CurrentNeighborhood Health Plan of Rhode IslandPolicy UM ONC_1382

Eculizumab products (Soliris, Epysqli) coverage criteria

Defines accepted indications, exclusion criteria, and authorization/approval process for eculizumab products for Neighborhood Health Plan of Rhode Island; applies to UM processing of medication requests for network providers.

Policy Summary

PayerNeighborhood Health Plan of Rhode Island

PolicyEculizumab products (Soliris, Epysqli) coverage criteria

Policy CodePolicy UM ONC_1382

Change TypeNo material change

Effective DateSeptember 27, 2024

Next Review DateN/A

Key ActionSubmit prior authorization requests to Evolent/Utilization Management for final approval by the Utilization Management Committee.

SourceLink

POLICY UPDATE CHANGES

No material clinical or coverage changes in this revision.

2eculizumab product names listed

PNH, aHUScovered indications

900/1,200 mgsingle-dose maximums

UM Committeefinal approval authority

Sep 18, 2024approval date

Trek Health ingests and normalizes Transparency in Coverage data and payer policy updates to give provider organizations a clear view of how commercial reimbursement behaves across markets, payers, and services. Our platform transforms raw payer disclosures into structured intelligence that supports contract evaluation, payer negotiations, and service line strategy. By combining market benchmarks with ongoing policy visibility, Trek helps teams identify variability, risk, and opportunity in commercial reimbursement. The result is faster insight, stronger negotiating positions, and more informed financial decisions.

Coverage Criteria for Eculizumab Products

Initial and continuation coverage criteria

Covered when ALL of the following are met for each indication group

Continuation exemption: Continuation requests are exempt if: (1) the requested medication was used within the last year AND (2) the member has not experienced disease progression or intolerance to the requested medication AND (3) additional medication(s) are not being added to the continuation request

II.A

PNH indication: The member has hemolytic paroxysmal nocturnal hemoglobinuria (PNH) and eculizumab or an eculizumab biosimilar is being used to reduce hemolysis

II.B

aHUS indication: Confirmed diagnosis of atypical hemolytic uremic syndrome (aHUS) with evidence of hemolysis (LDH above normal OR haptoglobin below normal OR schistocytes on peripheral smear) AND impaired renal function (serum creatinine above normal)

II.C

Disease progression while receiving eculizumab or its biosimilar is an exclusion when there is a lack of hemoglobin response with continued need for blood transfusions. Continuation of eculizumab after documented disease progression is also excluded if the same regimen or another anti‑complement therapy (for example, ravulizumab/Ultomiris) is being used following progression. Additionally, dosing that exceeds the single‑dose limits — 900 mg for PNH and 1,200 mg for aHUS — is excluded and may trigger denial of authorization.

Requests for investigational or off‑label uses of eculizumab or its biosimilar that are not supported by sufficient evidence may be considered not medically necessary. The policy specifies that adequate evidence excludes uncontrolled case reports and meeting abstracts without full peer‑reviewed publication; acceptable support requires representation of the patient population and regimen in the published evidence and clinically meaningful outcomes as defined by standard criteria. In practice, indications not supported by FDA labeling, CMS‑recognized compendia, or acceptable peer‑reviewed literature may be denied.

When determining whether an off‑label use is supported, reviewers will consider study design and relevance: randomized or well‑designed nonrandomized trials with appropriate populations and meaningful outcomes carry more weight, whereas case reports and abstracts alone are generally insufficient to establish accepted use.

