By Counterforce Health Team in Exondys 51 (eteplirsen) —

Getting Exondys 51 (Eteplirsen) Covered by Blue Cross Blue Shield in New York: Complete PA Guide

Answer Box: Quick Path to Coverage

Exondys 51 (eteplirsen) requires prior authorization from Blue Cross Blue Shield in New York. Your fastest path: 1) Confirm genetic testing shows DMD with exon 51 mutation amenable to skipping, 2) Have a pediatric neurologist or neuromuscular specialist complete the BCBS Medical Authorization Request Form (MARF), and 3) Submit with complete documentation including ICD-10 code G71.01, genetic reports, and baseline function tests. If denied, New York's external appeal process through DFS offers binding review within 30 days.

Table of Contents

Coverage Requirements at a Glance

Requirement What It Means Where to Find It
Prior Authorization Required for all BCBS plans BCBS Provider Portal
Diagnosis Code ICD-10: G71.01 (Duchenne muscular dystrophy) CDC ICD-10 Tool
HCPCS Code J1428 (Injection, eteplirsen, 10 mg) Empire BCBS Policy
Age Requirement Typically 7-13 years at initiation Empire BCBS Criteria
Prescriber Pediatric neurologist or neuromuscular specialist BCBS Medical Policies
Genetic Testing Confirmed exon 51 mutation amenable to skipping Required Documentation
Site of Care Outpatient infusion center, physician office Medical Benefit Coverage

Step-by-Step: Fastest Path to Approval

1. Confirm Eligibility Requirements

Who does it: Prescribing physician
Timeline: 1-2 days

Verify your patient meets BCBS criteria:

  • Confirmed Duchenne muscular dystrophy diagnosis
  • Genetic testing showing mutation amenable to exon 51 skipping
  • Age typically between 7-13 years at treatment start
  • Concurrent corticosteroid therapy (unless contraindicated)

2. Gather Required Documentation

Who does it: Medical team and family
Timeline: 3-5 days

Collect these essential documents:

  • Genetic test results confirming exon 51 mutation
  • Recent weight measurement (within 30 days)
  • Baseline cardiac and pulmonary function tests
  • Documentation of corticosteroid use or contraindications
  • Previous therapy attempts and outcomes

3. Complete the BCBS Prior Authorization Form

Who does it: Prescribing physician
Timeline: 1-2 days

Download and complete the Medical Authorization Request Form (MARF). Include:

  • Patient demographics and insurance information
  • Complete diagnosis with ICD-10 code G71.01
  • Detailed clinical rationale
  • Dosing calculation (30 mg/kg weekly)

4. Submit Through Proper Channel

Who does it: Provider's office
Timeline: Same day

Submit via:

  • BCBS provider portal (preferred)
  • Fax to plan-specific number (verify current number)
  • Include all supporting documentation as attachments

5. Follow Up on Decision

Who does it: Provider's office
Timeline: 5-15 business days

BCBS typically responds within 15 business days for standard requests, 72 hours for urgent cases. Track your request through the provider portal.

ICD-10 Coding and Documentation

Primary Diagnosis Code

Use ICD-10 code G71.01 for Duchenne muscular dystrophy. This is the most specific billable code for DMD, including cases with exon 51 mutations.

Essential Documentation Requirements

Your medical records must include:

Genetic Confirmation

  • Laboratory report showing DMD gene mutation
  • Specific notation of exon 51 amenability
  • Testing laboratory name and date

Clinical Evidence

  • Progressive muscle weakness history
  • Age at symptom onset (typically before age 5)
  • Current functional status
  • Elevated creatine kinase (CK) levels

Treatment History

  • Previous therapies tried and outcomes
  • Current corticosteroid regimen
  • Contraindications to other treatments
Clinician Tip: Always specify "exon 51 mutation" in clinical notes and attach the complete genetic test report. This specificity is crucial for Exondys 51 approval since the drug only works for mutations amenable to exon 51 skipping.

HCPCS Billing and Dosing Calculations

Billing Code and Units

HCPCS Code: J1428 (Injection, eteplirsen, 10 mg)

Dosing Calculation Steps

  1. Determine patient weight (most recent, within 30 days)
  2. Calculate weekly dose: Patient weight (kg) × 30 mg = total mg needed
  3. Convert to billing units: Total mg ÷ 10 = billing units (round up)
  4. Determine vials needed: Total mg ÷ 100 mg per vial = vials required (round up)

Example:

  • 40 kg patient
  • Weekly dose: 40 kg × 30 mg = 1,200 mg
  • Billing units: 1,200 mg ÷ 10 = 120 units
  • Vials needed: 1,200 mg ÷ 100 mg = 12 vials

Common Billing Errors to Avoid

  • Wrong unit conversion: Bill by 10 mg units (J1428), not by vials
  • Outdated weight: Use current weight; adjust dose for weight changes
  • Missing modifiers: Include appropriate site-of-care modifiers
  • Incorrect site coding: Ensure infusion site matches prior authorization

