The Requirements Checklist to Get Strimvelis (Autologous CD34+ ADA-SCID Gene Therapy) Covered by Blue Cross Blue Shield in Pennsylvania
Answer Box: Fast Track to Approval
Getting Strimvelis (autologous CD34+ ADA-SCID gene therapy) covered by Blue Cross Blue Shield in Pennsylvania requires prior authorization via medical exception due to non-FDA approval and international treatment location (SR-TIGET, Milan). Success depends on: (1) confirmed ADA-SCID diagnosis with biallelic ADA mutations and enzyme activity <1% normal, (2) documented HSCT failure/ineligibility, and (3) comprehensive medical necessity packet including genetic testing, immunology workup, and specialist letter. Submit through your plan's provider portal with billing code J3590 (unclassified biologics). If denied, Pennsylvania's new external review program has a 50% overturn rate for specialty therapies. Start today: Contact your Blue Cross case management team at 1-800-ASK-BLUE and request assignment of a complex-case coordinator.
Table of Contents
- Who Should Use This Checklist
- Member & Plan Basics
- Clinical Criteria Requirements
- Coding and Billing Requirements
- Documentation Packet Checklist
- Submission Process
- After Submission: What to Expect
- Common Denial Reasons & How to Fix Them
- Pennsylvania Appeals Process
- Printable Quick Reference
Who Should Use This Checklist
This checklist is designed for patients with confirmed ADA-SCID and their healthcare teams seeking coverage for Strimvelis gene therapy through any Blue Cross Blue Shield plan in Pennsylvania, including:
- Highmark Blue Shield (western PA)
- Independence Blue Cross (Philadelphia region)
- Blue Cross plans serving central and northeastern Pennsylvania
Expected outcome: With complete documentation meeting all clinical criteria, initial approval rates improve significantly. If denied, Pennsylvania's external review process has overturned 50% of specialty therapy denials since launching in 2024.
Key challenge: Strimvelis is not FDA-approved and administered only at SR-TIGET in Milan, Italy, requiring navigation of experimental/investigational exclusions and international care policies.
Member & Plan Basics
Coverage Verification Requirements
| Requirement | What to Check | Where to Find It |
|---|---|---|
| Active coverage | Current enrollment status | Member services: 1-800-ASK-BLUE |
| Plan type | HMO, PPO, EPO, self-funded | Benefits summary or HR department |
| International coverage | Out-of-network/overseas benefits | Policy booklet Section 7-8 |
| Gene therapy coverage | Specialty drug benefits | Formulary or medical policy section |
| Deductible status | Applied amounts, remaining | Online portal or member services |
Critical: Self-funded employer plans may not be eligible for Pennsylvania's external review process. Verify with HR whether your plan is fully insured (eligible) or self-funded (ERISA appeals only).
Prior Authorization Requirements
All Blue Cross plans in Pennsylvania require prior authorization for Strimvelis due to:
- Non-formulary status (no specific HCPCS code)
- High cost (€594,000 / ~$650,000 USD)
- International site of care
- Experimental/investigational classification
Clinical Criteria Requirements
Primary Diagnostic Confirmation
ADA-SCID diagnosis requires ONE of the following:
- Biochemical confirmation: ADA enzyme activity <1% of normal in erythrocytes or plasma
- Genetic confirmation: Biallelic pathogenic variants in the ADA gene
Supporting Clinical Evidence
| Criterion | Required Documentation | Source |
|---|---|---|
| Immune phenotype | T⁻ B⁻ NK⁻ SCID pattern on flow cytometry | Immunology lab report |
| Clinical severity | Recurrent infections, failure to thrive | Pediatric immunology notes |
| HSCT ineligibility | No matched donor or high-risk contraindications | HLA typing and transplant evaluation |
| Treatment history | PEG-ADA trials/failures if applicable | Pharmacy records and response data |
Age and Functional Status
- No specific age limits in Blue Cross policies
- Performance status adequate for international travel and procedure
- Cardiac/hepatic/renal function within SR-TIGET protocol requirements
Coding and Billing Requirements
Primary Billing Codes
| Code Type | Code | Description | Units |
|---|---|---|---|
| HCPCS | J3590 | Unclassified biologics | 1 per administration |
| ICD-10 | D81.110 | Severe combined immunodeficiency due to adenosine deaminase deficiency | Primary diagnosis |
| CPT | 96365 | IV infusion, initial | If applicable for preparation |
Note: Include detailed description: "Strimvelis autologous CD34+ gene therapy for ADA-SCID" with all J3590 submissions.
