Prior Authorization of Alpha-1 Proteinase Inhibitors
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Governs prior authorization requirements and medical necessity criteria for prescriptions of Alpha-1 Proteinase Inhibitors for Medical Assistance (fee-for-service) providers and pharmacies in Pennsylvania's MA program.
Pi*SZ was added to the list of example high-risk genetic variants associated with alpha-1 antitrypsin deficiency.
The phenotypes listed for high‑risk alpha-1 antitrypsin deficiency are examples and not a complete list.
Coverage Criteria
inv-01: Initial Therapy
Covered when ALL of the following are met for initial authorization:
If the beneficiary does not meet the clinical review guidelines above, a physician reviewer may approve the request if, in their professional judgment, the services are medically necessary to meet the beneficiary's needs.
inv-02: Continuation/Renewal Therapy
Renewal authorization covered when ALL of the following are met:
If the beneficiary does not meet the clinical review guidelines above, a physician reviewer may approve the renewal if, in their professional judgment, the services are medically necessary.
No separate exclusion list is provided beyond the inverse of the clinical criteria in Section B. Practically, this means patients who do not meet key clinical thresholds—such as those without a baseline alpha‑1 antitrypsin level below the specified assay thresholds or those who are active smokers—would not meet the coverage criteria for initiation or renewal of augmentation therapy.
Providers must document the required elements (baseline AAT level by an accepted assay, genotype/phenotype indicating a high‑risk variant, pulmonologist involvement, absence of contraindications, age and dose per package labeling, and smoking status) because absence of these items effectively functions as an exclusion from coverage unless a physician reviewer determines medical necessity.
Prior authorization personnel will apply the clinical guidelines in Section B when assessing medical necessity. If the request does not meet those guidelines, it will be referred to a physician reviewer and may be denied unless the physician reviewer, in their professional judgment, determines the requested service is medically necessary to meet the beneficiary’s needs.
Coding and Laboratory Thresholds
Provider Actions and Requirements
Prior authorization required for all Alpha-1 Proteinase Inhibitor prescriptions
All prescriptions for an Alpha-1 Proteinase Inhibitor must be prior authorized through the MA Prior Authorization of Pharmaceutical Services procedures; follow the handbook Section I procedures and SECTION II clinical review guidelines when submitting requests.
- Requests must follow the procedures in the Prior Authorization of Pharmaceutical Services Handbook (Section I).
- Prior authorization personnel will apply the Section B clinical guidelines when reviewing requests.
No step therapy rules specified
This bulletin specifies that no step therapy rules are defined for Alpha-1 Proteinase Inhibitors.
Required documentation for initial authorization — baseline AAT level and clinical information
For initial authorization, submit baseline (pre-treatment) alpha-1 antitrypsin plasma/serum level measured by an accepted assay and supporting clinical information per the handbook.
- Acceptable baseline AAT thresholds: rocket immunoelectrophoresis <11 μmol/L; radial immunodiffusion <80 mg/L; nephelometry <57 mg/dL.
- Include evidence of diagnosis of clinically evident emphysema secondary to severe AATD and documentation of genotype/phenotype as applicable.
Required documentation for renewals — evidence of improvement or stabilization
For renewals, provide documentation demonstrating improvement or stabilization of emphysema signs/symptoms and continued appropriate specialist involvement.
- Examples: annual spirometry showing slowed progression or decreased frequency/duration/severity of pulmonary exacerbations.
- Continued prescribing by or in consultation with a pulmonologist, smoking status, absence of contraindications, and dose consistent with package labeling.
Requests not meeting criteria will be referred to physician reviewer and may be denied
If the request does not meet the clinical guidelines in Section B, it will be referred to a physician reviewer and may be denied unless the physician reviewer determines the service is medically necessary.
- Prior authorization personnel apply the Section B guidelines; nonconforming requests are escalated to physician review for a medical necessity determination.
Background
Alpha‑1 antitrypsin deficiency (AATD) is a genetic condition that can result in clinically evident emphysema. Alpha‑1 Proteinase Inhibitors are augmentation therapies used to treat emphysema secondary to severe AATD by restoring circulating alpha‑1 antitrypsin levels to help protect the lungs from protease‑mediated injury.
This policy defines medical necessity for Alpha‑1 Proteinase Inhibitor therapy by requiring documentation of severe AATD (a baseline AAT plasma/serum level below accepted thresholds by specified assays and a high‑risk genotype), appropriate dosing and age per package labeling, absence of contraindications, pulmonologist involvement, and a non‑smoker or ex‑smoker status. For renewals, continued benefit is demonstrated by documentation of improvement or stabilization of emphysema (for example, slowed progression on annual spirometry or fewer/existing pulmonary exacerbations).
Definitions
Initial Therapy Criteria
inv-13: Initial
Initial authorization criteria
If the beneficiary does not meet the clinical review guidelines listed above, the physician reviewer may still approve the request if, in their professional judgment, the service is medically necessary.
Continuation / Renewal Criteria
inv-14: Renewal
Renewal criteria for previously approved Alpha-1 Proteinase Inhibitor prescriptions
If the beneficiary does not meet the clinical review guidelines listed above, the physician reviewer may approve the renewal if, in their professional judgment, the services are medically necessary.
Step Therapy
| Step | Requirement |
|---|---|
| No step therapy steps specified | This bulletin does not define any step therapy requirements for Alpha-1 Proteinase Inhibitors; prior authorization procedures are governed by the MA Prior Authorization of Pharmaceutical Services Handbook. |
Site of Care
No site‑of‑care restrictions specified
No site‑of‑care restrictions are specified in this bulletin; follow the prior authorization handbook procedures for site‑of‑care handling.
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