By Counterforce Health Team in Upstaza (eladocagene exuparvovec) —

The Requirements Checklist to Get Upstaza (Eladocagene Exuparvovec) Covered by UnitedHealthcare in Pennsylvania: Complete Prior Authorization Guide

Answer Box: Getting Upstaza Covered by UnitedHealthcare in Pennsylvania

To get Upstaza (eladocagene exuparvovec) covered by UnitedHealthcare in Pennsylvania, you'll need: genetic confirmation of biallelic DDC mutations, documented AADC deficiency symptoms, prior authorization through OptumRx, and treatment at a certified gene therapy center. Start today by: confirming your plan type (fully-insured vs. self-funded), gathering genetic test results and clinical documentation, then submitting the OptumRx prior authorization form via fax to 1-844-403-1027. If denied, Pennsylvania's external review process has a 50% overturn rate for advanced therapies.

Table of Contents

  1. Who Should Use This Checklist
  2. Member & Plan Basics
  3. Clinical Criteria Requirements
  4. Coding & Documentation
  5. Documentation Packet Essentials
  6. Submission Process
  7. Gene Therapy Centers in Pennsylvania
  8. After Submission: What to Track
  9. Common Denial Pitfalls
  10. Appeals Process for Pennsylvania
  11. Printable Checklist

Who Should Use This Checklist

This guide is designed for patients, families, and clinicians seeking UnitedHealthcare coverage for Upstaza (eladocagene exuparvovec) in Pennsylvania. Note that while Upstaza is the European brand name, UnitedHealthcare's current policy specifically covers Kebilidi™ (eladocagene exuparvovec-tneq), the FDA-approved version for AADC deficiency.

Use this checklist if you:

  • Have confirmed or suspected AADC deficiency
  • Hold UnitedHealthcare coverage in Pennsylvania
  • Need a one-time gene therapy administered at a certified center
  • Want to avoid common prior authorization denials

Expected outcome: With complete documentation and proper submission, most medically appropriate requests receive approval. If denied initially, Pennsylvania's external review process has overturned approximately 50% of advanced therapy denials in 2024.

Member & Plan Basics

Coverage Verification Checklist

  • Active UnitedHealthcare coverage (check member ID card)
  • Plan type confirmed: Fully-insured commercial plans are eligible for Pennsylvania external review; self-funded employer plans are not
  • Pharmacy benefit managed by OptumRx (most UnitedHealthcare plans)
  • Prior authorization requirement confirmed for gene therapy (required for all gene therapies)

Key Plan Details to Document

Requirement What to Verify Where to Find
Deductible status Annual/family deductible met? Member portal or EOB
Specialty tier Gene therapy coverage tier UnitedHealthcare formulary
Network requirements Centers of Excellence mandate UnitedHealthcare member services
Note: Self-funded employer plans follow federal ERISA rules and cannot use Pennsylvania's external review process. Contact your HR department to confirm your plan type.

Clinical Criteria Requirements

UnitedHealthcare requires strict clinical documentation for Kebilidi/Upstaza coverage based on their medical benefit drug policy.

Diagnostic Confirmation Required

  • Genetic testing: Biallelic pathogenic DDC gene mutations documented
  • Clinical presentation: Autonomic dysfunction and developmental delay
  • Age requirement: Patient ≥18 months (per FDA labeling)
  • Biochemical confirmation: At least one additional test:
    • Plasma AADC enzyme activity assay showing decreased/absent activity
    • CSF neurotransmitter metabolites (low 5-HIAA, HVA; high 3-OMD, L-dopa)

Step Therapy Considerations

While no direct alternatives exist for AADC deficiency gene therapy, document:

  • Supportive care trials: Physical therapy, medications for symptom management
  • Contraindications to other interventions
  • Disease progression despite supportive measures

Coding & Documentation

ICD-10 Diagnostic Codes

  • E70.8 - Other disorders of aromatic amino-acid metabolism (primary)
  • G93.89 - Other specified disorders of brain (if neurological complications)

Procedural Codes

  • HCPCS J-code: To be determined (verify with manufacturer)
  • CPT codes for administration: Typically 96365-96368 for therapeutic infusions

Required Documentation Elements

  • Diagnosis confirmation with genetic test results
  • Prior treatment history and outcomes
  • Clinical rationale for gene therapy timing
  • Treatment plan including monitoring protocols

Documentation Packet Essentials

Provider Letter of Medical Necessity Must Include:

  1. Patient identification: Name, DOB, UnitedHealthcare member ID
  2. Diagnosis details: AADC deficiency with genetic confirmation
  3. Clinical presentation: Specific symptoms (autonomic dysfunction, developmental delays)
  4. Genetic testing results: Laboratory report showing biallelic DDC mutations
  5. Treatment rationale: Why gene therapy is medically necessary now
  6. Center certification: Confirmation of treatment at approved facility
  7. Monitoring plan: Post-treatment follow-up protocols

Required Attachments Checklist

  • Complete genetic testing report
  • Biochemical test results (enzyme activity or CSF metabolites)
  • Clinical notes documenting AADC deficiency symptoms
  • Previous treatment attempts and outcomes
  • Specialist consultation notes (neurologist, geneticist)
  • Center of Excellence certification documentation
Tip: Counterforce Health helps patients and clinicians create evidence-backed appeals by analyzing denial reasons and crafting targeted rebuttals using payer-specific criteria and clinical evidence.