Required Modifiers
- International care modifier (if required by specific plan)
- Site of service: SR-TIGET Milan, Italy
- Provider NPI: Use treating immunologist's NPI for PA submission
Documentation Packet Checklist
Core Medical Necessity Letter Elements
Your immunologist's letter must include:
□ Patient identification and diagnosis
- ADA-SCID confirmed by [genetic/biochemical testing]
- ICD-10: D81.110
- Date of diagnosis and clinical presentation
□ Diagnostic evidence (attach reports)
- ADA enzyme activity <1% normal OR biallelic ADA mutations
- Lymphocyte subset analysis showing T⁻ B⁻ NK⁻ pattern
- Immunoglobulin levels and vaccine response data
□ Treatment history and failures
- HSCT evaluation: donor search results, contraindications
- PEG-ADA therapy history and limitations
- Current clinical status and prognosis
□ Medical necessity rationale
- Life-threatening nature of untreated ADA-SCID
- Strimvelis as standard of care internationally
- No equivalent FDA-approved alternative available
□ Treatment plan specifics
- SR-TIGET acceptance letter and protocol
- Cost estimate (€594,000)
- Expected length of stay and follow-up requirements
Required Attachments
□ Laboratory reports
- Genetic testing confirming biallelic ADA mutations
- ADA enzyme activity results
- Complete lymphocyte subset analysis
- Metabolic markers (adenosine/deoxyadenosine if available)
□ Clinical documentation
- Immunology consultation notes
- Infection history and hospitalizations
- Growth charts and developmental assessments
- HSCT evaluation and donor search records
□ Literature support
- Key peer-reviewed studies on Strimvelis efficacy
- International treatment guidelines citing gene therapy
- Comparative effectiveness data vs. long-term enzyme replacement
□ International treatment documentation
- SR-TIGET acceptance letter
- Detailed cost estimate in USD
- Treatment timeline and logistics plan
Submission Process
Step-by-Step Submission
1. Contact Blue Cross case management (1-2 days)
- Call member services: 1-800-ASK-BLUE
- Request assignment of complex case coordinator
- Explain need for international gene therapy PA review
2. Identify correct submission pathway (same day)
- Provider portal vs. fax submission
- Correct PA form version for your specific Blue plan
- Required fields and attachment formats
3. Compile complete documentation (1-2 weeks)
- Medical necessity letter from immunologist
- All laboratory and clinical reports
- International treatment documentation
- Literature support package
4. Submit via provider portal (same day)
- Upload all documents as single PDF when possible
- Include detailed HCPCS description with J3590
- Request expedited review if clinically urgent
5. Obtain confirmation (within 24 hours)
- PA request number and submission date
- Assigned reviewer contact information
- Expected timeline for initial determination
Common Submission Errors to Avoid
- Incomplete genetic testing: Must show biallelic pathogenic variants
- Missing HSCT evaluation: Required to justify gene therapy over transplant
- Insufficient cost justification: Include lifetime cost comparison with enzyme replacement
- Vague international care rationale: Specify why US alternatives are inadequate
After Submission: What to Expect
Review Timeline
| Review Type | Standard Timeline | Expedited Timeline |
|---|---|---|
| Initial review | 15-30 business days | 72 hours |
| Clinical review | Additional 10-15 days | 24-48 hours |
| Final determination | 30-45 days total | 3-5 days total |
Status Monitoring
□ Week 1: Confirm receipt and completeness □ Week 2: Check for additional information requests □ Week 3: Follow up if no preliminary decision □ Week 4: Request peer-to-peer review if needed □ Week 5+: Prepare appeal documentation if denied
Tip: Document all phone calls with confirmation numbers and representative names.
Common Denial Reasons & How to Fix Them
| Denial Reason | How to Overturn | Required Documentation |
|---|---|---|
| Experimental/investigational | Cite EMA approval and published long-term data | Peer-reviewed studies, international guidelines |
| No US alternative tried | Document HSCT ineligibility and PEG-ADA limitations | Transplant evaluation, enzyme replacement records |
| International care exclusion | Request medical necessity exception | Letter stating no equivalent US option available |
| Insufficient genetic confirmation | Provide complete variant analysis | CLIA lab report with pathogenicity assessment |
| Cost not justified | Compare lifetime costs vs. alternatives | Economic analysis including hospitalizations |
Appeal Preparation
If your initial request is denied, immediately begin preparing your internal appeal:
□ Request complete denial rationale in writing □ Address each cited reason point-by-point □ Gather additional supporting literature □ Consider independent medical review from ADA-SCID expert □ Prepare for peer-to-peer discussion with plan medical director
Pennsylvania Appeals Process
Pennsylvania's new external review program offers strong protections for denied specialty therapies:
Internal Appeals (Required First Step)
Timeline: Must be filed within 60 days of denial Process: Submit through Blue Cross provider portal or member services Documentation: Complete appeal packet addressing each denial reason
External Review (After Internal Appeals)
Eligibility: Available after Final Adverse Benefit Determination Timeline: Must request within 4 months of final internal denial Success rate: 50% of appeals overturned in favor of patients Cost: Free to consumers
Expedited review available: If delay poses serious health threat, can be filed immediately with physician certification
How to File External Review
- Complete internal appeals and obtain Final Adverse Benefit Determination
- Submit request via Pennsylvania Insurance Department portal
- Provide supporting documentation within 15 business days of IRO assignment
- Receive decision within 45 days (standard) or 72 hours (expedited)
For assistance, contact Pennsylvania Insurance Department Consumer Services at the number listed on their official website.
Printable Quick Reference
Pre-Submission Checklist
□ Active Blue Cross coverage verified □ ADA-SCID diagnosis confirmed (genetic + biochemical) □ HSCT evaluation documenting ineligibility □ SR-TIGET acceptance letter obtained □ Medical necessity letter completed □ All lab reports and clinical notes gathered □ Literature support package compiled □ Case coordinator assigned
Post-Submission Monitoring
□ PA confirmation number obtained □ Weekly status checks scheduled □ Appeal documentation prepared □ Pennsylvania external review timeline noted □ Alternative funding sources researched
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Sources & Further Reading
- Pennsylvania Insurance Department External Review Process
- Highmark Blue Shield Medical Policies
- Independence Blue Cross Formulary Exception Process
- ADA-SCID Diagnostic Criteria (HRSA)
- Strimvelis EMA Product Information
Disclaimer: This guide provides general information about insurance coverage processes and should not be considered medical or legal advice. Coverage decisions vary by individual plan and clinical circumstances. Always consult with your healthcare team and insurance plan directly for guidance specific to your situation. For questions about Pennsylvania's external review process, contact the Pennsylvania Insurance Department directly through their official website.
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