By Counterforce Health Team in Strimvelis (autologous CD34+ ADA-SCID gene therapy) —

How to Get Strimvelis Gene Therapy Covered by Aetna in Pennsylvania: Complete Prior Authorization and Appeals Guide

Quick Answer: Getting Strimvelis Covered by Aetna in Pennsylvania

Strimvelis gene therapy for ADA-SCID requires extensive prior authorization from Aetna, including genetic confirmation, enzyme testing, and proof of no suitable bone marrow donor. Since treatment is only available in Milan, Italy, you'll need international coverage approval. Start with genetic testing documentation, secure a medical necessity letter from an immunologist, and submit through Aetna's provider portal. If denied, Pennsylvania's new external review program has overturned ~50% of appeals in 2024. First step today: Contact your immunologist to begin genetic confirmation and enzyme testing documentation.

Table of Contents

  1. Understanding Strimvelis and Coverage Requirements
  2. Reading Your Aetna Denial Letter
  3. Required Documentation for Prior Authorization
  4. First-Level Appeal Process
  5. Peer-to-Peer Review Strategy
  6. Pennsylvania External Review Process
  7. Appeal Templates and Scripts
  8. International Treatment Coverage
  9. Tracking Your Appeal
  10. When Appeals Fail: Next Steps
  11. FAQ

Understanding Strimvelis and Coverage Requirements

Strimvelis is a one-time gene therapy for adenosine deaminase deficiency severe combined immunodeficiency (ADA-SCID), available exclusively at San Raffaele Hospital in Milan, Italy. The therapy costs approximately €594,000 (~$650,000) and is administered only to patients who lack suitable bone marrow donors.

Coverage at a Glance

Requirement What It Means Where to Find It Source
Genetic confirmation Biallelic ADA gene mutations Genetic testing lab report Mayo Clinic Labs SCID Panel
Enzyme testing ADA activity <1% of normal Blood enzyme assay ADA-SCID Info for HCPs
No suitable donor HLA-matched related donor unavailable Transplant center evaluation Primary Immune Guidelines
International approval Out-of-network coverage for Milan treatment Aetna international benefits Contact Aetna member services
Appeals deadline 180 days from denial Denial letter Aetna Appeals Process

Reading Your Aetna Denial Letter

When Aetna denies Strimvelis, common reasons include:

  • "Experimental/Investigational" - Despite EU approval, not FDA-approved in the US
  • "Out-of-network" - Treatment only available internationally
  • "Not medically necessary" - Insufficient documentation of failed alternatives
  • "No prior authorization" - Missing required pre-approval for specialty therapy
Tip: Look for the specific denial code on your Explanation of Benefits (EOB). This determines your appeal strategy and required documentation.

Required Documentation for Prior Authorization

Essential Medical Records

Genetic Testing:

  • Biallelic pathogenic variants in the ADA gene
  • Next-generation sequencing SCID panel results
  • Genetic counselor interpretation letter

Enzyme Confirmation:

  • ADA enzyme activity test showing <1% of normal levels
  • Laboratory certification and reference ranges
  • Date of testing and methodology used

Clinical Documentation:

  • Complete immunologic workup (T-B-NK- phenotype)
  • History of severe/recurrent infections
  • Growth charts showing failure to thrive
  • ICD-10 diagnosis code D81.31 (Adenosine deaminase deficiency)

Treatment History:

  • Documentation of enzyme replacement therapy (ERT) trials
  • Evidence of ERT failure or intolerance
  • Bone marrow transplant evaluation showing no suitable HLA-matched related donor
  • Contraindications to alternative therapies

Specialist Requirements

Your medical necessity letter must come from:

  • Board-certified immunologist or geneticist
  • Physician familiar with ADA-SCID management
  • Specialist affiliated with a recognized SCID treatment center

First-Level Appeal Process

Timeline and Submission

Deadline: 180 days from denial date Response time: 45-60 business days (standard); 36-72 hours (expedited) How to submit: Aetna provider portal or mail/fax

Required Components

  1. Completed Aetna appeal form (state-specific Pennsylvania version)
  2. Copy of denial letter and original claim
  3. Medical necessity letter addressing each denial reason
  4. Supporting documentation (genetic tests, enzyme assays, specialist evaluations)
  5. Authorization form if provider is appealing on patient's behalf
Note: Pennsylvania residents benefit from stronger appeal protections under Act 146 of 2022, which established the state's independent external review program.

Peer-to-Peer Review Strategy

Before filing a formal appeal, request a peer-to-peer discussion with Aetna's medical reviewer.

How to Request

Call Aetna CVS Caremark customer service and specifically request:

  • "Peer-to-peer review for utilization management decision"
  • Reference your case number and denial letter
  • Request to speak with a physician reviewer familiar with rare genetic disorders

Preparation Checklist

  • Patient's complete medical history
  • Genetic testing results and interpretation
  • Evidence of failed standard therapies
  • Documentation of no suitable bone marrow donor
  • Published guidelines supporting gene therapy for ADA-SCID
  • Treatment protocol from San Raffaele Hospital

Pennsylvania External Review Process

Pennsylvania's Independent External Review Program, launched in 2024, has overturned approximately 50% of appealed denials.

