CurrentNeighborhood Health Plan of Rhode IslandPolicy N/A

Alpha1-Proteinase Inhibitors (augmentation therapy) for AAT deficiency

Covers FDA-approved indications and compendial uses for chronic augmentation/maintenance therapy with alpha1-proteinase inhibitor products (Aralast NP, Glassia, Prolastin-C, Zemaira) for adults with clinically evident emphysema due to severe alpha1-antitrypsin deficiency, subject to prior authorization and specified clinical criteria.

Policy Summary

PayerNeighborhood Health Plan of Rhode Island

PolicyAlpha1-Proteinase Inhibitors (augmentation therapy) for AAT deficiency

Policy CodePolicy N/A

Change TypeNo material change

Effective Date

Next Review Date

Key ActionProvide pretreatment post-bronchodilation FEV1, pretreatment serum AAT level, and AAT protein phenotype or genotype as part of the prior authorization submission to initiate review.

POLICY UPDATE CHANGES

No material clinical/coverage changes

4Products named (brand)

1Initial approval duration (years)

2Key required labs/types

Coverage Summary

This policy covers FDA-approved augmentation/maintenance therapy with alpha1-proteinase inhibitor products (Aralast NP, Glassia, Prolastin-C, Zemaira) for adults with clinically evident emphysema due to severe congenital or hereditary alpha1-antitrypsin (AAT) deficiency, subject to prior authorization and the specified clinical criteria. All other (non‑FDA) indications are considered experimental/investigational and not medically necessary.

Initial Therapy Criteria (Authorization of 12 months)

Initial Approval (12 months)

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

ALL of the following

FEV1 percent predicted: Member's pretreatment post-bronchodilation forced expiratory volume in one second (FEV1) is greater than or equal to 25% and less than or equal to 80% of predicted value.>= 25% and <= 80% of predicted
Serum AAT level: Member's pretreatment serum alpha1-antitrypsin (AAT) level is less than 11 micromol/L (80 mg/dL by radial immunodiffusion or 50 mg/dL by nephelometry).< 11 micromol/L (80 mg/dL by radial immunodiffusion or 50 mg/dL by nephelometry)
Qualifying phenotypes: Member has a documented PiZZ, PiZ (null), or Pi (null,null) (homozygous) AAT deficiency or other phenotype/genotype associated with serum AAT concentrations < 11 micromol/L (80 mg/dL by radial immunodiffusion or 50 mg/dL by nephelometry).
See policy definitions for phenotype/genotype qualifying definitions (PiZZ, PiZ(null), Pi(null,null) or other genotypes associated with AAT < 11 micromol/L).
Excluded phenotypes: Member does not have the PiMZ or PiMS AAT deficiency.

Continuation Therapy Criteria (Authorization of 12 months)

Continuation of Therapy (12 months)

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

Clinical benefit: Member is experiencing beneficial clinical response from therapy.

AAT level thresholds: qualifies when pretreatment serum AAT is 11 micromol/L (80 mg/dL by radial immunodiffusion or 50 mg/dL by nephelometry).

Qualifying phenotypes: members with documented homozygous AAT deficiency such as PiZZ, PiZ(null), or Pi(null,null) or other genotypes associated with serum AAT < 11 micromol/L qualify; PiMZ and PiMS are excluded.

Unproven / Exclusions

Exclusion: All non‑FDA indications for alpha1‑proteinase inhibitor augmentation are considered experimental/investigational and not medically necessary.

Explicit ineligibility: members with PiMZ or PiMS AAT deficiency are not eligible for coverage and claims may be denied.

Provider Actions

Prior Authorization

Prior authorization required

Submission of pretreatment data (post-bronchodilation FEV1, pretreatment serum AAT level, and AAT protein phenotype or genotype) is necessary to initiate prior authorization review; authorization may be granted for 12 months if criteria are met.

Documentation Required

Required diagnostic documentation

Provide pretreatment post-bronchodilation FEV1, pretreatment serum AAT level, and AAT protein phenotype or genotype as part of the PA submission.

Denial Risk

Not eligible phenotypes

Claims may be denied if the member has PiMZ or PiMS AAT deficiency or pretreatment serum AAT levels above the specified threshold (< 11 micromol/L; 80 mg/dL by radial immunodiffusion or 50 mg/dL by nephelometry).

Clinical Evidence & References

Supporting references include the package inserts for Glassia, Aralast NP, Prolastin‑C, and Zemaira, plus professional guidance and literature such as the American Thoracic Society/European Respiratory Society (ATS/ERS) statement, the Canadian Thoracic Society clinical practice guideline, and related publications cited.

Background

Augmentation therapy with alpha1‑proteinase inhibitor is indicated for chronic augmentation/maintenance therapy in adults with clinically evident emphysema due to severe congenital or hereditary alpha1‑antitrypsin deficiency. Non‑FDA indications are considered experimental/investigational and not medically necessary.