CurrentAlliance HealthPolicy 1S-13

Cell & Gene Therapies

NC Medicaid clinical coverage policy for FDA-approved cell and gene therapies, defining coverage criteria, exclusions, prior approval requirements, provider qualifications, CGT Access Model requirements, and claims/billing instructions (partial document: attachments and codes included).

Policy Summary

PayerAlliance Health

PolicyCell & Gene Therapies

Policy CodePolicy 1S-13

Change TypeDefinitions, PA & billing clarifications

Effective DateJan 1, 2025

Next Review Date

Key ActionObtain prior approval and submit PA with supporting records to the DHHS Utilization Review Contractor, using product-specific CPT/HCPCS (or J3590 if none) and attaching invoice/AAC/NDC as required.

POLICY UPDATE CHANGES

Added references to related clinical coverage policies and specified State Selected Model Drugs (LYFGENIA and CASGEVY).

Definitions updated to include Candidate Beneficiary, CMS-Designated Patient Registry, Model Beneficiary, Model Drug, QTC/ATC, SCD, SRA, VBP.

Added note that PA criteria may be more specific than FDA label and that PA criteria take precedence; if none, follow FDA label. Clarified J3590 use when no product-specific HCPCS exists.

Clarified repeat treatment non-coverage does not apply to therapies that require multiple infusions/doses as part of a single treatment course; updated Qualified Treatment Center language to include Authorized Treatment Center.

Attachment A amended to add ICD-10-PCS and HCPCS; added billing and reimbursement instructions requiring AAC submission, invoice attachment, and referencing NC Select Drug List for outpatient methodology.

Added requirement that CGT manufacturers be enrolled in Medicaid Drug Rebate Program (42 U.S.C. § 1396r-8 and 42 C.F.R. § 447.509).

Clarified CGT Access Model requirements for provider registry registration and beneficiary consent; payment contingent on continued compliance with Model requirements.

Amended Date: April 1, 2025 reflecting reimbursement methodology details for Cell & Gene Therapies.

FDA-approvedCovered Indications

PA requiredAuthorization Requirement

One-time (generally)Therapy Frequency

QTC/ATCSite Requirement

Inpatient & OutpatientService Settings Covered

PA/AAC/HCPCS/NDC/invoiceRequired claim elements

Coverage Summary

Policy: NC Medicaid Clinical Coverage Policy No. 1S-13 — Subject: Cell & Gene Therapies (Medicaid). Status: CURRENT. Original effective date: January 1, 2025; Amended date / last review: April 1, 2025.

Scope summary: NC Medicaid covers FDA-approved cell and gene therapies when medically necessary and when specific policy criteria are met; the policy defines coverage criteria, exclusions, prior approval requirements, provider qualifications, CGT Access Model requirements, and claims/billing instructions (attachments and codes included).

Key facts: Prior approval is required before rendering therapies and providers must use product-specific CPT/HCPCS codes (or J3590 if no HCPCS assigned). Generally therapies are treated as a one-time, lifetime treatment (except when multiple infusions/doses are part of a single course).

Key claims and payment requirements: inpatient and outpatient claims must include the PA number, HCPCS, NDC, AAC and attached manufacturer invoice; inpatient drug reimbursement is at the invoice (AAC) price with no markup, outpatient is reimbursed at the lesser of ASP (if available) or invoice price with no markup for therapies on the NC Select Drug List.

Key Policy Facts

Covered IndicationsFDA-approved therapies only

Authorization RequirementPrior approval required before rendering Cell & Gene Therapies; use product-specific CPT/HCPCS or J3590 if none

Therapy FrequencyOne-time lifetime treatment generally; exception for therapies requiring multiple infusions/doses as single course

Site RequirementAdministration at Qualified Treatment Center (QTC) or Authorized Treatment Center (ATC) when manufacturer requires

Required claim elementsPA number, AAC, HCPCS (or J3590), NDC, and manufacturer invoice must be submitted/attached

State-Selected Model DrugsLYFGENIA (lovotibeglogene autotemcel) and CASGEVY (exagamglogene autotemcel)

