By Counterforce Health Team in Brineura (cerliponase alfa) —

How to Get Brineura (Cerliponase Alfa) Covered by Blue Cross Blue Shield in Pennsylvania: Complete Prior Authorization and Appeals Guide

Answer Box: Getting Brineura Covered by Blue Cross Blue Shield in Pennsylvania

Brineura (cerliponase alfa) requires prior authorization from Blue Cross Blue Shield plans in Pennsylvania, including Highmark and Independence Blue Cross. Coverage is approved for pediatric patients with confirmed CLN2 disease (TPP1 deficiency) when administered at specialized centers by neurologists experienced in intraventricular therapy. First step today: Contact your child's neurologist to initiate the PA request with confirmed CLN2 diagnosis, baseline functional assessment, and documentation of no contraindications. If denied, Pennsylvania's new external review program has a 50% overturn rate for specialty drug appeals.

Table of Contents

  1. Coverage Requirements at a Glance
  2. Step-by-Step: Fastest Path to Approval
  3. Common Denial Reasons & How to Fix Them
  4. Appeals Playbook for Pennsylvania Blue Cross Blue Shield
  5. Medical Necessity Letter Checklist
  6. Pennsylvania External Review Process
  7. Scripts & Templates
  8. Costs & Patient Support Options
  9. Frequently Asked Questions

Coverage Requirements at a Glance

Requirement What It Means Where to Find It Source
Prior Authorization Required for all Brineura prescriptions Provider portal or member services Highmark Policy
Diagnosis Confirmed CLN2 disease (TPP1 deficiency) Enzyme testing or genetic confirmation UHC PA Policy
Specialist Required Neurologist with CLN2/lysosomal expertise Hospital neurology departments PA Health & Wellness
Age Requirement All pediatric ages (FDA expanded 2021) FDA labeling FDA Access Data
Administration Site Intraventricular infusion center FDA-approved facilities BioMarin Coverage Guide
Initial Approval 6 months typical PA determination letter BCBS PA Policy

Step-by-Step: Fastest Path to Approval

1. Confirm CLN2 Diagnosis (Neurologist)

What's needed: TPP1 enzyme deficiency test or genetic testing showing pathogenic CLN2/TPP1 mutations Timeline: Results typically available in 2-3 weeks Submit to: Include with initial PA request

2. Baseline Assessment (Medical Team)

What's needed: CLN2 Clinical Impairment Score, ambulation status, seizure history Who does it: Pediatric neurologist specializing in lysosomal disorders Documentation: Functional assessment showing potential for benefit

3. Prior Authorization Submission (Provider)

Submit via: Blue Cross Blue Shield provider portal or fax Include: Diagnosis confirmation, specialist recommendation, baseline scores, treatment plan Timeline: 30-45 days for standard review; request expedited if urgent

4. Identify Infusion Center (Care Team)

Requirement: FDA-approved center with intraventricular capability Contact: Children's Hospital of Philadelphia or UPMC Children's Hospital neurology departments Verify: Center accepts your Blue Cross Blue Shield plan

5. Device Placement Coordination (Neurosurgery)

What's needed: Intraventricular reservoir/catheter placement Pre-approval: Ensure both drug and surgical procedure are authorized Timeline: Schedule after drug approval confirmed

6. Treatment Initiation (Infusion Center)

Dosing: 300mg every 2 weeks (or age-appropriate dose) Monitoring: 4+ hour infusion with hydrocephalus monitoring Documentation: Track functional outcomes for reauthorization

7. Reauthorization Planning (Provider)

Timeline: Submit 30 days before 6-month approval expires Evidence needed: Functional stability or improvement, continued medical necessity Documentation: Compare baseline to current CLN2 scores

Common Denial Reasons & How to Fix Them

Denial Reason How to Overturn Required Documentation
"Age/diagnosis mismatch" Submit genetic testing confirmation TPP1 enzyme assay + pathogenic mutations
"Lack of specialist care" Obtain neurologist evaluation Letter from CLN2-experienced neurologist
"Inappropriate setting" Identify FDA-approved center Confirmation from qualified infusion facility
"Not medically necessary" Document functional decline Baseline vs. current CLN2 Clinical Impairment Scores
"Experimental/investigational" Cite FDA approval Reference FDA label for CLN2 indication
"Missing prior authorization" Resubmit with complete packet All required forms and clinical documentation
From our advocates: We've seen families successfully overturn denials by ensuring the neurologist's letter explicitly states the child has "confirmed CLN2 disease with potential for ambulation benefit" rather than generic language about "neurological decline." This specific phrasing aligns with payer criteria and significantly improves approval odds.

