By Counterforce Health Team in Defitelio (defibrotide) —

Do You Qualify for Defitelio Coverage by UnitedHealthcare in Pennsylvania? Decision Tree & Next Steps

Answer Box: Your Path to Defitelio Coverage

Getting Defitelio (defibrotide) covered by UnitedHealthcare in Pennsylvania requires prior authorization for hepatic VOD/SOS with organ dysfunction post-HSCT. You're likely eligible if you have confirmed hepatic veno-occlusive disease with renal or pulmonary dysfunction following hematopoietic stem cell transplant. Start by gathering your transplant records, lab results showing organ dysfunction, and having your hematologist/oncologist submit a PA request through the UnitedHealthcare provider portal. If denied, Pennsylvania's new external review program overturns about 50% of appealed denials.

First step today: Contact your transplant center to confirm your VOD diagnosis meets FDA criteria and request they initiate the prior authorization process immediately.

Table of Contents

  1. How to Use This Decision Tree
  2. Eligibility Triage: Do You Qualify?
  3. If You're Likely Eligible: Document Checklist
  4. If You're Possibly Eligible: Tests to Request
  5. If Not Yet Eligible: Alternatives to Discuss
  6. If Denied: Pennsylvania Appeal Path
  7. Coverage Requirements at a Glance
  8. Common Denial Reasons & How to Fix Them
  9. Frequently Asked Questions
  10. Sources & Further Reading

How to Use This Decision Tree

This guide helps you determine if you qualify for Defitelio coverage and what steps to take next. Defitelio (defibrotide) is an FDA-approved treatment for hepatic veno-occlusive disease (VOD) or sinusoidal obstruction syndrome (SOS) with organ dysfunction following hematopoietic stem cell transplantation (HSCT).

Start here: Answer the eligibility questions in order. Each section will direct you to the next appropriate step based on your specific situation.

Note: This is not medical advice. Always consult with your transplant team and insurance representative for personalized guidance.

Eligibility Triage: Do You Qualify?

Work through these questions with your medical team to determine your coverage pathway:

1. Diagnosis Confirmed?

  • Yes, I have hepatic VOD/SOS diagnosed by my transplant center → Continue to question 2
  • No, but I have concerning symptoms after HSCT → Jump to Tests to Request
  • No HSCT performed → Jump to Not Yet Eligible

2. Severity Assessment

  • I have both elevated bilirubin (≥2 mg/dL) AND organ dysfunction (kidney or lung problems) → Continue to question 3
  • I have elevated bilirubin but unclear organ dysfunction → Jump to Tests to Request
  • I have mild symptoms only → Jump to Not Yet Eligible

3. Transplant Timeline

  • VOD developed within 30 days of HSCT → You're Likely Eligible → Continue to Document Checklist
  • VOD developed 30+ days after HSCT → You're Possibly Eligible → Jump to Tests to Request

4. Prior Therapy Status

  • This is first-line treatment for VOD with organ dysfunction → You're Likely Eligible
  • I've tried other treatments first → Document what was tried and why it failed

If You're Likely Eligible: Document Checklist

You meet FDA criteria for Defitelio. Here's what your medical team needs to submit for UnitedHealthcare prior authorization:

Required Clinical Documentation

  • HSCT procedure details (type, date, conditioning regimen)
  • VOD diagnosis confirmation with ICD-10 code K76.5
  • Organ dysfunction evidence:
    • Renal: Elevated creatinine levels and dates
    • Pulmonary: Oxygen requirements, imaging results, or pulmonary function tests
  • Laboratory results showing bilirubin ≥2 mg/dL
  • Clinical symptoms documented: weight gain ≥5%, hepatomegaly, right upper quadrant pain
  • Treatment plan specifying 6.25 mg/kg IV every 6 hours, hospital administration

Prescriber Requirements

Your hematologist/oncologist must provide:

  • Medical necessity letter referencing FDA indication
  • Specialist attestation confirming diagnosis and organ dysfunction
  • Treatment duration justification (typically up to 21 days)
  • Safety monitoring plan for bleeding risk

Submission Process

  1. Submit through UnitedHealthcare provider portal at uhcprovider.com
  2. Alternative: Fax to 1-844-403-1027 (verify current number)
  3. Timeline: Submit 3-4 weeks in advance; standard review takes 5-7 business days
  4. Urgent cases: Request expedited review within 72 hours
Tip: Include references to published survival data showing improved Day +100 survival with Defitelio (up to 45% vs. 21-31% with supportive care alone).

If You're Possibly Eligible: Tests to Request

You may qualify but need additional documentation. Work with your transplant team to obtain:

Additional Diagnostic Tests

  • Liver biopsy or imaging to confirm VOD if diagnosis is uncertain
  • Comprehensive metabolic panel to document kidney function changes
  • Chest X-ray or CT scan if pulmonary dysfunction suspected
  • Coagulation studies to assess bleeding risk before treatment
  • Daily weights and abdominal measurements to track fluid retention

Timeline to Re-evaluate

  • Within 48-72 hours: Obtain missing lab results and imaging
  • Within 1 week: Reassess symptoms and repeat eligibility triage
  • If symptoms worsen: Request urgent PA submission

What to Track

Document any worsening of:

  • Bilirubin levels (trending upward)
  • Kidney function (rising creatinine)
  • Breathing difficulties or oxygen needs
  • Abdominal pain or liver enlargement

If Not Yet Eligible: Alternatives to Discuss

If you don't currently meet FDA criteria, discuss these options with your transplant team:

Supportive Care Options

  • Fluid management and diuretics for weight gain
  • Pain management for abdominal discomfort
  • Monitoring protocols for early VOD detection
  • Prophylactic measures if at high risk for VOD development

Preparing for Exception Requests

If your case is complex, your doctor may request coverage based on:

  • Compassionate use criteria for severe cases
  • Off-label use with strong clinical justification
  • Literature support for expanded indications
Note: Off-label prophylaxis typically requires extensive documentation and may face higher denial rates.

