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

Getting Strimvelis Gene Therapy Covered by Blue Cross Blue Shield in North Carolina: Complete Guide to International Coverage and Appeals

Answer Box: Getting Strimvelis Covered by Blue Cross Blue Shield in North Carolina

Strimvelis gene therapy for ADA-SCID is not FDA-approved in the U.S. and requires treatment at San Raffaele Hospital in Milan, Italy. Blue Cross Blue Shield of North Carolina (Blue Cross NC) does not have a specific coverage policy for Strimvelis, classifying it as investigational. However, coverage may be possible through international benefits if you have GeoBlue® or Global Core coverage. First step today: Call Blue Cross NC member services to verify your international benefits and request a formulary exception form. If denied, North Carolina's Smart NC program provides free external review assistance.

Table of Contents

  1. What Drives Coverage Decisions
  2. Investigating Your Benefits
  3. Assistance and Support Options
  4. Requesting Formulary Exceptions
  5. International Treatment Coordination
  6. Appeals Process in North Carolina
  7. Planning for Treatment Costs
  8. Conversation Scripts
  9. FAQ

What Drives Coverage Decisions

Blue Cross Blue Shield of North Carolina evaluates coverage for Strimvelis based on several key factors that directly impact your approval chances.

Medical Necessity Criteria

Blue Cross NC requires documentation that Strimvelis is medically necessary for your specific case of ADA-SCID. This includes:

  • Confirmed genetic diagnosis of adenosine deaminase deficiency
  • Laboratory evidence showing ADA enzyme deficiency
  • Documentation of failed or contraindicated standard treatments (enzyme replacement therapy, stem cell transplant)
  • Specialist recommendation from an immunologist or geneticist

Investigational Status

Currently, Blue Cross NC does not have a specific medical policy for Strimvelis, classifying most gene therapies as investigational unless explicitly covered. This creates a higher bar for approval but doesn't make coverage impossible.

International Treatment Requirements

Since Strimvelis is only available in Milan, Italy, coverage depends on your plan's international benefits. Plans with GeoBlue® or Global Core typically offer broader international coverage than basic emergency-only policies.

Note: The €594,000 list price makes this one of the most expensive single treatments, requiring exceptional documentation for any coverage consideration.

Investigating Your Benefits

Before pursuing coverage, gather comprehensive information about your specific Blue Cross NC plan benefits.

Coverage Investigation Checklist

Call Blue Cross NC Member Services and document:

  • International benefits: Does your plan include GeoBlue® or Global Core coverage?
  • Out-of-network maximums: What are your annual limits for international care?
  • Gene therapy coverage: Are any gene therapies covered, even investigationally?
  • Prior authorization requirements: What forms and documentation are needed?
  • Appeals timelines: Internal appeal deadlines and external review rights

Information to Record:

  • Representative name and reference number
  • Date and time of call
  • Specific policy language about international and experimental treatments
  • Required forms and submission methods
  • Coverage determination timelines

Key Questions for Your Plan

  1. "Does my plan cover medically necessary treatments received internationally?"
  2. "What prior authorization is required for gene therapy not on your formulary?"
  3. "If I receive a denial, what are my appeal rights in North Carolina?"
  4. "Are there specific clinical criteria for ADA-SCID treatments?"

Assistance and Support Options

Multiple resources can help reduce your financial burden and navigate the coverage process.

Manufacturer Support

Orchard Assist Program

  • Contact: 1-833-672-4278
  • Services: Insurance navigation, financial assistance screening, clinical support
  • Eligibility: Varies by individual circumstances
  • Website: orchardassist.com

The program helps families understand insurance requirements and may provide financial assistance for qualifying patients.

Foundation and Grant Support

Telethon Foundation Since taking over Strimvelis distribution in 2023, the Telethon Foundation may provide support for travel, lodging, and ancillary costs associated with treatment in Milan.

National Organization for Rare Disorders (NORD) Offers patient assistance programs and advocacy support for rare disease treatments.

Patient Advocate Foundation Provides insurance navigation and may offer grants for treatment-related expenses.

State Resources

Smart NC Consumer Assistance

  • Phone: 1-855-408-1212
  • Services: Free insurance appeals assistance, external review filing, advocacy
  • Website: NC Department of Insurance

Smart NC staff can help you understand your appeal rights and assist with external review applications if your claim is denied.