Appeals Playbook for Pennsylvania Blue Cross Blue Shield

Internal Appeals Process

First-Level Internal Appeal

  • Deadline: 180 days from denial notice
  • Submit to: Blue Cross Blue Shield appeals department (address on denial letter)
  • Include: New medical evidence, specialist letter, peer-to-peer request
  • Timeline: 30 days for standard; 72 hours for urgent

Second-Level Internal Appeal

  • When: After first-level denial
  • Process: Automatic review by different medical director
  • Outcome: Final Adverse Benefit Determination letter
  • Next step: Enables external review eligibility

Peer-to-Peer Review Process

How to request: Call provider services during first-level appeal Who participates: Prescribing neurologist and Blue Cross Blue Shield medical director Preparation checklist:

  • Patient's specific CLN2 presentation
  • Why alternative treatments are inadequate
  • Evidence of functional decline without treatment
  • Brineura's mechanism and expected benefit

Medical Necessity Letter Checklist

Clinical Documentation Requirements

Patient Information:

  • Full name, DOB, member ID, group number
  • Confirmed CLN2 diagnosis with test results
  • Current functional status and progression

Medical History:

  • Age at symptom onset (typically 2-4 years)
  • Initial symptoms: seizures, ataxia, speech delay
  • Progressive motor and cognitive decline
  • Current CLN2 Clinical Impairment Score

Treatment Rationale:

  • Why Brineura is medically necessary for this patient
  • Expected outcomes: slowing ambulation loss
  • Risks of not treating: continued neurological decline
  • No alternative disease-modifying therapies available

Administration Plan:

  • Intraventricular delivery via implanted reservoir
  • 300mg every 2 weeks at qualified center
  • Monitoring plan for device complications

Supporting Evidence:

  • FDA approval for CLN2 disease
  • Clinical trial data showing 87% response rate
  • 2021 expert consensus supporting early treatment
  • Patient-specific factors supporting benefit potential

Counterforce Health specializes in crafting evidence-based appeals that address payer-specific denial criteria. Their platform analyzes denial letters and creates targeted rebuttals using the right clinical evidence and procedural requirements, significantly improving approval rates for complex specialty drugs like Brineura.

Pennsylvania External Review Process

New State-Run Program (Launched 2024)

Pennsylvania's Independent External Review program offers a powerful appeal option after internal appeals are exhausted. Key statistics: 50% of external reviews are overturned, requiring insurers to cover previously denied treatments.

Eligibility Requirements:

  • Commercial insurance (employer, Pennie, direct-purchase)
  • Completed internal appeals with Final Adverse Benefit Determination
  • Medical necessity, experimental treatment, or coverage disputes
  • Excludes: Self-funded plans, Medicaid, Medicare

How to Submit:

  1. Gather documents: Final denial letter, insurance card, medical records
  2. Submit online: PA.gov external review portal
  3. Timeline: Within 4 months of final internal denial

Review Process:

  • PID sends to insurer for eligibility (1 business day)
  • Insurer confirms eligibility (5 business days)
  • Independent Review Organization assigned (1 business day)
  • Submit additional evidence (15 days from assignment)
  • IRO decision (45 days from assignment)

Expedited Reviews (for urgent cases):

  • Requirement: Physician certification of serious health risk
  • Timeline: 72 hours from IRO assignment
  • Process: Direct contact with assigned IRO for additional information

Scripts & Templates

Patient Phone Script for Blue Cross Blue Shield

"Hello, I'm calling about prior authorization for my child's Brineura prescription. The member ID is [number]. Our neurologist has diagnosed confirmed CLN2 disease and recommended this FDA-approved treatment. Can you tell me the status of our PA request submitted on [date]? If it was denied, I'd like to understand the specific reason and request a peer-to-peer review with our neurologist."

