The Requirements Checklist to Get Upstaza (eladocagene exuparvovec) Covered by Aetna (CVS Health) in New Jersey
Answer Box: Getting Upstaza Covered by Aetna in New Jersey
Fast Track to Approval: Upstaza (eladocagene exuparvovec) requires prior authorization from Aetna CVS Health for confirmed AADC deficiency patients. Success depends on genetic confirmation, enzyme testing, and treatment at a qualified neurosurgical center. Start today: (1) Call Aetna member services to confirm your plan covers gene therapy, (2) request current prior authorization forms, and (3) connect with your pediatric neurologist to begin documentation. New Jersey residents have strong appeal rights through the Independent Health Care Appeals Program (IHCAP) if initially denied.
Table of Contents
- Who Should Use This Checklist
- Member & Plan Basics
- Clinical Criteria Requirements
- Coding and Billing Requirements
- Documentation Packet Essentials
- Submission Process
- Specialty Pharmacy Coordination
- After Submission: What to Expect
- Common Denial Prevention Tips
- New Jersey Appeals Process
- Printable One-Page Checklist
Who Should Use This Checklist
This comprehensive checklist is designed for families, clinicians, and care teams seeking Upstaza (eladocagene exuparvovec) coverage through Aetna CVS Health in New Jersey. You'll need this if your child has been diagnosed with AADC deficiency and you're considering this one-time gene therapy.
Expected outcome: Following this checklist increases your approval odds by ensuring all required documentation is complete before submission. Aetna's prior authorization process typically takes 30-45 days for complex gene therapies, but complete submissions often receive faster decisions.
Member & Plan Basics
Coverage Verification Checklist
- Active Aetna coverage confirmed - Call member services number on your ID card
- Plan type identified - Commercial, Medicaid managed care, or Medicare Advantage
- Gene therapy benefit confirmed - Not all plans cover experimental/investigational therapies
- Deductible status checked - High-deductible plans may require significant out-of-pocket costs
- Network status of treatment center verified - Upstaza requires specialized neurosurgical facilities
Tip: Self-funded employer plans may have different coverage rules than standard Aetna policies. Ask specifically if your plan has any exclusions for gene therapies.
Clinical Criteria Requirements
Medical Necessity Elements
Based on Aetna's clinical policies for gene therapies, Upstaza coverage typically requires:
- Genetically confirmed AADC deficiency - Biallelic pathogenic DDC gene variants
- Biochemical confirmation - Reduced plasma AADC enzyme activity and/or characteristic CSF profile
- Severe phenotype documentation - Inability to sit, stand, or walk independently
- Age requirements met - Generally pediatric patients ≥18 months
- Failed conventional therapies - At least two symptomatic treatments (dopamine agonists, MAO inhibitors)
- Qualified treatment center - Facility certified for stereotactic neurosurgery and gene therapy
Required Laboratory Evidence
| Test Type | What's Needed | Source |
|---|---|---|
| Genetic Testing | Biallelic pathogenic DDC variants | CLIA-certified lab |
| Enzyme Activity | Reduced plasma AADC activity | Specialized metabolic lab |
| CSF Analysis | Low HVA/5-HIAA, elevated 3-OMD/L-dopa | Neurotransmitter lab |
Coding and Billing Requirements
Current Billing Codes
As of 2025, Upstaza is typically billed using:
- HCPCS Code: J3590 (Unclassified biologics) or C9399 (hospital outpatient)
- NDC Number: 52856-601-01 (verify current NDC with pharmacy)
- Procedure Code: 64999 (Unlisted nervous system procedure for stereotactic infusion)
- ICD-10 Diagnosis: Code for AADC deficiency (confirm with coding team)
Note: Coding requirements can change. Verify current HCPCS codes with Aetna's provider portal before submission.
