CurrentNeighborhood Health Plan of Rhode IslandPolicy N/A

Adakveo

Defines accepted indications, clinical criteria, exclusions, coding, and continuity rules for Adakveo (crizanlizumab-tmca) use primarily for sickle cell disease in members aged 16 and older across Commercial, Exchange/Marketplace, and Medicaid lines of business. Also describes evidence sources required for off-label uses and continuation rules.

Policy Summary

PayerNeighborhood Health Plan of Rhode Island

PolicyAdakveo

Policy CodePolicy N/A

Change TypeRevised (template conversion)

Effective Date

Next Review Date

Key ActionFor off-label uses, document adequate evidence per FDA labeling, CMS-recognized compendia, NCCN/ASCO guidelines, or peer-reviewed literature meeting CMS Medicare Benefit Policy Manual Chapter 15 standards.

SourceLink

POLICY UPDATE CHANGES

Converted to new Evolent guideline template and replaced prior UM ONC_1375 Adakveo (crizanlizumab).

1Covered Indication (Sickle Cell Disease)

4Exclusion criteria listed

1HCPCS drug code

Coverage Summary

Coverage stance: covered_with_criteria for Adakveo (crizanlizumab-tmca) for the prevention of vaso-occlusive crises in sickle cell disease. Scope: applies to Commercial, Exchange/Marketplace, and Medicaid lines of business for members aged ≥ 16 years. Key initial-therapy criteria include diagnosis of sickle cell disease (HbSS, HbSC, HbS/beta0-thalassemia, HbS/beta+thalassemia, or other genotypes) and ≥ 2 sickle cell–related pain crises in the past 12 months, with Hydroxyurea requirements when applicable (prescribed ≥ 6 months and on a stable dose ≥ 3 months). Off-label uses must be supported by FDA labeling, CMS-recognized compendia, NCCN/ASCO guidelines, or peer-reviewed literature that meet the CMS Medicare Benefit Policy Manual Chapter 15 standards.

Initial Therapy — Medical Necessity

Initial Therapy - Sickle Cell Disease

Adakveo (crizanlizumab-tmca) may be used when ALL of the following are met:

ALL of the following

Member is aged 16 years or older>= 16 years
Diagnosis of sickle cell disease (HbSS, HbSC, HbS/beta0-thalassemia, HbS/beta+thalassemia, or other less common genotypes)
Two or more sickle cell-related pain crises in the past 12 months>= 2 in past 12 months
Hydroxyurea requirement
- If receiving Hydroxyurea, it must have been prescribed for at least 6 months>= 6 months prescribed (if receiving Hydroxyurea)
- If receiving Hydroxyurea, the patient must be on a stable dose of Hydroxyurea for at least 3 monthsstable dose >= 3 months (if receiving Hydroxyurea)

Continuation Therapy

Continuation requests exempt from Evolent policy when ALL of the following are met:

ALL of the following

Member has not experienced disease progression on the requested medication
See definition: continuation request exemption
Requested medication was used within the last year without a lapse of more than 30 days of having an active authorization
See definition: continuation request exemption
No additional medications are being added to the continuation request
See definition: continuation request exemption

Exclusion Criteria / Not Medically Appropriate

Adakveo is excluded when ANY of the following apply:

ANY of the following

Adakveo is being used after disease progression with the same regimen
Receiving chronic anticoagulation therapy (e.g., warfarin, heparin) other than aspirin
Member has a hemoglobin level less than 4.0 g/dL< 4.0 g/dL
Dosing exceeds single dose limit of Adakveo (crizanlizumab-tmca) 5 mg/kg> 5 mg/kg
Investigational/Insufficient Evidence: Investigational use for an off-label indication that is not supported by CMS-recognized compendia or acceptable peer-reviewed literature
Evidence insufficiency considerations included (e.g., representativeness, regimen representation, clinically meaningful outcomes per ASCO definitions, study design, adequacy of non-randomized trials, limits of case reports/abstracts)

Provider Actions & Documentation

Documentation Required

Support for off-label use

Off‑label indications must be supported by FDA labeling, CMS‑recognized compendia, NCCN or ASCO clinical guidelines, or peer‑reviewed literature that meets CMS Medicare Benefit Policy Manual Chapter 15 standards. Documentation should explicitly state the evidence used, why it is adequate for the requested off‑label use, and how the patient is represented in the supporting evidence (e.g., disease characteristics and regimen match).

Include an assessment of evidence adequacy and representativeness (patient characteristics, regimen, and clinically meaningful outcomes).

Documentation Required

Continuation request documentation

For continuation requests, document that the member has not experienced disease progression while on the requested medication; that the medication was used within the last year without a lapse of more than 30 days in active authorization; and that no additional medications are being added to the continuation request.

Continuation criteria definition: no progression, prior use within last 12 months without >30‑day lapse, and no added medications.

Denial Risk

Exclusion-based denial risks

Claims may be denied if any exclusion criterion is met. Exclusions include use after disease progression on the same regimen; concurrent chronic anticoagulation other than aspirin (e.g., warfarin, heparin); hemoglobin less than 4.0 g/dL; dosing that exceeds the single‑dose limit of 5 mg/kg; or investigational/off‑label use lacking sufficient evidence or not accepted by the medical community.

Affected code: J0791

Clinical Evidence & References

Evidence: Key trial: Ataga KI et al., NEJM 2017 (Crizanlizumab for prevention of pain crises). Prescribing information: Adakveo prescribing information, Novartis 2024. Note: off-label indications require support from FDA labeling, CMS-recognized compendia, NCCN/ASCO guidelines, or acceptable peer-reviewed literature meeting Medicare Benefit Policy Manual Chapter 15 standards.

Background

Background: Adakveo (crizanlizumab-tmca) is indicated for prevention of vaso-occlusive crises in sickle cell disease for patients aged ≥ 16 years who have had ≥ 2 pain crises in the prior 12 months; if on Hydroxyurea it must have been prescribed ≥ 6 months and be on a stable dose ≥ 3 months. Off-label use of Adakveo is permitted only when supported by FDA labeling, CMS-recognized compendia, NCCN/ASCO guidelines, or peer-reviewed literature that meets the CMS Medicare Benefit Policy Manual Chapter 15 standards; investigational or off-label indications lacking sufficient evidence as defined in the policy (representativeness, regimen representation, clinically meaningful outcomes, study design, and quality of evidence) are excluded.