By Counterforce Health Team in Exondys 51 (eteplirsen) —

How to Get Exondys 51 (Eteplirsen) Covered by UnitedHealthcare in New York: Complete Prior Authorization and Appeals Guide

Answer Box: Getting Exondys 51 Covered by UnitedHealthcare in New York

UnitedHealthcare requires prior authorization for Exondys 51 (eteplirsen) with specific criteria: confirmed DMD with exon 51-amenable mutation, ambulatory status, specialist prescriber, and baseline testing. First step: Contact a DMD specialist at Mount Sinai or NYU Langone to order genetic testing and functional assessments. If denied, New York residents can file external appeals through the Department of Financial Services within 4 months. Success rate improves with complete genetic documentation and specialist support letters.

Table of Contents

  1. UnitedHealthcare's Exondys 51 Policy Overview
  2. Medical Necessity Requirements
  3. Step Therapy and Exception Pathways
  4. Required Diagnostic Testing
  5. Specialist Prescriber Requirements
  6. Site of Care and Administration
  7. Evidence to Support Your Case
  8. Common Denial Reasons and Solutions
  9. New York Appeals Process
  10. Cost Assistance Options
  11. FAQ

UnitedHealthcare's Exondys 51 Policy Overview

UnitedHealthcare (through OptumRx) classifies Exondys 51 as a specialty medication requiring prior authorization for all plan types in New York, including commercial, Medicare Advantage, and Medicaid managed care plans.

Coverage at a Glance

Requirement Details Documentation Needed Timeline
Prior Authorization Required for all plans Complete PA form + clinical notes 5-15 business days
Formulary Status Specialty tier (high copay) Insurance verification N/A
Step Therapy None explicitly required Document corticosteroid use N/A
Quantity Limits Per FDA dosing (30 mg/kg weekly) Weight-based calculation Ongoing
Site of Care Infusion center or home health Provider network verification Before first dose
Age Restrictions Typically ambulatory patients Functional assessments Initial approval

Medical Necessity Requirements

UnitedHealthcare's medical policy requires meeting all of these criteria:

Core Eligibility Criteria

  1. Confirmed DMD diagnosis with genetic testing showing a mutation amenable to exon 51 skipping
  2. Ambulatory status without assistive devices (no cane, walker, or wheelchair)
  3. Functional capacity: North Star Ambulatory Assessment (NSAA) score >17 OR time to rise <7 seconds
  4. No prior gene therapy (like Elevidys) or documented lack of benefit if previously used
  5. Specialist prescriber experienced in DMD management
  6. Appropriate dosing per FDA labeling (30 mg/kg weekly IV)

Genetic Testing Requirements

Your genetic test report must specifically state:

  • Pathogenic DMD mutation with exact exons affected
  • Confirmation that the mutation is amenable to exon 51 skipping
  • Lab interpretation stating eligibility for eteplirsen therapy
Tip: If your genetic report doesn't explicitly mention exon 51 skipping, ask your neurologist to request an addendum from the genetics lab clarifying amenability.

Step Therapy and Exception Pathways

Unlike many specialty drugs, UnitedHealthcare doesn't require formal step therapy for Exondys 51. However, they do expect documentation of:

  • Corticosteroid therapy (current or previous use with deflazacort or prednisone)
  • Standard DMD care including physical therapy and cardiac monitoring
  • Reason for Exondys 51 over other available treatments

Medical Exceptions

If corticosteroids are contraindicated, document:

  • Specific contraindication (growth concerns, behavioral issues, etc.)
  • Previous trial and intolerance details
  • Alternative therapies attempted

Required Diagnostic Testing

Baseline Testing Requirements

Renal Function (required for approval):

  • Urinalysis showing absence of proteinuria
  • Serum creatinine and BUN (normal values)
  • Repeat every 6-12 months for renewals

Cardiac Assessment:

  • Echocardiogram or cardiac MRI within 12 months
  • Documentation of "stable cardiac function"
  • Cardiology consultation notes

Functional Testing:

  • 6-minute walk test (6MWT) for ambulatory patients
  • North Star Ambulatory Assessment (NSAA)
  • Timed function tests (time to rise, climb stairs)

Pulmonary Function:

  • Forced vital capacity (FVC) if non-ambulatory
  • Documentation of stable respiratory status
Note: Counterforce Health helps patients and clinicians organize these complex testing requirements into targeted appeals that address UnitedHealthcare's specific criteria.

Specialist Prescriber Requirements

Required Prescriber Qualifications

UnitedHealthcare requires prescription by or consultation with a neurologist experienced in DMD. In New York, consider these MDA-certified centers:

Mount Sinai Duchenne Program

NYU Langone Neuromuscular Center

University of Rochester

  • Emma Ciafolini, MD (Pediatric Neurologist)
  • DMD expertise available

Verifying Network Status

Always confirm your chosen specialist is in-network through the UnitedHealthcare provider directory or by calling member services.

