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

Getting Strimvelis Gene Therapy Covered by Blue Cross Blue Shield in New Jersey: Complete 2025 Guide with Forms and Appeals

Answer Box: Your Path to Strimvelis Coverage in New Jersey

Strimvelis gene therapy coverage through Blue Cross Blue Shield in New Jersey requires a medical exception request due to its Milan-only availability and lack of FDA approval. Success depends on thorough documentation of ADA-SCID diagnosis, failed standard treatments, and medical necessity. If denied, New Jersey's Independent Health Care Appeals Program (IHCAP) offers external review with 20-40% overturn rates for specialty drugs. Start today by: 1) Confirming your BCBS plan includes international coverage, 2) Gathering genetic testing and enzyme deficiency documentation, 3) Requesting medical exception forms from member services at 1-800-355-2583.

Table of Contents

Understanding Strimvelis and Insurance Challenges

Strimvelis represents a groundbreaking but complex coverage scenario. This one-time gene therapy, developed originally by GSK and now managed by Orchard Therapeutics, is the only approved treatment for ADA-SCID (adenosine deaminase deficiency severe combined immunodeficiency) in Europe. However, it's exclusively available at the SR-TIGET center in Milan, Italy, creating unique insurance hurdles.

The therapy works by extracting a patient's bone marrow cells, genetically modifying them to produce the missing ADA enzyme, and reinfusing them. With a list price of approximately €594,000 ($650,000), it requires extensive documentation and often multiple appeals to secure coverage through Blue Cross Blue Shield plans.

Counterforce Health specializes in turning insurance denials into successful appeals by analyzing denial letters, plan policies, and clinical notes to craft targeted, evidence-backed rebuttals. For complex cases like Strimvelis, where international treatment coordination meets rare disease coverage, having expert support can make the difference between approval and denial.

Pre-Authorization Requirements

Essential Documentation Checklist

Before submitting your request, gather these critical documents:

Medical Documentation:

  • Confirmed ADA-SCID diagnosis via genetic testing showing biallelic pathogenic variants in the ADA gene
  • ADA enzyme activity levels (typically <1% of normal range)
  • Complete immunologic profile showing T-B-NK- phenotype
  • Clinical history documenting severe/recurrent infections and failure to thrive

Treatment History:

  • Documentation of failed or contraindicated hematopoietic stem cell transplantation (HSCT)
  • Records of enzyme replacement therapy trials and outcomes
  • Evidence of lack of suitable bone marrow donors
  • Specialist letters explaining why standard treatments are insufficient

Insurance Verification:

  • Confirm your BCBS plan includes international coverage (Blue Cross Blue Shield Global Core or GeoBlue)
  • Verify your plan type (fully insured vs. self-funded ERISA plans have different appeal rights)
  • Obtain medical exception and international treatment authorization forms

Step-by-Step Approval Process

Phase 1: Initial Authorization Request (Days 1-30)

Step 1: Contact Member Services Call your BCBS member services line to request:

  • Medical exception forms for non-formulary treatments
  • International treatment authorization paperwork
  • Confirmation of your plan's international coverage benefits

Step 2: Physician Preparation Your specialist should prepare a comprehensive letter of medical necessity addressing:

  • Confirmed ADA-SCID diagnosis with genetic and enzymatic evidence
  • Clinical urgency and prognosis without treatment
  • Why domestic alternatives (HSCT, enzyme replacement) are unsuitable
  • Supporting literature from EMA approval and peer-reviewed studies

Step 3: Submit Complete Package Submit through your BCBS provider portal or designated submission method:

  • Completed medical exception request
  • Physician letter of medical necessity
  • All diagnostic documentation
  • Treatment history and failure documentation
  • SR-TIGET eligibility confirmation from Milan

Phase 2: Review and Decision (Days 30-60)

BCBS typically responds within 30 days for standard requests, 72 hours for expedited cases. The review focuses on medical necessity criteria and plan coverage limitations for international treatments.

Common Denial Reasons and Solutions

Denial Reason Solution Strategy Required Documentation
"Experimental/Investigational" Emphasize EMA approval and published efficacy data EMA approval documents, peer-reviewed studies, FDA orphan drug designation
"Treatment available domestically" Document HSCT contraindications or donor unavailability Transplant center evaluation, donor search results, specialist letter
"Not medically necessary" Provide comprehensive clinical justification Genetic testing, enzyme levels, infection history, growth charts
"International coverage exclusion" Request medical exception for life-saving treatment SR-TIGET documentation, treatment protocol, medical urgency letter

New Jersey Appeals Process (IHCAP)

If your initial request is denied, New Jersey offers robust appeal protections through the Independent Health Care Appeals Program (IHCAP).