Submission Process

Step-by-Step Prior Authorization

  1. Download current form: Get the latest OptumRx Prior Authorization Request form
  2. Complete all sections: Patient info, prescriber details, clinical rationale
  3. Submit documentation:
    • Fax: 1-844-403-1027 (standard)
    • Electronic: UnitedHealthcare Provider Portal or CoverMyMeds
    • Urgent cases: Call 1-800-711-4555
  4. Confirm receipt: Document confirmation number and submission date

Common Submission Errors to Avoid

  • Incomplete genetic testing documentation
  • Missing clinical symptom details
  • Outdated prior authorization forms
  • Insufficient provider attestation
  • Missing center certification

Gene Therapy Centers in Pennsylvania

UnitedHealthcare requires gene therapy administration at certified Centers of Excellence. Major facilities in Pennsylvania include:

Primary Options

  • Children's Hospital of Philadelphia (CHOP)
    • Cell and Gene Therapy Collaborative
    • 3501 Civic Center Blvd, Philadelphia, PA 19104
  • University of Pennsylvania Gene Therapy Program
    • 125 S. 31st Street, Suite 1200, Philadelphia, PA 19104
Important: Verify current UnitedHealthcare network status and Upstaza/Kebilidi certification with both the center and UnitedHealthcare member services before scheduling.

After Submission: What to Track

Timeline Expectations

  • Standard review: 15 business days
  • Expedited review: 72 hours (urgent medical necessity)
  • Appeals processing: 30 days internal, 45 days external review

Status Monitoring

  • Confirmation number documented
  • Review timeline calendared
  • Status check schedule (weekly for standard, daily for urgent)
  • Contact information for follow-up calls

What to Record

  • All communication dates and reference numbers
  • Names of representatives spoken with
  • Additional documentation requests
  • Decision notifications and next steps

Common Denial Pitfalls

Denial Reason Prevention Strategy Documentation Fix
Insufficient genetic confirmation Submit complete genetic test report with biallelic DDC mutations Laboratory report with pathogenic variant details
Missing clinical correlation Document specific AADC symptoms and severity Detailed clinical notes with symptom timeline
Center not certified Verify UnitedHealthcare COE status before treatment Letter from center confirming certification
Incomplete prior therapy documentation Show supportive care attempts and limitations Treatment history with outcomes
Administrative errors Use current forms and complete all fields Double-check member ID, provider NPI, diagnosis codes

Appeals Process for Pennsylvania

If your initial prior authorization is denied, Pennsylvania offers robust appeal rights for fully-insured plans.

Internal Appeals (Required First Step)

  • Timeline: 180 days from denial receipt
  • Process: Submit appeal through UnitedHealthcare member portal
  • Documentation: Address specific denial reasons with additional evidence

Pennsylvania External Review

Pennsylvania's Independent External Review Program launched in January 2024 with strong patient outcomes.

Key Statistics: In 2024, approximately 50% of external appeals were overturned, with 259 out of 517 cases resulting in coverage approval.

External Review Requirements:

  • Completed internal appeals with final denial letter
  • Fully-insured plan (not self-funded employer plan)
  • Submit within 4 months of final denial
  • Complete documentation addressing medical necessity

How to Submit:

  • Online: Pennsylvania Insurance Department portal
  • Phone: (877) 881-6388 for assistance
  • Timeline: 45 days for standard review, 72 hours for expedited
From our advocates: "We've seen several Pennsylvania families successfully overturn UnitedHealthcare denials for gene therapies by thoroughly documenting the genetic diagnosis and working with certified centers. The key is addressing every point in the denial letter with specific clinical evidence and ensuring all procedural requirements are met."

Printable Checklist

Pre-Submission Checklist

  • UnitedHealthcare coverage active and verified
  • Plan type confirmed (fully-insured for appeal rights)
  • Genetic testing shows biallelic DDC mutations
  • Clinical symptoms documented (autonomic dysfunction, developmental delay)
  • Treatment center certified for gene therapy
  • Current OptumRx PA form downloaded
  • Complete documentation packet assembled
  • Provider letter of medical necessity completed
  • Submission method chosen (fax/electronic)

Post-Submission Tracking

  • Confirmation number recorded
  • Review timeline calendared
  • Status check schedule established
  • Appeal strategy prepared if needed
  • Pennsylvania external review information gathered

For patients and families navigating complex coverage decisions, Counterforce Health provides specialized support in turning insurance denials into successful appeals through evidence-backed documentation and payer-specific strategies.

FAQ

Q: How long does UnitedHealthcare prior authorization take for Upstaza? A: Standard review takes 15 business days. Urgent cases can be expedited to 72 hours by calling 1-800-711-4555.

Q: What if Upstaza isn't on UnitedHealthcare's formulary? A: UnitedHealthcare covers Kebilidi (the FDA-approved version) under their medical benefit drug policy when clinical criteria are met.

Q: Can I appeal if my employer plan is self-funded? A: Self-funded plans cannot use Pennsylvania's external review process. You'll need to follow the plan's internal appeal procedures and potentially federal external review.

Q: Do I need to try other treatments first? A: No direct alternatives exist for AADC deficiency gene therapy, but document any supportive care attempts and their limitations.

Q: What happens if the external review approves coverage? A: UnitedHealthcare must provide coverage immediately, including retroactive coverage if you paid out-of-pocket during the appeal.

Sources & Further Reading

Disclaimer: This information is for educational purposes only and does not constitute medical or legal advice. Coverage decisions depend on individual plan terms and clinical circumstances. Always consult with your healthcare provider and insurance company for specific guidance. For additional support with Pennsylvania insurance appeals, contact the Pennsylvania Insurance Department Consumer Services at (877) 881-6388.

Powered by Counterforce Health—AI that turns drug denials into evidence-based appeals patients and clinicians can submit today.