Requesting Formulary Exceptions

Since Strimvelis isn't on Blue Cross NC's formulary, you'll need to request a formulary exception with comprehensive medical documentation.

Step-by-Step Exception Process

1. Gather Required Documentation

  • Genetic testing confirming ADA-SCID diagnosis
  • Laboratory results showing ADA enzyme deficiency
  • Records of failed standard treatments
  • Specialist letters supporting Strimvelis necessity
  • Treatment plan from San Raffaele Hospital

2. Complete Exception Request

  • Use Blue Cross NC's formulary exception form
  • Include detailed medical necessity justification
  • Attach all supporting clinical documentation

3. Submit via Multiple Channels

  • Online: Through provider portal or CoverMyMeds.com
  • Fax: 888-348-7332
  • Phone Follow-up: 800-672-7897

4. Track Your Request

  • Standard review: 72 hours
  • Urgent review: 24 hours
  • Document all communications

Medical Necessity Letter Components

Your physician's letter should include:

  • Diagnosis: Confirmed ADA-SCID with genetic and enzymatic evidence
  • Prior Treatments: Detailed history of enzyme replacement therapy trials, outcomes, and any contraindications to stem cell transplant
  • Clinical Rationale: Why Strimvelis is the most appropriate treatment option
  • Treatment Plan: Coordination with San Raffaele Hospital Milan
  • Supporting Literature: FDA orphan drug designation, EU approval data, published outcomes
Clinician Tip: Include specific lab values (ADA enzyme levels, lymphocyte counts) and cite the European Medicines Agency approval as evidence of established efficacy and safety.

International Treatment Coordination

Successfully obtaining coverage for treatment in Milan requires careful coordination between your U.S. insurance and the Italian treatment center.

Pre-Authorization Requirements

Blue Cross NC International Coverage If your plan includes international benefits, you may need:

  • Pre-authorization for non-emergency international treatment
  • Confirmation that San Raffaele Hospital accepts your coverage
  • Understanding of direct billing vs. reimbursement processes

Documentation for International Claims

  • Complete medical records in English
  • Treatment authorization from San Raffaele
  • Detailed cost estimates
  • Travel and accommodation documentation (if covered)

Working with San Raffaele Hospital

Patient Coordination Office San Raffaele has experience working with international patients and insurance systems. Contact their international patient services to:

  • Verify treatment eligibility and scheduling
  • Understand payment requirements and timing
  • Coordinate with your U.S. insurance for coverage verification

Required Medical Documentation The Milan center will need:

  • Complete genetic analysis confirming ADA-SCID
  • Detailed treatment history
  • Current clinical status and laboratory values
  • Insurance pre-authorization (if obtained)

Appeals Process in North Carolina

If Blue Cross NC denies coverage, North Carolina offers robust appeal rights through both internal and external review processes.

Internal Appeals with Blue Cross NC

First-Level Internal Appeal

  • Deadline: 180 days from denial
  • Required: Denial letter, medical records, physician support letter
  • Timeline: 30 days for standard, 72 hours for urgent
  • Submission: Member services or provider portal

Second-Level Internal Appeal

  • Automatic: If first-level appeal is denied
  • Timeline: Additional 30 days
  • Review: Independent medical reviewers

External Review Through Smart NC

Eligibility Requirements

  • Completed Blue Cross NC internal appeals
  • Request within 120 days of final internal denial
  • State-regulated plan (most individual and small group plans qualify)
  • Denial based on medical necessity or experimental determination

External Review Process

  • Timeline: 45 days for standard review, 72 hours for expedited
  • Cost: Free to patients
  • Reviewers: Independent medical experts with relevant specialty knowledge
  • Decision: Binding on Blue Cross NC if coverage is approved

How to Request External Review

  1. Complete Smart NC external review form
  2. Include all denial letters and medical documentation
  3. Submit within 120 days of final internal denial
  4. Contact Smart NC at 1-855-408-1212 for assistance
Success Tip: External reviews for specialty medications with strong clinical documentation have historically achieved 40-60% overturn rates in North Carolina.

Planning for Treatment Costs

Understanding potential costs helps you prepare financially and explore all available resources.

