CurrentNeighborhood Health Plan of Rhode IslandPolicy N/A

Orkambi (lumacaftor/ivacaftor) for cystic fibrosis

Policy governs coverage and prior authorization criteria for Orkambi (lumacaftor/ivacaftor) for treatment of cystic fibrosis in members meeting FDA‑approved genotype and age criteria, including documentation and prescriber specialty requirements, authorization duration, and continuation criteria.

Policy Summary

PayerNeighborhood Health Plan of Rhode Island

PolicyOrkambi (lumacaftor/ivacaftor) for cystic fibrosis

Policy CodePolicy N/A

Change TypeNo material change

Effective Date

Next Review Date

Key ActionSubmit genetic testing report confirming F508del mutation on both alleles and ensure prescription is written by or in consultation with a pulmonologist when requesting prior authorization.

SourceLink

POLICY UPDATE CHANGES

No material clinical/coverage changes.

1Covered Indication (homozygous F508del)

>=2Minimum age for coverage

12Authorization duration (months)

other genotypesNot covered / experimental

Coverage Summary

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Policy stance: Covered with criteria for Orkambi (lumacaftor/ivacaftor). Scope summary: Orkambi is covered for the treatment of cystic fibrosis in patients who are homozygous for the F508del CFTR mutation and are aged >= 2 years. Coverage requires genetic confirmation of the F508del mutation on both alleles, prescriber involvement by or in consultation with a pulmonologist, and adherence to other specified criteria (e.g., not used with other ivacaftor-containing products). Authorization may be granted for up to 12 months for initial and continuation requests.

Key thresholds and quick facts

Minimum age for coverage>= 2 years

Authorization duration12 months

Covered indicationCystic fibrosis - homozygous F508del (F508del/F508del)

Not covered / ExperimentalAll other genotypes (non‑homozygous F508del)

Prescriber requirementPrescription by or in consultation with a pulmonologist

Required documentationGenetic testing report confirming F508del on both alleles

Initial Therapy Criteria

Initial Authorization - Cystic Fibrosis (FDA‑approved indication)

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

ALL of the following

Diagnosis of cystic fibrosis
Genetic testing was conducted to detect a mutation in the CFTR gene
Submission of genetic testing report confirming the presence of the appropriate CFTR gene mutation is necessary for prior authorization review
Genotype: Member is positive for the F508del mutation on both alleles of the CFTR gene
Age: Member is at least 2 years of ageAge >= 2 years
Document states indication per FDA label (Orkambi indicated for patients aged 2 years and older)
Drug combination: Orkambi will not be used in combination with other medications containing ivacaftor
Prescriber: Prescription written by or in consultation with a pulmonologist
Prescriber specialty requirement; include documentation of consultation when applicable

Continuation Therapy Criteria

Continuation / Reauthorization

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

ALL of the following

Indication: Member is requesting reauthorization for an indication listed in the initial criteria (Section IV)
Clinical benefit: Member is experiencing benefit from therapy as evidenced by disease stability or clinical benefit
Clinical documentation of continued benefit required for reauthorization

Documentation Required

Continuation evidence

For reauthorization, provide documentation demonstrating the member is experiencing benefit from therapy. Acceptable evidence includes documentation of disease stability or clinical improvement attributable to treatment.

Evidence of disease stability
Clinical improvement documented in notes

Unproven / Exclusions

Not Medically Necessary / Experimental

All other indications beyond the FDA‑approved homozygous F508del population are considered:

Not medically necessary: Use of Orkambi for cystic fibrosis in patients who are not homozygous for the F508del mutation is considered experimental/investigational and not medically necessary

Efficacy and safety not established for other genotypes

The efficacy and safety of Orkambi have not been established for patients with cystic fibrosis who are not homozygous for the F508del mutation; this policy aligns coverage to the FDA‑approved indication and limits use to the specified genotype.

Provider Actions

Prior Authorization

Prior authorization required with genetic test report

Prior authorization requires submission of a genetic testing report confirming the presence of the F508del mutation on both alleles of the CFTR gene to initiate review.

Genetic testing report
Confirm homozygous F508del (F508del on both alleles)

Documentation Required

Prescriber specialty documentation

The prescription must be written by a pulmonologist or written in consultation with a pulmonologist. When prescribed in consultation, include documentation of the consultation with the prior authorization submission.

Documentation of pulmonologist consultation when applicable

Prior Authorization

Duration of authorization

Authorizations, both initial and for continuation, may be granted for up to 12 months.

Background

Background: Orkambi (lumacaftor/ivacaftor) is indicated for treatment of cystic fibrosis in patients who are homozygous for the F508del mutation in the CFTR gene. The efficacy and safety of Orkambi have not been established for other genotypes. Prior authorization requires submission of genetic testing confirming the F508del mutation on both alleles and the prescription must be written by or in consultation with a pulmonologist. Definition: F508del — the F508del mutation in the CFTR gene (also written F508del / F508del homozygous).