CurrentNeighborhood Health Plan of Rhode IslandPolicy N/A

Kalydeco (ivacaftor) for cystic fibrosis

Defines coverage and prior authorization criteria for ivacaftor (Kalydeco) for treatment of cystic fibrosis, including required genetic testing, prescriber specialty, age requirements, combination exclusions, duration of authorization, and continuation criteria.

Policy Summary

PayerNeighborhood Health Plan of Rhode Island

PolicyKalydeco (ivacaftor) for cystic fibrosis

Policy CodePolicy N/A

Change Typeclarified

Effective Date

Next Review Date

Key ActionFor initial requests, submit genetic testing report confirming presence of the appropriate CFTR gene mutation (if genotype unknown, submit FDA-cleared CF mutation test results and verification sequencing when recommended).

POLICY UPDATE CHANGES

Policy references and formatting from CVS Caremark template appear; no clinical policy statement changes documented.

1Covered Indication (CF)

12 monthsAuthorization Duration

Multiple listedEligible Mutations

PulmonologistPrescriber

Coverage Summary

Scope: Ivacaftor (Kalydeco) is covered with criteria for FDA-approved cystic fibrosis indications when all approval criteria are met, including documented CFTR mutation status and required prescriber specialty; authorization may be granted for up to 12 months when criteria are met. Genetic testing is required if the patient's genotype is unknown — an FDA-cleared CF mutation test with recommended sequencing verification should be used. The source document's age threshold text is incomplete and therefore the specific numeric age requirement is not fully specified in the policy text.

Initial Therapy Criteria (FDA-Approved Indication / Initial Authorization)

FDA-Approved Indication / Initial Authorization

Covered when ALL of the following are met:

ALL of the following

Diagnosis of cystic fibrosis (CF) for treatment with ivacaftor (Kalydeco).
Genetic testing was conducted to detect a mutation in the CFTR gene.
If genotype unknown, an FDA-cleared CF mutation test should be used followed by verification with bi-directional sequencing when recommended by the test instructions for use.
Member has at least one of the listed CFTR mutations
- A120T
- A234D
- A349V
- A455E
- F311del
- F311L
- F508C
- Q237H
- Q359R
- Q1291R
- R74W
- R75Q
- R352Q
- R553Q
- R668C
- R792G
- R933G
- R1070Q
- R1070W
- R1162L
- R1283M
- S549N
- S549R
- S589N
Member is at least [age not fully specified in document] month(s) of age.
Document shows missing numeric age; member must meet the age threshold per FDA labeling.
Kalydeco will not be used in combination with other medications containing ivacaftor.
Prescribed by or in consultation with a pulmonologist.

Duration of Authorization

Authorization limits

Initial authorization: up to 12 months may be granted when all initial criteria are met.12 months

Continuation Therapy Criteria (Reauthorization)

Continuation / Reauthorization

Covered for reauthorization when ALL of the following are met:

ALL of the following

Member requests reauthorization for an indication listed in the initial criteria (Section IV).
Member is experiencing benefit from therapy as evidenced by disease stability or disease improvement (e.g., improvement in FEV1 from baseline).
Authorization of 12 months may be granted for continued treatment.12 months

Not Covered / Investigational

All other indications are not covered:

Any indication other than the FDA-approved indications and compendial uses listed is considered experimental/investigational and not medically necessary.

Kalydeco will not be used in combination with other medications containing ivacaftor; do not bill for Kalydeco when used in combination with other ivacaftor-containing products.

Provider Actions / Prior Authorization

Prior Authorization

Prior authorization required with genetic test documentation

Submit a genetic testing report confirming the presence of an appropriate CFTR mutation. If the genotype is unknown, submit results from an FDA-cleared CF mutation test and include verification with bi-directional sequencing when recommended by the test instructions for use.

Genetic testing report confirming presence of appropriate CFTR mutation.
If genotype unknown, FDA-cleared CF mutation test results plus verification sequencing when recommended.

Documentation Required

Prescriber specialty documentation

Medication must be prescribed by or in consultation with a pulmonologist; include documentation of pulmonologist involvement with the prior authorization request.

Documentation Required

Continuation evidence of benefit

For reauthorization, provide objective evidence that the member is benefiting from therapy, such as disease stability or improvement (for example, improvement in FEV1 from baseline).

Billing Rule

Combination therapy exclusion

Do not bill for Kalydeco when used in combination with other medications that contain ivacaftor; combination use with other ivacaftor-containing products is not allowed.

Clinical Evidence & References

Guideline referenced: Cystic fibrosis pulmonary guidelines: chronic medications for maintenance of lung health. Am J Respir Crit Care Med. 2013;187:680-689.

Note: FDA approval for ivacaftor is for patients with CF who have at least one CFTR mutation responsive to ivacaftor potentiation; if genotype is unknown, an FDA-cleared CF mutation test with recommended verification sequencing should be used.

Background & Definitions

Kalydeco (ivacaftor) is FDA-approved for treatment of cystic fibrosis in patients with at least one CFTR mutation that is responsive to ivacaftor potentiation. If the patient's genotype is unknown, an FDA-cleared CF mutation test should be used to detect the CFTR mutation followed by verification with bi-directional sequencing when recommended by the mutation test instructions for use.

CFTR mutation testing: Use of an FDA-cleared CF mutation test followed by verification with bi-directional sequencing when recommended by the mutation test instructions for use.