By Counterforce Health Team in Translarna (ataluren) —

Getting Translarna (Ataluren) Covered by Humana in Virginia: Prior Authorization Guide and Appeal Process

Answer Box: How to Get Translarna (Ataluren) Covered by Humana in Virginia

Translarna (ataluren) requires prior authorization from Humana and is typically not on standard formularies. Your fastest path to coverage:

  1. Confirm genetic testing shows nonsense mutation Duchenne muscular dystrophy (nmDMD)
  2. Document corticosteroid trial/failure to meet step therapy requirements
  3. Submit comprehensive PA request via Humana provider portal with clinical notes, genetic report, and medical necessity letter

Start today: Call the number on your Humana ID card to verify formulary status and PA requirements. If denied, Virginia's State Corporation Commission Bureau of Insurance provides external review within 120 days.

Table of Contents

Humana's Coverage Criteria for Translarna

Plan Types and Policy Application

Humana operates primarily Medicare Advantage and Part D plans in Virginia, with some commercial coverage. Prior authorization requirements apply across all plan types for specialty medications like Translarna, which typically falls under specialty pharmacy tiers due to its high cost and limited indication.

FDA and Regulatory Status

As of 2024, Translarna (ataluren) lacks FDA approval in the United States, though PTC Therapeutics submitted an NDA in October 2024 that remains under review. The European Medicines Agency (EMA) recommended non-renewal of marketing authorization in 2024, though the drug remains available in some regions through compassionate use programs.

This regulatory uncertainty creates additional coverage hurdles, as insurers typically require either FDA approval or established compendia listing for coverage consideration.

Coverage at a Glance

Requirement Details Documentation Needed Source
Prior Authorization Required for all plans PA form via provider portal Humana PA Policy
Formulary Status Typically non-formulary Exception request required Humana Drug Search
Genetic Testing Confirmed nonsense mutation DMD Genetic lab report Clinical guidelines
Step Therapy Corticosteroid trial required Treatment history documentation Humana medical policy
Ambulatory Status Must be ambulatory Functional assessment Clinical notes
Specialist Prescriber Neuromuscular specialist preferred Provider credentials PA requirements

Step Therapy Requirements and Exceptions

Standard Step Therapy Protocol

Humana typically requires patients to try and fail corticosteroids (prednisone or deflazacort) before considering Translarna, as corticosteroids are the established first-line therapy for all DMD patients and can delay loss of ambulation by 2-5 years.

Medical Exception Pathways

You can bypass step therapy if you can document:

  • Contraindications to corticosteroids (severe osteoporosis, uncontrolled diabetes, active infection)
  • Previous intolerance with specific adverse effects documented
  • Clinical urgency where delay would compromise ambulatory status
Tip: Document any corticosteroid trial with specific dates, doses, duration, and reason for discontinuation. Even a brief trial with documented side effects can satisfy step therapy requirements.

Required Documentation for Medical Necessity

Essential Clinical Documentation

Genetic Testing Requirements:

  • Laboratory report confirming nonsense mutation in DMD gene
  • Explicit statement that variant is "pathogenic" or "likely pathogenic"
  • HGVS notation showing premature stop codon
  • Lab interpretation confirming Duchenne muscular dystrophy diagnosis

Functional Status Documentation:

  • Current ambulatory status with objective measures
  • Timed 10-meter walk test results if available
  • Motor Function Measure (MFM) scores, particularly D1 domain
  • Documentation that patient can walk independently without assistive devices

Treatment History:

  • Previous corticosteroid therapy with dates, doses, and outcomes
  • Any other DMD treatments attempted
  • Reason for discontinuation of prior therapies

Clinician Corner: Medical Necessity Letter Checklist

When preparing your medical necessity letter, include:

  1. Clear diagnosis: "Duchenne muscular dystrophy, nonsense mutation, ambulatory phase"
  2. Genetic confirmation: Reference specific lab report and mutation type
  3. Functional status: Current ambulatory capacity with objective measures
  4. Treatment rationale: Why Translarna is appropriate for this specific mutation type
  5. Clinical urgency: Risk of losing ambulation without treatment
  6. Monitoring plan: How treatment response will be assessed

Counterforce Health specializes in turning insurance denials into targeted, evidence-backed appeals by analyzing denial letters, plan policies, and clinical notes to draft point-by-point rebuttals aligned to each plan's specific rules and requirements.

Prior Authorization Process

Step-by-Step Submission Process

  1. Verify current formulary status via Humana's drug search tool or call member services
  2. Gather required documentation (genetic testing, clinical notes, treatment history)
  3. Submit PA request through Humana provider portal or designated fax line
  4. Include comprehensive medical necessity letter addressing all coverage criteria
  5. Follow up within 5-7 business days if no initial response received

Submission Methods

  • Provider Portal: Log in at Humana.com (preferred method for tracking)
  • Fax: Check current PA fax number on provider portal (verify with source)
  • Phone: For urgent cases, call provider services for expedited review

Expected Timelines

  • Standard PA decisions: 30 days for Medicare Advantage pre-service decisions
  • Expedited review: 72 hours if clinical urgency documented
  • Part D appeals: 7 days standard, 72 hours expedited

Appeals Process: Internal and External Review

Humana Internal Appeals

If your initial PA request is denied, you have 65 days from the denial notice to file an internal appeal with Humana. The internal appeal should include:

  • Copy of original denial letter
  • Additional clinical documentation supporting medical necessity
  • Peer-reviewed literature supporting Translarna use in nmDMD
  • Updated functional assessments if available

When to Request Peer-to-Peer Review

Before filing a formal appeal, request a peer-to-peer review where your prescribing physician can speak directly with Humana's medical director. This often resolves coverage issues more quickly than formal appeals.

