- mutation: A234D
- mutation: A349V
- mutation: A455E
- mutation: A1067T
- mutation: D110E
- mutation: D110H
- mutation: D192G
- mutation: D579G
- mutation: D924N
- mutation: D1152H
- mutation: D1270N
- mutation: E56K
- mutation: E193K
- mutation: E822K
- mutation: E831X
- mutation: F311del
- mutation: F311L
- mutation: F508C
- mutation: F508C;S1251N
- mutation: F1052V
- mutation: F1074L
- mutation: G178E
- mutation: G178R
- mutation: G194R
- mutation: G314E
- mutation: G551D
- mutation: G551S
- mutation: G576A
- mutation: G970D
- mutation: G1069R
- mutation: G1244E
- mutation: G1249R
- mutation: G1349D
- mutation: H939R
- mutation: H1375P
- mutation: I148T
- mutation: I175V
- mutation: I807M
- mutation: I1027T
- mutation: I1139V
- mutation: K1060T
- mutation: L206W
- mutation: L320V
- mutation: L967S
- mutation: L997F
- mutation: L1480P
- mutation: M152V
- mutation: M952I
- mutation: M952T
- mutation: P67L
- mutation: Q237E
- mutation: Q237H
- mutation: Q359R
- mutation: Q1291R
- mutation: R74W
- mutation: R75Q
- mutation: R117C
- mutation: R117G
- mutation: R117H
- mutation: R117L
- mutation: R117P
- mutation: R170H
- mutation: R347H
- mutation: R347L
- mutation: R352Q
- mutation: R553Q
- mutation: R668C
- mutation: R792G
- mutation: R933G
- mutation: R1070Q
- mutation: R1070W
- mutation: R1162L
- mutation: R1283M
- mutation: S549N
- mutation: S549R
- mutation: S589N
- mutation: S737F
- mutation: S945L
- mutation: S977F
- mutation: S1159F
- mutation: S1159P
- mutation: S1251N
- mutation: S1255P
- mutation: T338I
- mutation: T1053I
- mutation: V232D
- mutation: V562I
- mutation: V754M
- mutation: V1293G
- mutation: W1282R
- mutation: Y1014C
- mutation: Y1032C
- mutation: 711+3A→G
- mutation: 2789+5G→A
- mutation: 3272 -26A→G
- mutation: 3849+10kbC→T
Minimum age: Member is at least 1 months of age.>= 1 month
Combination prohibition: Kalydeco will not be used in combination with other ivacaftor containing medications.
F508del exclusion: Member does not have the F508del mutation on both alleles of the CFTR gene.
Granules age constraint: If requesting granules, member is between 1 months of age to under 6 years of age.1 month to <6 years

ref

Continuation Therapy Criteria (Reauthorization)

Continuation of Therapy (Reauthorization)

Authorization of 12 months may be granted when ALL of the following are met:

ALL of the following

Covered indication: Member is requesting reauthorization for an indication listed in Section III (covered indication).
Evidence of benefit: Member is experiencing benefit from therapy as evidenced by disease stability or disease improvement (e.g., improvement in FEV1 from baseline).

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title":

Provider Actions & Billing Rules

Prior Authorization

Prior authorization required

Authorization is required. Initial authorization may be granted for up to 6 months. Continuation (reauthorization) may be granted for up to 12 months when the member is experiencing benefit from therapy (e.g., disease stability or improvement such as FEV1 improvement). Prescriptions must be by or in consultation with a pulmonologist; documentation of pulmonologist prescribing or consult is required for approval and for reauthorization evidence of benefit must be provided.

Documentation Required

Genetic testing documentation

Document that genetic testing was conducted to detect a mutation in the CFTR gene. If the patient's genotype is unknown, use an FDA-cleared CF mutation test to detect the CFTR mutation and, when recommended by the mutation test instructions for use, verify results with bi-directional sequencing.

Billing Rule

Quantity limit enforcement

A quantity limit is enforced: maximum of 2 tablets or packets per day.

Denial Risk

Exclusion for non-approved indications

Claims for indications that are not FDA-approved or compendial uses are considered experimental/investigational and are not medically necessary; such claims will be subject to denial.

Documentation Required

Special formulation age constraint

When requesting the granules formulation, document that the member's age is between 1 month and under 6 years.

Background & Definitions

Kalydeco (ivacaftor) is a CFTR potentiator indicated for treatment of cystic fibrosis in patients aged 1 month and older who have one CFTR mutation responsive to ivacaftor potentiation. Genetic testing is required to identify responsive mutations; if genotype is unknown, use an FDA-cleared CF mutation test with verification by bi-directional sequencing when recommended. Kalydeco must not be used in combination with other ivacaftor-containing medications, and patients who are F508del homozygotes (F508del on both alleles) are excluded. Granules formulation is restricted to patients aged 1 month to under 6 years.

Responsive to ivacaftor potentiation: Mutations in the CFTR gene that are responsive based on clinical and/or in vitro assay data as listed in the policy or per FDA labeling.

Revision History

2019-01-01effective_date

Policy effective date set to 1/1/2019.

2019-08review

Policy review conducted in 8/2019.

2020-06review

Policy review conducted in 6/2020.

2020-10review

Policy review conducted in 10/2020.

2021-02review

Policy review conducted in 02/2021.

2022-02review

Policy review conducted in 02/2022.

2023-03review

Policy review conducted in 03/2023.

2023-05review

Policy review conducted in 05/2023.

2024-02review

Policy review conducted in 02/2024.

2025-05reviewLatest

Most recent policy review conducted in 5/2025.

2019-01-01clarificationLatest

Continuation (reauthorization) authorization duration clarified as up to 12 months for members experiencing benefit from therapy.