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

If Strimvelis (ADA-SCID Gene Therapy) Isn't Approved by Blue Cross Blue Shield in Florida: Alternative Treatments & Exception Paths

Answer Box: Your Options When Strimvelis Gene Therapy Is Denied

If Blue Cross Blue Shield (BCBS) in Florida denies Strimvelis gene therapy for ADA-SCID, you have three main paths: PEG-ADA enzyme replacement therapy as a bridge treatment, allogeneic stem cell transplant if a donor is available, or formulary exception appeal with comprehensive medical necessity documentation. Start by requesting written denial reasons and contacting your treating immunologist to discuss alternative treatments. Most patients begin with PEG-ADA while pursuing definitive therapy through appeals or transplant evaluation.

Table of Contents

When Alternatives Make Sense

ADA-SCID is a life-threatening condition requiring immediate intervention. When Strimvelis gene therapy isn't initially approved by Blue Cross Blue Shield, alternative treatments can stabilize your child's condition while you work toward securing coverage for definitive therapy.

Consider alternatives when:

  • Initial prior authorization is denied pending additional documentation
  • Strimvelis is not available at a BCBS-contracted facility
  • You're waiting for appeals or external review decisions
  • Your child needs immediate metabolic stabilization

Don't delay treatment while appealing. PEG-ADA enzyme replacement can begin within days of diagnosis and significantly improves survival outcomes while you pursue gene therapy or transplant options.

Note: All ADA-SCID treatments should be coordinated through a specialized pediatric immunology or transplant center experienced with this rare condition.

Treatment Alternatives for ADA-SCID

PEG-ADA Enzyme Replacement Therapy

What it is: Weekly or twice-weekly injections of pegylated adenosine deaminase that replaces the missing enzyme and reduces toxic metabolite buildup.

Coverage status: Generally covered by BCBS plans as a specialty drug requiring prior authorization. Florida Blue typically covers PEG-ADA (Revcovi/Adagen) when diagnostic criteria are met.

Typical authorization requirements:

  • Confirmed ADA-SCID diagnosis by genetic testing and enzyme assay
  • Documentation of severe immunodeficiency
  • Prescribed by pediatric immunologist
  • Regular monitoring plan for ADA levels and immune function

Allogeneic Hematopoietic Stem Cell Transplant (HSCT)

What it is: Curative transplant using stem cells from a matched donor, typically a sibling or unrelated donor from registries.

Coverage status: Blue Cross Blue Shield plans generally cover HSCT for SCID when performed at designated transplant centers.

Requirements:

  • Confirmed ADA-SCID diagnosis
  • Suitable HLA-matched donor identified
  • Treatment at Blue Distinction Center for Transplants
  • Comprehensive pre-transplant evaluation

Clinical Trial Participation

What it is: Access to investigational ADA-SCID gene therapies through research protocols.

Coverage considerations: Clinical trial costs are often covered for routine care elements, with sponsors covering investigational treatments.

How to find trials: Search ClinicalTrials.gov for ADA-SCID studies or ask your immunologist about compassionate use programs.

Pros and Cons Overview

Treatment Pros Cons Access Considerations
PEG-ADA ERT • Rapid stabilization
• Widely available
• Generally covered		 • Requires lifelong treatment
• Incomplete immune correction
• High ongoing costs		 Usually approved within 1-2 weeks
HSCT • Potentially curative
• Well-established
• Covered when criteria met		 • Requires suitable donor
• Transplant-related risks
• Complex procedure		 Donor search can take months
Clinical Trials • Access to cutting-edge therapy
• Comprehensive monitoring
• Often no cost to family		 • Limited availability
• Strict eligibility criteria
• Unknown outcomes		 Geographic limitations

Exception Strategy for Strimvelis

Building Your Case

When pursuing a formulary exception for Strimvelis gene therapy, your appeal must demonstrate medical necessity and lack of appropriate alternatives.

Key documentation needed:

  1. Diagnostic confirmation - Genetic testing results showing ADA gene mutations and enzyme activity levels
  2. Disease severity - Infection history, hospitalizations, immune function tests
  3. Treatment rationale - Why gene therapy is superior to alternatives for your child
  4. Center expertise - Documentation that treatment facility has appropriate experience

Medical Necessity Letter Components

Your physician's letter should address:

Clinical urgency: "ADA-SCID is uniformly fatal without definitive treatment. Current enzyme replacement provides only partial immune correction and requires lifelong administration."

Treatment comparison: Reference long-term gene therapy outcomes showing sustained immune reconstitution and discontinuation of enzyme replacement in most patients.

Cost-effectiveness: Document projected lifetime costs of chronic PEG-ADA versus one-time gene therapy, emphasizing reduced infection-related healthcare utilization.

