CurrentNeighborhood Health Plan of Rhode IslandPolicy N/A

Drug Policy: Empaveli (pegcetacoplan) — Coverage Criteria

Defines clinical indications, inclusion and exclusion criteria, and utilization management requirements for Empaveli (pegcetacoplan) for members; applies to UM processing of medication requests by network providers.

Policy Summary

PayerNeighborhood Health Plan of Rhode Island

PolicyEmpaveli (pegcetacoplan) — Drug Policy (coverage criteria)

Policy CodePolicy N/A

Change TypeMinor/administrative (no material clinical changes)

Effective DateMay 31, 2024

Next Review DateN/A

Key ActionSubmit medication requests to Evolent UM with documentation supporting FDA labeling, CMS‑recognized compendia, NCCN/ASCO guidelines, or acceptable peer‑reviewed literature.

SourceLink

POLICY UPDATE CHANGES

No material clinical or coverage changes in this revision.

PNHprimary covered indication

1,080 mgsingle dose maximum

16 wksHgb improvement interval

4 wksmax concurrent overlap

multiplecommittee review dates

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Coverage Criteria for Empaveli (pegcetacoplan)

inv-01: Initial/Primary Indication

Covered when ALL of the following are met

PNH treatment criteria: Member has a confirmed diagnosis of Paroxysmal Nocturnal Hemoglobinuria (PNH) and Empaveli (pegcetacoplan) is being used to treat the member's hemolytic anemia due to PNH.

inv-02: Continuation Requests

Continuation therapy exemption

Continuation exemption: Requested medication was used within the last year; the member has not experienced disease progression and/or intolerance to the requested medication; no additional medication(s) are being added to the continuation request.

Applies to continuation requests for a not-approvable medication.

Coverage will be denied when any of the following exclusion criteria apply. Lack of a hemoglobin response is defined as failure to achieve a ≥2.0 g/dL increase in Hgb at 16 weeks of therapy with Empaveli (pegcetacoplan). Concurrent administration with Soliris (eculizumab) beyond a 4‑week overlap is not permitted; when switching from eculizumab to Empaveli a 4‑week run‑in period is recommended to reduce hemolysis risk. Dosing that exceeds a single dose of 1,080 mg for Empaveli will also be excluded.

Empaveli (pegcetacoplan) use for indications that are off‑label or investigational will be considered not medically necessary unless supported by adequate evidence as defined in this policy. Acceptable supporting evidence includes FDA labeling, CMS‑recognized compendia, or peer‑reviewed literature that adequately represents the patient population and therapy, reports clinically meaningful outcomes, and uses appropriate study design. The policy specifically excludes reliance on uncontrolled case reports and meeting abstracts without the full published article as sufficient evidence for coverage decisions.

Coding and Measurable Criteria

inv-05: Hemoglobin improvement — threshold for evaluation

Hemoglobin improvement thresholdIncrease in Hgb ≥2.0 g/dL at 16 weeks

Evaluation timepoint16 weeks after initiating Empaveli

Consequence of not meeting thresholdRequest excluded (denied) if Hgb improvement <2.0 g/dL at 16 weeks

inv-06: Single dose maximum — dosing cap

Single dose maximum (mg)1,080 mg

Policy action if exceededDosing that exceeds single dose limit of 1,080 mg will be excluded (denied)

Initial Therapy Requirements

inv-13: Initial Therapy

Initial coverage requirement

Initial therapy for PNH: Confirmed diagnosis of Paroxysmal Nocturnal Hemoglobinuria (PNH) and use of Empaveli (pegcetacoplan) to treat hemolytic anemia due to PNH.

Continuation Therapy Requirements

inv-14: Continuation Therapy

Continuation request exemption conditions

Continuation exemption nodes: Medication used within last year; no disease progression and/or intolerance; no additional medications added to the continuation request.

Continuation requests for a not-approvable medication are exempt if all conditions are met.

Provider Actions and Authorization

Prior Authorization

Prior authorization and review authority

All medication requests for Empaveli (pegcetacoplan) are processed by Evolent’s Utilization Management department; final approval authority resides with the Utilization Management Committee.

Step Therapy

Continuation exemption — renewal conditions

Continuation (renewal) requests for a medication that would otherwise be not-approvable are exempt if all of the following are met: the requested medication was used within the last year, the member has not experienced disease progression or intolerance to the medication, and no additional medications are being added to the continuation request.

Requested medication used within the last year
No disease progression and/or no intolerance to the medication
No additional medications added to the continuation request

Background

Empaveli (pegcetacoplan) is a complement C3 inhibitor indicated for treatment of hemolytic anemia in members with Paroxysmal Nocturnal Hemoglobinuria (PNH). This policy frames Empaveli within its clinical context by requiring a confirmed diagnosis of PNH and referencing clinical trial evidence and prescribing information to define accepted use, as well as prescribing specific utilization controls (response assessment, overlap management when switching from eculizumab, and a single‑dose maximum).

Definitions

inv-12: Paroxysmal Nocturnal Hemoglobinuria (PNH) — definition and requirement for confirmed diagnosis

DefinitionParoxysmal Nocturnal Hemoglobinuria (PNH): confirmed diagnosis required for coverage when Empaveli (pegcetacoplan) is used to treat hemolytic anemia due to PNH.

Coverage requirementMember must have a confirmed diagnosis of PNH and Empaveli must be used to treat hemolytic anemia due to PNH

Source referencePolicy indication section specifying confirmation of PNH diagnosis for coverage

Quantity Limits

inv-15: Empaveli (pegcetacoplan) — quantity limit / single dose maximum reference

Quantity limit / single dose1,080 mg per single dose

Policy placementIdentified in the exclusion criteria as the maximum allowable single dose

Denial conditionDosing that exceeds this 1,080 mg single-dose limit will result in exclusion (denial)