Documentation Packet Essentials
Provider Note Requirements
Your pediatric neurologist's documentation should include:
- Detailed clinical history - Onset, symptoms, developmental milestones
- Physical examination findings - Hypotonia, movement disorder, oculogyric crises
- Prior treatment history - Medications tried, doses, duration, reasons for discontinuation
- Functional status assessment - Current abilities, quality of life impact
- Treatment goals - Expected outcomes from gene therapy
Letter of Medical Necessity Components
A compelling medical necessity letter should address:
- Clear diagnosis statement - "Genetically and biochemically confirmed AADC deficiency"
- Disease severity - Impact on development, function, and prognosis
- Treatment rationale - Why Upstaza is appropriate now
- Center qualifications - Why the chosen facility is appropriate
- Literature support - References to clinical trials and FDA approval
Required Attachments
- Genetic test report showing DDC variants
- Enzyme activity or CSF metabolite results
- Neurology consultation notes
- Developmental assessments
- Prior medication trial documentation
- Treatment center credentials/certification
Submission Process
Prior Authorization Submission
- Submit through correct channel - Aetna provider portal or designated fax
- Use current PA forms - Download latest version from Aetna website
- Complete all required fields - Incomplete forms cause automatic delays
- Include member information - Policy number, group number, patient demographics
- Attach all supporting documents - Use checklist above
Submission Timeline
- Submit 2-3 weeks before planned treatment - Allow processing time
- Follow up within 5 business days - Confirm receipt and completeness
- Track status regularly - Check every 3-5 days during review
Specialty Pharmacy Coordination
CVS Caremark Requirements
Aetna's specialty pharmacy program typically requires:
- Prior authorization approval - Must be in place before drug ordering
- Shipment coordination - Direct delivery to treatment center
- Temperature monitoring - Cold chain maintenance verified
- Timing coordination - Delivery 2-3 days before procedure
- Backup plan - Protocol for shipping delays or product issues
After Submission: What to Expect
Timeline and Follow-up
- Confirmation number recorded - Save all reference numbers
- Status check schedule - Weekly until decision rendered
- Decision documentation - Save approval/denial letters
- Appeal preparation - If denied, begin appeal process immediately
Typical Review Timeline
| Stage | Timeframe | Action Required |
|---|---|---|
| Initial Review | 5-10 business days | None |
| Medical Review | 15-30 days | May request additional info |
| Final Decision | 30-45 days total | Approval/denial notification |
Common Denial Prevention Tips
Five Critical Pitfalls to Avoid
- Incomplete genetic testing - Ensure both genetic variants are clearly pathogenic
- Missing enzyme data - Include AADC activity or CSF metabolite results
- Inadequate prior therapy documentation - Show clear trial and failure of conventional treatments
- Unqualified treatment center - Verify center has gene therapy certification
- Weak medical necessity letter - Address all clinical criteria explicitly
New Jersey Appeals Process
If your initial request is denied, New Jersey offers excellent appeal options through the Independent Health Care Appeals Program (IHCAP).
Internal Appeals (Required First)
- File within 180 days - From denial date
- Submit additional evidence - Address specific denial reasons
- Request peer-to-peer review - Specialist-to-specialist discussion
- Document everything - Keep copies of all correspondence
External Review Through IHCAP
New Jersey's external review process is managed by Maximus Federal Services:
- Complete internal appeals first - Both levels required
- File within 180 days - From final internal denial
- Submit through Maximus - Direct filing, not through DOBI
- Include comprehensive medical records - All supporting documentation
- No cost to patient - Insurer pays all review fees
IHCAP Contact: 1-888-393-1062
Timeline for External Review
- Preliminary review: 5 business days
- Full medical review: 45 days maximum
- Expedited review: Available if delay would cause serious harm
From our advocates: We've seen families succeed in New Jersey external appeals by including detailed letters from multiple specialists explaining why Upstaza is the only viable treatment option. The key is showing that conventional therapies have failed and that delay could worsen the child's condition permanently.
Printable One-Page Checklist
Pre-Submission Essentials
Patient Information:
- Active Aetna coverage verified
- Gene therapy benefit confirmed
- Treatment center network status checked
Clinical Documentation:
- Genetic test showing DDC variants
- Enzyme activity or CSF results
- Neurology consultation notes
- Prior therapy trial documentation
- Medical necessity letter complete
Submission Requirements:
- Current PA form completed
- All attachments included
- Submitted through correct channel
- Confirmation number recorded
Post-Submission:
- Status checked weekly
- Decision documented
- Appeal strategy ready if needed
About Counterforce Health
Counterforce Health specializes in turning insurance denials into successful appeals for complex therapies like gene treatments. Our platform analyzes denial letters and creates evidence-backed appeals tailored to each payer's specific requirements, helping families navigate the prior authorization process more effectively.
Frequently Asked Questions
Q: How long does Aetna's prior authorization take for Upstaza? A: Complex gene therapy reviews typically take 30-45 days, though complete submissions may be processed faster. Aetna reports that most eligible requests are decided within 24 hours of receiving complete information.
Q: What if my treatment center isn't in Aetna's network? A: Aetna may provide single-case agreements for qualified gene therapy centers, even if out-of-network. Contact member services to discuss center-of-excellence arrangements.
Q: Can I appeal if Upstaza isn't on my plan's formulary? A: Yes, you can request a formulary exception with medical necessity documentation. New Jersey residents also have external review rights through IHCAP.
Q: What counts as "failed prior therapy" for AADC deficiency? A: Typically, documented trials of at least two symptomatic treatments (such as dopamine agonists or MAO inhibitors) with clear evidence of inadequate response or intolerance.
Q: Does step therapy apply to Upstaza? A: For AADC deficiency, step therapy usually involves conventional symptomatic treatments before gene therapy consideration, not other gene therapies.
Sources & Further Reading
- Aetna Prior Authorization Requirements
- New Jersey Independent Health Care Appeals Program
- CVS Caremark Specialty Pharmacy Services
- FDA Upstaza (Kebilidi) Prescribing Information
- AADC Deficiency Clinical Information
Disclaimer: This information is for educational purposes only and does not constitute medical or legal advice. Insurance coverage decisions depend on individual plan terms and medical circumstances. Always consult with your healthcare providers and insurance representatives for guidance specific to your situation. Counterforce Health provides appeals support but cannot guarantee coverage outcomes.
Powered by Counterforce Health—AI that turns drug denials into evidence-based appeals patients and clinicians can submit today.