By Counterforce Health Team in Exondys 51 (eteplirsen) —

Work With Your Doctor to Get Exondys 51 (eteplirsen) Covered by UnitedHealthcare in New Jersey: Complete Guide

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

Eligibility: Exondys 51 requires prior authorization from UnitedHealthcare for patients with Duchenne muscular dystrophy (DMD) and a confirmed mutation amenable to exon 51 skipping.

Fastest approval path:

  1. Ensure prescriber is a neurologist with DMD expertise
  2. Gather genetic testing confirming exon 51 amenability
  3. Complete required functional assessments (6-minute walk test, cardiac/pulmonary baselines)

First step today: Schedule an appointment with your neurologist to review UnitedHealthcare's specific documentation requirements and begin collecting baseline testing results. If denied, New Jersey's IHCAP external review through Maximus Federal Services provides binding appeals within 4 months.

Table of Contents

Set Your Goal: Understanding UnitedHealthcare's Requirements

Getting Exondys 51 approved requires a strong partnership between you and your doctor. UnitedHealthcare has specific criteria that must be met, and your success depends on thorough documentation and clinical expertise.

Coverage Requirements at a Glance

Requirement What It Means Where to Find It
Prior Authorization Must be approved before treatment UnitedHealthcare Provider Portal
Specialist Prescriber Neurologist with DMD expertise required Medical policy documentation
Genetic Testing Confirmed exon 51-amenable mutation Genetic test report
Functional Assessments 6MWT ≥300m, cardiac/pulmonary baselines Recent test results (within 30 days)
Step Therapy May require corticosteroid trial first Plan-specific formulary
Note: UnitedHealthcare's Medicare Advantage plans may have additional step therapy requirements, requiring patients to try corticosteroids like deflazacort before accessing Exondys 51.

Your role is to work closely with your neurologist to ensure every piece of required documentation is complete and submitted properly. This isn't just about getting a prescription—it's about building a comprehensive medical case.

Preparing for Your Doctor Visit

Come prepared with a detailed timeline of your or your child's symptoms and treatment history. This preparation will help your doctor write a stronger medical necessity letter and avoid delays.

What to Bring

Symptom Timeline

  • When DMD symptoms first appeared
  • How symptoms have progressed over time
  • Current functional abilities and limitations
  • Any recent changes in mobility or strength

Previous Treatments

  • All medications tried for DMD (especially corticosteroids)
  • Reasons for stopping any treatments (side effects, lack of effectiveness)
  • Physical therapy and other supportive care
  • Any hospitalizations or emergency care

Current Status Documentation

  • Recent lab results and imaging
  • Cardiac function tests (echocardiogram, ECG)
  • Pulmonary function tests (spirometry/FVC)
  • Any recent functional assessments

Questions to Ask Your Doctor

  1. "Do you have experience managing DMD patients and prescribing Exondys 51?"
  2. "What baseline tests do we need to complete for UnitedHealthcare's requirements?"
  3. "Have you submitted prior authorizations to UnitedHealthcare for Exondys 51 before?"
  4. "What's your success rate with these approvals?"

If your current doctor isn't comfortable with the prior authorization process, ask for a referral to a pediatric neuromuscular specialist or DMD center of excellence.

Building Your Evidence Kit

Strong clinical documentation is essential for approval. Work with your healthcare team to gather comprehensive evidence that addresses every aspect of UnitedHealthcare's policy.

Required Clinical Evidence

Genetic Testing Results

  • Complete genetic analysis report showing DMD mutation
  • Specific confirmation that mutation is amenable to exon 51 skipping
  • Lab name and date of testing

Functional Assessment Battery

  • 6-Minute Walk Test: Results showing ≥300 meters (for ambulatory patients)
  • Time to Rise: Must be <7 seconds, or North Star Ambulatory Assessment score >17
  • Cardiac Function: Recent echocardiogram with LVEF/FS results
  • Pulmonary Function: Forced vital capacity (FVC) ≥30% predicted

Specialist Documentation

  • Chart notes from neurologist specializing in DMD
  • Documentation of prescriber's expertise and board certification
  • Recent consultation notes addressing current disease status

Supporting Literature

Your doctor should reference current clinical guidelines and FDA labeling in the medical necessity letter. Key sources include:

Medical Necessity Letter Structure

The medical necessity letter is the cornerstone of your prior authorization request. It must be comprehensive, well-organized, and directly address UnitedHealthcare's coverage criteria.