Internal Appeals (Required First Step)

Timeline: Must file within 60 days of denial notice Process: Two levels of internal BCBS review required Decision timeframe: 30 days standard, 72 hours expedited

External Review Through IHCAP

Eligibility: Available for fully insured New Jersey plans (not self-funded ERISA plans) Filing deadline: Within 4 months (180 days) of final internal denial Cost: Free for most plans; $25 fee for some HMO members Success rate: 20-40% overturn rate for specialty drugs and gene therapies

IHCAP Contact Information:

  • Phone: 888-866-5205
  • Online portal: njihcap.maximus.com
  • Mail: Maximus Federal Services, Attn: NJ IHCAP, 3750 Monroe Ave, Suite 705, Pittsford, NY 14534

Timeline:

  • Preliminary review: 5 business days
  • Final decision: 45 days from complete submission
  • Expedited cases: Much shorter timeframe when delay could cause serious harm
From our advocates: "We've seen families succeed with IHCAP by focusing on the unique aspects of their child's case—not just meeting general criteria, but explaining why this specific patient needs this specific treatment right now. The physician reviewers understand rare diseases better than insurance company administrators."

International Coverage Considerations

Blue Cross Blue Shield Global Coverage

Most BCBS plans in New Jersey include some level of international coverage through:

Blue Cross Blue Shield Global Core:

  • Emergency and urgent care coverage abroad
  • Access to international provider networks
  • Pre-authorization requirements for planned treatments

GeoBlue Programs:

  • Enhanced international coverage option
  • Broader network access in Europe
  • Coordination services for complex treatments

Key Steps for International Authorization:

  1. Verify your specific plan's international benefits
  2. Contact the Global Core Service Center before treatment
  3. Obtain pre-authorization for the Milan facility
  4. Coordinate with SR-TIGET for treatment scheduling

For questions about international coverage, contact:

  • From US: 1-800-810-BLUE
  • From abroad: +1-804-673-1177 (collect calls accepted)

Costs and Financial Support

Even with insurance approval, families often face significant out-of-pocket costs for:

  • Travel and accommodation in Milan
  • Extended stay for treatment and monitoring
  • Additional medical expenses not covered by international benefits

Financial Support Resources:

  • Patient advocacy organizations for ADA-SCID
  • Orchard Therapeutics patient support programs
  • Charitable foundations supporting rare disease treatments
  • Hospital financial assistance programs at treating facilities

FAQ

How long does the BCBS prior authorization process take in New Jersey? Standard requests: 30 days. Expedited requests (when delay could harm the patient): 72 hours. The clock starts when BCBS receives your complete submission.

What if Strimvelis isn't on my plan's formulary? Request a medical exception based on medical necessity. Include documentation showing why formulary alternatives aren't appropriate for ADA-SCID treatment.

Can I request an expedited appeal if my child's condition is worsening? Yes. Both internal appeals and IHCAP external reviews offer expedited processes when delays could cause serious harm. Your physician must provide documentation supporting the urgency.

Does New Jersey require step therapy for gene therapies? Most BCBS plans require documentation of failed standard treatments before approving gene therapies. For ADA-SCID, this typically means showing HSCT contraindications or donor unavailability.

What happens if IHCAP overturns the denial? The IHCAP decision is legally binding. BCBS must provide coverage as determined by the external review panel. This is mandated by New Jersey statute (N.J.S.A. 26:2S-11).

How does self-funded vs. fully insured affect my appeal rights? Self-funded ERISA plans follow federal appeal rules and aren't eligible for New Jersey's IHCAP external review. Fully insured plans offer more robust state-level protections.

Patient Advocacy Resources

New Jersey Department of Banking and Insurance:

  • Consumer Hotline: 1-800-446-7467
  • IHCAP-specific support: 1-888-393-1062
  • Website: dobi.nj.gov

Additional Support:

  • New Jersey Managed Care Ombudsman (Department of Human Services)
  • ALS Association New Jersey (provides general appeal guidance)
  • Patient advocacy organizations specific to primary immunodeficiencies

Counterforce Health offers specialized support for families navigating complex gene therapy approvals, helping transform denials into successful appeals through targeted documentation and evidence-based advocacy.

Timeline Overview

Phase Duration Key Activities
Preparation 2-4 weeks Gather documentation, verify coverage
Initial Submission 1-2 weeks Submit complete authorization request
Insurance Review 30 days BCBS medical necessity evaluation
Internal Appeal (if needed) 30-60 days Two levels of internal review
External Review (if needed) 45 days IHCAP independent physician review

Conclusion

Getting Strimvelis covered through Blue Cross Blue Shield in New Jersey requires persistence, thorough documentation, and understanding of both insurance processes and state appeal rights. While challenging, the combination of medical exception requests, comprehensive clinical documentation, and New Jersey's robust external review process through IHCAP provides multiple pathways to approval.

Remember that each case is unique, and success often depends on the specific details of your medical situation and insurance plan. Don't hesitate to seek support from patient advocacy organizations and consider professional assistance with complex appeals.

Sources & Further Reading

Disclaimer: This information is for educational purposes only and does not constitute medical or legal advice. Insurance coverage decisions depend on individual plan terms, medical circumstances, and state regulations. Always consult with your healthcare providers and insurance representatives for guidance specific to your situation. For questions about New Jersey insurance regulations, contact the Department of Banking and Insurance at 1-800-446-7467.

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