CurrentNeighborhood Health Plan of Rhode IslandPolicy N/A

Ayvakit (avapritinib) utilization guideline — coverage criteria

Defines accepted indications, coverage criteria, exclusions, coding, and prior authorization expectations for Ayvakit (avapritinib) for payers and providers requesting medication authorization.

Policy Summary

PayerNeighborhood Health Plan of Rhode Island

PolicyAyvakit (avapritinib) utilization guideline — coverage criteria

Policy CodePolicy N/A

Change TypeNo material changes

Effective DateN/A

Next Review DateN/A

Key ActionSubmit prior authorization and supporting documentation (FDA labeling, CMS compendia, NCCN/ASCO guidance or peer‑reviewed literature) and ensure continuation requests meet criteria.

SourceLink

POLICY UPDATE CHANGES

No material clinical or coverage changes in this revision.

PDGFRA exon 18GIST molecular indication

≥50Minimum platelet

J8999Billing code

Commercial/MedicaidLines of business

Coverage Criteria for Ayvakit (avapritinib)

GIST (PDGFRA exon 18 mutant) — Initial coverage

Policy Summary

PayerNeighborhood Health Plan of Rhode Island

PolicyAyvakit (avapritinib) utilization guideline — coverage criteria

Policy CodePolicy N/A

Change TypeNo material changes

Effective DateN/A

Next Review DateN/A

Key ActionSubmit prior authorization and supporting documentation (FDA labeling, CMS compendia, NCCN/ASCO guidance or peer‑reviewed literature) and ensure continuation requests meet criteria.

SourceLink

On This Page

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The policy lists specific situations where Ayvakit (avapritinib) is excluded from coverage. Exclusions include use after disease progression on the same regimen, use for PDGFRA wild‑type GIST, and concurrent administration with another tyrosine kinase inhibitor (examples cited: imatinib, sunitinib, regorafenib, sorafenib, dasatinib, pazopanib). Dose‑related limits are enforced: single‑dose maximums of 300 mg for GIST, 200 mg for advanced systemic mastocytosis, and 25 mg for indolent systemic mastocytosis, and monthly tablet limits (various strengths) must not be exceeded. Finally, investigational or off‑label uses lacking sufficient evidence are excluded.

The policy defines criteria for adequate evidence and unacceptable evidence types. Requests based only on case reports or meeting abstracts without full peer‑reviewed publications are specifically noted as insufficient. The guideline explains that acceptable evidence should be supported by CMS‑recognized compendia or peer‑reviewed literature and should address whether the patient population, regimen, and clinically meaningful outcomes are appropriately represented in the published studies.

Use of Ayvakit for indications that are not supported by the policy’s evidence standards is considered investigational or off‑label and is therefore not approvable. The policy explicitly identifies PDGFRA wild‑type GIST as a noncovered use and states that investigational/off‑label requests lacking sufficient supporting evidence are not medically necessary.

The policy clarifies what evidence is required to consider off‑label uses: supportive data must come from CMS‑recognized compendia or acceptable peer‑reviewed literature. It further states that case reports are generally inadequate and that abstracts without full peer‑reviewed articles do not provide sufficient clinical evidence to establish an accepted use.

Regimen	Indication	Coverage status
Avapritinib monotherapy (dosing limits apply by indication)	Unresectable or metastatic gastrointestinal stromal tumor (GIST) with documented PDGFRA exon 18 mutation (including PDGFRA D842V)	Covered with criteria

Regimen	Indication / requirement	Coverage status
Avapritinib monotherapy (platelet threshold applies)	Indolent systemic mastocytosis (ISM) OR advanced systemic mastocytosis (AdvSM) including SM-AHN or mast cell leukemia (MCL); member's platelet count >= 50 × 10^9 /L prior to start of therapy	Covered with criteria

Provider Actions, Authorization & Documentation

Prior Authorization

Prior Authorization Required

Prior authorization is required for Ayvakit (avapritinib). Continuation (renewal) requests may be considered when there has been no disease progression on the requested medication, the medication was used within the last year without a lapse of more than 30 days of active authorization, and no additional medications are being added to the continuation request.