Essential Components

Patient Information and Diagnosis

  • Complete patient demographics and insurance details
  • Clear statement of DMD diagnosis with ICD-10 code
  • Genetic testing results confirming exon 51 amenability

Clinical Rationale

  • Current disease status and functional limitations
  • Explanation of why Exondys 51 is medically necessary
  • Discussion of expected benefits and treatment goals

Previous Treatments

  • Detailed history of corticosteroid use (if applicable)
  • Reasons for discontinuation or contraindications
  • Documentation of inadequate response to standard therapies

Supporting Evidence

  • Baseline functional, cardiac, and pulmonary assessments
  • References to clinical literature and FDA approval
  • Prescriber qualifications and DMD expertise

Clinician Corner: Medical Necessity Checklist

For Healthcare Providers: Ensure your letter includes:Specific genetic mutation details (not just "DMD diagnosis")Quantified functional assessments with datesClear statement of prognosis without treatmentDocumentation of your DMD expertise and patient volumeReferences to current clinical guidelines and FDA labeling

Supporting Peer-to-Peer Reviews

If UnitedHealthcare requests a peer-to-peer review, be prepared to support your doctor during this critical conversation.

How to Help

Provide Availability Windows

  • Give your doctor several time slots when you're available for questions
  • Be prepared to join the call if the medical director wants patient input
  • Have all relevant documents easily accessible

Prepare a Concise Case Summary

  • One-page summary of key points for your doctor to reference
  • Timeline of disease progression and treatment failures
  • Specific functional limitations affecting daily life

Key Messages for the Review

  • Emphasize the progressive nature of DMD without treatment
  • Highlight genetic testing confirming exon 51 amenability
  • Discuss functional decline and impact on quality of life
  • Reference successful outcomes in clinical trials

After Your Visit: Next Steps

Once your doctor submits the prior authorization, stay actively involved in tracking the process and maintaining communication.

What to Save and Track

Documentation

  • Copy of the prior authorization request
  • All supporting clinical documents
  • Confirmation of submission (reference numbers, dates)
  • Any correspondence from UnitedHealthcare

Communication Records

  • Notes from phone calls with UnitedHealthcare
  • Messages sent through patient portal
  • Responses from your doctor's office

Portal Communication Tips

When messaging your doctor through the patient portal:

  • Be specific about what information you need
  • Include relevant dates and reference numbers
  • Ask for copies of any denials or requests for additional information
  • Request updates on expected timelines

Respectful Persistence and Follow-Up

Approval processes can take time, but appropriate follow-up helps ensure your case doesn't get delayed or forgotten.

Follow-Up Cadence

Week 1-2: Confirm submission and ask for reference number Week 3: Check on status if no response received Week 4+: Weekly check-ins until determination made

How to Escalate Politely

  1. Start with your doctor's office: Ask case managers or nurses for updates
  2. Contact UnitedHealthcare directly: Use member services number on your insurance card
  3. Request supervisor review: If getting conflicting information
  4. Document everything: Keep records of all communications
From our advocates: We've seen families succeed by maintaining a collaborative relationship with their healthcare team while staying appropriately persistent. One family created a shared document with their neurologist's office to track all communications and deadlines, which helped ensure nothing fell through the cracks during a complex appeal process.

New Jersey Appeals Process

If UnitedHealthcare denies your initial request, New Jersey offers robust appeal rights through the Independent Health Care Appeals Program (IHCAP).