Clinic Staff Script for Peer-to-Peer

"I'm requesting a peer-to-peer review for [patient name], member ID [number], regarding denied Brineura coverage. Our pediatric neurologist Dr. [name] has extensive experience with CLN2 disease and can discuss the medical necessity. The patient has confirmed TPP1 deficiency with progressive functional decline. When can we schedule this clinical discussion?"

Appeal Letter Template Opening

"Dear Medical Director,

I am writing to formally appeal the denial of Brineura (cerliponase alfa) coverage for [patient name], member ID [number]. This pediatric patient has confirmed CLN2 disease (TPP1 deficiency) and meets all criteria in your policy for this FDA-approved, life-extending therapy. The denial appears to be based on [specific reason from letter], which I will address with clinical evidence below."

Costs & Patient Support Options

Financial Assistance Programs

BioMarin RareConnections

  • Copay assistance for eligible commercial insurance
  • Case management support for PA and appeals
  • Financial hardship programs
  • Contact: 1-866-906-6100

National Organization for Rare Disorders (NORD)

  • Patient assistance programs
  • Rare Disease Coping Fund
  • State-specific resources
  • Website: rarediseases.org

Pennsylvania Resources

  • CHIP/Medicaid coverage for eligible families
  • Pennsylvania Insurance Department consumer assistance
  • Children's hospitals often have financial counselors

Manufacturer Support

BioMarin provides comprehensive support through their RareConnections program, including:

  • Prior authorization assistance
  • Appeals support and documentation
  • Reimbursement specialists
  • Patient access coordinators

Frequently Asked Questions

How long does Blue Cross Blue Shield PA take in Pennsylvania? Standard prior authorization decisions are made within 30-45 days. Expedited reviews for urgent cases are completed within 72 hours when medical urgency is documented.

What if Brineura is non-formulary on my plan? Non-formulary drugs can still be covered through formulary exceptions. Submit a PA request with medical necessity documentation explaining why no formulary alternatives are appropriate for CLN2 disease.

Can I request an expedited appeal if my child is declining rapidly? Yes. Pennsylvania allows expedited appeals when delay could seriously jeopardize health. Include physician certification of urgency with your appeal request.

Does step therapy apply to Brineura? Most Blue Cross Blue Shield plans don't require step therapy for Brineura since no alternative disease-modifying treatments exist for CLN2 disease. However, verify your specific plan's policy.

What happens if both internal appeals are denied? You can file an external review through Pennsylvania's Independent External Review program within 4 months. This state-supervised process has a 50% success rate for overturning denials.

Which hospitals in Pennsylvania can administer Brineura? Contact Children's Hospital of Philadelphia (CHOP) or UPMC Children's Hospital neurology departments to verify their Brineura infusion capabilities and your insurance acceptance.

How much does Brineura cost without insurance? List price is approximately $27,000 per biweekly infusion ($702,000 annually), plus additional costs for the infusion center and monitoring. Patient assistance programs may help reduce out-of-pocket costs.

Can I appeal to the state if Blue Cross Blue Shield keeps denying coverage? Yes. After exhausting internal appeals, Pennsylvania's external review program provides independent medical review. You can also file complaints with the Pennsylvania Insurance Department if you believe the insurer is not following proper procedures.

When navigating complex specialty drug approvals like Brineura, having expert support can make the difference between approval and denial. Counterforce Health transforms insurance denials into targeted, evidence-backed appeals by analyzing payer policies and crafting point-by-point rebuttals that address specific denial criteria. Their platform helps patients, clinicians, and specialty pharmacies get prescription drugs approved by turning the appeals process from guesswork into a systematic, data-driven approach.

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 and medical circumstances. Always consult with your healthcare provider and insurance company for specific guidance. For additional help with Pennsylvania insurance appeals, contact the Pennsylvania Insurance Department Consumer Services at 1-877-881-6388.

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