Eligibility and Timeline

When to use: After receiving Final Adverse Benefit Determination from Aetna's internal appeal Deadline: 4 months from final denial Decision timeline: 45 days (standard); 72 hours (expedited) Cost: Free to consumers

How to Submit

  1. Online portal: pa.gov/reviewmyclaim
  2. Supporting documents: Submit within 15 days of case assignment
  3. Contact for help: Pennsylvania Insurance Department at 1-877-881-6388

When working with complex cases like Strimvelis gene therapy, many families benefit from professional advocacy support. Counterforce Health specializes in turning insurance denials into targeted, evidence-backed appeals by analyzing denial letters, plan policies, and clinical notes to craft point-by-point rebuttals aligned with each payer's specific requirements.

Appeal Templates and Scripts

Medical Necessity Letter Template

RE: Appeal for Strimvelis Gene Therapy - [Patient Name], DOB: [Date]
Policy Number: [Number] | Case ID: [ID]

Dear Medical Director,

I am writing to appeal the denial of Strimvelis (autologous CD34+ gene therapy) for my patient with confirmed ADA-SCID. This denial was based on [specific reason from denial letter].

PATIENT SUMMARY:
- Confirmed ADA-SCID with biallelic ADA gene mutations
- ADA enzyme activity <1% of normal (attach lab report)
- Severe immunodeficiency with recurrent life-threatening infections
- No suitable HLA-matched related donor available for HSCT

MEDICAL NECESSITY:
Strimvelis is the only viable treatment option because:
1. Standard HSCT requires matched donor (unavailable)
2. Enzyme replacement therapy has failed/caused intolerance
3. Without treatment, ADA-SCID is uniformly fatal

EVIDENCE BASE:
- EMA approval for ADA-SCID patients without matched donors
- NICE recommendation for this exact indication
- 100% survival at 3 years in pivotal studies
- Only treatment available at authorized center (Milan)

REQUEST:
Approve coverage for Strimvelis treatment including:
- International travel for treatment at San Raffaele Hospital
- Pre-treatment workup and post-treatment monitoring
- Coordination of care between US and Italian physicians

[Physician signature and credentials]

Patient Phone Script for Aetna

"Hello, I'm calling about a prior authorization denial for Strimvelis gene therapy. My policy number is [X]. The denial letter states [reason]. I'd like to request a peer-to-peer review with a medical director who has experience with rare genetic disorders. Can you connect me with someone who can schedule this discussion with my specialist?"

International Treatment Coverage

Since Strimvelis is only available in Milan, securing international coverage is crucial.

Key Steps for International Approval

  1. Verify benefits: Confirm your Aetna plan includes international/out-of-network coverage
  2. Medical necessity: Document why treatment cannot be obtained in the US
  3. Center authorization: Obtain eligibility confirmation from San Raffaele Hospital
  4. Care coordination: Establish communication between US and Italian physicians
  5. Travel logistics: Note that travel and lodging costs are typically not covered
Important: International treatment approvals often require extensive documentation and may involve higher patient cost-sharing (typically 50-70% after deductible).

For families navigating the complex international treatment approval process, Counterforce Health helps patients and clinicians compile the specific evidence and operational details that international payers expect, including NDC codes, site of care documentation, and administration plans that meet procedural requirements.

Tracking Your Appeal

Documentation Log

Keep detailed records of all communications:

Date Contact Method Person/Department Summary Follow-up Needed

Important Deadlines

  • Initial appeal: 180 days from denial
  • External review: 4 months from final adverse determination
  • Expedited appeals: Request immediately for urgent medical situations

When Appeals Fail: Next Steps

If all appeals are unsuccessful, consider:

Alternative Options

  1. Compassionate use programs: Contact Orchard Therapeutics
  2. Clinical trials: Search ClinicalTrials.gov for ADA-SCID studies
  3. Foundation support: Apply for grants from rare disease organizations
  4. Insurance change: Consider plans with broader international coverage during open enrollment

Regulatory Complaints

File complaints with:

  • Pennsylvania Insurance Department: Consumer complaint process
  • Department of Health and Human Services: Federal oversight of insurance practices

FAQ

How long does Aetna prior authorization take for Strimvelis? Standard PA decisions typically take 30-45 days. Expedited reviews for urgent cases can be completed in 72 hours.

What if Strimvelis is considered experimental by Aetna? Challenge this determination by citing EMA approval, NICE recommendations, and published clinical outcomes. The therapy has established efficacy for the approved indication.

Can I get an expedited appeal in Pennsylvania? Yes, if delay in treatment could jeopardize your health. Both Aetna internal appeals and Pennsylvania external reviews offer expedited timelines for urgent cases.

Does step therapy apply to ADA-SCID treatments? Typically yes - you must document failure or contraindications to standard treatments like enzyme replacement therapy and bone marrow transplant evaluation.

What happens if I pay out-of-pocket during appeals? If your appeal is successful, Aetna must provide retroactive coverage and reimburse out-of-pocket expenses incurred during the appeal process.

How successful are appeals in Pennsylvania? Pennsylvania's new external review program has overturned approximately 50% of appealed denials in its first year, significantly higher than many other states.

Sources & Further Reading

Disclaimer: This guide provides general information about insurance coverage and appeals processes. It is not medical advice or legal counsel. Always consult with your healthcare providers and insurance plan documents for specific guidance. For additional help with complex appeals, contact the Pennsylvania Insurance Department Consumer Services at 1-877-881-6388.

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