By Counterforce Health Team in Upstaza (eladocagene exuparvovec) —

Aetna (CVS Health) Coverage for Upstaza (eladocagene exuparvovec) in Washington: Complete Guide to Getting Gene Therapy Approved

Answer Box: Getting Upstaza Covered by Aetna in Washington

Upstaza (eladocagene exuparvovec) requires prior authorization from Aetna (CVS Health) in Washington. To get approved, you need confirmed AADC deficiency diagnosis through genetic testing and enzyme/CSF studies, documentation of severe phenotype, and treatment at a certified center. If denied, Washington provides binding external review through an Independent Review Organization (IRO).

Three steps to start today:

  1. Gather genetic testing results and AADC deficiency confirmation
  2. Contact your neurologist to initiate Aetna prior authorization via Availity portal
  3. If denied, file Level 1 appeal within 180 days using Aetna's member portal

Table of Contents

Aetna's Coverage Policy Overview

Aetna (CVS Health) classifies Upstaza as a specialty gene therapy requiring prior authorization across all plan types—HMO, PPO, and Medicare Advantage. The drug falls under Aetna's Clinical Policy Bulletin 1073, which governs gene therapies for AADC deficiency.

Key policy points:

  • Prior authorization required for all Upstaza requests
  • Specialty pharmacy routing through CVS Caremark/CVS Specialty
  • Center of excellence requirements for administration
  • Medical necessity review by CVS Health Solutions clinicians
Note: As of July 2025, formulary requirements may have changed. Always verify current status through Aetna's precertification lists.

Medical Necessity Requirements

To meet Aetna's medical necessity criteria for Upstaza, you must demonstrate:

Diagnostic Confirmation

  • Genetic testing: Biallelic pathogenic variants in the DDC gene
  • Biochemical confirmation: Either plasma AADC enzyme activity assay OR cerebrospinal fluid neurotransmitter metabolite panel showing characteristic patterns
  • Expert interpretation: Results reviewed by a metabolic or genetic specialist

Clinical Severity

  • Severe phenotype: Inability to sit, stand, or walk unassisted
  • Age requirement: 18 months or older with skull maturity achieved
  • Motor impairment documentation: Detailed neurological assessments showing significant developmental delays

Treatment History

  • Prior therapies: Documentation of symptomatic treatments tried (if any)
  • Lack of alternatives: Evidence that no other disease-modifying therapies are available

Counterforce Health specializes in helping patients and clinicians compile this exact documentation for gene therapy appeals. Their platform automatically identifies which specific evidence Aetna requires and formats it into targeted, policy-aligned medical necessity letters.

Prior Authorization Process

Step-by-Step Submission

  1. Prepare documentation (2-3 weeks before needed)
    • Complete genetic and biochemical test results
    • Neurological assessments and imaging
    • Treatment history and prior therapy records
  2. Submit via Availity portal (provider responsibility)
    • Use Novologix system for electronic submission
    • Include all required clinical documentation
    • Specify urgency if treatment needed within 48 hours
  3. Track authorization status
    • Standard review: 30-45 days for complex cases
    • Expedited review: ≤72 hours for urgent requests
    • Medicare Advantage: ≤72 hours per CMS requirements
  4. Receive determination
    • Approval includes specific authorized treatment center
    • Denial includes reason codes and appeal rights

Required Documentation Checklist

  • Genetic testing report with DDC gene variants
  • Enzyme activity or CSF metabolite results
  • Neurological examination notes
  • Developmental milestone assessments
  • Brain imaging (MRI) if available
  • Treatment center confirmation of acceptance

Common Denial Reasons & Solutions

Denial Reason Solution Required Evidence
Insufficient diagnostic proof Submit both genetic AND biochemical confirmation Complete DDC gene analysis + enzyme/CSF studies
Non-certified treatment center Verify center designation Contact PTC Therapeutics for current authorized sites
Experimental/investigational Cite FDA approval status Reference FDA approval letter and product labeling
Not medically necessary Demonstrate severe phenotype Motor function assessments, developmental evaluations
Missing prior authorization Ensure submission before treatment Complete PA process through Availity portal

Appeals Process in Washington

Washington provides robust consumer protections for insurance denials, including binding external review.