Prior authorization required
Continuation allowed if no disease progression and continuous prior use (<= 30 day lapse) within last 12 months
No additional medications may be added to continuation requests

Documentation Required

Required Supporting Documentation

Requests must be supported by one or more of the following: FDA-approved product labeling, CMS-recognized compendia, NCCN or ASCO clinical guidelines, or acceptable peer-reviewed literature that meets CMS standards. Documentation should include citations (e.g., guideline or compendium references) and relevant clinical information demonstrating the patient meets the referenced indication or evidence basis.

Acceptable evidence: FDA label, CMS-recognized compendia, NCCN/ASCO guidelines, or peer-reviewed literature meeting CMS Medicare Benefit Policy Manual Chapter 15 standards
Include specific citations and clinical rationale in the request

Denial Risk

Concurrent TKI Use Excluded

Concurrent use of Ayvakit (avapritinib) with another tyrosine kinase inhibitor is excluded and will result in denial. Requests indicating combination therapy with agents such as imatinib, sunitinib, regorafenib, sorafenib, dasatinib, pazopanib, or other TKIs should be denied unless clear clinical justification and explicit prior approval documentation demonstrating safety and medical necessity are provided (which is not supported by this policy).

Concurrent TKI use excluded (e.g., imatinib, sunitinib, regorafenib, sorafenib, dasatinib, pazopanib)
Single-agent use expected per policy; concurrent TKI therapy is a denial trigger

Denial Risk

Denial / Exclusion Triggers

Requests for investigational or off-label uses must have sufficient supporting evidence as defined by CMS compendia or acceptable peer-reviewed literature. Use after disease progression on the same regimen, use in PDGFRA wild-type GIST, dosing above single-dose or monthly tablet limits, or investigational indications without adequate evidence are exclusion criteria and may be denied.

Excluded: use after disease progression on same regimen
Excluded: PDGFRA wild-type GIST
Excluded: dosing above single-dose or monthly tablet limits
Excluded: investigational/off-label uses lacking sufficient CMS-compendia or peer-reviewed evidence

Background

Avapritinib (Ayvakit) is an oral tyrosine kinase inhibitor approved for molecularly defined indications and intended for single‑agent (monotherapy) use in the covered indications. The policy supports use for gastrointestinal stromal tumor (GIST) with a documented PDGFRA exon 18 mutation, including D842V, and for specified subtypes of systemic mastocytosis (indolent SM and advanced SM including SM‑AHN and mast cell leukemia).

The policy emphasizes monotherapy dosing and enforces indication‑specific dose limits and monitoring requirements (for example, platelet thresholds for systemic mastocytosis are specified elsewhere in the guideline). Concurrent use with other tyrosine kinase inhibitors is excluded, reflecting that Ayvakit is intended as a single‑agent targeted therapy in these indications.

Definitions and Biomarker Requirements

inv-12: PDGFRA exon 18 mutant GIST — definition

DefinitionGastrointestinal stromal tumor (GIST) with documented PDGFRA exon 18 mutation, including PDGFRA D842V

Disease settingUnresectable or metastatic GIST

Therapy typeAvapritinib (Ayvakit) may be used as monotherapy for this mutation-defined GIST

inv-13: Systemic Mastocytosis subtypes — ISM and AdvSM including SM-AHN and mast cell leukemia

Indolent systemic mastocytosis (ISM)Included as an indication for avapritinib monotherapy

Advanced systemic mastocytosis (AdvSM)Included (examples: systemic mastocytosis with an associated hematological neoplasm [SM-AHN], or mast cell leukemia [MCL])

Therapy typeAvapritinib is specified as monotherapy for these SM subtypes

inv-15: PDGFRA exon 18 (including D842V) — biomarker requirement

Required biomarkerPDGFRA exon 18 mutation (including D842V)

Indication tied to biomarkerRequired for coverage of avapritinib in unresectable or metastatic GIST

Therapy implicationMonotherapy use specified when PDGFRA exon 18 mutation is present

OpenPayer

Ayvakit (avapritinib) utilization guideline — coverage criteria

Coverage Criteria for Ayvakit (avapritinib)

Coding and Billing Information

Provider Actions, Authorization & Documentation

Background

Definitions and Biomarker Requirements

Line of Therapy