Site of Care and Administration

Exondys 51 requires weekly IV infusion over 35-60 minutes. UnitedHealthcare typically covers administration at:

  • Hospital outpatient infusion centers
  • Freestanding infusion clinics
  • Home health services (with prior approval)

Verify your chosen site accepts UnitedHealthcare and has experience with Exondys 51 administration.

Supporting Evidence for Medical Necessity

Clinical Documentation Checklist

Your neurologist's medical necessity letter should include:

  1. Patient history: DMD diagnosis, genetic testing results, current functional status
  2. Treatment rationale: Why Exondys 51 is appropriate for this specific mutation
  3. Baseline assessments: Functional testing results, cardiac/renal status
  4. Monitoring plan: How clinical benefit will be measured
  5. Literature support: FDA approval basis, clinical trial data

Key References to Include

  • FDA Exondys 51 label and approval rationale
  • DMD care guidelines from neuromuscular societies
  • Patient-specific genetic testing interpretation

Common Denial Reasons and Solutions

Denial Reason Solution Required Documentation
"Genetic testing insufficient" Submit complete genetic report with exon 51 amenability statement Full molecular testing results
"Non-specialist prescriber" Transfer care or obtain specialist consultation Neuromuscular neurologist notes
"Missing baseline testing" Complete required assessments Cardiac, renal, functional testing
"Not ambulatory" Document current functional status Recent 6MWT, NSAA scores
"Experimental/investigational" Cite FDA approval and medical necessity FDA label, clinical guidelines

New York Appeals Process

Internal Appeals with UnitedHealthcare

Timeline: 180 days from denial notice Process:

  1. Submit appeal via UnitedHealthcare member portal or mail
  2. Include additional clinical documentation
  3. Request peer-to-peer review if available
  4. Await determination (typically 30 days)

External Appeals Through New York DFS

If UnitedHealthcare upholds the denial, New York residents have strong appeal rights through the Department of Financial Services.

Key Benefits:

  • Independent medical expert review
  • Binding decision on UnitedHealthcare
  • Documented success with Exondys 51 appeals

Filing Requirements:

  • Submit within 4 months of final internal denial
  • Use NY External Appeal Application
  • Include physician attestation and medical records
  • Fee: $25 (waived for Medicaid/hardship)

Expedited Appeals: Available for urgent medical needs (72-hour decision)

From our advocates: "We've seen New York external appeals succeed when families include a detailed genetic counselor letter explaining exactly why their child's mutation requires exon 51 skipping, along with the neurologist's treatment rationale. The independent reviewers appreciate this level of genetic detail."

Getting Help with Appeals

Community Health Advocates: Free assistance at 888-614-5400 NY DFS Consumer Hotline: 800-400-8882

Cost Assistance Options

Manufacturer Support

Sarepta Therapeutics offers patient assistance through their Access360 program. Contact Exondys 51 support for eligibility screening.

Foundation Grants

  • Patient Access Network Foundation
  • HealthWell Foundation
  • National Organization for Rare Disorders (NORD)

New York State Programs

Medicaid expansion covers many families; check eligibility through NY State of Health.

When insurance denials become overwhelming, Counterforce Health specializes in turning complex rare disease denials into successful appeals by analyzing payer policies and crafting evidence-backed responses that speak directly to each plan's criteria.

FAQ

How long does UnitedHealthcare prior authorization take for Exondys 51? Typically 5-15 business days for standard review. Expedited review available for urgent medical needs.

What if Exondys 51 isn't on my formulary? It should be covered as a specialty medication. If denied as non-formulary, request medical exception based on lack of therapeutic alternatives.

Can I get emergency supplies during appeals? Yes, request emergency fills through OptumRx specialty pharmacy, especially for progressive rare diseases.

Do I need to repeat genetic testing? No, but ensure your report clearly states exon 51 amenability. If unclear, request lab interpretation addendum.

What if I move between ambulatory and non-ambulatory status? Notify your care team immediately. Coverage criteria may change, but continuation therapy often has different requirements than initial approval.

How often do I need renewal approvals? Typically every 12 months, requiring updated functional testing and specialist evaluation.

What if my neurologist isn't in-network? Request single-case agreement for specialist care or transfer to in-network DMD expert.

Can I appeal if I'm over the age limit? Yes, especially if you were previously stable on therapy. Document continued clinical benefit and medical necessity.

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. Always consult with your healthcare provider and insurance plan directly. For additional support with insurance appeals in New York, contact Community Health Advocates at 888-614-5400.

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