CurrentNeighborhood Health Plan of Rhode IslandPolicy N/A

Orkambi 1885 A Sgm P2022B

Covers Orkambi for treatment of cystic fibrosis in members aged ≥1 year who are homozygous for the F508del CFTR mutation when all prior authorization criteria are met; other uses are considered investigational. Authorization and reauthorization are for 12 months when criteria and benefit are met.

Policy Summary

PayerNeighborhood Health Plan of Rhode Island

PolicyOrkambi 1885 A Sgm P2022B

Policy CodePolicy N/A

Change TypeNo material change

Effective Date

Next Review Date

Key ActionPrior authorization requires submission of a genetic testing report confirming F508del mutation on both alleles.

SourceLink

POLICY UPDATE CHANGES

No material clinical/coverage changes

1Minimum Age (years)

F508del homozygousCovered Genotype Requirement

12 moAuthorization Duration

Not coveredOther indications

Coverage Summary

Covers Orkambi for treatment of cystic fibrosis in members aged ≥1 year who are homozygous for the F508del CFTR mutation when all prior authorization criteria are met; other uses are considered investigational. Authorization and reauthorization are for 12 months when criteria and benefit are met.

Initial Therapy Criteria

Initial Authorization — Cystic Fibrosis

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

Genetic testing was conducted to detect a mutation in the CFTR gene.
Genotype requirement: The member is positive for the F508del mutation on both alleles of the CFTR gene.
Coverage requires positivity on both alleles.
Age requirement: The member is at least 1 year of age.>= 1 year
Minimum age threshold from policy.
Therapy combination restriction: Orkambi will not be used in combination with other medications containing ivacaftor.

Continuation / Reauthorization Criteria

Continuation / Reauthorization

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

Indication match: Member requests reauthorization for an indication listed in Initial Authorization (Cystic Fibrosis).
Documented benefit: Member is experiencing benefit from therapy as evidenced by disease stability or disease improvement (e.g., improvement in FEV1 from baseline).

Not Medically Necessary / Exclusions

Not covered:

Non‑homozygous indications: Any indication other than homozygous F508del mutation (all other indications are considered experimental/investigational and not medically necessary).
Combination with ivacaftor: Use in combination with other medications containing ivacaftor.

Efficacy and safety have not been established for patients with cystic fibrosis who are not homozygous for the F508del mutation.

Provider Actions / Requirements

Prior Authorization

Prior authorization required

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

Genetic testing report confirming F508del on both alleles

Documentation Required

Prescriber specialty requirement

This medication must be prescribed by or in consultation with a pulmonologist.

Documentation Required

Reauthorization documentation

For reauthorization, provide documentation of clinical benefit or stability (for example, improvement in FEV1 from baseline).

Denial Risk

Denial for non-approved indications

Claims for any indication other than cystic fibrosis in patients homozygous for the F508del mutation are considered experimental/investigational and not medically necessary and may be denied.

Clinical Evidence / References

Reference: Orkambi [package insert]. Vertex Pharmaceuticals Inc.; July 2019. Note: Efficacy and safety have not been established for CF patients who are not homozygous for the F508del mutation.

Background

Orkambi is the combination of lumacaftor and ivacaftor indicated for treatment of cystic fibrosis in patients aged 1 year and older who are homozygous for the F508del mutation in the CFTR gene. The efficacy and safety of Orkambi have not been established in patients with CF other than those homozygous for the F508del mutation.

F508del mutation: a specific deletion mutation in the CFTR gene; coverage requires positivity on both alleles.