By Counterforce Health Team in Exondys 51 (eteplirsen) —

Blue Cross Blue Shield Ohio's Coverage Criteria for Exondys 51: What Counts as "Medically Necessary"?

Answer Box

Blue Cross Blue Shield of Ohio (Anthem) requires prior authorization for Exondys 51 (eteplirsen) with specific medical necessity criteria: confirmed DMD with exon 51-amenable genetic mutation, stable corticosteroid therapy ≥6 months, meaningful motor function, and neurologist prescriber. Submit PA via Anthem provider portal with genetic test results, baseline labs (kidney function, urinalysis), and treatment history. Standard review takes 15 business days; expedited within 72 hours for urgent cases. Appeal denials through Ohio's external review process within 180 days. First step today: Contact your DMD specialist to order genetic testing confirming exon 51 skipping eligibility and gather baseline cardiac/pulmonary assessments.

Table of Contents

Policy Overview

Blue Cross Blue Shield of Ohio, operating as Anthem, covers Exondys 51 (eteplirsen) as a specialty tier medication requiring prior authorization across all plan types—HMO, PPO, and marketplace plans. The drug targets Duchenne muscular dystrophy (DMD) patients with confirmed genetic mutations amenable to exon 51 skipping, affecting approximately 13% of DMD patients.

Anthem's clinical criteria align with FDA accelerated approval requirements and focus on genetic confirmation, specialist oversight, and baseline safety assessments. Coverage policies are updated regularly; the most recent review cycle occurred in Q2 2024 with next updates scheduled for 2026.

Note: Self-funded employer plans may have different criteria. Verify your specific plan's formulary through the Anthem Formulary Navigator.

Coverage at a Glance

Requirement What It Means Where to Find It Source
Prior Authorization Required for all Exondys 51 prescriptions Anthem provider portal Anthem PA Guidelines
Formulary Tier Specialty Tier 4-5 (higher copays) Plan-specific formulary Verify with plan
Step Therapy Stable corticosteroids ≥6 months Clinical documentation FDA Label Requirements
Quantity Limits 30 mg/kg weekly maximum Weight-based dosing FDA prescribing information
Site of Care Non-hospital facility or specialty pharmacy network Network directory Anthem provider portal
Age Limits Typically pediatric/young adult focus Case-by-case review Clinical policy
Diagnosis Code ICD-10: G71.01 (DMD) Medical records Required for PA
Appeals Deadline 180 days for external review in Ohio Ohio Department of Insurance ODI Appeal Process

Indication Requirements

Exondys 51 received FDA accelerated approval for DMD patients with confirmed mutations amenable to exon 51 skipping. Anthem requires:

Primary Diagnosis

  • Duchenne muscular dystrophy (ICD-10: G71.01) confirmed by:
    • Clinical presentation consistent with DMD
    • Elevated creatine kinase levels
    • Muscle biopsy showing dystrophin deficiency (if performed)

Genetic Testing Mandate

The cornerstone requirement is genetic confirmation of exon 51 skipping eligibility. Acceptable mutations include:

  • Deletions of exons 45-50, 48-50, 49-50, or exon 50 alone
  • Specific duplications that restore reading frame when exon 51 is skipped
  • Results from validated DMD gene sequencing or MLPA testing
Tip: Use the Exondys 51 genetic eligibility tool to verify mutation compatibility before PA submission.

Step Therapy & Exceptions

Required Prior Therapy

Anthem mandates stable corticosteroid therapy for ≥6 months unless contraindicated or intolerated. Acceptable corticosteroids include:

  • Deflazacort (Emflaza) - preferred
  • Prednisone or prednisolone
  • Other corticosteroids with documented rationale

Medical Exception Pathways

Exceptions to corticosteroid requirements are granted for:

  • Documented intolerance: Growth suppression, behavioral changes, bone density loss
  • Medical contraindications: Active infection, diabetes complications, severe osteoporosis
  • Prior treatment failure: Lack of benefit or disease progression despite compliance

Document exceptions with:

  • Clinical notes detailing adverse effects
  • Growth charts or bone density scans
  • Specialist consultation notes

Required Diagnostics

Baseline Laboratory Requirements

Submit recent results (within 30 days of PA request):

Renal Function Assessment:

  • BUN and serum creatinine
  • BUN/creatinine ratio
  • Urinalysis without proteinuria
  • Estimated GFR if applicable

Cardiac and Pulmonary Baseline:

  • Echocardiogram with ejection fraction
  • Electrocardiogram (ECG)
  • Pulmonary function tests (if age-appropriate)
  • Six-minute walk test (6MWT) distance

Functional Status Documentation

Anthem requires evidence of meaningful voluntary motor function, typically demonstrated by:

  • Ambulatory status with ≥300m 6MWT distance
  • NSAA (North Star Ambulatory Assessment) score >17
  • Ability to perform activities of daily living
  • Stable or slowly progressive disease course
Clinician Corner: Include baseline assessments in your medical necessity letter, emphasizing the patient's current functional capacity and potential for benefit from dystrophin restoration therapy.