Effective / Last reviewEffective: 2025-01-01; Last review / Amended: 2025-04-01

Medical Necessity / Coverage Criteria

General Medical Necessity Criteria

Medicaid shall cover the procedure, product, or service related to this policy when medically necessary, and:

ALL of the following

The procedure, product, or service is individualized, specific, and consistent with symptoms or confirmed diagnosis of the illness or injury under treatment, and not in excess of the beneficiary's needs.
The procedure, product, or service can be safely furnished, and no equally effective and more conservative or less costly treatment is available statewide.
The procedure, product, or service is furnished in a manner not primarily intended for the convenience of the beneficiary, the beneficiary's caretaker, or the provider.

Specific Criteria Covered

Medicaid shall cover Cell & Gene Therapies when the beneficiary meets the following specific criteria:

Prior Authorization, Submission & Provider Obligations

Prior Authorization

Obtain prior approval before therapy

Obtain prior approval before therapy. PA number must be obtained and included on claims; attach the supplier invoice to the claim when instructed. Inpatient drug costs should be reported and reimbursed at invoice price; outpatient drug reimbursement will be at the lesser of average sales price (ASP) or invoice price. Follow state billing instructions for State-Selected Model Drugs. Failure to follow these billing and reporting requirements may result in claim denial or recoupment.

Codes affected: J3590 and therapy-specific HCPCS/NDC codes listed in Attachment A
Include PA number on all claims for the service
Attach the drug invoice to the claim when submitting for reimbursement
Inpatient reimbursement: report the drug cost and will be reimbursed at the invoice price
Outpatient reimbursement: reimbursed at the lesser of ASP or invoice price
Follow state billing instructions for State-Selected Model Drugs
Providers must report inpatient/outpatient drug costs on claims as required by payer reporting rules

Coding, Claim Submission & Reimbursement Conditions

ICD-10-PCS Product-Specific Codes (Inpatient PCS)ICD-10Covered

XW133H9	Transfusion of Lovotibeglogene Autotemcel into Peripheral Vein, Percutaneous Approach, New Technology Group 9
XW143H9	Transfusion of Lovotibeglogene Autotemcel into Central Vein, Percutaneous Approach, New Technology Group 9
XW133J8	Transfusion of Exagamglogene Autotemcel into Peripheral Vein, Percutaneous Approach, New Technology Group 8
XW143J8	Transfusion of Exagamglogene Autotemcel into Central Vein, Percutaneous Approach, New Technology Group 8
XW133G8	Transfusion of Atidarsagene Autotemcel into Peripheral Vein, Percutaneous Approach, New Technology Group 8
XW143G8	Transfusion of Atidarsagene Autotemcel into Central Vein, Percutaneous Approach, New Technology Group 8
XW133B8	Transfusion of Betibeglogene Autotemcel into Peripheral Vein, Percutaneous Approach, New Technology Group 8
XW143B8	Transfusion of Betibeglogene Autotemcel into Central Vein, Percutaneous Approach, New Technology Group 8

HCPCS / J-codes (Outpatient / PA submission)HCPCSCovered

J3590	Unclassified biologics (use when product-specific HCPCS not assigned)
J3392	Injection, exagamglogene autotemcel, per treatment
J3393	Injection, betibeglogene autotemcel, per treatment
J3394	Injection, lovotibeglogene autotemcel, per treatment
J3398	Injection, voretigene neparvovec-rzyl, 1 billion vector genomes
J3399	Injection, onasemnogene abeparvovec-xioi, per treatment, up to 5x10
J1411	Injection, etranacogene dezaparvovec-drlb, per therapeutic dose
J1413	Injection, delandistrogene moxeparvovec-rokl, per therapeutic dose

Claim Forms / Transactionsmixed

UB-04	Institutional claim form for inpatient and outpatient hospital services
837I	Electronic transaction for institutional claims

Required Drug Identifiers on Claimmixed

HCPCS	HCPCS code must be submitted on claim line for drug
NDC	National Drug Code must be submitted on claim line for drug