Internal Appeals (Aetna)

Level 1 Appeal:

  • Timeline: 180 days from denial to file
  • Method: Aetna member portal or written request
  • Decision: 30-60 days (expedited: ≤72 hours)

Level 2 Appeal:

  • Timeline: 60 days from Level 1 denial
  • Process: Same submission methods
  • Peer review: May include physician-to-physician discussion

External Review (IRO) - Washington State

After exhausting internal appeals, Washington law guarantees independent review:

Consumer Advocacy: Call Washington OIC at 800-562-6900 for appeal assistance and guidance throughout the process.

Treatment Centers & Site of Care

Currently, no certified Upstaza treatment centers operate in Washington state. Approved centers include:

  • Boston Children's Hospital (Massachusetts)
  • Texas Children's Hospital (Texas)

Aetna Network Considerations

  • Verify out-of-state center coverage with Aetna case management
  • Obtain prior authorization for both the drug and the facility
  • Confirm travel and lodging coverage if applicable

Contact PTC Therapeutics patient support for current center locations and referral coordination.

From our advocates: "We've seen Washington families successfully get coverage for out-of-state gene therapy when they worked closely with both their local care team and Aetna's case management early in the process. Starting the conversation about logistics and coverage 3-4 months before planned treatment helps avoid last-minute denials."

Cost Support Options

Given Upstaza's multimillion-dollar cost, explore all available support:

Manufacturer Support

  • PTC Cares program: Patient assistance and case management
  • Financial assistance: Income-based support programs
  • Travel grants: May cover treatment-related travel expenses

State and Federal Programs

  • Washington Apple Health (Medicaid): May provide coverage for eligible families
  • CHIP: Children's health insurance program coverage
  • Foundation grants: Rare disease organizations often provide emergency assistance

FAQ

How long does Aetna prior authorization take for Upstaza in Washington? Standard review takes 30-45 days. For urgent cases requiring treatment within 48 hours, specify expedited review for ≤72-hour decisions.

What if Upstaza is non-formulary on my Aetna plan? Submit a formulary exception request with your prior authorization, including medical necessity documentation and evidence that no formulary alternatives exist for AADC deficiency.

Can I appeal if there's no treatment center in Washington? Yes. Document medical necessity and include evidence that out-of-state treatment is the only available option. Aetna may cover travel to approved centers.

Does Washington require step therapy for gene therapies? Step therapy typically doesn't apply to orphan drugs like Upstaza when no therapeutic alternatives exist. Document this in your prior authorization request.

How do I request expedited appeals in Washington? Contact Aetna directly and specify that delayed treatment poses serious health risks. Provide supporting physician documentation of urgency.

What happens after IRO external review? If the IRO approves coverage, Aetna must provide the service. If denied, your remaining option is legal action, though you can contact the Washington OIC for additional guidance.

When to Get Help

Consider professional assistance when:

  • Initial prior authorization is denied
  • You're unfamiliar with Washington's external review process
  • Clinical documentation seems incomplete
  • You need help coordinating out-of-state treatment

Counterforce Health transforms insurance denials into evidence-backed appeals specifically for specialty medications like gene therapies. Their platform ingests denial letters and clinical notes to draft point-by-point rebuttals aligned with Aetna's specific policies, incorporating the right citations from FDA labeling, peer-reviewed studies, and specialty guidelines that Washington IRO reviewers expect to see.

Disclaimer: This information is for educational purposes and does not constitute medical or legal advice. Insurance policies and state regulations may change. Always consult with your healthcare provider and insurance plan for current requirements and personalized guidance.

Sources & Further Reading

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