CurrentNeighborhood Health Plan of Rhode IslandPolicy 1884-A

Kalydeco (ivacaftor) coverage policy

Defines coverage, prior authorization documentation, prescriber specialty, initial and continuation approval criteria, excluded uses, and treatment duration for Kalydeco (ivacaftor) for cystic fibrosis patients age 6gt;=1 month with specified CFTR mutations.

Policy Summary

PayerNeighborhood Health Plan of Rhode Island

PolicyKalydeco (ivacaftor) coverage policy

Policy CodePolicy 1884-A

Change TypeNo material changes

Effective Date

Next Review Date

Key ActionFor initial requests, submit genetic testing report confirming presence of an appropriate CFTR gene mutation to initiate prior authorization review.

SourceLink

POLICY UPDATE CHANGES

No material clinical or coverage changes

1Minimum age (months)

12 monthsAuthorization duration

~70Listed CFTR mutations

Coverage Summary

Scope: This policy (Reference 1884-A) defines coverage for Kalydeco (ivacaftor) for treatment of cystic fibrosis in patients aged ≥1 month. Authorization may be granted for 12 months. Coverage is conditional on demonstration of a CFTR mutation that is responsive to ivacaftor based on clinical and/or in vitro assay data; when the patient's genotype is unknown an FDA-cleared CF mutation test with verification by bi-directional sequencing when recommended must be used. The policy applies to brand and generic ivacaftor across dosage forms and strengths and requires prescribing by or in consultation with a pulmonologist.

Initial Therapy Criteria - Cystic Fibrosis

Initial Authorization - Cystic Fibrosis

Authorization of 12 months may be granted for treatment of cystic fibrosis when all the following criteria are met:

ALL of the following

Genetic testing was conducted to detect a mutation in the CFTR gene.
ANY of the following
- A120T
- A234D
- A349V
- A455E
- A1067T
- D110E
- D110H
- D192G
- D579G
- D924N
- D1152H
- D1270N
- E56K
- E193K
- E822K
- E831X
- F311del
- F311L
- F508C
- S1251N
- F1052V
- F1074L
- G178E
- G178R
- G194R
- G314E
- G551D
- G551S
- G576A
- G970D
- G1069R
- G1244E
- G1249R
- G1349D
- H939R
- H1375P
- I148T
- I175V
- I807M
- I1027T
- I1139V
- K1060T
- L206W
- L320V
- L967S
- L997F
- L1480P
- M152V
- M952I
- M952T
- P67L
- Q237E
- Q237H
- Q359R
- Q1291R
- R74W
- R75Q
- R117C
- R117G
- R117H
- R117L
- R117P
- R170H
- R347H
- R347L
- R352Q
- R553Q
- R668C
- R792G
- R933G
- R1070Q
- R1070W
- R1162L
- R1283M
- S549N
- S549R
- S589N
- S737F
- S945L
- S977F
- S1159F
- S1159P
- S1251N
- S1255P
- T338I
- T1053I
- V232D
- V562I
- V754M
- V1293G
- W1282R
- Y1014C
- Y1032C
- 711+3A → G
- 2789+5G → A
- 3272-26A → G
- 3849+10kbC → T
The member is at least 1 month of age.
If genotype is unknown, an FDA-cleared CF mutation test should be used to detect the presence of CFTR mutation followed by verification with bi-directional sequencing when recommended by the mutation test instructions for use.

Continuation Therapy Criteria (Reauthorization)

Continuation of Therapy (Reauthorization)

Authorization of 12 months may be granted for continued treatment in members requesting reauthorization for an indication listed in the coverage criteria section who are experiencing benefit from therapy as evidenced by:

ANY of the following

Disease stability
Disease improvement (e.g., improvement in FEV1 from baseline)

Excluded / Not Medically Necessary Uses

Excluded/Not Medically Necessary Uses

Other indications beyond the listed FDA-approved indication are considered not medically necessary:

ALL of the following

All other indications are considered experimental/investigational and are not medically necessary.
Kalydeco will 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 disallowed.

Provider Actions

Prior Authorization

Prior authorization submission

For initial requests, submit the genetic testing report confirming the presence of the appropriate CFTR gene mutation to initiate prior authorization review.

Documentation Required

Prescriber specialty requirement

Medication must be prescribed by or in consultation with a pulmonologist.

Denial Risk

Not covered if mutation not listed

Requests for indications or CFTR mutations not listed in the policy are considered experimental/investigational and will be denied as not medically necessary.

Denial Risk

Combination therapy exclusion

Claims for Kalydeco used in combination with another CFTR modulator (e.g., Orkambi, Symdeko) will not be approved.

Required documentation for initial prior authorization: submit the genetic testing report confirming the presence of an appropriate CFTR gene mutation to initiate review. Prescriptions should be from or in consultation with a pulmonologist. For reauthorization/continuation requests, provide evidence of continued benefit such as disease stability or disease improvement (e.g., improvement in FEV1 from baseline). Include any prescriber consultation notes as applicable and note that requests for mutations or indications not listed in the policy will be denied as not medically necessary.

Background & Definitions

Background: Kalydeco (ivacaftor) is indicated for cystic fibrosis patients age 1 month and older who have at least one CFTR 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 to detect CFTR mutations with verification by bi-directional sequencing when recommended by the test instructions.

Definitions

TermResponsive mutation

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