If Denied: Pennsylvania Appeal Path

Pennsylvania offers strong consumer protections with a new external review program that overturns about 50% of insurance denials.

Internal Appeals (Required First Step)

  1. File within 180 days of UnitedHealthcare's denial letter
  2. Submit electronically through UnitedHealthcare provider portal (required as of December 2024)
  3. Include all documentation: denial letter, medical evidence, provider support
  4. Timeline: UnitedHealthcare has 30 days to respond (72 hours for urgent cases)

Peer-to-Peer Review Option

  • Request before formal appeal to discuss case with UnitedHealthcare medical director
  • Prepare your specialist to articulate guideline-based justification
  • Reference clinical evidence including NCCN guidelines and FDA labeling
  • Document the conversation for potential use in formal appeals

Pennsylvania External Review (New Program)

If your internal appeal is denied:

  1. Eligibility: Must have Final Adverse Benefit Determination letter from UnitedHealthcare
  2. Timeline: Request within 4 months of final denial
  3. Process: Submit through Pennsylvania Insurance Department website
  4. Decision: Independent Review Organization decides within 45 days (72 hours for urgent)
  5. Binding: If IRO rules in your favor, UnitedHealthcare must provide coverage immediately

Free Help Available

Pennsylvania Health Law Project offers free assistance:

  • Helpline: 1-800-274-3258 or email [email protected]
  • Services: Appeal guidance, documentation help, legal support for Medicaid members
  • Expertise: Specialty drug denials and "experimental" treatment challenges

Coverage Requirements at a Glance

Requirement What It Means Where to Find It
Prior Authorization Required for all Defitelio use UnitedHealthcare Provider Portal
Specialty Tier Hospital/infusion center only OptumRx formulary documents
Step Therapy May require documentation of alternatives Denial letter will specify if applicable
Medical Necessity FDA indication: VOD with organ dysfunction post-HSCT FDA Defitelio labeling
Site of Care Inpatient hospital or approved infusion center UnitedHealthcare policy documents
Age Limits None (FDA approved for all ages) FDA labeling
Diagnosis Code ICD-10: K76.5 (hepatic veno-occlusive disease) Medical coding references

Common Denial Reasons & How to Fix Them

Denial Reason How to Overturn
"Not medically necessary" Submit specialist letter with FDA indication, organ dysfunction labs, and survival data
"Experimental/investigational" Reference FDA approval (2016) and include package insert
"Step therapy required" Document contraindications to alternatives or lack of effective alternatives for VOD
"Site of care restriction" Confirm hospital/infusion center administration in treatment plan
"Missing documentation" Provide complete HSCT records, VOD diagnosis, and organ dysfunction evidence
"Off-label use" If using for prophylaxis, provide literature support and compassionate use justification

Frequently Asked Questions

Q: How long does UnitedHealthcare prior authorization take in Pennsylvania? A: Standard review takes 5-7 business days. Urgent cases are reviewed within 72 hours. Submit 3-4 weeks before needed treatment when possible.

Q: What if Defitelio is non-formulary on my plan? A: Non-formulary drugs can still be covered with prior authorization and medical necessity documentation. The appeals process remains the same.

Q: Can I request an expedited appeal if my health is deteriorating? A: Yes. For life-threatening situations, request expedited review through UnitedHealthcare (72 hours) and Pennsylvania's external review program (72 hours for urgent cases).

Q: Does step therapy apply if I've never tried other VOD treatments? A: Step therapy may not apply for FDA-approved indications where Defitelio is first-line therapy, but document any contraindications to supportive care measures.

Q: What happens if I'm on Medicaid managed care through UnitedHealthcare? A: The same PA process applies, but you have additional appeal rights including fair hearings. Contact Pennsylvania Health Law Project for free assistance.

Q: How much does Defitelio cost if not covered? A: As a buy-and-bill hospital drug, costs vary by institution. Contact Jazz Pharmaceuticals patient support programs for assistance options.

When navigating complex specialty drug approvals like Defitelio, having expert support can make the difference between approval and denial. Counterforce Health helps patients, clinicians, and specialty pharmacies turn insurance denials into targeted, evidence-backed appeals by analyzing denial letters, plan policies, and clinical notes to create compelling medical necessity arguments that align with each payer's specific requirements.

Whether you're just starting the prior authorization process or facing a denial, this decision tree provides the framework to advocate effectively for Defitelio coverage. Remember that Pennsylvania's strengthened consumer protections, including the new external review program, give you powerful tools to challenge unfair denials.

The key to success is thorough documentation, understanding your plan's specific requirements, and knowing when to escalate through Pennsylvania's appeals system. With proper preparation and the right support, patients with legitimate medical needs for Defitelio can successfully navigate UnitedHealthcare's approval process.

Sources & Further Reading

Disclaimer: This information is for educational purposes only and does not constitute medical or legal advice. Always consult with your healthcare provider and insurance representative for personalized guidance. Coverage policies and procedures may change; verify current requirements with your specific plan.

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