Internal Appeals First

Level 1 Internal Appeal

  • File within 180 days of denial
  • Submit through UnitedHealthcare member portal or by mail
  • Include all supporting documentation
  • Decision within 30 days (expedited: 72 hours)

Level 2 Internal Appeal (if required)

  • Must complete before external review
  • Same timeline and documentation requirements

External Review Through IHCAP

New Jersey contracts with Maximus Federal Services to manage independent external reviews.

Eligibility

  • Completed UnitedHealthcare's internal appeals (or qualified exception)
  • Denial based on medical necessity or experimental/investigational status
  • Filed within 4 months of final internal denial

Process and Timeline

  • Submit official IHCAP form with $25 filing fee
  • Standard review: Decision within 45 calendar days
  • Expedited review: Decision within 48 hours for urgent cases
  • UnitedHealthcare must comply within 10 business days if overturned

How to File

  • Online through Maximus IHCAP portal
  • By mail to address provided on forms
  • Include final denial letter and supporting medical records

Common Denial Reasons & Solutions

Understanding typical denial reasons helps you address them proactively or in appeals.

Denial Reason How to Address
Missing genetic testing Submit complete genetic analysis confirming exon 51 amenability
Non-specialist prescriber Provide documentation of neurologist's DMD expertise
Incomplete functional assessments Complete all required baseline tests (6MWT, cardiac, pulmonary)
Step therapy not completed Document corticosteroid trial/failure or contraindication
Insufficient medical necessity Strengthen clinical rationale with disease progression data

Step Therapy Exceptions

UnitedHealthcare may require trying corticosteroids first. Exceptions include:

  • Previous intolerance or adverse reactions
  • Medical contraindications
  • Continued therapy from previous insurer
  • Documented inadequate response to corticosteroids

Cost Support Options

Even with insurance coverage, Exondys 51 can involve significant out-of-pocket costs. Several programs may help reduce your financial burden.

Manufacturer Support

Sarepta Therapeutics Patient Access Program

  • Copay assistance for eligible patients
  • Prior authorization support services
  • Financial counseling and appeals assistance

Foundation Grants

  • Parent Project Muscular Dystrophy: Emergency financial assistance
  • MDA (Muscular Dystrophy Association): Equipment and care grants
  • Patient Advocate Foundation: Copay relief program

State Programs

New Jersey residents may qualify for additional assistance through state pharmaceutical assistance programs, particularly for Medicare beneficiaries.

FAQ

How long does UnitedHealthcare prior authorization take in New Jersey? Standard determinations are made within 72 hours, with urgent requests processed within 24 hours. Complex cases may take longer if additional information is requested.

What if Exondys 51 is non-formulary on my plan? Non-formulary medications can still be covered through the medical exception process. Your doctor must demonstrate medical necessity and that formulary alternatives are inappropriate.

Can I request an expedited appeal if my child's condition is worsening? Yes, both UnitedHealthcare internal appeals and New Jersey IHCAP external reviews offer expedited processes for urgent medical situations.

Does step therapy apply if we tried corticosteroids with a previous insurer? Documentation of previous treatment trials with other insurers should qualify for step therapy exceptions, but you'll need to provide detailed records.

What happens if IHCAP overturns UnitedHealthcare's denial? The decision is binding, and UnitedHealthcare must provide coverage within 10 business days. This is required by New Jersey law.

Can our doctor file the external appeal for us? Yes, healthcare providers can file IHCAP appeals on behalf of patients with proper consent and authorization.

About Counterforce Health

Counterforce Health helps patients, clinicians, and specialty pharmacies turn insurance denials into successful appeals. Our platform analyzes denial letters, plan policies, and clinical notes to create targeted, evidence-backed appeals that align with each payer's specific requirements. We specialize in complex prior authorizations for specialty medications like Exondys 51, providing the clinical documentation and regulatory expertise needed to navigate UnitedHealthcare's approval process effectively.

Sources & Further Reading

This information is for educational purposes only and does not constitute medical or legal advice. Always consult with your healthcare provider and insurance company for the most current requirements and procedures. For additional help with insurance appeals in New Jersey, contact the NJ Department of Banking and Insurance Consumer Hotline at 1-800-446-7467.

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