Initial Therapy Requirements

Initial therapy

Initial therapy coverage requires documented diagnosis and relevant laboratory evidence as applicable to the indication

PNH initial: Documented hemolytic paroxysmal nocturnal hemoglobinuria (PNH) and intent to use eculizumab or an eculizumab biosimilar to reduce hemolysis

II.B

aHUS initial: Confirmed diagnosis of atypical hemolytic uremic syndrome (aHUS) with evidence of hemolysis (LDH above normal OR haptoglobin below normal OR schistocytes on peripheral smear) AND impaired renal function (serum creatinine above normal)

II.C

Continuation Therapy Requirements

Continuation requests for a previously used medication are eligible when ALL of the following are met:

{

"operator":"all",

"children":[

{"text":"The requested medication was used within the last year (documentation of prior use must be provided in the medical record).","threshold":"<= 12 months"},

{"text":"The member has not experienced disease progression and has not had intolerance to the requested medication (clinical notes must document stability or improvement and absence of adverse events)."},

Provider Documentation, Authorization, and Exclusions

Prior Authorization

Submit prior authorization to Evolent/UM; Utilization Management Committee issues final approval

Prior authorization requests for eculizumab products are processed by Evolent/Utilization Management; final approval authority rests with the Utilization Management Committee.

Evolent (Utilization Management) processes medication requests
Final approval — Utilization Management Committee

Step Therapy

Step/alternate therapy — no coverage after progression or switch to other anti‑complement agents

Requests for continued use after disease progression on eculizumab or after switching to another anti‑complement therapy (for example, ravulizumab/Ultomiris) are excluded and will be denied.

Use after disease progression with the same regimen is excluded
Use after progression with other anti‑complement therapies (e.g., ravulizumab) is excluded

Coding and Quantity Limits

Single dose maximums

Single-dose maximum — PNH900 mg per single dose

Single-dose maximum — aHUS1,200 mg per single dose

Dosing exceedance consequenceDosing that exceeds these single-dose limits may trigger denial under exclusion criteria

Eculizumab / biosimilar quantity limits

Product scopeEculizumab and eculizumab biosimilars (policy applies to both Soliris and biosimilar products)

Quantity limit — PNHMaximum single-dose: 900 mg

Quantity limit — aHUS

Step Therapy and Prior Use Restrictions

Step	Requirement / Restriction
1	Use of eculizumab or eculizumab biosimilar after disease progression on eculizumab (defined by lack of hemoglobin response and continued need for blood transfusions) is excluded. Continuation on the same regimen after progression is not permitted.
	Use of eculizumab/eculizumab biosimilar following disease progression with other anti-complement therapies (e.g., Ultomiris (ravulizumab)) is excluded.

Key Definitions

aHUS (diagnostic criteria and lab evidence)

DefinitionAtypical hemolytic uremic syndrome (aHUS)

Required diagnostic evidenceConfirmed aHUS diagnosis with laboratory evidence of hemolysis (elevated LDH, low haptoglobin, schistocytes on peripheral smear)

Renal involvementImpaired renal function demonstrated by serum creatinine above normal

PNH (paroxysmal nocturnal hemoglobinuria)

DefinitionParoxysmal nocturnal hemoglobinuria (PNH)

Clinical summaryHemolytic condition for which eculizumab/eculizumab biosimilar is used to reduce hemolysis

Background and Drug Information

Eculizumab is a monoclonal terminal complement inhibitor used to reduce intravascular hemolysis in paroxysmal nocturnal hemoglobinuria (PNH) and to treat atypical hemolytic uremic syndrome (aHUS) with laboratory evidence of hemolysis and impaired renal function. The policy notes that use must be supported by appropriate evidence sources and also lists important exclusions, including treatment of STEC‑HUS or TTP (ADAMTS‑13 activity <5%) and continuation after disease progression or when dosing exceeds the single‑dose maximums.

Biosimilar Coverage

Note

Coverage includes Soliris and the biosimilar Epysqli when criteria are met

Both the reference product Soliris (eculizumab) and the biosimilar Epysqli (eculizumab‑aagh) are covered under this policy when the coverage criteria are met.