Supporting Evidence

Include peer-reviewed articles demonstrating:

Switching Between Treatments

From PEG-ADA to Gene Therapy

Timeline considerations:

  • PEG-ADA is typically stopped 1 week before gene therapy
  • Close monitoring required during transition period
  • Most patients don't need to restart enzyme replacement

Insurance coordination:

  • Notify BCBS of treatment plan changes
  • Ensure both therapies are authorized during transition
  • Document medical necessity for timing

From PEG-ADA to HSCT

Preparation phase:

  • Continue PEG-ADA until conditioning begins
  • Coordinate with transplant center financial team
  • Verify coverage for donor search and procurement
Tip: Work with your treatment center's financial coordinators who specialize in BCBS authorizations and can navigate complex approval processes.

Re-Trying for Strimvelis Later

What to Document During Alternative Treatment

While receiving PEG-ADA or preparing for HSCT, maintain detailed records for future Strimvelis appeals:

Treatment response:

  • ADA enzyme levels and toxic metabolite measurements
  • Infection frequency and severity
  • Hospitalizations and complications
  • Quality of life impacts

Ongoing medical necessity:

  • Evidence of incomplete immune correction with alternatives
  • Treatment burden and adherence challenges
  • Long-term prognosis concerns

Changed circumstances:

  • New clinical data supporting gene therapy
  • Availability at additional treatment centers
  • Updated insurance policies or coverage criteria

Resubmission Strategy

Timing: Wait at least 6 months between appeals unless significant new information is available.

New evidence: Include updated clinical trials, expanded FDA approvals, or additional safety data.

Provider support: Consider switching to a physician with more gene therapy experience if needed.

Appeals Process in Florida

Internal Appeals

Timeline: 180 days from denial notice to file internal appeal

Required elements:

  • Written request for reconsideration
  • Additional medical documentation
  • Physician letter addressing denial reasons

Expedited process: Available for urgent cases where delays pose serious health risks

External Review

If internal appeal fails, Florida law provides independent external review through contracted organizations.

Timeline: 4 months after final internal denial to request external review

Process: Independent medical experts review case without insurer involvement

Cost: No charge to consumers for external review

How to request: Contact Florida Department of Financial Services at 877-693-5236

Financial Assistance Options

Manufacturer Support

Orchard Therapeutics may offer patient assistance programs for eligible families. Contact their patient support services to inquire about:

  • Copay assistance programs
  • Compassionate use access
  • Travel and lodging support

Foundation Grants

Organizations providing assistance for rare disease treatments:

  • Immune Deficiency Foundation - Patient support and advocacy
  • National Organization for Rare Disorders (NORD) - Financial assistance programs
  • Be The Match - Transplant-related support if HSCT is pursued

Hospital-Based Programs

Many pediatric centers offer:

  • Charity care programs based on income
  • Payment plans for out-of-pocket costs
  • Social work services to navigate coverage

When to Escalate

Contact Florida insurance regulators if you experience:

  • Repeated denials without adequate review
  • Delays beyond stated timeframes
  • Requests for inappropriate documentation

Florida Office of Insurance Regulation:

Important: Keep detailed records of all communications, including dates, representative names, and reference numbers.

FAQ

How long does Blue Cross Blue Shield prior authorization take in Florida? Standard reviews typically take 15 business days, though urgent cases may be expedited to 72 hours. Complex cases like gene therapy may require additional time for specialist review.

What if Strimvelis is completely non-formulary? You can request a formulary exception based on medical necessity. This requires comprehensive documentation showing no appropriate formulary alternatives exist.

Can I request an expedited appeal for ADA-SCID? Yes, given the life-threatening nature of untreated ADA-SCID, you can request expedited internal and external reviews with decisions typically within 72 hours.

Does step therapy apply if my child failed treatments in another state? Florida Blue should accept documented treatment failures from other states. Provide detailed records of prior therapies, dosing, duration, and reasons for discontinuation.

How much does PEG-ADA cost with Blue Cross Blue Shield coverage? As a specialty tier drug, expect significant copays or coinsurance. Typical out-of-pocket costs range from hundreds to thousands monthly, depending on your specific plan benefits.

What documentation do I need for a peer-to-peer review? Your physician will need complete medical records, treatment history, current clinical status, and rationale for requested therapy. The insurance plan's medical director will review with your doctor directly.

About Counterforce Health: Counterforce Health specializes in turning insurance denials into successful appeals for complex therapies like gene treatments. Our platform analyzes denial letters, identifies specific coverage criteria, and helps families build evidence-backed appeals that align with payer requirements. For ADA-SCID cases involving Blue Cross Blue Shield, we can help coordinate the medical necessity documentation and appeals strategy while you focus on your child's care.

When facing a Strimvelis denial, remember that alternative treatments can provide crucial stabilization while you pursue definitive therapy through appeals or clinical trials. Work closely with experienced immunology centers that understand both the medical complexities of ADA-SCID and the insurance landscape in Florida. With persistence and proper documentation, many families ultimately secure coverage for their preferred treatment option.

Sources & Further Reading

Disclaimer: This information is for educational purposes and does not constitute medical or legal advice. Insurance coverage varies by plan and individual circumstances. Always consult with your healthcare provider and insurance representative for guidance specific to your situation. For assistance with insurance complaints in Florida, contact the Department of Financial Services at 877-693-5236.

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