CurrentNeighborhood Health Plan of Rhode IslandPolicy N/A

Um Onc_1325 Mylotarg Gemtuzumab Ozogamicin_08302024

Defines accepted indications, continuation and exclusion criteria, dosing limits, evidence requirements, and prior authorization/approval authority for use of Mylotarg (gemtuzumab ozogamicin) for Acute Myeloid Leukemia (AML) including pediatric use and relapsed/refractory disease.

Policy Summary

PayerNeighborhood Health Plan of Rhode Island

PolicyUm Onc_1325 Mylotarg Gemtuzumab Ozogamicin_08302024

Policy CodePolicy N/A

Change Typeclarified

Effective DateAug 30, 2024

Next Review Date

Key ActionUse must be supported by FDA labeling, CMS-recognized compendia, NCCN or ASCO guidelines, or peer-reviewed literature that meets CMS Medicare Benefit Policy Manual Chapter 15 requirements.

SourceLink

POLICY UPDATE CHANGES

Policy approval date August 14, 2024 and effective date August 30, 2024 noted; committee review history updated through 08/14/24.

1Primary Indication (AML)

2017-2024Committee review years

4Dose rules listed

Coverage Summary

Scope: This policy defines accepted indications, continuation and exclusion criteria, dosing limits, evidence requirements, and prior authorization/approval authority for Mylotarg (gemtuzumab ozogamicin) for the treatment of Acute Myeloid Leukemia (AML), including pediatric use and relapsed/refractory disease. Coverage is covered with criteria when the member has CD33-positive AML and Mylotarg is used as a single agent or in combination with chemotherapy for newly diagnosed AML (age 1 month and older) or relapsed/refractory AML (age 2 years and older) in members who have not previously received Mylotarg.

Medical Necessity - Acute Myeloid Leukemia (AML)

Covered when ALL of the following are met:

ALL of the following

ALL of the following
- ONE of
  - Used for newly diagnosed AML (age 1 month and older)
  - Used for relapsed/refractory AML (age 2 years and older) who have not received Mylotarg previously

Continuation Therapy Criteria

Continuation requests exemption

Continuation requests for a not-approvable medication shall be exempt provided ALL of the following:

ALL of the following

The requested medication was used within the last year
The member has not experienced disease progression and/or no intolerance to the requested medication
Additional medication(s) are not being added to the continuation request

Exclusions and Regimens Not Supported

Regimens Not Supported

Not supported by policy (these uses are not approved by Evolent):

ANY of the following

Induction therapy, less than 60 years of age: Fludarabine + HiDAC + idarubicin + G-CSF + gemtuzumab ozogamicin
Induction/Consolidation therapy, greater than or equal to 60 years of age: Single agent Mylotarg (gemtuzumab ozogamicin)

Exclusion Criteria

Not covered when ANY of the following are present:

ANY of the following

Disease progression on or following Mylotarg (gemtuzumab ozogamicin) or Mylotarg-containing regimen
Dosing in adult members exceeds single dose limit of Mylotarg combination therapy 3 mg/m2 (max dose is 4.5 mg) or 6 mg/m2 as single agent
- ANY of the following
  - 3 mg/m2 for pediatric members with BSA >= 0.6 m2
  - 0.1 mg/kg for pediatric members with BSA < 0.6 m2
Investigational use with an off-label indication that is not supported by sufficient evidence or not generally accepted by the medical community

Provider Actions & Prior Authorization

Documentation Required

Evidence supporting use required

Use must be supported by FDA labeling, CMS-recognized compendia, NCCN or ASCO guidelines, or peer-reviewed literature that meets CMS Medicare Benefit Policy Manual Chapter 15 requirements.

Prior Authorization

Utilization Management review required

Evolent (UM Dept) processes medication requests; medications not authorized by Evolent may be deemed not approvable and not reimbursable. Final approval authority resides with the Utilization Management Committee; Evolent UM handles review processing.

Denial Risk

Requests exceeding dose limits

Claims may be excluded/denied if single dose limits specified in policy are exceeded.

Adult combination therapy single dose limit: 3 mg/m2 (max dose 4.5 mg)
Adult single-agent single dose limit: 6 mg/m2
Pediatric single dose limit (BSA ≥ 0.6 m2): 3 mg/m2
Pediatric single dose limit (BSA < 0.6 m2): 0.1 mg/kg

Denial Risk

Investigational/off-label not supported by evidence

Requests for off-label indications lacking sufficient evidence or not supported by recognized compendia/guidelines may be denied as investigational.

Clinical Evidence & References

Evidence sources: Key references supporting covered uses include the FDA prescribing information (Pfizer 2021), randomized and phase III trials such as ALFA-0701 and COG AAML0531, and clinical compendia (NCCN 2024, Clinical Pharmacology 2024, Micromedex 2024). FDA label, NCCN/ASCO guidance, CMS-recognized compendia, and peer-reviewed trials form the evidentiary basis for the policy's covered uses.

The policy requires that uses be supported by FDA labeling, CMS-recognized compendia, NCCN or ASCO guidelines, or peer-reviewed literature meeting CMS Medicare Benefit Policy Manual Chapter 15 standards.

Definitions

Sufficient evidenceEvidence supported by FDA label, CMS-approved compendia, NCCN/ASCO guidelines, or peer-reviewed literature that meets CMS Medicare Benefit Policy Manual Chapter 15 standards; excludes case reports and abstracts without full articles.

CD33-positiveTumor expression of CD33 antigen as required for Mylotarg use in AML per policy.

Background

Background and scope: The policy establishes the intended scope to define accepted indications for Mylotarg including FDA-approved uses and off-label indications supported by recognized compendia or guidelines. Accepted indications include use as a single agent or in combination for CD33-positive AML in specified age groups and exclude regimens or indications not supported by sufficient evidence.

Utilization management: Evolent's UM Department processes medication requests and the Utilization Management Committee holds final approval authority; medications not authorized by Evolent may be deemed not approvable and not reimbursable.

Dose Thresholds

Adult combination therapy single dose limit3 mg/m2 (max dose 4.5 mg)

Adult single agent single dose limit6 mg/m2

Pediatric single dose limit (BSA >= 0.6 m2)3 mg/m2

Pediatric single dose limit (BSA < 0.6 m2)0.1 mg/kg