Pricing Methodologies Referencedmixed

AAC	Actual Acquisition Cost (per 42 CFR § 447.502) — submitted on claim and supported by invoice
ASP	Average Sales Price — used as comparator for outpatient reimbursement if available
DRG	Diagnosis-Related Group — inpatient reimbursement grouping
RCC	Ratio Cost to Charge — outpatient payment methodology

Inpatient Hospital Reimbursement Conditions

Reimbursement will follow the State Plan and apply when all of the following are met:

ALL of the following

Inpatient stay and services are related to the administration of Cell & Gene Therapies and billed on Institutional Claim (UB-04/837I).
Reimbursement uses existing diagnosis-related group (DRG) payment methodology and is based on the primary diagnosis code grouped to the appropriate DRG.
The prior authorization (PA) number is included on the claim.
PA number must be present on claim to be reimbursed under this methodology.
Actual Acquisition Cost (AAC) of the drug is submitted on the inpatient claim with corresponding HCPCS code and NDC.

Background & Definitions

Regulatory context: Gene and cell therapy products fall under the FDA Center for Biologics Evaluation and Research (CBER). Clinical investigation requires an IND and marketing approval requires a BLA; the policy references that therapies must be U.S. FDA–approved to be covered.

CMS CGT Access Model: The CMS Cell and Gene Therapy Access Model prioritizes gene therapy treatments for beneficiaries with sickle cell disease (SCD). Under the Model, states and CMS negotiate terms for Model Drugs and participating providers must register with a CMS-designated patient registry and obtain beneficiary consent for CMS-specified studies.

Manufacturer and registry requirements: The policy identifies State-Selected Model Drugs (NC's: LYFGENIA and CASGEVY) and requires that certain therapies be administered only at manufacturer-designated Qualified Treatment Centers (QTC) or Authorized Treatment Centers (ATC) when manufacturer policy dictates. Participating manufacturers must comply with Medicaid rebate enrollment and, for Model Beneficiaries, provide required supports such as fertility preservation payment per CMS in the CGT Access Model.

Term	Definition
Candidate Beneficiary
Beneficiary with documented sickle cell disease and whose primary payer for a State-Selected Model Drug is the State Medicaid program.
Model Beneficiary
Candidate Beneficiary who received infusion of a State-Selected Model Drug with State Medicaid as primary payer at time of infusion and VBP SRA in effect.
Model Drug / State-Selected Model Drug
FDA-approved gene therapy indicated for SCD chosen by the state for inclusion in the CGT Access Model; NC's are LYFGENIA and CASGEVY.
Qualified Treatment Center (QTC) / Authorized Treatment Center (ATC)
Facility meeting manufacturer and regulatory requirements to administer CGT; may be manufacturer designated.
CMS-designated patient registry
Data collection system identified/approved by CMS for tracking outcomes and safety; providers must participate when administering State-Selected Model Drugs.
Supplemental Rebate Agreement (SRA)
CMS-authorized supplemental rebate agreement as described in 42 C.F.R. § 447.502.
Value-Based Purchasing (VBP)
Value-based purchasing arrangement as described in 42 C.F.R. § 447.502.
Fertility Preservation Services
Safeguarding or storing eggs, sperm, or reproductive tissue for future biological children; generally not covered except manufacturer payment required for Model Beneficiaries under CGT Access Model per CMS.
Actual Acquisition Cost (AAC)
As defined in 42 CFR § 447.502; the cost submitted on the claim that must match the invoice.
NC Select Drug List
List of drugs subject to specific reimbursement methodology, located at the referenced Medicaid provider pharmacy services URL.

Additional Coverage Items & Exclusions

Remaining / Model Beneficiary & NEMT Exceptions

If additional criteria trees remain, surface noted exceptions; otherwise include fertility preservation exception for Model Beneficiaries and NEMT exclusions.

ALL of the following

Fertility preservation services are not covered generally but CMS requires participating manufacturers to provide payment for fertility preservation services for a Model Beneficiary who receive therapies under the CGT Access Model.
Non-Emergency Medical Transportation (NEMT) is covered for medical appointments but NEMT is not covered for fertility preservation service appointments.