Common Denial Reasons & How to Fix Them

Denial Reason How to Overturn Required Documentation
No genetic confirmation Submit complete genetic test report Lab report showing exon 51 mutation amenable to skipping
Age outside criteria Provide clinical justification Medical necessity letter explaining benefit-risk ratio
Non-specialist prescriber Transfer care or obtain co-management Referral to pediatric neurologist or neuromuscular specialist
Missing baseline tests Complete required assessments Cardiac function, pulmonary function, baseline 6-minute walk test
Quantity/dosing issues Recalculate and justify dose Current weight, dosing calculation, clinical rationale
Site of care not approved Request approved infusion site Documentation of appropriate outpatient facility

Medical Necessity Letter Checklist

When appealing a denial, your medical necessity letter should include:

✓ Patient's specific DMD mutation and exon 51 amenability
✓ Clinical progression and current functional status
✓ Previous treatments tried and reasons for discontinuation
✓ Evidence supporting Exondys 51 efficacy for this mutation
✓ Dosing rationale based on current weight
✓ Monitoring plan and expected outcomes
✓ Citations from FDA labeling and clinical guidelines

New York Appeals Process

If your initial prior authorization is denied, New York offers robust appeal rights through a multi-level process.

Internal Appeal (Level 1)

  • Timeline: Must file within 180 days of denial
  • Decision time: 15 business days (72 hours if urgent)
  • How to file: Through BCBS member portal or by phone

External Appeal Through DFS (Level 2)

  • Timeline: Within 4 months of final internal denial
  • Decision time: 30 days (72 hours if expedited)
  • Cost: $25 fee (waived for hardship cases)
  • Binding: Yes, insurer must comply with decision

To file an external appeal:

  1. Complete the DFS External Appeal Application
  2. Include all medical records and denial letters
  3. Submit within 4 months of final adverse determination
  4. Request expedited review if health is at serious risk
Note: New York's external appeal process has helped overturn many specialty drug denials. The DFS External Appeals Database allows you to research similar cases for precedent.

Getting Help with Appeals

Community Health Advocates
Phone: 888-614-5400
Free assistance with insurance denials and appeals for New York residents

New York Department of Financial Services
Phone: 800-400-8882
Official external appeal applications and guidance

Cost Support Options

Manufacturer Support Programs

Sarepta Therapeutics Patient Support

  • Copay assistance for eligible patients
  • Prior authorization support services
  • Reimbursement specialists
  • Visit: Exondys51.com

Foundation Grants and Assistance

Many rare disease foundations offer financial assistance for DMD treatments. Research organizations like Parent Project Muscular Dystrophy maintain lists of available programs.

State Programs

New York residents may qualify for additional support through:

  • Medicaid coverage (if eligible)
  • State pharmaceutical assistance programs
  • Hospital charity care programs

Frequently Asked Questions

How long does BCBS prior authorization take in New York?
Standard requests: 15 business days. Urgent requests: 72 hours. Track status through the provider portal for updates.

What if Exondys 51 is not on my BCBS formulary?
Request a formulary exception. Provide medical necessity documentation showing why preferred alternatives are inappropriate.

Can I request an expedited appeal?
Yes, if delaying treatment would seriously jeopardize your health. Your physician must certify the urgent medical need.

Does step therapy apply to Exondys 51?
Some BCBS plans may require trial of corticosteroids first. Document any previous steroid use or contraindications.

What happens if I move from another state to New York?
Continue your appeals process in New York if you switch to a New York BCBS plan. Gather all previous medical records and denial letters.

How much does Exondys 51 cost without insurance?
Typical cash price is approximately $7,822 for a 10 mL vial, though actual costs vary by pharmacy and patient weight-based dosing needs.

From our advocates: We've seen families successfully overturn initial Exondys 51 denials by working closely with their neuromuscular specialist to provide comprehensive genetic documentation and detailed clinical rationale. The key is often demonstrating that the patient's specific mutation profile makes them an appropriate candidate for exon 51 skipping therapy, with thorough documentation of previous treatment attempts.

For families navigating the complex world of rare disease insurance coverage, platforms like Counterforce Health are transforming how patients and providers approach prior authorizations and appeals. By analyzing denial letters and payer policies, these specialized services help create targeted, evidence-backed appeals that address the specific reasons for coverage denials. This approach can significantly improve approval rates for specialty medications like Exondys 51, where success often depends on presenting the right clinical evidence in the format that payers expect.

The prior authorization process for Exondys 51 requires attention to detail, but with proper preparation and documentation, most eligible patients can achieve coverage. When working with your healthcare team, remember that Counterforce Health and similar services specialize in turning insurance denials into successful appeals by leveraging payer-specific requirements and clinical evidence standards.

Sources & Further Reading

Disclaimer: This information is for educational purposes only and does not constitute medical or legal advice. Insurance policies and coverage criteria change frequently. Always verify current requirements with your specific BCBS plan and consult with your healthcare provider for medical decisions. For personalized assistance with insurance appeals in New York, contact Community Health Advocates at 888-614-5400 or the New York Department of Financial Services at 800-400-8882.

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