Script for requesting peer-to-peer: "I'm calling to request a peer-to-peer review for the denied prior authorization for Translarna. The prescribing physician would like to discuss the clinical rationale with your medical director."

Virginia External Review Process

Overview of Virginia's External Review System

Virginia's State Corporation Commission Bureau of Insurance provides an independent external review process for health insurance denials. This is a powerful tool when Humana's internal appeals process has been exhausted.

Step-by-Step External Review Process

  1. Complete Humana's internal appeals and receive final adverse determination
  2. Submit external review request within 120 days using Form 216-A
  3. Include all supporting documentation: denial letters, medical records, clinical notes
  4. SCC assigns Independent Review Organization (IRO) with relevant medical expertise
  5. IRO makes binding decision within 45 days (72 hours for expedited reviews)

Submission Details

Contact Information:

  • Email: [email protected]
  • Fax: (804) 371-9915
  • Mail: SCC Bureau of Insurance – External Review, P.O. Box 1157, Richmond, VA 23218

Expedited External Review

For cases where waiting 45 days could seriously jeopardize health, Virginia offers expedited external review with decisions within 72 hours. This may apply to Translarna cases where rapid functional decline threatens loss of ambulation.

Note: Virginia's external review process is free to consumers, and the IRO's decision is binding on both the insurer and the patient.

Common Denial Reasons and Solutions

Denial Reason How to Overturn Required Documentation
"Experimental/Investigational" Cite published efficacy data and FDA review status Peer-reviewed studies, FDA correspondence
"Not medically necessary" Provide functional assessments and progression data Timed walk tests, MFM scores, clinical notes
"Step therapy not completed" Document corticosteroid trial/contraindication Treatment records, adverse event documentation
"Non-formulary drug" Request formulary exception with clinical justification Medical necessity letter, alternative therapy failures
"Genetic testing insufficient" Provide comprehensive genetic report Lab report with HGVS notation, pathogenicity assessment

Appeals Success Strategies

Strengthen your appeal with:

  • Meta-analysis data showing 15-31 meter improvement in 6-minute walk distance
  • Documentation of ambulatory status preservation
  • Comparison to alternative therapy costs and outcomes
  • Specialist letters from neuromuscular disease experts

When dealing with complex appeals, Counterforce Health helps patients and clinicians by identifying specific denial bases and drafting targeted rebuttals that address each plan's unique coverage criteria and procedural requirements.

Cost Assistance and Patient Support

Manufacturer Support Programs

PTC Therapeutics may offer patient assistance programs for eligible patients. Contact their patient support services for current program availability and eligibility requirements (verify current contact information with manufacturer).

Foundation Resources

  • Muscular Dystrophy Association: May provide financial assistance for medications and equipment
  • Parent Project Muscular Dystrophy: Advocacy and resource navigation
  • CureDuchenne: Patient support and clinical trial information

Virginia State Resources

Virginia's Office of the Managed Care Ombudsman can assist with understanding appeal rights and may help resolve coverage disputes informally.

From Our Advocates

"We've seen success when families work closely with their neuromuscular specialist to document not just the genetic confirmation, but the functional trajectory. One family's appeal was approved after they included serial 10-meter walk test results showing declining speed over six months, combined with a detailed letter explaining how Translarna specifically targets their child's nonsense mutation. The key was making the clinical urgency clear while addressing every coverage criterion systematically."

Frequently Asked Questions

Q: How long does Humana's prior authorization process take? A: Standard PA decisions take up to 30 days for Medicare Advantage plans. Expedited reviews are available within 72 hours if clinical urgency is documented.

Q: What if Translarna is not on Humana's formulary? A: You can request a formulary exception by demonstrating medical necessity and providing clinical justification for why covered alternatives are inappropriate.

Q: Can I appeal if I live in Virginia but have coverage through an employer plan? A: Self-funded employer plans may opt into Virginia's external review process. If not, federal external review through HHS may be available.

Q: Does step therapy apply if I tried corticosteroids with a previous insurer? A: Yes, treatment history from other insurers typically counts toward step therapy requirements if properly documented.

Q: How much does Translarna cost without insurance? A: Annual costs can exceed $300,000. Manufacturer assistance programs and foundation support may help with costs.

Q: What genetic testing is required for coverage? A: You need comprehensive DMD gene analysis showing a confirmed pathogenic nonsense mutation, typically performed after negative deletion/duplication testing.

Q: Can I get expedited review for Translarna appeals? A: Yes, if your physician documents that delay could jeopardize your health or ambulatory status, both Humana and Virginia's external review process offer expedited timelines.

Q: What happens if the external review upholds the denial? A: The decision is final and binding, though you retain rights to pursue legal action separately if you believe laws were violated.

Sources & Further Reading

Disclaimer: This guide provides general information about insurance coverage processes and should not be considered medical or legal advice. Coverage decisions depend on individual plan terms, medical circumstances, and current regulatory status. Always consult with your healthcare provider and insurance plan for specific coverage determinations. For assistance with complex appeals, contact Virginia's State Corporation Commission Bureau of Insurance or consider working with advocacy organizations that specialize in insurance coverage issues.

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