Revision History

Amended Date: April 1, 2025addedLatest

Definitions updated to include Candidate Beneficiary, CMS-Designated Patient Registry, Model Beneficiary, Model Drug, QTC/ATC, SCD, SRA, VBP. Added references to related clinical coverage policies and specified State Selected Model Drugs (LYFGENIA and CASGEVY).

Amended Date: April 1, 2025revisedLatest

Clarified PA request process and use of J3590 when no HCPCS assigned; noted PA criteria may be more specific than FDA label and take precedence.

Amended Date: April 1, 2025

Policy Summary

PayerAlliance Health

PolicyCell & Gene Therapies

Policy CodePolicy 1S-13

Change TypeDefinitions, PA & billing clarifications

Effective DateJan 1, 2025

Next Review Date

On This Page

ALL of the following

The Cell or Gene Therapy has received approval from the United States Food & Drug Administration (U.S. FDA).
Administration per label: Therapy is administered per the U.S. FDA approved label regarding indications and usage; dosage and administration; dosage forms and strengths; and warnings and precautions.
Note: If prior approval (PA) criteria have been established for a Cell or Gene Therapy they take precedence over the FDA label; if no PA criteria exist, follow the FDA label.
Administration occurs at a Qualified Treatment Center (QTC) or Authorized Treatment Center (ATC) that has received approval for administering the therapy, if required by the manufacturer.

Medicaid Additional Coverage

Additional coverage items

ANY of the following

Non-Emergency Medical Transportation (NEMT) for beneficiary transportation to medical appointments (subject to NEMT policy).
See NEMT policy: https://medicaid.ncdhhs.gov/NEMT-policy.

General Criteria Not Covered

Medicaid shall not cover when:

ANY of the following

The beneficiary does not meet the eligibility requirements listed in Section 2.0.
The beneficiary does not meet the criteria listed in Section 3.0.
The procedure, product, or service duplicates another provider's procedure, product, or service.
The procedure, product, or service is experimental, investigational, or part of a clinical trial.

Specific Criteria Not Covered

Medicaid shall not cover Cell & Gene Therapies for ANY one of the following:

ANY of the following

The Cell or Gene Therapy has not received approval from the U.S. FDA.
Administration outside label: Administered outside the U.S. FDA label regarding indications and usage; dosage and administration; or dosage forms and strengths.
Administered at a facility that has not been approved as a QTC or ATC for that therapy, when required by the manufacturer.
Repeat treatment in a beneficiary who has received the same or another Cell or Gene Therapy previously, except for therapies that require multiple infusions or doses as part of a single treatment course.
When the beneficiary's psychosocial history limits the beneficiary's ability to comply with pre- and post-infusion medical care.
Current beneficiary or caretaker non-compliance that would make compliance with a disciplined medical regime improbable.

Medicaid Additional Not Covered Items

Additional items not covered by Medicaid under this policy

ALL of the following

Fertility preservation services associated with Cell & Gene Therapy administration.
NEMT for fertility preservation service appointments.
NOTE: CMS requires participating manufacturers to provide payment for fertility preservation services for a Model Beneficiary who receive therapies administered under the CGT Access Model.

Documentation Required

Submit supporting records with PA

Submit supporting clinical records and administrative documentation with the PA request and retain copies for claims-level audits. Claims submitted without the required supporting documentation or invoice may be denied.

Attach: clinical notes, diagnostic test results, prior treatments, FDA label (if applicable), and the drug invoice with PA submission when requested
For J3590, supply the drug name, manufacturer, formulation, and invoice to substantiate the charge
Retain all supporting records; provide them upon request for retrospective review and audits

Documentation Required

Registry registration and consent for Model Beneficiaries

Registry registration and documented informed consent for Model Beneficiaries must be completed prior to billing. Provide registry documentation with the PA and include evidence of consent in the medical record and with claims when required.

Register beneficiary in the CMS-designated patient registry and include registry ID with the PA submission
Document informed consent that meets CMS/state Model Beneficiary requirements and retain in the medical record
Claims for Model Drugs submitted without registry documentation and consent may be denied until documentation is provided

AAC as defined in 42 CFR § 447.502 must equal invoice drug cost.

An invoice is attached to the claim to support the drug cost and the AAC submitted must equal the drug cost on the invoice.