Soliris (eculizumab)
Epysqli (eculizumab‑aagh)

Policy Summary

PayerNeighborhood Health Plan of Rhode Island

PolicyEculizumab products (Soliris, Epysqli) coverage criteria

Policy CodePolicy UM ONC_1382

Change TypeNo material change

Effective DateSeptember 27, 2024

Next Review DateN/A

Key ActionSubmit prior authorization requests to Evolent/Utilization Management for final approval by the Utilization Management Committee.

SourceLink

On This Page

{"text":"No additional medication(s) are being added to the continuation request; continuation is limited to the same agent and regimen previously approved."},

{"text":"For members with a diagnosis of atypical hemolytic uremic syndrome (aHUS), provider must include diagnostic documentation supporting the aHUS diagnosis (e.g., ADAMTS13 level to exclude TTP, complement testing results, genetic testing if available, and relevant clinical notes).","note":"Failure to submit aHUS diagnostic documentation may result in denial or delay."},

Provider should document consideration of step or alternate therapies where clinically appropriate (e.g., prior trials of approved alternatives, rationale for continuing current therapy over alternatives, contraindications or intolerance to alternatives).

If step therapy is required by the plan, documentation of trials of required alternatives or justification for bypass must be provided.

Prior Authorization

Prior Authorization Required / Documentation

Provider Action: Ensure all required documentation accompanies the continuation request to avoid denial or delay.

Include records showing prior use within the last 12 months (start/stop dates, dosing).
Include clinical progress notes documenting no disease progression and absence of intolerance or adverse effects.
Do not add new medications to the continuation request; submit a separate request for additions.
For aHUS, include ADAMTS13 results, complement testing, and relevant genetic or specialty consultation notes.
Document consideration of step/alternate therapies and provide rationale if bypassing step therapy requirements.

Documentation Required

Document confirmed aHUS with specific hemolysis and renal labs

For aHUS, provide documentation of a confirmed diagnosis plus laboratory evidence of hemolysis (LDH above normal, haptoglobin below normal, or schistocytes on peripheral smear) and impaired renal function (serum creatinine above normal).

Confirmed aHUS diagnosis
Evidence of hemolysis: LDH above normal OR low haptoglobin OR schistocytes on peripheral smear
Impaired renal function: serum creatinine above normal

Documentation Required

Document hemolytic PNH as the indication

Document hemolytic paroxysmal nocturnal hemoglobinuria (PNH) as the indication for therapy in the member’s record to support coverage.

Clinical documentation that the member has hemolytic PNH
Intent that eculizumab/eculizumab biosimilar is being used to reduce hemolysis

Denial Risk

Off‑label/investigational requests require sufficient evidence or may be denied

Requests for investigational or off‑label indications without sufficient supporting evidence (not supported by FDA labeling, CMS‑recognized compendia, NCCN/ASCO guidelines, or acceptable peer‑reviewed literature) may be denied.

Off‑label use must have supporting evidence from FDA label, CMS compendia, NCCN/ASCO, or acceptable peer‑reviewed literature
Insufficient evidence as defined in policy (e.g., lack of representative clinical characteristics, inadequate regimen representation, or lack of clinically meaningful outcomes) may prompt denial

Denial Risk

Excluded diagnoses — STEC‑HUS and TTP (ADAMTS‑13 <5%) not covered

Do not request coverage for STEC‑HUS or TTP with ADAMTS‑13 activity <5%; these diagnoses are not indicated and may be denied.

STEC‑HUS (Shiga toxin E. coli‑related HUS) is excluded
TTP defined as ADAMTS‑13 activity less than 5% is excluded

Billing Rule

Excess dosing — adhere to single‑dose maximums to avoid denial

Dosing that exceeds the single‑dose limits—900 mg for PNH and 1,200 mg for aHUS—may trigger denial of the request.

Single‑dose maximum for PNH: 900 mg
Single‑dose maximum for aHUS: 1,200 mg

Maximum single-dose: 1,200 mg

Authorization impactRequests exceeding these single-dose maximums may be excluded from coverage

Documentation requirement

Documentation of hemolytic PNH as the indication for therapy is required