Cost Components

Treatment Costs

  • Strimvelis therapy: €594,000 (approximately $650,000 USD)
  • Hospital fees in Milan
  • Pre-treatment evaluation and testing
  • Post-treatment monitoring

Travel and Logistics

  • International travel for patient and family
  • Extended stay in Milan (typically several weeks)
  • Accommodation and living expenses
  • Potential emergency medical evacuation coverage

Financial Planning Strategies

Insurance Coordination

  • Maximize in-network benefits where possible
  • Understand out-of-pocket maximums for international care
  • Coordinate with any secondary insurance coverage

Payment Options

  • Work with Orchard Assist for manufacturer support programs
  • Explore foundation grants and assistance programs
  • Consider medical financing options if partial coverage is obtained

Tax Considerations

  • Medical expense deductions for qualifying costs
  • Coordination with Health Savings Accounts (HSAs) or Flexible Spending Accounts (FSAs)

Conversation Scripts

Use these scripts to communicate effectively with insurance representatives, healthcare providers, and support programs.

Blue Cross NC Member Services Script

"Hello, I'm calling about coverage for my child who has ADA-SCID, a rare genetic immune deficiency. We need coverage for Strimvelis gene therapy, which is only available in Milan, Italy. Can you help me understand:

  1. Does my plan include international coverage through GeoBlue or Global Core?
  2. What prior authorization process applies to non-formulary gene therapies?
  3. How do I request a formulary exception for a treatment not available in the U.S.?
  4. What documentation will be required for medical necessity review?"

Orchard Assist Program Script

"I'm calling about my child with ADA-SCID who may be a candidate for Strimvelis therapy. We're working on insurance coverage with Blue Cross Blue Shield in North Carolina. Can you help us understand:

  1. What financial assistance programs are available?
  2. How can you help with our insurance prior authorization process?
  3. What support is available for travel and treatment coordination in Milan?
  4. Are there any grants or foundations you can connect us with?"

Smart NC Appeals Assistance Script

"I'm calling because Blue Cross Blue Shield denied coverage for Strimvelis gene therapy for my child with ADA-SCID. The treatment is only available in Italy, and we believe it's medically necessary. Can Smart NC help us:

  1. Understand our external review rights?
  2. File an external review application?
  3. Gather the right medical documentation?
  4. Navigate the appeals timeline and requirements?"

FAQ

How long does Blue Cross NC take to review Strimvelis prior authorization requests? Standard formulary exception reviews take up to 72 hours. For urgent requests involving serious medical conditions, Blue Cross NC must respond within 24 hours. International treatment coordination may require additional processing time.

What if Strimvelis is considered experimental by Blue Cross NC? Even if classified as investigational, coverage may still be possible through formulary exceptions based on medical necessity. Focus on documenting failed standard treatments and the lack of viable alternatives in the U.S.

Can I request an expedited appeal if my child's condition is deteriorating? Yes. Both Blue Cross NC internal appeals and Smart NC external reviews offer expedited processes for urgent medical situations. Document the urgency with current clinical status and physician recommendations.

Does North Carolina's external review apply to international treatments? Yes, Smart NC external review covers denials for medically necessary treatments regardless of location, as long as you have a state-regulated insurance plan and meet other eligibility requirements.

What happens if Blue Cross NC approves coverage but San Raffaele Hospital isn't in-network? You may receive out-of-network benefits, which typically involve higher cost-sharing. Work with both Blue Cross NC and San Raffaele to understand direct billing options or reimbursement processes.

How can Counterforce Health help with my Strimvelis appeal? Counterforce Health specializes in turning insurance denials into targeted, evidence-backed appeals. Their platform can help identify the specific denial basis and draft point-by-point rebuttals aligned to Blue Cross NC's policies, incorporating the right clinical evidence and procedural requirements for North Carolina appeals.

Are there alternatives if all appeals fail? If insurance coverage isn't successful, explore compassionate use programs, clinical trials, or work with advocacy organizations for alternative funding sources. The Telethon Foundation and other rare disease organizations may provide additional support options.

What documentation should I keep throughout this process? Maintain copies of all insurance communications, medical records, denial letters, appeal submissions, and correspondence with San Raffaele Hospital. This documentation is crucial for appeals and external review processes.

Getting coverage for rare disease treatments like Strimvelis requires persistence and comprehensive documentation. Counterforce Health helps patients and clinicians navigate complex insurance appeals by turning denials into targeted, evidence-backed requests that align with payer policies and procedural requirements.

Disclaimer: This information is for educational purposes only and does not constitute medical or legal advice. Insurance coverage decisions depend on individual plan benefits and medical circumstances. Always consult with your healthcare providers and insurance representatives for guidance specific to your situation.

Sources & Further Reading

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