Drug will be reimbursed at the attached invoice price with no markup.

Outpatient Hospital Reimbursement Conditions

Reimbursement will follow the State Plan and apply when all of the following are met:

ALL of the following

Outpatient stay and services are related to the administration of Cell & Gene Therapies and billed on Institutional Claim (UB-04/837I).
Reimbursement uses existing Ratio Cost to Charge (RCC) outpatient payment methodology.
The prior authorization (PA) number is included on the claim.
Actual Acquisition Cost (AAC) of the drug is submitted on the outpatient claim with corresponding HCPCS code and NDC.
AAC per 42 CFR § 447.502 must equal invoice drug cost.
An invoice is attached to the claim to support the drug cost and the AAC submitted must equal the drug cost on the invoice.
The drug will be reimbursed at the lesser of the Average Sales Price (ASP) (if available) or the attached invoice price, with no markup.
Only Cell & Gene Therapies on the NC Select Drug List are subject to the above outpatient reimbursement methodology; all other covered Cell & Gene Therapies follow standard payment methodology.
Refer to NC Select Drug List at https://medicaid.ncdhhs.gov/providers/pharmacy-services.

Billing Rule

Use product-specific inpatient PCS or most specific code

File inpatient claims with the product-specific ICD-10-PCS code if assigned; if not assigned, file with the most specific available billing code. Include the prior authorization (PA) number and NDC on the claim and attach the manufacturer's invoice to support the drug cost.

Use product-specific ICD-10-PCS codes when assigned (examples listed below).
Include PA number and NDC on the inpatient claim.
Attach manufacturer invoice to the claim to support drug cost.

Billing Rule

Outpatient billing and PA number

File outpatient claims with the product-specific HCPCS code; if none assigned use HCPCS J3590 (UNCLASSIFIED BIOLOGICS). Include the prior authorization (PA) number and NDC on the claim and attach the manufacturer's invoice. HCPCS J-codes (or J3590 when applicable) should be reported on the claim line.

Use product-specific HCPCS for outpatient claims; if none, use J3590.
Include PA number and NDC on the outpatient claim.
Attach manufacturer invoice to the claim; report HCPCS (or J3590) on the claim line.

Billing Rule

Submit AAC with invoice, HCPCS, and NDC

Submit the Actual Acquisition Cost (AAC) of the drug on the claim with the corresponding HCPCS code and NDC and attach the manufacturer invoice; the AAC must equal the invoice drug cost. Attach the invoice to support the acquisition cost. Include the prior authorization (PA) number on the claim. Inpatient drugs will be reimbursed at the attached invoice price with no markup; outpatient drugs will be reimbursed at the lesser of ASP (if available) or the attached invoice price, with no markup.

Submit AAC on the claim line with corresponding HCPCS and NDC and attach invoice; AAC must equal invoice drug cost.
Attach manufacturer invoice to the claim to support drug cost; claims lacking an invoice or with AAC not matching invoice may be at risk of denial or incorrect reimbursement.
Include the PA number on both inpatient and outpatient claims.
Inpatient reimbursement: drug paid at attached invoice price, no markup.
Outpatient reimbursement: drug paid at the lesser of ASP (if available) or attached invoice price, no markup.

Clarified repeat treatment exclusion exception for therapies requiring multiple infusions/doses as part of single course and updated QTC language to include ATC and manufacturer-specific requirement.

Amended Date: April 1, 2025revisedLatest

Attachment A amended to add ICD-10-PCS and HCPCS; removed NDCs from one section; reimbursement terminology updates and added Medicaid Drug Rebate Program enrollment requirement for CGT manufacturers (42 U.S.C. § 1396r-8 and 42 C.F.R. § 447.509).

Amended Date: April 1, 2025clarifiedLatest

Specified billing instructions and payment contingent on compliance; clarified CGT Access Model requirements for provider registry registration and beneficiary consent.

Amended Date: April 1, 2025revisedLatest

Clarifies inpatient reimbursement at invoice price and outpatient reimbursement at lesser of ASP or invoice for therapies on NC Select Drug List; requires AAC submission and invoice attachment.