Site of Care & Specialty Pharmacy

Specialty Pharmacy Networks

Exondys 51 must be sourced through Anthem's designated specialty pharmacy network:

  • Amber Specialty Pharmacy
  • Option Care Health
  • CVS Specialty (preferred for some plans)
  • Orsini Pharmaceutical Services

Administration Requirements

Anthem's Site of Care policy mandates administration in non-hospital facility-based locations:

  • Ambulatory infusion centers
  • Specialty clinics
  • Home infusion (via network providers)
  • Hospital outpatient only for initial doses or documented instability
Note: Using out-of-network pharmacies or inappropriate sites of care will trigger automatic denials.

Evidence to Support Necessity

Clinical Guidelines to Cite

Reference these authoritative sources in medical necessity letters:

  • FDA prescribing information for Exondys 51
  • Duchenne Registry data supporting dystrophin increase
  • Parent Project MD Care Considerations for DMD management
  • MDA Clinical Practice Guidelines for neuromuscular disorders

Sample Medical Necessity Paragraph

"This 12-year-old patient with genetically confirmed DMD (deletion of exons 45-50, amenable to exon 51 skipping per genetic testing dated [DATE]) has been on stable deflazacort 0.9 mg/kg/day for 8 months with good tolerance. Current 6MWT distance is 350 meters, NSAA score 19, demonstrating meaningful motor function. Baseline renal function normal (creatinine 0.6 mg/dL, normal urinalysis), cardiac function stable (EF 58% on recent echo). Exondys 51 30 mg/kg weekly IV is indicated per FDA labeling to increase dystrophin expression and potentially slow disease progression in this exon 51-amenable patient."

At Counterforce Health, we help patients and clinicians navigate complex prior authorization requirements by analyzing denial patterns and crafting evidence-based appeals tailored to each payer's specific criteria. Our platform identifies the exact documentation needed to meet medical necessity standards and streamlines the approval process for specialty medications like Exondys 51.

Appeals Playbook for Ohio

Internal Appeals with Anthem

  • Timeline: 60 days to file from denial notice
  • Decision deadline: 15 business days (standard), 72 hours (expedited)
  • Submission: Anthem provider portal or member services
  • Required documents: Denial letter, additional clinical evidence, peer-reviewed studies

Ohio External Review Process

If Anthem upholds the denial:

  1. Request external review within 180 days of final adverse determination
  2. Submit through Anthem (not directly to Ohio DOI)
  3. Timeline: 30 days for standard review, 72 hours for expedited
  4. Cost: No charge to patient or provider
  5. Decision: Binding on Anthem if overturned

Ohio Department of Insurance Consumer Hotline: 1-800-686-1526

Expedited Appeals

Request expedited review if delay would:

  • Seriously jeopardize patient's health
  • Compromise ability to regain maximum function
  • Create risk of permanent disability

Common Denial Reasons & Fixes

Denial Reason How to Overturn Required Documentation
Missing genetic testing Submit validated DMD gene analysis Lab report showing exon 51 amenability
Non-specialist prescriber Transfer care or obtain co-signature Neurologist/neuromuscular specialist credentials
Insufficient prior therapy Document corticosteroid trial Treatment records showing ≥6 months stable dosing
Inadequate baseline assessments Complete required testing Recent labs, cardiac echo, pulmonary function
Quantity/dosing concerns Justify weight-based calculation Current weight, dosing rationale, FDA guidelines
Site of care issues Coordinate with network providers Confirmation of approved infusion center
From Our Advocates: "We've seen successful appeals where families initially received denials for 'experimental' status. The key was demonstrating that Exondys 51 has full FDA approval for the patient's specific genetic subtype and citing the accelerated approval pathway. Including dystrophin expression data from clinical trials alongside the patient's genetic test results created a compelling medical necessity case."

FAQ

How long does Anthem PA take in Ohio? Standard review: 15 business days. Expedited review: 72 hours for urgent cases. Submit complete documentation to avoid delays.

What if Exondys 51 is non-formulary on my plan? Request formulary exception with medical necessity letter citing lack of therapeutic alternatives for exon 51-amenable DMD.

Can I request an expedited appeal? Yes, if delay would jeopardize health or functional capacity. Provide physician certification of urgency.

Does step therapy apply if I've failed corticosteroids outside Ohio? Prior treatment history from other states counts. Submit complete medical records documenting trials and outcomes.

What's the cost without insurance coverage? Approximately $300,000-400,000 annually. SareptAssist offers patient support programs and financial assistance.

How often do I need to renew PA approval? Typically every 6-12 months. Renewal requires demonstration of continued clinical benefit and absence of unacceptable toxicity.

When navigating complex specialty drug approvals, Counterforce Health provides expert support by analyzing your specific denial, identifying gaps in documentation, and crafting targeted appeals that address payer-specific medical necessity criteria. Our platform helps ensure your Exondys 51 appeal includes all required evidence and follows Ohio's procedural requirements.

Sources & Further Reading

Disclaimer: This information is for educational purposes only and does not constitute medical or legal advice. Coverage decisions depend on individual circumstances and plan specifics. Consult your healthcare provider and insurance plan for personalized guidance. For additional help with Ohio insurance appeals, contact the Ohio Department of Insurance at 1-800-686-1526.

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